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In the face of COVID-19, cell and gene therapy space shows ‘remarkable resilience:’ report – FierceBiotech

In the early days of COVID-19, the Alliance for Regenerative Medicine (ARM) was unsure how the pandemic and its accompanying economic downturn would affect the cell and gene therapy space.

It was a really specific time when the world and the markets were clearly reeling from the first appreciation for the seriousness of COVID-19, Janet Lambert, the organizations CEO said.

Now, the numbers are inand theyre better than ever. In the first half of 2020, the regenerative medicine sector raised $10.7 billion, more than the total capital raised in 2019 and a 120% jump over the first half of 2019, ARM found in a new report titled, Innovation in the Time of COVID-19. The proceeds were shared pretty evenly between cell therapy companies ($7.5 billion) and gene and gene-modified cell therapy companies ($7.9 billion), with companies focused on tissue engineering reeling in $84 million.

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RELATED: Biotech IPO bonanza: Legend's $350M offering as Repare, Forma get in on the action

That $10.7 billion was driven by a couple of outsize deals and includes $1.4 billion raised in five IPOs, $1.6 billion in follow-on offerings and $3 billion in venture capital. Chinese CAR-T player Legend Biotech led the pack with its mammoth $487 million Wall Street debut in June, but its peers netted considerable sums too. That same month, gene therapy companies Generation Bio and Akouos raised $230 million and $244 million, respectively. In February, another gene therapy outfit, Passage Bio, raised $284 million and gene-editing biotech Beam Therapeutics bagged $207 million.

On the venture side, Sana Biotechnology scored $700 millionalmost as much as the five next largest private rounds raised by Orca Bio Elevate Bio, Legend, Freeline Therapeutics and Poseida, the report found. Like Legend, Generation Bio and Akouos also completed sizable private rounds the same year they went public.

RELATED: 'The silver lining': Biotech IPOs in the time of coronavirus

All this enthusiasm for this sector right now is evidenced by these really astonishing financing numbers I think the drivers of that enthusiasm remain in place and make me optimistic for the second half of 2020, Lambert said. We continue to see really promising clinical results. We continue to see products making it to market. We continue to see patient, regulator and payer enthusiasm for these products.

Part of that enthusiasm stems from an appreciation for the biotech sector generally, Lambert said.

Attention is being paid to what the biopharma sector can do for us all as we try to weather and get out of the pandemic, she said, echoing the sentiments of venture capitalists whove managed to raise life sciences funds in spite of the pandemic.

The other side of the equation is the nature of biotechbecause the drug development cycle is long, biotech investors arent looking for quarter-to-quarter returns, but at milestone readouts that can come more than a year after IPO, Jordan Saxe, head of healthcare listings at Nasdaq, said in a previous interview.

Biotech is actually fairly well positioned to weather these kinds of events because youre not relying on day-to-day consumer spending. Youre relying on meaningful clinical catalysts at the end of the day to really generate value, and thats still going to be there in this environment, said Jason Pitts, Ph.D., a principal at Sofinnova, in ARMs report.

RELATED: Flagship raises $1.1B to create biotechs for post-pandemic world

All this gas in the tank isnt just bankrolling existing cell and gene therapies, but also driving company formation, Lambert said. For the first time, ARM counts more than 1,000 companies working in the sector, with more than 1,000 clinical trials going on worldwide. More than half of those studies are in phase 2, with just over a third in phase 1 and the remainder in phase 3.

Of those studies, 11 are testing regenerative medicine approaches against COVID-19, with several academic research centers and biopharma companies working on new treatments to treat the disease in the short and long term.

Most of them are using cell therapies to address ARDS, or acute respiratory distress syndrome, which is a consequence of COVID-19, Lambert said. Unlike other prospects in the pipeline, such as antibodies, which could potentially be used to prevent infection as well as treat it, regenerative treatments focus on repairing damage to the lungs or other organs that patients can suffer as part of COVID-19.

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In the face of COVID-19, cell and gene therapy space shows 'remarkable resilience:' report - FierceBiotech

Gene Therapy Promising in BCG-Unresponsive Bladder Cancer – Medscape

More than half the patients with high-grade bacillus Calmette-Gurin (BCG)-unresponsive nonmuscle invasive bladder cancer (NMIBC) treated with nadofaragene firadenovec (Instiladrin), an investigational intravesical viral gene therapy, achieved a clinical response at 3 months in a phase3 trial.

The results "provide a significant efficacy benefit that, pending regulatory approval, might offer patients with a difficult-to-treat bladder cancer a bladder-sparing alternative," said Neal Shore, MD, medical director for the Carolina Urologic Research Center in Myrtle Beach, South Carolina.

There is clearly an unmet need for new bladder-sparing treatments in these patients, said Fred Witjes, MD, from the Radboud Institute for Molecular Life Science in Nijmegen, the Netherlands, who discussed trial findings during the virtual European Association of Urology 2020 Congress.

"The drugs that we have are old and there is a limited availability for both MMC [mitomycin-C] and BCG. We need some alternatives for initial adjuvant therapy," he explained. "The unmet need is, of course, especially there in BCG-unresponsive patients or BCG-unresponsive CIS [carcinoma insitu]."

"Clinically appropriate patients with BCG-unresponsive NMIBC are currently faced with radical cystectomy," Shore explained during his presentation at the congress.

Nadofaragene firadenovec is a viral-based gene therapy that consists of a replication-deficient adenovirus that delivers the gene for interferon alpha-2b (IFN2b). When administered with the polyamide compound Syn3, the viral vector can deliver the IFN2b gene to the epithelial lining of the bladder. The gene is subsequently incorporated into cellular DNA, meaning that large amounts of the IFN2b protein can be produced locally.

For their open-label, randomized trial, Shore and his colleagues looked at 157 patients with a mean age around70 years. All participants had carcinoma insitu or high-grade Ta (noninvasive) or T1 (invasive) papillary disease with or without carcinoma insitu, and all had been unresponsive to standard intravesical treatment with BCG in the previous 12 months.

Nadofaragene firadenovec was administered once every 3 months, for up to four doses in the first year. If patients showed no signs of high-grade disease recurrence at 12 months, they were offered continued treatment.

For patients with high-grade carcinoma insitu, the complete response rate was 53.4% at 3 months and 24.3% at 12 months. For patients with papillary tumors, the response rate was 73.0% at 3 months and 44% at 12 months.

The majority of study participants (72%) received two or three courses of BCG overall; that rate was 68.3% for those with carcinoma insitu and 80.0% for those with papillary disease. Just over half the patients with carcinoma insitu were refractory to BCG, as were 70% of those with papillary disease.

Almost one third of patients will not respond to BCG, and more than 50% will experience recurrence and progression during long-term follow-up, according to results from the phase2 study of nadofaragene firadenovec, which Shore was involved in.

In that trial of 40 patients, the response rate was 30% at 12 months for those with carcinoma insitu, and durable responses were seen out to 36 months. Investigators reported no dose-limiting toxicities or immune toxicity.

In the phase3 study, treatment-emergent adverse events were experienced by 93% of participants, but the vast majority were transient and grade1 or 2 events; approximately 17% were grade3. There was one grade4 event, but this was not related to the study treatment.

The most common treatment-emergent adverse events were instillation-site discharge, reported by 33.1% of the patients; fatigue, reported by 23.6%; bladder spasm, reported by 19.7%; micturition urgency, reported by 17.8%; and hematuria, reported by 16.6%.

"Follow-up and treatment of these patients is ongoing in an extension study," Shore said.

"We do really need something new in nonmuscle invasive bladder cancer," Witjes observed. "There has to be an alternative to cystectomy and, fortunately, news is coming."

"There is a highly unmet need, but we have to realize that there is a lot in the pipeline," he explained. "We have trials with immune checkpoint blockade, vaccines, genetic therapy, and drug-delivery systems."

Nadofaragene firadenovec creates "adaptive immunity that may be lifelong," Witjes reported. "Instiladrin has a good basis, has consistent, good results, and it has a good safety profile. In light of current developments, I think this certainly is an interesting option."

The study was sponsored by FKD Therapies Oy and conducted in collaboration with the Society of Urologic Oncology Clinical Trials Consortium. Shore reports receiving research and consulting fees from Amgen, Astellas, Bayer, BMS, Dendreon, Fergene, Ferring, Janssen, Merck, Myovant, Nymox, Pacific Edge, Nucleix, Pfizer, Sanofi-Genzyme, Sun Pharma, and Tolmar. Witjtes reports receiving advisory or lecturer fees from multiple companies, but none relevant to his comments.

European Association of Urology (EAU) 2020 Congress. Presented July17, 2020.

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Gene Therapy Promising in BCG-Unresponsive Bladder Cancer - Medscape

Evolution and Expansion of Therapies in the Global Cell and Gene Therapy Tools and Reagents Market 2020-2024 – PRNewswire

DUBLIN, Aug. 4, 2020 /PRNewswire/ -- The "Cell and Gene Therapy Tools, and Reagents: Global Markets" report has been added to ResearchAndMarkets.com's offering.

Gene and cell therapy are emerging as important tools to treat human health. Techniques such as CAR-T therapy have emerged as key ways of treating many different types of cancers. The promise of gene therapy using technologies such as CRISPR is starting to be realized in clinical trials, and markets are scaling up to treat other diseases as well, particularly rare gene-based diseases. As these therapies are coming to the fore, a new market for tools to develop these therapies using standard methodologies is emerging. This report will cover what those tools are, how they impact the larger life science tools market, and how they will evolve over the next five years.

The scope of this study encompasses an investigation of the market's cell and gene therapy tools such as GMP proteins, media, cell separation and activation reagents, viral and non-viral, cytokine release syndrome monitoring products, GMP antibodies, leukapheresis instrumentation, immunoassays (multiplex and singleplex) and bioreactors. This research analyzes each tool type, determines its current market status, examines its impact on future markets, and presents forecasts of growth over the next five years. Technological issues, including the latest trends, are discussed. The report analyzes the industry on a worldwide basis, from both application and demand perspectives, in the major regions of the world.

The Report Includes:

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background

Chapter 4 Market Breakdown by Region

Chapter 5 Market Breakdown by End User

Chapter 6 Government Regulations

Chapter 7 Patent Review/New Developments

Chapter 8 Analysis of Market Opportunities

Chapter 9 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/p5fqx6

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Evolution and Expansion of Therapies in the Global Cell and Gene Therapy Tools and Reagents Market 2020-2024 - PRNewswire

How Gene Therapy Helped Conner Run : Short Wave – NPR

Conner Curran, 9, (right) and his brother Will, 7, at their home in Ridgefield, Conn. The gene therapy treatment that stopped the muscle wasting of Conner's muscular dystrophy two years ago took more than 30 years of research to develop. Kholood Eid for NPR hide caption

Conner Curran, 9, (right) and his brother Will, 7, at their home in Ridgefield, Conn. The gene therapy treatment that stopped the muscle wasting of Conner's muscular dystrophy two years ago took more than 30 years of research to develop.

Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, it could change the lives of thousands of children with Duchenne muscular dystrophy. NPR's Jon Hamilton tells us about Conner and his family...and one of the scientists who helped develop the treatment, a pioneer in the field of gene therapy.

This episode was produced by Abby Wendle, edited by Viet Le and fact-checked by Berly McCoy.

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How Gene Therapy Helped Conner Run : Short Wave - NPR

Preparing for an influx of cell and gene therapy approvals – – pharmaphorum

Cell and gene therapies offer some of the most groundbreaking advancements in patient care the pharma industry has ever seen. However, to fully realise the potential of these innovative therapies, integration across the supply chain is critical particularly with reimbursement and logistics.

As of the end of 2019, there were 17 cell and gene therapy products approved by the FDA. Now, there is more momentum than ever to bring these innovative medicines to market, and the FDA anticipates that it will approve 10 to 20 cell and gene therapy products a year within the next five years.

These therapies can offer new opportunities to patients with conditions where there are few treatment options and no cures. But the potential these products offer could remain largely unrealised if manufacturers and their partners are not prepared. Cell and gene therapy innovators and other stakeholders across the supply chain need to set themselves up for the greatest chance of success by addressing three key challenges: access barriers; logistics; and the need for stakeholder education.

Addressing access barriers through innovative payment models

While cell and gene therapies offer novel treatment to patients who have limited options, the cost associated with each product anywhere between $375,000 and $2 million can create significant access barriers. This challenge is compounded compared to traditional treatments that typically require multiple doses, as many cell and gene therapies are one-time treatments.

This situation increases the risk for payers covering the cell and gene therapy, given that the long-term magnitude and durability of the product is not known at the time of first regulatory approval and patients switch insurance carriers throughout their lifetimes.

Stakeholders across the industry have recognised the increasing need to consider alternatives to the standard payment system if cell and gene therapies are to become widely available

Stakeholders across the industry, such as manufacturers and payers, have recognised the increasing need to consider alternatives to the standard payment system if cell and gene therapies are to become widely available. As a result, a variety of payment models have been discussed:

We have already begun to see payers and manufacturers of cell and gene therapies attempt to adopt alternative payment models for their products, and more should continue to do so as additional therapies come through the approval pipeline. With a range of interdependencies that affect the success of cell and gene therapies, manufacturers need to develop their reimbursement strategy early in the commercialisation process. Its critical for manufacturers to consider various payment models for cell and gene therapies ahead of approvals so that they can maximise patient access for their products.

Ensuring therapies reach their patients

Manufacturers have noted that the delivery of critical shipments is one of the biggest challenges facing the advanced therapy industry, as if you cannot connect cell and gene therapies with patients their efficacy is irrelevant. The inclusion of patients into the cell and gene therapy supply chain, the potentially life-altering impact of the therapies and their high cost leaves no room for failure.

These therapies require timely delivery and maintaining precise temperature control is integral for the patient and the product. It calls for near-perfect execution ranging from mapping the best transportation route and planning for multiple contingencies (such as closed international borders), to how the packaging itself is evaluated, validated and used to maintain product integrity in all conditions.

Successful execution of these processes requires both manufacturers and other supply chain partners to maintain a robust logistics platform. Currently, many manufacturers are developing different logistics plans for each of the stages of a clinical trial, only to find out these processes dont scale when it is time to commercialise. Developing a plan early that can scale will position a product for success as more therapies are reviewed and approved. Manufacturers need to work with their 3PL and distribution partners to ensure control and oversight throughout the product journey to the patient failure to do so will put patient outcomes and commercial success at risk.

Promoting stakeholder education

Many stakeholders spanning payers, providers and patients do not understand the full clinical, logistical, operational, financial or reimbursement components associated with cell and gene therapies. Manufacturers can leverage the preliminary data theyve gathered throughout their initial commercialisation journey to support education and awareness efforts with these key stakeholders.

As payers conduct product reviews earlier and earlier in the development lifecycle, their demand for pre-approval information continues to grow. However, recent research shows that a gap still exists between the evidence sought by healthcare decision makers and what is being shared by manufacturers. COVID-19 has also caused delays in providing information in a timely and relevant manner, causing even more challenges for stakeholders.

The use of Pre-approval Information Exchange (PIE) is one way to combat these challenges. PIE allows manufacturers to communicate ahead of approval to partners with accurate, and unbiased information on products or indications, and share information early that may result in a place saved at the table for their product. This information equips stakeholders with the education needed to understand a products value story and positioning. Partners embedded in the industry particularly those with a patient-centric focus can also offer manufacturers the information they need to showcase the value of these products to patients.

The cell and gene therapy space is continuing to evolve. Through analysing payment models, working with partners to navigate logistical challenges and leveraging data, patients will have more opportunities than ever to access the next generation of medicines. Overall, the collaboration between stakeholders across the supply chain will facilitate a world in which we see 10 to 20 cell and gene therapies not only approved each year but out in the market directly impacting patients.

About the authors

Alex Guite is vice president services and alliances at World Courier. As strategy and services lead, Alex is responsible for developing and executing key strategic initiatives.Before joining World Courier in 2013 as head of pricing, Alex spent nearly 3 years with Oliver Wyman as a consultant in the Health and Life Sciences practice.

Ana Stojanovska is vice president, reimbursement & policy insights at Xcenda. She has extensive practical knowledge in working with key stakeholders to motivate local coverage of new products by both public and private payers and providing strategic compendia analyses and ongoing coding support. Prior to Xcenda, Ana worked for a bipartisan, non-profit health policy organization in Washington DC, where she helped lead research, health policy analysis, media outreach, and fundraising.

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Preparing for an influx of cell and gene therapy approvals - - pharmaphorum

Catalent’s Harmans Site Approved To Manufacture AveXis’ Gene Therapy – Contract Pharma

Catalent was approved by the U.S. FDA to produce commercial drug substance intermediate for AveXis spinal muscular atrophy (SMA) gene therapy at its manufacturing facility in Harmans, MD.

The approval follows an FDA inspection of the Harmans commercial-scale gene therapy manufacturing center in June. Under Catalents partnership with AveXis, a Novartis company, a dedicated suite space has been prepared at the Harmans facility for the commercial manufacture of this adeno-associated virus (AAV) gene therapy.

This is a significant milestone for Catalent and the gene therapy industry as a whole. Catalent is proud to be the first contract development and manufacturing organization to be approved for commercial gene therapy production, commented Manja Boerman, Ph.D., President of Catalent Cell & Gene Therapy. This approval allows us to leverage our now-licensed, state-of-the-art GMP commercial manufacturing facility, and our deep AAV expertise, to support AveXis as it delivers a life-changing treatment for patients.

Given the complexity and length of time required to make gene therapies, manufacturing is critically important, said Dannielle Appelhans, Chief Technical Officer for AveXis. This approval further complements our internal manufacturing capacity and, over time, will allow us to increase supply to meet growing patient needs.

Catalents Harmans commercial manufacturing facility, located near BWI airport, is equipped with single-use technology, and houses over 200,000 sq.-ft. of late-stage clinical and commercial-stage gene therapy production. The facility is one of Catalents five gene therapy facilities in Maryland providing clinical through commercial scale services, and houses multiple CGMP manufacturing suites, including fill/finish, central services and testing labs, warehousing, and supply chain capabilities.

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Catalent's Harmans Site Approved To Manufacture AveXis' Gene Therapy - Contract Pharma

Sangamo and Novartis partner on gene therapies for autism – BioPharma-Reporter.com

The targets will be three undisclosed genes that are associated with certain neurodevelopmental disorders, such as autism spectrum disorder (ASD) and intellectual disability.

Sangamo Therapeutics will bring its genome regulation technology to upregulate the expression of key genes involved in neurodevelopmental disorders.

To gain access to this technology, Novartis will pay $75m (63m) upfront to Sangamo. Beyond this, Novartis could end up paying an additional $720m in developmental milestones.

According to Sangamo, the hope is that its zing-finger technology can active the expression of genes that are inadequately expressed in individuals with certain types of neurodevelopmental disorders.

Sangamos potentially therapy is currently delivered through adeno-associated viruses (AAVs) to the DNA level.

The terms of the agreement see the collaboration playing out over a three-year period, with Novartis holding the exclusive rights to zinc finger protein transcription factors (ZFP-TFs) targeting the three undisclosed genes at the center of the deal.

In addition, Novartis will have the option to license Sangamo proprietary AAVs delivery technology.

In terms of development, Sangamo will remain in the lead of research and associated manufacturing activities, though with funding from Novartis, and the latter company will perform supplementary research activities, before stepping in to take the lead in regulatory and commercial activities later in development.

Jay Bradner, president of the Novartis Institutes for BioMedical Research, said, The goal [of the partnership] is to create new gene regulation therapies that act at the genomic level, moving us beyond the symptom-focused treatments of today and toward therapies that can address some of the most challenging neurodevelopmental disorders.

Gene therapy has become a focus area for Novartis over the last few years, as the company invests into its cell and gene manufacturing capabilities and opens new treatment avenues for patients with genetic conditions.

Alongside these new developments, Novartis has also called for rethink of how healthcare is costed and what the value such treatments provide to society are, a debate that has come to the fore as the issue of the pricing of such therapies has arisen.

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Sangamo and Novartis partner on gene therapies for autism - BioPharma-Reporter.com

Research shows promising results for LHON gene therapy – Ophthalmology Times

This article was reviewed by Alvin Luk, PhD, MBA, CCRA

Results from two investigator-initiated studies demonstrate that a single intravitreal injection of rAAV2-ND4 (Neurophth Therapeutics Inc.) is associated with long-term safety and durable efficacy for improving vision in patients with Leber hereditary optic neuropathy (LHON), according to Alvin Luk, PhD, MBA, CCRA.

The research is comprised of a single center study including 9 patients followed for 75 to 90 months and a multicenter, international trial including 159 patients followed for up to 12 months, with observation ongoing.

Related: TANGO: Helping target genes produce more protein

Across the entire cohort, there were no serious or severe adverse events, and efficacy data showed that a majority of patients benefited with significant and sustained improvement in BCVA.

LHON is a rare inherited visual disorder that leads to bilateral vision loss and for which there is currently no effective treatment, said Luk. These 2 studies contain the largest and longest follow-up of patients treated withthis gene therapy. Based on the results, we are very excited about its potential impact for restoring vision and greater independence for patients with LHON.

Luk, the CEO at Neurophth Therapeutics Inc., noted gene therapy also is offering a positive impact on treatment.

The gene therapy restores function of the mitochondrial respiratory chain in retinal ganglion cells by delivering the NADH ubiquinone oxidoreductase subunit 4 (ND4) gene.

A series of preclinical studies confirmed that the intravitreal treatment resulted in targeted gene delivery and provided evidence of its safety.

Related: Gene therapy zeroes in as LHON treatment

Importantly, the confocal microscopy analysis confirms that rAAV2-ND4 reaches the targeted areas in the eyes where the pathological changes of LHON occur, Luk said. Therefore, providing support to the hypothesis that treatment with rAAV2-ND4 may alleviate the underlying cause of the vision loss in patients with LHON.

The clinical studies of rAAV2-ND4 gene therapy reported by Luk have been led by Bin Li, MD, PhD, Tongji Hospital, Huazhong University of Science and Technology, Wuhan, China.

The first study was initiated in 2011. Known as SEE4 LHON (NCT01267422), it included 3 patients aged younger than 12 years who received 0.5 x 1010 vector genome (vg)/eye and six patients aged older than 12 years who were treated with a dose of 1.0 x 1010vg/eye.

According to investigators, the injections were given into 1 eye in an outpatient procedure and had a volume of 0.05 mL.

Although planned follow-up was initially for 12 months, 8 of the 9 patients have continued follow-up until today, Luk said.

Related: Study targets ocular damage from chronic intravitreal injections

According to Luk, there have been no late toxicities noted nor any abnormalities in intraocular pressure (IOP) in extensive laboratory testing, which includes assessments of hepatic, renal, and immune function.

Efficacy is being evaluated with measurement of the logarithm of the minimum angle of resolution (logMAR) in best-corrected visual acuity (BCVA).

Luk pointed out that at 3 months post-injection, 8 patients (89%) demonstrated improvement from baseline, and 6 of 9 patients (67%) maintained improvement at 3 years.

They also noted that the BCVA response was maintained at month 70 by 5 of the 6 responders who achieved a mean BCVA gain of 0.68 logMAR.

Patients in this study also benefited with some improvement in the untreated eye, Luk said. This kind of bilateral response was also seen in the second larger trial of this therapy and is consistent with observations by other groups working on gene therapy for this inherited eye disease.

Based on the encouraging results of SEE4LHON, a larger scale study named 4-HOPE was launched in 2017.

Related: Research targets precision dosing for gene, cell therapy

All patients were treated at three investigational sites in China and 10 patients are continuing follow-up at their local centers in Argentina.

Luk pointed out that the study enrolled 159 patients aged 6 years or older who received a unilateral injection with 1.0 x 1010 vg/eye.

The safety review showed there were no drug-related adverse events. Ocular hypertension was the most common adverse event that patients experienced, but it is related to the course of oral steroid treatment that is given in conjunction with the injection, Luk explained.

The IOP elevations are generally mild and resolve spontaneously once the steroid treatment is ended, he said.

Of the 159 enrolled patients, 106 had data available from a 12-month follow-up visit. Of the 106 patients, Luk noted that 63% showed an improvement from baseline BCVA with an average gain of 0.3 logMAR.

Related: Greater IOP-lowering with iStent inject

It is important to point out that the patients enrolled in this study represent a heterogenous group with a wide range of ages, time since diagnosis, and baseline BCVA values, Luk concluded. We would not be surprised to see even better efficacy results in a cohort enrolled using narrower inclusion criteria.

Luk noted some of the patients in the study also benefited with bilateral BCVA improvement.

Taking improvements ofinjected and noninjected eyes into consideration from baseline to 12 months post treatment, 43.7% patients who classified as legally blind by World Health Organizationcriteria ( > 1.3 logMAR) were recovered to low (> 0.5 to 1.3 logMAR) or normal ( 0.5 logMAR) vision.Read more by Lynda Charters

--

Alvin Luk, PhD, MBA, CCRAe:alvin.luk@neuropth.comLuk is an employee of Neurophth Therapeutics Inc, but has no other relevant financial interests to disclose.

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Research shows promising results for LHON gene therapy - Ophthalmology Times

Global Gene Therapy in Oncology Market 2020 Growth Statistics, New Opportunities, Competitive Outloo – PharmiWeb.com

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The report delivers brief information on the competitors and the specific growth opportunities with key market drivers. Complete market analysis is given by segmenting the report by companies, region, type, and applications in the report. Top players also analyzed by splitting the globalGene Therapy in Oncologymarket by product type and applications/end industries. The overall report encompasses many aspects of the industry like market size, market status, market trends, and forecast. Additionally, development trends, competitive landscape analysis, and key regions development status has been demonstrated. It also focuses on a product analysis, application analysis, competitive strategies, and strategies impacting the industry. An expert and in-depth analysis of key business trends and future market development prospects, key drivers and restraints, profiles of major market players, and forecasting for 2020 to 2025 time-period has been given.

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Geographically, this market report studies the following key geographical regions:North America (United States, Canada and Mexico), Europe (Germany, France, United Kingdom, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), Middle East & Africa (Saudi Arabia, UAE, Egypt and South Africa)

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Global Gene Therapy in Oncology Market 2020 Growth Statistics, New Opportunities, Competitive Outloo - PharmiWeb.com

The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 – Yahoo Finance

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included in this Global Cell and Gene Therapy Market Report. The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.

New York, Aug. 04, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell & Gene Therapy Market - Global Outlook and Forecast 2020-2025" - https://www.reportlinker.com/p05827567/?utm_source=GNW 90% during the period 20192025.

The global cell and gene therapy market is one of the fastest-growing segments in the regenerative medicine market. The market is expected to grow at a faster pace during the forecast period. The demand can be attributed to the growing prevalence of several chronic diseases such as cancer, cartilage related problems, wounds, diabetic foot ulcer, genetic disorders, and other rare diseases across the globe. The prevalence of cancer and diabetes is increasing in the global population, which is influencing the growth of the market. There is a large unmet need in the treatment available, which is filled by cell and gene therapies. The market is growing due to the increased availability of funding from various public and private institutions. Besides, there is increased support from regulatory bodies for product approval. Several governments are creating awareness of cell and gene therapies in the population.

The following factors are likely to contribute to the growth of the cell and gene therapy market during the forecast period: Increase in Strategic Acquisition Activities Increased Funding for Cell & Gene Therapy Products Expanding Applications of Cell and Gene Therapies Increased in the Patient Pool

The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2019?2025. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market. Cell And Gene Therapy Market Segmentation The global cell and gene therapy market research report includes a detailed segmentation by product, disease, end-user, and geography. In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and gene therapy market. The segment is expected to grow at a steady rate during the forecast period due to the increase in the target population and the rise in the number of countries preferring cell therapies in their patients. Increased therapeutic benefits are attracting several countries to invest in this technology and conduct a high number of clinical trials. However, the lack of advanced infrastructure in developing countries is hindering the growth of the segment.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

Segmentation by Product Cell Therapy Gene Therapy Segmentation by Disease Dermatology Musculoskeletal Oncology Genetic Disorders Others Segmentation by End-user Hospitality Cancer Care Centers Wound Care Centers Ambulatory Surgical Centers Others

INSIGHTS BY GEOGRAPHY In 2019, North America accounted for a share of over 60% of the global cell and gene therapy market. There are more than 530 regenerative medicine companies, including cell and gene therapy manufacturing developers. The number of products approved in North America grew significantly in 2019, with developers filed for marketing authorization for 10+ regenerative medicines, many of which we expect to be approved in 2020. Within the next 12 years, the number of approved gene therapies is expected to double. The US and Canada are the major contributors to the cell and gene therapy market in North America. Regulatory bodies are supporting several investigational products, fast track approvals, RMAT designation for the faster approval of the product into the market. The alliance for regenerative medicine and Medicare and Medicaid is working together to bring the structured reimbursement channels for cell and gene therapies.

Segmentation by Geography North America o US o Canada Europe o UK o Germany o France o Spain o Italy APAC o China o Japan o South Korea o Australia o India Latin America o Brazil o Mexico Middle East & Africa o Saudi Arabia o Turkey o South Africa o UAE

INSIGHTS BY VENDORS The global cell and gene therapy market is highly dynamic and characterized by the presence of several global, regional, and local vendors offering a wide range of therapies. Dendreon, Gilead Sciences, Novartis, Organogenesis, Osiris Therapeutics, Vericel, Amgen, and Spark Therapeutics are the leading players in the market with significant shares. Vendors such as NuVasive, APAC Biotech, Nipro, Orthocell, bluebird bio, J-TEC, and Terumo are the other prominent players in the market with a presence, especially in the cell therapy market. Most leading players are focusing on implementing strategies such as product launches and approvals, marketing and promotional activities, acquisitions, increased R&D investments, and strengthening their distribution networks to enhance their share and presence in the market.

Prominent Vendors Gilead Sciences Spark Therapeutics Novartis Organogenesis Amgen Osiris Therapeutics Dendreon Vericel

Other Prominent Vendors Anterogen Tego Sciences Japan Tissue Engineering JCR Pharmaceuticals Medipost MolMed AVITA Medical CollPlant Biosolution Stempeutics Research Kolon Tissue Gene Orchard Therapeutics Sibiono GeneTech NuVasive Corestem Pharmicell Shanghai Sunway Biotech RMS Regenerative Medical System Takeda Pharmaceutical Company CHIESI Farmaceutici CO.DON AnGes GC Pharma Human Stem Cells Institute JW CreaGene APAC Biotech Nipro Terumo Orthocell bluebird bio

Key Questions Answered 1. What is the cell and gene therapy market size and growth rate during the forecast period? 2. What are the factors impacting the growth of the cell and gene therapy market share? 3. How is the growth of the healthcare segment affecting the growth of the cell and gene therapy market? 4. Who are the leading vendors in the cell and gene therapy market, and what are their market shares? 5. Which product type/ end-user type/region is generating the largest revenue in the Asia Pacific region?Read the full report: https://www.reportlinker.com/p05827567/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 - Yahoo Finance

Detailed Information on Gene Therapy Market 2020 | Covid-19 Impact Analysis | Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio,…

Global Gene Therapy Market report forecast to 2026 investigate the Impact of COVID-19 on Industry further market size, manufactures, types, applications and key regions like North America, Europe, Asia Pacific, Central & South America and Middle East & Africa, focuses on the consumption of Gene Therapy in these regions. This report also studies the global Gene Therapy market share, competition landscape, status share, growth rate, future trends, market drivers, opportunities and challenges, sales channels and distributors.

COVID-19 can affect the global economy in 3 main ways: by directly affecting production and demand, by creating supply chain and market disturbance, and by its financial impact on firms and financial markets.

Get Sample copy along with Few Details- https://www.worldwidemarketreports.com/sample/364919

Leading Players from the market are covered in this report- Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon

Impact of Covid-19 on Gene Therapy Industry 2020

Gene Therapy Market report analyses the impact of Coronavirus (COVID-19) on the Gene Therapy industry. Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost 180+ countries around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Gene Therapy market in 2020.

The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans and quarantines; restaurants closed; all indoor events restricted; emergency declared in many countries; massive slowing of the supply chain; stock market unpredictability; falling business assurance, growing panic among the population, and uncertainty about future.

Download Sample TOC to understand the CORONA Virus/COVID19 impact and be smart in redefining business strategies-https://www.worldwidemarketreports.com/sample/364919

Market Segments:

Based on Types, the Gene Therapy Market is Classsified as Ex vivo, In vivo

Based on Application, this report focuses on the status and outlook for major applications/end users, consumption (sales), market share and growth rate for each application, including Cancer Diseases, Monogenic Diseases, Infectious Diseases, Cardiovascular Diseases, Others

Gene Therapy Market Report Provides Comprehensive Analysis as Following:

Study on Table of Contents:

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Detailed Information on Gene Therapy Market 2020 | Covid-19 Impact Analysis | Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio,...

Global Cell & Gene Therapy Market Outlook and Forecast 2020-2025 – GlobeNewswire

Dublin, Aug. 07, 2020 (GLOBE NEWSWIRE) -- The "Cell & Gene Therapy Market - Global Outlook and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included

The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2019-2025. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Key Questions Answered

1. What is the cell and gene therapy market size and growth rate during the forecast period?2. What are the factors impacting the growth of the cell and gene therapy market share?3. How is the growth of the healthcare segment affecting the growth of the cell and gene therapy market?4. Who are the leading vendors in the cell and gene therapy market, and what are their market shares?5. Which product type/ end-user type/region is generating the largest revenue in the Asia-Pacific region?

The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.9% during the period 2019-2025

The global cell and gene therapy market is one of the fastest-growing segments in the regenerative medicine market. The market is expected to grow at a faster pace during the forecast period. The demand can be attributed to the growing prevalence of several chronic diseases such as cancer, cartilage related problems, wounds, diabetic foot ulcer, genetic disorders, and other rare diseases across the globe.

The prevalence of cancer and diabetes is increasing in the global population, which is influencing the growth of the market. There is a large unmet need in the treatment available, which is filled by cell and gene therapies. The market is growing due to the increased availability of funding from various public and private institutions. Besides, there is increased support from regulatory bodies for product approval. Several governments are creating awareness of cell and gene therapies in the population.

Cell and Gene Therapy Market Segmentation

The global cell and gene therapy market research report includes a detailed segmentation by product, disease, end-user, and geography.

In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and gene therapy market. The segment is expected to grow at a steady rate during the forecast period due to the increase in the target population and the rise in the number of countries preferring cell therapies in their patients. Increased therapeutic benefits are attracting several countries to invest in this technology and conduct a high number of clinical trials. However, the lack of advanced infrastructure in developing countries is hindering the growth of the segment.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

Segmentation by Product

Segmentation by Disease

Segmentation by End-user

Insights by Geography

In 2019, North America accounted for a share of over 60% of the global cell and gene therapy market. There are more than 530 regenerative medicine companies, including cell and gene therapy manufacturing developers. The number of products approved in North America grew significantly in 2019, with developers filed for marketing authorization for 10+ regenerative medicines, many of which we expect to be approved in 2020. Within the next 1-2 years, the number of approved gene therapies is expected to double.

The US and Canada are the major contributors to the cell and gene therapy market in North America. Regulatory bodies are supporting several investigational products, fast track approvals, RMAT designation for the faster approval of the product into the market. The alliance for regenerative medicine and Medicare and Medicaid is working together to bring the structured reimbursement channels for cell and gene therapies.

Segmentation by Geography

Insights by Vendors

The global cell and gene therapy market is highly dynamic and characterized by the presence of several global, regional, and local vendors offering a wide range of therapies. Dendreon, Gilead Sciences, Novartis, Organogenesis, Osiris Therapeutics, Vericel, Amgen, and Spark Therapeutics are the leading players in the market with significant shares.

Vendors such as NuVasive, APAC Biotech, Nipro, Orthocell, bluebird bio, J-TEC, and Terumo are the other prominent players in the market with a presence, especially in the cell therapy market. Most leading players are focusing on implementing strategies such as product launches and approvals, marketing and promotional activities, acquisitions, increased R&D investments, and strengthening their distribution networks to enhance their share and presence in the market.

Prominent Vendors

Other Prominent Vendors

Market Dynamics

Opportunities & Trends

Growth Enablers

Growth Restraints

For more information about this report visit https://www.researchandmarkets.com/r/1tjzij

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global Cell & Gene Therapy Market Outlook and Forecast 2020-2025 - GlobeNewswire

Coronavirus Impact Editon of Gene Therapy for Mucopolysaccharidosis Market Research Study 2020 Future Development, Top Manufacturers, Technological…

Rising number of corona virus cases has impacted numerous lives and led to numerous fatalities, and has affected the overall economic structure globally. The Gene Therapy for Mucopolysaccharidosis has analyzed and published the latest report on the global Gene Therapy for Mucopolysaccharidosis market. Change in the market has affected the global platform. Along with the Gene Therapy for Mucopolysaccharidosis market, numerous other markets are also facing similar situations. This has led to the downfall of numerous businesses, because of the widespread increase of the number of cases across the globe.href=mailto:nicolas.shaw@cognitivemarketresearch.com>nicolas.shaw@cognitivemarketresearch.com or call us on +1-312-376-8303.

Request Free Sample Copy of Gene Therapy for Mucopolysaccharidosis Market Research Report@ https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-mucopolysaccharidosis-market-report

The major players in the Gene Therapy for Mucopolysaccharidosis market are Sangamo Therapeutics, Swedish Orphan Biovitrum, uniQure . Some of the players have adopted new strategies to sustain their position in the Gene Therapy for Mucopolysaccharidosis market. A detailed research study is done on the each of the segments, and is provided in Gene Therapy for Mucopolysaccharidosis market report. Based on the performance of the Gene Therapy for Mucopolysaccharidosis market in various regions, a detailed study of the Gene Therapy for Mucopolysaccharidosis market is also analyzed and covered in the study.

Report Scope:Some of the key types analyzed in this report are as follows: Intravenous, ICV, Intracerebral, Intracisternal

Some of the key applications as follow: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III A, Mucopolysaccharidosis III B

Following are the major key players: Sangamo Therapeutics, Swedish Orphan Biovitrum, uniQure

An in-depth analysis of the Gene Therapy for Mucopolysaccharidosis market is covered and included in the research study. The study covers an updated and a detailed analysis of the Gene Therapy for Mucopolysaccharidosis market. It also provides the statistical information of the Gene Therapy for Mucopolysaccharidosis market. The study of the report consists of the detailed definition of the market or the overview of the Gene Therapy for Mucopolysaccharidosis market. Furthermore, it also provides detailed information for the target audience dealing with or operating in this market is explained in the next section of the report.

Read Detailed Index of full Research Study @: https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-mucopolysaccharidosis-market-report#download_report

The report also provides detailed information on the research methodologies, which are used for the analysis of the Gene Therapy for Mucopolysaccharidosis market. The methods are covered in detail in this section of the report. For the analysis of the market, several tools are used for the extraction of the market numbers. Among the several tools, primary and secondary research studies were also incorporated for the research study. These were further analyzed and validated by the market experts, to increase precision and make the data more reliable.

Moreover, the report also highlights and provides a detailed analysis of the drivers, restrains, opportunities, and challenges of the Gene Therapy for Mucopolysaccharidosis market. This section of Gene Therapy for Mucopolysaccharidosis market also covers the updated information, in accordance with the present situation of the market.

According to the estimation and the analysis of the market, the Gene Therapy for Mucopolysaccharidosis market is likely to have some major changes in the estimated forecasts period. Moreover, these changes can be attributed to the changes due to economic and trading conditions across the globe. Moreover, several market players operating in the Gene Therapy for Mucopolysaccharidosis market will have to strategically change their business strategies in order to survive in the market.

If Any Inquiry of Gene Therapy for Mucopolysaccharidosis Report @: https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-mucopolysaccharidosis-market-report#download_report

Reasons for Buying this Gene Therapy for Mucopolysaccharidosis Report1. Gene Therapy for Mucopolysaccharidosis market advertise report helps with understanding the Basic product segments alongside likewise their potential future.2. This global Gene Therapy for Mucopolysaccharidosis report offers pin-point evaluation for changing competitive dynamics.3. The Gene Therapy for Mucopolysaccharidosis market supplies pin point analysis of changing competition dynamics and keeps you in front of competitors4. Original images and illustrated a SWOT evaluation of large segments supplied by the Gene Therapy for Mucopolysaccharidosis market.5. This report supplies a forward-looking perspective on different driving factors or controlling Gene Therapy for Mucopolysaccharidosis market gain.6. This report assists to make wise business choices using whole insights of the Gene Therapy for Mucopolysaccharidosis and also from creating a comprehensive evaluation of market sections.Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

*If you have any special requirements, please let us know and we will offer you the report as you want Click Here>Download Customized Sample Report of Gene Therapy for Mucopolysaccharidosis Market Report 2020 (COVID-19 Impact Analysis Updated Edition May 2020)

About Us: Cognitive Market Research is one of the finest and most efficient Market Research and Consulting firm. The company strives to provide research studies which include syndicate research, customized research, round the clock assistance service, monthly subscription services, and consulting services to our clients. We focus on making sure that based on our reports, our clients are enabled to make most vital business decisions in easiest and yet effective way. Hence, we are committed to delivering them outcomes from market intelligence studies which are based on relevant and fact-based research across the global market.

Contact Us: +1-312-376-8303Email: nicolas.shaw@cognitivemarketresearch.comWeb: https://www.cognitivemarketresearch.com

**********Download the Entire Report*************************************************https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-mucopolysaccharidosis-market-report

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Coronavirus Impact Editon of Gene Therapy for Mucopolysaccharidosis Market Research Study 2020 Future Development, Top Manufacturers, Technological...

The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 – Press Release – Digital…

New York, Aug. 04, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell & Gene Therapy Market - Global Outlook and Forecast 2020-2025" - https://www.reportlinker.com/p05827567/?utm_source=GNW 90% during the period 20192025.

The global cell and gene therapy market is one of the fastest-growing segments in the regenerative medicine market. The market is expected to grow at a faster pace during the forecast period. The demand can be attributed to the growing prevalence of several chronic diseases such as cancer, cartilage related problems, wounds, diabetic foot ulcer, genetic disorders, and other rare diseases across the globe. The prevalence of cancer and diabetes is increasing in the global population, which is influencing the growth of the market. There is a large unmet need in the treatment available, which is filled by cell and gene therapies. The market is growing due to the increased availability of funding from various public and private institutions. Besides, there is increased support from regulatory bodies for product approval. Several governments are creating awareness of cell and gene therapies in the population.

The following factors are likely to contribute to the growth of the cell and gene therapy market during the forecast period: Increase in Strategic Acquisition Activities Increased Funding for Cell & Gene Therapy Products Expanding Applications of Cell and Gene Therapies Increased in the Patient Pool

The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2019?2025. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market. Cell And Gene Therapy Market Segmentation The global cell and gene therapy market research report includes a detailed segmentation by product, disease, end-user, and geography. In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and gene therapy market. The segment is expected to grow at a steady rate during the forecast period due to the increase in the target population and the rise in the number of countries preferring cell therapies in their patients. Increased therapeutic benefits are attracting several countries to invest in this technology and conduct a high number of clinical trials. However, the lack of advanced infrastructure in developing countries is hindering the growth of the segment.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

Segmentation by Product Cell Therapy Gene Therapy Segmentation by Disease Dermatology Musculoskeletal Oncology Genetic Disorders Others Segmentation by End-user Hospitality Cancer Care Centers Wound Care Centers Ambulatory Surgical Centers Others

INSIGHTS BY GEOGRAPHY In 2019, North America accounted for a share of over 60% of the global cell and gene therapy market. There are more than 530 regenerative medicine companies, including cell and gene therapy manufacturing developers. The number of products approved in North America grew significantly in 2019, with developers filed for marketing authorization for 10+ regenerative medicines, many of which we expect to be approved in 2020. Within the next 12 years, the number of approved gene therapies is expected to double. The US and Canada are the major contributors to the cell and gene therapy market in North America. Regulatory bodies are supporting several investigational products, fast track approvals, RMAT designation for the faster approval of the product into the market. The alliance for regenerative medicine and Medicare and Medicaid is working together to bring the structured reimbursement channels for cell and gene therapies.

Segmentation by Geography North America o US o Canada Europe o UK o Germany o France o Spain o Italy APAC o China o Japan o South Korea o Australia o India Latin America o Brazil o Mexico Middle East & Africa o Saudi Arabia o Turkey o South Africa o UAE

INSIGHTS BY VENDORS The global cell and gene therapy market is highly dynamic and characterized by the presence of several global, regional, and local vendors offering a wide range of therapies. Dendreon, Gilead Sciences, Novartis, Organogenesis, Osiris Therapeutics, Vericel, Amgen, and Spark Therapeutics are the leading players in the market with significant shares. Vendors such as NuVasive, APAC Biotech, Nipro, Orthocell, bluebird bio, J-TEC, and Terumo are the other prominent players in the market with a presence, especially in the cell therapy market. Most leading players are focusing on implementing strategies such as product launches and approvals, marketing and promotional activities, acquisitions, increased R&D investments, and strengthening their distribution networks to enhance their share and presence in the market.

Prominent Vendors Gilead Sciences Spark Therapeutics Novartis Organogenesis Amgen Osiris Therapeutics Dendreon Vericel

Other Prominent Vendors Anterogen Tego Sciences Japan Tissue Engineering JCR Pharmaceuticals Medipost MolMed AVITA Medical CollPlant Biosolution Stempeutics Research Kolon Tissue Gene Orchard Therapeutics Sibiono GeneTech NuVasive Corestem Pharmicell Shanghai Sunway Biotech RMS Regenerative Medical System Takeda Pharmaceutical Company CHIESI Farmaceutici CO.DON AnGes GC Pharma Human Stem Cells Institute JW CreaGene APAC Biotech Nipro Terumo Orthocell bluebird bio

Key Questions Answered 1. What is the cell and gene therapy market size and growth rate during the forecast period? 2. What are the factors impacting the growth of the cell and gene therapy market share? 3. How is the growth of the healthcare segment affecting the growth of the cell and gene therapy market? 4. Who are the leading vendors in the cell and gene therapy market, and what are their market shares? 5. Which product type/ end-user type/region is generating the largest revenue in the Asia Pacific region?Read the full report: https://www.reportlinker.com/p05827567/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 - Press Release - Digital...

Orgenesis Second Quarter 2020 Revenue Increases 55% Reflecting Progress of POCare Platform – GlobeNewswire

Provides update on COVID-19 therapeutic programs

Reports cash and cash equivalents of $97.5 million as of June 30, 2020

GERMANTOWN, Md., Aug. 07, 2020 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS) (Orgenesis or the Company), a pioneering, global biotech company committed to accelerating commercialization and transforming the delivery of cell and gene therapies (CGTs), today provided a business update for the second quarter ended June 30, 2020.

Vered Caplan, CEO of Orgenesis, stated, We continue to implement our Point of Care (POCare) cell and gene therapy strategy, including the expansion of our network, as evidenced by the expected revenue growth. Revenue for the second quarter of 2020 increased 55% to $1.7 million compared to $1.1 million for the second quarter of 2019. We have also maintained a solid balance sheet with over $97.5 million of cash and cash equivalents as of June 30, 2020. Our true progress can be seen in the advancement of our POCare Therapeutics pipeline, including immuno-oncology, metabolic, and anti-viral therapies. Our mission is to make these therapies available to large numbers of patients at reduced costs using the point-of-care model. Our POCare Network also continues to grow via new partnerships with leading hospitals and research institutes around the world.

We have made progress towards our goal of adapting our cell-based and antiviral technologies to address the COVID-19 pandemic. First, we are focused on advancing our cell-based vaccine platform in order to target COVID-19, as well as other potential existing and emerging viral diseases. Second, we recently launched our BioShield Program, which is designed to potentially accelerate discovery and set up a first line of defense against the spread of viral pathogens, such as COVID-19. And finally, we are engaging with industry partners for the development of Ranpirnase for the potential treatment of COVID-19, as well as other viruses.

The Company remained active through the second quarter and into the third quarter of 2020, reporting the following recent advances:

The Companys complete financial results are available in the Companys Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 6, 2020, which is available at http://www.sec.gov and on the Companys website.

About Orgenesis

Orgenesis is a pioneering global biotech company that is working to unlock the full potential of personalized therapies and closed processing systems through its Cell & Gene Therapy Biotech Platform, with the ultimate aim of providing life changing treatments at the Point of Care to a large number of patients at lower costs. The Platform consists of: (a) POCare Therapeutics, a pipeline of licensed cell and gene therapies (CGTs), and proprietary scientific knowhow; (b) POCare Technologies, a suite of proprietary and in-licensed technologies which are engineered to create customized processing systems for affordable point of care therapies; and (c) POCare Network, a collaborative, international ecosystem of leading research institutes and hospitals committed to clinical development and supply of CGTs at the point of care. By combining science, technologies and a collaborative network, Orgenesis is able to identify the most promising new therapies and provide a pathway for them to reach patients more quickly, more efficiently and at scale, thereby unlocking the power of cell and gene therapy for all. Additional information is available at: http://www.orgenesis.com.

Notice Regarding Forward-Looking Statements

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, the risk that the acquisition of Tamirs assets will not be successfully integrated with our technologies or that the potential benefits of the acquisition will not be realized; our ability to further develop ranpirnase; our reliance on, and our ability to grow, our point-of-care cell therapy platform; our ability to effectively use the net proceeds from the sale of Masthercell; our ability to achieve and maintain overall profitability; the development of our POCare strategy; the sufficiency of working capital to realize our business plans; the development of our transdifferentiation technology as therapeutic treatment for diabetes which could, if successful, be a cure for Type 1 Diabetes; our technology not functioning as expected; our ability to retain key employees; our ability to satisfy the rigorous regulatory requirements for new procedures; our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading "RISK FACTORS" in Item 1A of our Annual Report on Form 10-K for the fiscal year ended December 31, 2019, and in our other filings with the Securities and Exchange Commission. We undertake no obligation to revise or update any forward-looking statement for any reason.

Contact for Orgenesis:David WaldmanCrescendo Communications, LLCTel: 212-671-1021ORGS@crescendo-ir.com

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Regulatory Focus, July issue: Cell and gene therapy – Regulatory Focus

Feature articles during July focused on global regulatory strategy for cell and gene therapy, with articles on US and EU regulations and guidances and the development and manufacture of the therapies. Also included were articles on recasting the corrective and preventive action (CAPA) process as a continuous improvement process, a military-civilian perspective on real-world evidence (RWE) to support regulatory decision making, and regulatory reporting in multinational trials during COVID-19.Advanced therapy medicinal products (ATMPs), including cell therapies, gene therapies, and tissue-engineered products, are highly complex treatments that differ from traditional medicines, both in how they are made and administered and in the benefits they may provide. Regulations for these products were established relatively recently and are still evolving in many jurisdictions globally. The novelty of these products, the inherent complexities of cell and gene therapy products, and the lack of experience with such products pose many challenges for developers.In part one of this two-part series, this months expert authors address these challenges and offer hands-on, practical advice and guidance on regulation and production of ATMPs. If there is one clear, take-home message to developers, it is that early and frequent collaboration with regulatory agencies, during both the approval and development phases, is paramount. It saves time and money, and it reduces the risk of a negative impact on the trajectory of a clinical trial. Part 2 of the series will cover upstream manufacturing and process controls for biologics, the ATMP regulatory landscape in China, EU GMP requirements for autologous cell therapies and parenteral biologics, and regulatory challenges and opportunities in the US.Regulations and guidancesThe regenerative medicine advanced therapy (RMAT) field has the potential to provide profound benefits to patients with serious diseases and disorders, and the RMAT designation is helping drive these transformative technologies to market. In Update on RMAT designations, William K. Sietsema and Janet Lynch Lambert discuss the scope and purpose of the special designation for RMATs created by the passage of the 21st Century Cures Act and provide a tally of products that have received the special designation to date. However, while the promise of regenerative medicines to cure disease is propelling the field at a rapid pace, developing these therapies requires a rigorous, carefully planned approach to ensure a seamless progression to regulatory approval and commercial success.Siegfried Schmitt expands on that point about carefully navigating the complex and nuanced regulatory environment in two articles on US and EU regulations for RMATs and ATMPs, respectively. In US regulations for regenerative medicine advanced therapies, he provides a user-friendly, quick-access list of RMAT-related regulations and guidances. The article includes a useful introduction to the application process and descriptions of the features and criteria for various expedited program options, including breakthrough therapy, fast track, advanced approval, and priority review.In Regulation of advanced therapy medicinal products in the EU, Schmitt explains some of the terminology relating to ATMPs before documenting the key EU regulations and guidances for each therapy type. He concludes with discussions on marketing authorization, accelerated regulatory pathways, and market access. Again, he urges companies and developers to engage with the regulatory agencies early and often throughout the approval process and to seek external regulatory support, especially if the developer has limited in-house regulatory resources.Development pathways and manufacturingTwo articles shift the focus from the regulatory landscape to development and manufacturing pathways. The drug manufacturing facility environment presents one of the major sources of potential contaminants in the final biologic drug product, so it is critical to design facilities with cleanroom environmental controls and monitoring that adhere to the highest standards of current good manufacturing practice (cGMP) quality guidelines, write Mo Heidaran and colleagues. In Designing a biologics manufacturing facility: Early planning for success, the authors lay out the planning steps for compliance with cGMP to readiness for chemistry, manufacturing, and controls (CMC). The authors warn that the pressure to reduce time to market put considerable stress on all aspects of commercial operations and commercial-scale manufacturing development, so yet again, early tactical and strategic planning essential.In Advanced therapies: Trip hazards along the development pathway, Kirsten Messmer and Richard Dennett focus on the challenges and complexities of ferrying advanced therapies along the developmental pathway, which they call the trip. They examine the importance of some of the fundamental building blocks for the development program and highlight some commonly encountered challenges, or trip hazards, for cell and gene therapies. The suggest developers establish sound technical and regulatory strategies to better anticipate and avoid the trip hazards, which could prove costly, both in time and money, and have a negative impact the overall clinical study program.CAPA, RWE, and COVID-19Todays CAPA process has become highly focused on compliance, which has manufacturers struggling to determine which issues require a structured CAPA process and which can be resolved in alternative ways, writes Kathryn Merrill in Recasting CAPA as a continuous improvement process. Merrill summarizes a white paper developed by the Medical Device Innovation Consortium, in which the CAPA process is recast as a continuous improvement process for driving higher product quality and improved patient safety. It is intended to enable organizations make a greater number of improvements more quickly, and over time, which will have a favorable impact on product quality in the field.During the Afghanistan and Iraq wars, the US Military Health System used an approach known as focused empiricism to develop new approaches for casualty care. In doing so, it implemented real-world data (RWD) and RWE into a system of performance improvement and product development to achieve historic rates of survival, write Todd E. Rasmussen, a colonel in the US Air force, and Brian J. Young. In A military-civilian perspective on real-world evidence to support regulatory decision making, the authors summarize the framework promoting the collection and analysis of RWD in the healthcare system and describe a new era of collaboration between the US Department of Defense and the FDA, within the context of a new Public Law 115-92, to coordinate on the delivery of military-relevant medical products. The article reviews the FDA evidentiary standards for medical product approval and gives examples of how RWE can help meet those standards.COVID-19 continues to disrupt and redefine the regulatory process and activity. In Managing uncertainty: Regulatory reporting in multinational trials during COVID-19, Ioana Ionita discusses regulatory reporting challenges for multinational clinical trials during the pandemic, as well as the challenge of assessing what is reportable and how to submit COVID-19 risk mitigation measures. She offers real-world experience on how she and her colleagues stopped and restarted recruitment in ongoing multinational clinical trials, and how those actions were reported globally. Ionita concludes that close collaboration between sponsors, CROs, local affiliates, investigational sites, and health authorities is important in choosing strategies under challenging circumstances and when no precedent applies.Whats coming in August?Articles during August will focus on global clinical trials and clinical trial applications. Despite ICH efforts to produce guidelines for the development of drugs and biologics and to standardize the format of marketing applications, there remain considerable differences among countries in the format of clinical trial applications and health authority review processes. This collection of articles will address these divergent formats and processes and provide options for navigating the regulatory aspects of clinical trials. Look for these topics and more throughout August at http://www.raps.org.October call for articlesFor October, Regulatory Focus will look at the regulatory toolboxthe tools regulatory professionals need and where to find them, with an emphasis on websites, guidances, meeting minutes and FDA correspondence, including warning letters, enforcement actions, 483s, and notices. Articles will discuss how to interpret the meaning behind regulatory agency actions and available options for documentation. The submission deadline for articles is 1 September 2020. To contribute to the October issue or suggest a topic, contact Rene Matthews at rmatthews@raps.org.Citation Matthews R. Regulatory Focus, July issue: Cell and gene therapy. Regulatory Focus. July 2020. Regulatory Affairs Professionals Society.

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G-CON PODs Selected for Expression Therapeutics’ Gene Therapy Manufacturing Facility – PR Web

Photo courtesy of Expression Therapeutics

COLLEGE STATION, Texas (PRWEB) August 05, 2020

G-CON Manufacturing, the leader in prefabricated, flexible cleanroom solutions, announced today that it has been selected by Expression Therapeutics to support its cleanroom build-out at its new clinical manufacturing facility in Cincinnati, Ohio. The PODs will provide the cleanroom infrastructure for the production of Expression Therapeutics cell and gene therapies.

For this project, time was a critical factor for Expression Therapeutics. Offsite manufacturing of the cleanroom infrastructure allowed for concurrent preparation of their new host facility, significantly reducing the projects overall timeline. Future expandability was also key, as their POD system is being designed to accommodate Expression Therapeutics cleanroom needs for its next phase. Removable panels incorporated into the POD walls will easily allow integration of additional POD clean space. Since PODs are autonomous and completely assembled offsite, this expansion will result in minimal downtime and disruption to the functioning first phase POD cleanrooms.

Its great to work in an industry where innovation moves so fast and helps people live better lives, said Tim Rasmussen, Sales Engineer at G-CON Manufacturing. Companies like Expression Therapeutics need infrastructure to develop breakthrough therapies, and they need them fast. We are proud to play a part in making patients lives better by delivering state of the art cleanroom solutions more quickly than any other solution on the market.

We decided to utilize advanced pre-built modular cleanrooms from G-CON to accelerate our buildout and commence vector manufacturing this year. With vector GMP manufacturing backlogs today typically exceeding 18 months, we wanted to bring on additional capacity as soon as possible to serve clients said Bill Swaney, Vice President of Manufacturing for Expression Therapeutics.

About G-CON ManufacturingG-CON Manufacturing designs, builds and installs prefabricated G-CON POD cleanrooms. G-CONs POD portfolio provides cleanrooms in a number of dimensions for a variety of uses, from laboratory environments to personalized medicine and production process platforms. G-CON POD cleanroom units surpass traditional cleanroom structures in scalability, mobility and the possibility of repurposing the PODs once the production process reaches its lifecycle end. For more information, please visit G-CONs website at http://www.gconbio.com.

G-CON Manufacturing... BUILDING FOR LIFE

About Expression TherapeuticsExpression Therapeutics is a biotechnology company based in Atlanta and Cincinnati. The current therapeutic pipeline includes advanced gene therapies for hemophilia, neuroblastoma, T-cell leukemia/lymphoma, acute myeloid leukemia (AML), and primary immunodeficiencies such as hemophagocytic lymphohistiocytosis (HLH).

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G-CON PODs Selected for Expression Therapeutics' Gene Therapy Manufacturing Facility - PR Web

CRISPR co-discoverer on the gene editor’s pandemic push – Axios

The coronavirus pandemic is accelerating the development of CRISPR-based tests for detecting disease and highlighting how gene-editing tools might one day fight pandemics, one of its discoverers, Jennifer Doudna, tells Axios.

Why it matters: Testing shortages and backlogs underscore a need for improved mass testing for COVID-19. Diagnostic tests based on CRISPR which Doudna and colleagues identified in 2012, ushering in the "CRISPR revolution" in genome editing are being developed for dengue, Zika and other diseases, but a global pandemic is a proving ground for these tools that hold promise for speed and lower costs.

Driving the news: Last week, the NIH awarded $250 million for the development of COVID-19 diagnostic tests to a handful of companies, including Mammoth Biosciences, which is working on a CRISPR-based test that CEO Trevor Martin says will deliver 200 tests per hour per machine.

The challenge now is "getting it into a format where it can be used easily either in a laboratory or at the point-of-care," like the doctor's office or home, she says.

How it works: Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR, are sequences of genetic code that bacteria naturally use to find and destroy viruses.

That editing ability is viewed as having vast potential for treating disease, a nascent use of CRISPR.

But there's a persistent problem: Getting the sizable CRISPR system through the membranes and to the DNA of the cells that need editing.

And, there are other concerns about off-target editing with currently available enzymes and unknown long-term effects of gene editing directly in the body.

The intrigue: CRISPR could one day be wielded in future pandemics.

Yes, but: That would require sophisticated understanding of how a virus changes and the immune system's complex response to it.

The big picture: Such "genetic vaccination" is a long way off, but it could eliminate having to wait until a virus shows up, make a vaccine to that virus and then vaccinate people, she says.

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Voyager Therapeutics Provides Update on AbbVie Vectorized Antibody Collaborations – GlobeNewswire

CAMBRIDGE, Mass., Aug. 03, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced the termination of its tau and alpha-synuclein vectorized antibody collaborations with AbbVie. Voyager retains full rights to the vectorization technology and certain novel vectorized antibodies developed as part of the collaborations.

Our efforts to harness AAV-based gene therapy to produce antibodies directly in the brain and overcome major limitations with delivery of current biologics across the blood-brain barrier have been highly productive, said Omar Khwaja, M.D., Ph.D., Chief Medical Officer and Head of R&D at Voyager. Through the tau and alpha-synuclein collaborations, we believe we have made considerable progress against targets for neurodegenerative diseases with this novel approach, reinforcing our enthusiasm for its potential to deliver therapeutically efficacious levels of biologics to the brain and central nervous system. We believe our continued work on discovery and design of novel AAV capsids with substantially improved blood-brain barrier penetrance will also considerably broaden the potential of AAV-based gene therapy, including vectorized antibodies or other biologics, for the treatment of severe neurological diseases.

The tau and alpha-synuclein research collaborations were formed in 2018 and 2019, respectively. Under the terms of the collaboration agreements, Voyager received upfront payments to perform research and preclinical development of vectorized antibodies directed against tau and alpha-synuclein. With the conclusion of the collaborations, Voyager has regained full clinical development and commercialization rights to certain product candidates developed within the context of the collaboration for the tau program. Voyager is free to pursue vectorized antibody programs for tau and alpha-synuclein alone or in collaboration with another partner.

Voyager does not anticipate any changes to its cash runway guidance due to the termination of the agreements. As of March 31, 2020, the Company had cash, cash equivalents and marketable debt securities of $250.9 million, which, along with amounts expected to be received for reimbursement of development costs from Neurocrine Biosciences, is expected to be sufficient to meet Voyagers projected operating expenses and capital expenditure requirements into mid-2022.

About Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, Friedreichs ataxia, and other severe neurological diseases. For more information, please visit http://www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, plan, anticipate, believe, estimate, undoubtedly, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the ability of Voyager to maintain research and development activities currently included within the collaboration agreements with AbbVie; Voyagers ability to advance its AAV-based gene therapies and its ability to continue to develop its gene therapy platform; the scope of the intellectual property rights and other rights that will be available to Voyager following the termination of the AbbVie collaboration agreements; the anticipated effects of the termination of the AbbVie collaboration agreements on Voyagers anticipated financial results, including Voyagers available cash, cash equivalents and marketable debt securities; and Voyagers ability to fund its operating expenses with its current cash, cash equivalents and marketable debt securities through a stated time period are forward looking. All forward-looking statements are based on estimates and assumptions by Voyagers management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the continued cooperation of AbbVie in activities arising from the termination of the AbbVie collaboration agreements, the development of the gene therapy platform; Voyagers scientific approach and general development progress; Voyagers ability to create and protect its intellectual property; and the sufficiency of Voyagers cash resources. These statements are also subject to a number of material risks and uncertainties that are described in Voyagers most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investors:Paul CoxVP, Investor Relations857-201-3463pcox@vygr.com

Media:Sheryl SeapyW2Opure949-903-4750sseapy@purecommunications.com

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Gene therapy – Wikipedia

Medical field

Gene therapy (also called human gene transfer) is a medical field which focuses on the utilization of the therapeutic delivery of nucleic acids into a patient's cells as a drug to treat disease.[1][2] The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989.[3] The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. It is thought to be able to cure many genetic disorders or treat them over time.

Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in phase I.[4] As of 2017, Spark Therapeutics' Luxturna (RPE65 mutation-induced blindness) and Novartis' Kymriah (Chimeric antigen receptor T cell therapy) are the FDA's first approved gene therapies to enter the market. Since that time, drugs such as Novartis' Zolgensma and Alnylam's Patisiran have also received FDA approval, in addition to other companies' gene therapy drugs. Most of these approaches utilize adeno-associated viruses (AAVs) and lentiviruses for performing gene insertions, in vivo and ex vivo, respectively. ASO / siRNA approaches such as those conducted by Alnylam and Ionis Pharmaceuticals require non-viral delivery systems, and utilize alternative mechanisms for trafficking to liver cells by way of GalNAc transporters.

The concept of gene therapy is to fix a genetic problem at its source. If, for instance, in an (usually recessively) inherited disease a mutation in a certain gene results in the production of a dysfunctional protein, gene therapy could be used to deliver a copy of this gene that does not contain the deleterious mutation, and thereby produces a functional protein. This strategy is referred to as gene replacement therapy and is employed to treat inherited retinal diseases. [5][6]

While the concept of gene replacement therapy is mostly suitable for recessive diseases, novel strategies have been suggested that are capable of also treating conditions with a dominant pattern of inheritance.

Not all medical procedures that introduce alterations to a patient's genetic makeup can be considered gene therapy. Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients.[14] Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effect.

Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies.

The first attempt, an unsuccessful one, at gene therapy (as well as the first case of medical transfer of foreign genes into humans not counting organ transplantation) was performed by Martin Cline on 10 July 1980.[15][16] Cline claimed that one of the genes in his patients was active six months later, though he never published this data or had it verified[17] and even if he is correct, it's unlikely it produced any significant beneficial effects treating beta-thalassemia.[medical citation needed]

After extensive research on animals throughout the 1980s and a 1989 bacterial gene tagging trial on humans, the first gene therapy widely accepted as a success was demonstrated in a trial that started on 14 September 1990, when Ashi DeSilva was treated for ADA-SCID.[18]

The first somatic treatment that produced a permanent genetic change was initiated in 1993. The goal was to cure malignant brain tumors by using recombinant DNA to transfer a gene making the tumor cells sensitive to a drug that in turn would cause the tumor cells to die.[19]

The polymers are either translated into proteins, interfere with target gene expression, or possibly correct genetic mutations. The most common form uses DNA that encodes a functional, therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a "vector", which carries the molecule inside cells.[medical citation needed]

Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers' attention, although as of 2014[update], it was still largely an experimental technique.[20] These include treatment of retinal diseases Leber's congenital amaurosis[5][21][22][23] and choroideremia,[24] X-linked SCID,[25] ADA-SCID,[26][27] adrenoleukodystrophy,[28] chronic lymphocytic leukemia (CLL),[29] acute lymphocytic leukemia (ALL),[30] multiple myeloma,[31] haemophilia,[27] and Parkinson's disease.[32] Between 2013 and April 2014, US companies invested over $600 million in the field.[33]

The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers.[34] In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia.[35]In 2012 Glybera, a treatment for a rare inherited disorder, lipoprotein lipase deficiency became the first treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.[20][36]

Following early advances in genetic engineering of bacteria, cells, and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered replacing or disrupting defective genes.[37] Scientists focused on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia, and sickle cell anemia. Glybera treats one such disease, caused by a defect in lipoprotein lipase.[36]

DNA must be administered, reach the damaged cells, enter the cell and either express or disrupt a protein.[38] Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered virus to deliver the DNA into a chromosome.[39][40] Naked DNA approaches have also been explored, especially in the context of vaccine development.[41]

Generally, efforts focused on administering a gene that causes a needed protein to be expressed. More recently, increased understanding of nuclease function has led to more direct DNA editing, using techniques such as zinc finger nucleases and CRISPR. The vector incorporates genes into chromosomes. The expressed nucleases then knock out and replace genes in the chromosome. As of 2014[update] these approaches involve removing cells from patients, editing a chromosome and returning the transformed cells to patients.[42]

Gene editing is a potential approach to alter the human genome to treat genetic diseases,[7] viral diseases,[43] and cancer.[citation needed] As of 2016[update] these approaches were still years from being medicine.[44][45]

Gene therapy may be classified into two types:

In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete, germ cell, gametocyte, or undifferentiated stem cell. Any such modifications affect the individual patient only, and are not inherited by offspring. Somatic gene therapy represents mainstream basic and clinical research, in which therapeutic DNA (either integrated in the genome or as an external episome or plasmid) is used to treat disease.[medical citation needed]

Over 600 clinical trials utilizing SCGT are underway[when?] in the US. Most focus on severe genetic disorders, including immunodeficiencies, haemophilia, thalassaemia, and cystic fibrosis. Such single gene disorders are good candidates for somatic cell therapy. The complete correction of a genetic disorder or the replacement of multiple genes is not yet possible. Only a few of the trials are in the advanced stages.[46] [needs update]

In germline gene therapy (GGT), germ cells (sperm or egg cells) are modified by the introduction of functional genes into their genomes. Modifying a germ cell causes all the organism's cells to contain the modified gene. The change is therefore heritable and passed on to later generations. Australia, Canada, Germany, Israel, Switzerland, and the Netherlands[47] prohibit GGT for application in human beings, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations[47] and higher risks versus SCGT.[48] The US has no federal controls specifically addressing human genetic modification (beyond FDA regulations for therapies in general).[47][49][50][51]

The delivery of DNA into cells can be accomplished by multiple methods. The two major classes are recombinant viruses (sometimes called biological nanoparticles or viral vectors) and naked DNA or DNA complexes (non-viral methods).[medical citation needed]

In order to replicate, viruses introduce their genetic material into the host cell, tricking the host's cellular machinery into using it as blueprints for viral proteins. Retroviruses go a stage further by having their genetic material copied into the genome of the host cell. Scientists exploit this by substituting a virus's genetic material with therapeutic DNA. (The term 'DNA' may be an oversimplification, as some viruses contain RNA, and gene therapy could take this form as well.) A number of viruses have been used for human gene therapy, including retroviruses, adenoviruses, herpes simplex, vaccinia, and adeno-associated virus.[4] Like the genetic material (DNA or RNA) in viruses, therapeutic DNA can be designed to simply serve as a temporary blueprint that is degraded naturally or (at least theoretically) to enter the host's genome, becoming a permanent part of the host's DNA in infected cells.

Non-viral methods present certain advantages over viral methods, such as large scale production and low host immunogenicity. However, non-viral methods initially produced lower levels of transfection and gene expression, and thus lower therapeutic efficacy. Newer technologies offer promise of solving these problems, with the advent of increased cell-specific targeting and subcellular trafficking control.

Methods for non-viral gene therapy include the injection of naked DNA, electroporation, the gene gun, sonoporation, magnetofection, the use of oligonucleotides, lipoplexes, dendrimers, and inorganic nanoparticles.

More recent approaches, such as those performed by companies such as Ligandal, offer the possibility of creating cell-specific targeting technologies for a variety of gene therapy modalities, including RNA, DNA and gene editing tools such as CRISPR. Other companies, such as Arbutus Biopharma and Arcturus Therapeutics, offer non-viral, non-cell-targeted approaches that mainly exhibit liver trophism. In more recent years, startups such as Sixfold Bio, GenEdit, and Spotlight Therapeutics have begun to solve the non-viral gene delivery problem. Non-viral techniques offer the possibility of repeat dosing and greater tailorability of genetic payloads, which in the future will be more likely to take over viral-based delivery systems.

Companies such as Editas Medicine, Intellia Therapeutics, CRISPR Therapeutics, Casebia, Cellectis, Precision Biosciences, bluebird bio, and Sangamo have developed non-viral gene editing techniques, however frequently still use viruses for delivering gene insertion material following genomic cleavage by guided nucleases. These companies focus on gene editing, and still face major delivery hurdles.

BioNTech, Moderna Therapeutics and CureVac focus on delivery of mRNA payloads, which are necessarily non-viral delivery problems.

Alnylam, Dicerna Pharmaceuticals, and Ionis Pharmaceuticals focus on delivery of siRNA (antisense oligonucleotides) for gene suppression, which also necessitate non-viral delivery systems.

In academic contexts, a number of laboratories are working on delivery of PEGylated particles, which form serum protein coronas and chiefly exhibit LDL receptor mediated uptake in cells in vivo.[52]

Some of the unsolved problems include:

Three patients' deaths have been reported in gene therapy trials, putting the field under close scrutiny. The first was that of Jesse Gelsinger, who died in 1999 because of immune rejection response.[60][61] One X-SCID patient died of leukemia in 2003.[18] In 2007, a rheumatoid arthritis patient died from an infection; the subsequent investigation concluded that the death was not related to gene therapy.[62]

In 1972 Friedmann and Roblin authored a paper in Science titled "Gene therapy for human genetic disease?"[63] Rogers (1970) was cited for proposing that exogenous good DNA be used to replace the defective DNA in those who suffer from genetic defects.[64]

In 1984 a retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes.[65]

The first approved gene therapy clinical research in the US took place on 14 September 1990, at the National Institutes of Health (NIH), under the direction of William French Anderson.[66] Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency. The defective gene of the patient's blood cells was replaced by the functional variant. Ashanti's immune system was partially restored by the therapy. Production of the missing enzyme was temporarily stimulated, but the new cells with functional genes were not generated. She led a normal life only with the regular injections performed every two months. The effects were successful, but temporary.[67]

Cancer gene therapy was introduced in 1992/93 (Trojan et al. 1993).[68] The treatment of glioblastoma multiforme, the malignant brain tumor whose outcome is always fatal, was done using a vector expressing antisense IGF-I RNA (clinical trial approved by NIH protocol no.1602 24 November 1993,[69] and by the FDA in 1994). This therapy also represents the beginning of cancer immunogene therapy, a treatment which proves to be effective due to the anti-tumor mechanism of IGF-I antisense, which is related to strong immune and apoptotic phenomena.

In 1992 Claudio Bordignon, working at the Vita-Salute San Raffaele University, performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases.[70] In 2002 this work led to the publication of the first successful gene therapy treatment for adenosine deaminase deficiency (ADA-SCID). The success of a multi-center trial for treating children with SCID (severe combined immune deficiency or "bubble boy" disease) from 2000 and 2002, was questioned when two of the ten children treated at the trial's Paris center developed a leukemia-like condition. Clinical trials were halted temporarily in 2002, but resumed after regulatory review of the protocol in the US, the United Kingdom, France, Italy, and Germany.[71]

In 1993 Andrew Gobea was born with SCID following prenatal genetic screening. Blood was removed from his mother's placenta and umbilical cord immediately after birth, to acquire stem cells. The allele that codes for adenosine deaminase (ADA) was obtained and inserted into a retrovirus. Retroviruses and stem cells were mixed, after which the viruses inserted the gene into the stem cell chromosomes. Stem cells containing the working ADA gene were injected into Andrew's blood. Injections of the ADA enzyme were also given weekly. For four years T cells (white blood cells), produced by stem cells, made ADA enzymes using the ADA gene. After four years more treatment was needed.[72]

Jesse Gelsinger's death in 1999 impeded gene therapy research in the US.[73][74] As a result, the FDA suspended several clinical trials pending the reevaluation of ethical and procedural practices.[75]

The modified cancer gene therapy strategy of antisense IGF-I RNA (NIH n 1602)[69] using antisense / triple helix anti-IGF-I approach was registered in 2002 by Wiley gene therapy clinical trial - n 635 and 636. The approach has shown promising results in the treatment of six different malignant tumors: glioblastoma, cancers of liver, colon, prostate, uterus, and ovary (Collaborative NATO Science Programme on Gene Therapy USA, France, Poland n LST 980517 conducted by J. Trojan) (Trojan et al., 2012). This anti-gene antisense/triple helix therapy has proven to be efficient, due to the mechanism stopping simultaneously IGF-I expression on translation and transcription levels, strengthening anti-tumor immune and apoptotic phenomena.

Sickle-cell disease can be treated in mice.[76] The mice which have essentially the same defect that causes human cases used a viral vector to induce production of fetal hemoglobin (HbF), which normally ceases to be produced shortly after birth. In humans, the use of hydroxyurea to stimulate the production of HbF temporarily alleviates sickle cell symptoms. The researchers demonstrated this treatment to be a more permanent means to increase therapeutic HbF production.[77]

A new gene therapy approach repaired errors in messenger RNA derived from defective genes. This technique has the potential to treat thalassaemia, cystic fibrosis and some cancers.[78]

Researchers created liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane.[79]

In 2003 a research team inserted genes into the brain for the first time. They used liposomes coated in a polymer called polyethylene glycol, which unlike viral vectors, are small enough to cross the bloodbrain barrier.[80]

Short pieces of double-stranded RNA (short, interfering RNAs or siRNAs) are used by cells to degrade RNA of a particular sequence. If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced.[81]

Gendicine is a cancer gene therapy that delivers the tumor suppressor gene p53 using an engineered adenovirus. In 2003, it was approved in China for the treatment of head and neck squamous cell carcinoma.[34]

In March researchers announced the successful use of gene therapy to treat two adult patients for X-linked chronic granulomatous disease, a disease which affects myeloid cells and damages the immune system. The study is the first to show that gene therapy can treat the myeloid system.[82]

In May a team reported a way to prevent the immune system from rejecting a newly delivered gene.[83] Similar to organ transplantation, gene therapy has been plagued by this problem. The immune system normally recognizes the new gene as foreign and rejects the cells carrying it. The research utilized a newly uncovered network of genes regulated by molecules known as microRNAs. This natural function selectively obscured their therapeutic gene in immune system cells and protected it from discovery. Mice infected with the gene containing an immune-cell microRNA target sequence did not reject the gene.

In August scientists successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells.[84]

In November researchers reported on the use of VRX496, a gene-based immunotherapy for the treatment of HIV that uses a lentiviral vector to deliver an antisense gene against the HIV envelope. In a phase I clinical trial, five subjects with chronic HIV infection who had failed to respond to at least two antiretroviral regimens were treated. A single intravenous infusion of autologous CD4 T cells genetically modified with VRX496 was well tolerated. All patients had stable or decreased viral load; four of the five patients had stable or increased CD4 T cell counts. All five patients had stable or increased immune response to HIV antigens and other pathogens. This was the first evaluation of a lentiviral vector administered in a US human clinical trial.[85][86]

In May researchers announced the first gene therapy trial for inherited retinal disease. The first operation was carried out on a 23-year-old British male, Robert Johnson, in early 2007.[87]

Leber's congenital amaurosis is an inherited blinding disease caused by mutations in the RPE65 gene. The results of a small clinical trial in children were published in April.[5] Delivery of recombinant adeno-associated virus (AAV) carrying RPE65 yielded positive results. In May two more groups reported positive results in independent clinical trials using gene therapy to treat the condition. In all three clinical trials, patients recovered functional vision without apparent side-effects.[5][21][22][23]

In September researchers were able to give trichromatic vision to squirrel monkeys.[88] In November 2009, researchers halted a fatal genetic disorder called adrenoleukodystrophy in two children using a lentivirus vector to deliver a functioning version of ABCD1, the gene that is mutated in the disorder.[89]

An April paper reported that gene therapy addressed achromatopsia (color blindness) in dogs by targeting cone photoreceptors. Cone function and day vision were restored for at least 33 months in two young specimens. The therapy was less efficient for older dogs.[90]

In September it was announced that an 18-year-old male patient in France with beta-thalassemia major had been successfully treated.[91] Beta-thalassemia major is an inherited blood disease in which beta haemoglobin is missing and patients are dependent on regular lifelong blood transfusions.[92] The technique used a lentiviral vector to transduce the human -globin gene into purified blood and marrow cells obtained from the patient in June 2007.[93] The patient's haemoglobin levels were stable at 9 to 10 g/dL. About a third of the hemoglobin contained the form introduced by the viral vector and blood transfusions were not needed.[93][94] Further clinical trials were planned.[95] Bone marrow transplants are the only cure for thalassemia, but 75% of patients do not find a matching donor.[94]

Cancer immunogene therapy using modified antigene, antisense/triple helix approach was introduced in South America in 2010/11 in La Sabana University, Bogota (Ethical Committee 14 December 2010, no P-004-10). Considering the ethical aspect of gene diagnostic and gene therapy targeting IGF-I, the IGF-I expressing tumors i.e. lung and epidermis cancers were treated (Trojan et al. 2016).[96][97]

In 2007 and 2008, a man (Timothy Ray Brown) was cured of HIV by repeated hematopoietic stem cell transplantation (see also allogeneic stem cell transplantation, allogeneic bone marrow transplantation, allotransplantation) with double-delta-32 mutation which disables the CCR5 receptor. This cure was accepted by the medical community in 2011.[98] It required complete ablation of existing bone marrow, which is very debilitating.

In August two of three subjects of a pilot study were confirmed to have been cured from chronic lymphocytic leukemia (CLL). The therapy used genetically modified T cells to attack cells that expressed the CD19 protein to fight the disease.[29] In 2013, the researchers announced that 26 of 59 patients had achieved complete remission and the original patient had remained tumor-free.[99]

Human HGF plasmid DNA therapy of cardiomyocytes is being examined as a potential treatment for coronary artery disease as well as treatment for the damage that occurs to the heart after myocardial infarction.[100][101]

In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia; it delivers the gene encoding for VEGF.[102][35] Neovasculogen is a plasmid encoding the CMV promoter and the 165 amino acid form of VEGF.[103][104]

The FDA approved Phase 1 clinical trials on thalassemia major patients in the US for 10 participants in July.[105] The study was expected to continue until 2015.[95]

In July 2012, the European Medicines Agency recommended approval of a gene therapy treatment for the first time in either Europe or the United States. The treatment used Alipogene tiparvovec (Glybera) to compensate for lipoprotein lipase deficiency, which can cause severe pancreatitis.[106] The recommendation was endorsed by the European Commission in November 2012[20][36][107][108] and commercial rollout began in late 2014.[109] Alipogene tiparvovec was expected to cost around $1.6 million per treatment in 2012,[110] revised to $1 million in 2015,[111] making it the most expensive medicine in the world at the time.[112] As of 2016[update], only the patients treated in clinical trials and a patient who paid the full price for treatment have received the drug.[113]

In December 2012, it was reported that 10 of 13 patients with multiple myeloma were in remission "or very close to it" three months after being injected with a treatment involving genetically engineered T cells to target proteins NY-ESO-1 and LAGE-1, which exist only on cancerous myeloma cells.[31]

In March researchers reported that three of five adult subjects who had acute lymphocytic leukemia (ALL) had been in remission for five months to two years after being treated with genetically modified T cells which attacked cells with CD19 genes on their surface, i.e. all B-cells, cancerous or not. The researchers believed that the patients' immune systems would make normal T-cells and B-cells after a couple of months. They were also given bone marrow. One patient relapsed and died and one died of a blood clot unrelated to the disease.[30]

Following encouraging Phase 1 trials, in April, researchers announced they were starting Phase 2 clinical trials (called CUPID2 and SERCA-LVAD) on 250 patients[114] at several hospitals to combat heart disease. The therapy was designed to increase the levels of SERCA2, a protein in heart muscles, improving muscle function.[115] The FDA granted this a Breakthrough Therapy Designation to accelerate the trial and approval process.[116] In 2016 it was reported that no improvement was found from the CUPID 2 trial.[117]

In July researchers reported promising results for six children with two severe hereditary diseases had been treated with a partially deactivated lentivirus to replace a faulty gene and after 732 months. Three of the children had metachromatic leukodystrophy, which causes children to lose cognitive and motor skills.[118] The other children had WiskottAldrich syndrome, which leaves them to open to infection, autoimmune diseases, and cancer.[119] Follow up trials with gene therapy on another six children with WiskottAldrich syndrome were also reported as promising.[120][121]

In October researchers reported that two children born with adenosine deaminase severe combined immunodeficiency disease (ADA-SCID) had been treated with genetically engineered stem cells 18 months previously and that their immune systems were showing signs of full recovery. Another three children were making progress.[27] In 2014 a further 18 children with ADA-SCID were cured by gene therapy.[122] ADA-SCID children have no functioning immune system and are sometimes known as "bubble children."[27]

Also in October researchers reported that they had treated six hemophilia sufferers in early 2011 using an adeno-associated virus. Over two years later all six were producing clotting factor.[27][123]

In January researchers reported that six choroideremia patients had been treated with adeno-associated virus with a copy of REP1. Over a six-month to two-year period all had improved their sight.[6][124] By 2016, 32 patients had been treated with positive results and researchers were hopeful the treatment would be long-lasting.[24] Choroideremia is an inherited genetic eye disease with no approved treatment, leading to loss of sight.

In March researchers reported that 12 HIV patients had been treated since 2009 in a trial with a genetically engineered virus with a rare mutation (CCR5 deficiency) known to protect against HIV with promising results.[125][126]

Clinical trials of gene therapy for sickle cell disease were started in 2014.[127][128]

In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the disease.[129]

In March researchers delivered a recombinant gene encoding a broadly neutralizing antibody into monkeys infected with simian HIV; the monkeys' cells produced the antibody, which cleared them of HIV. The technique is named immunoprophylaxis by gene transfer (IGT). Animal tests for antibodies to ebola, malaria, influenza, and hepatitis were underway.[130][131]

In March, scientists, including an inventor of CRISPR, Jennifer Doudna, urged a worldwide moratorium on germline gene therapy, writing "scientists should avoid even attempting, in lax jurisdictions, germline genome modification for clinical application in humans" until the full implications "are discussed among scientific and governmental organizations".[132][133][134][135]

In October, researchers announced that they had treated a baby girl, Layla Richards, with an experimental treatment using donor T-cells genetically engineered using TALEN to attack cancer cells. One year after the treatment she was still free of her cancer (a highly aggressive form of acute lymphoblastic leukaemia [ALL]).[136] Children with highly aggressive ALL normally have a very poor prognosis and Layla's disease had been regarded as terminal before the treatment.[137]

In December, scientists of major world academies called for a moratorium on inheritable human genome edits, including those related to CRISPR-Cas9 technologies[138] but that basic research including embryo gene editing should continue.[139]

In April the Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis[140][141] and the European Commission approved it in June.[142] This treats children born with adenosine deaminase deficiency and who have no functioning immune system. This was the second gene therapy treatment to be approved in Europe.[143]

In October, Chinese scientists reported they had started a trial to genetically modify T-cells from 10 adult patients with lung cancer and reinject the modified T-cells back into their bodies to attack the cancer cells. The T-cells had the PD-1 protein (which stops or slows the immune response) removed using CRISPR-Cas9.[144][145]

A 2016 Cochrane systematic review looking at data from four trials on topical cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy does not support its clinical use as a mist inhaled into the lungs to treat cystic fibrosis patients with lung infections. One of the four trials did find weak evidence that liposome-based CFTR gene transfer therapy may lead to a small respiratory improvement for people with CF. This weak evidence is not enough to make a clinical recommendation for routine CFTR gene therapy.[146]

In February Kite Pharma announced results from a clinical trial of CAR-T cells in around a hundred people with advanced Non-Hodgkin lymphoma.[147]

In March, French scientists reported on clinical research of gene therapy to treat sickle-cell disease.[148]

In August, the FDA approved tisagenlecleucel for acute lymphoblastic leukemia.[149] Tisagenlecleucel is an adoptive cell transfer therapy for B-cell acute lymphoblastic leukemia; T cells from a person with cancer are removed, genetically engineered to make a specific T-cell receptor (a chimeric T cell receptor, or "CAR-T") that reacts to the cancer, and are administered back to the person. The T cells are engineered to target a protein called CD19 that is common on B cells. This is the first form of gene therapy to be approved in the United States. In October, a similar therapy called axicabtagene ciloleucel was approved for non-Hodgkin lymphoma.[150]

In December the results of using an adeno-associated virus with blood clotting factor VIII to treat nine haemophilia A patients were published. Six of the seven patients on the high dose regime increased the level of the blood clotting VIII to normal levels. The low and medium dose regimes had no effect on the patient's blood clotting levels.[151][152]

In December, the FDA approved Luxturna, the first in vivo gene therapy, for the treatment of blindness due to Leber's congenital amaurosis.[153] The price of this treatment was 850,000 US dollars for both eyes.[154][155]

A need was identified for high quality randomised controlled trials assessing the risks and benefits involved with gene therapy for people with sickle cell disease.[156]

In February, medical scientists working with Sangamo Therapeutics, headquartered in Richmond, California, announced the first ever "in body" human gene editing therapy to permanently alter DNA - in a patient with Hunter syndrome.[157] Clinical trials by Sangamo involving gene editing using Zinc Finger Nuclease (ZFN) are ongoing.[158]

In May, the FDA approved onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in children under two years of age. The list price of Zolgensma was set at US$2.125 million per dose, making it the most expensive drug ever.[159]

In May, the EMA approved betibeglogene autotemcel (Zynteglo) for treating beta thalassemia for people twelve years of age and older.[160][161]

In July, Allergan and Editas Medicine announced phase 1/2 clinical trial of AGN-151587 for the treatment of Leber congenital amaurosis 10.[162] It will be the world's first in vivo study of a CRISPR-based human gene editing therapy, where the editing takes place inside the human body.[163] The first injection of the CRISPR-Cas System was confirmed in March of 2020.[164] This marks the first instance of genome editing within an adult human in the context of a scientific study. The very first in-vivo human genome editing however likely took place outside of academia in the context of a self-administered therapy by Biophysicist Josiah Zayner, PhD.[165][166]

Speculated uses for gene therapy include:

Athletes might adopt gene therapy technologies to improve their performance.[167] Gene doping is not known to occur, but multiple gene therapies may have such effects. Kayser et al. argue that gene doping could level the playing field if all athletes receive equal access. Critics claim that any therapeutic intervention for non-therapeutic/enhancement purposes compromises the ethical foundations of medicine and sports.[168]

Genetic engineering could be used to cure diseases, but also to change physical appearance, metabolism, and even improve physical capabilities and mental faculties such as memory and intelligence. Ethical claims about germline engineering include beliefs that every fetus has a right to remain genetically unmodified, that parents hold the right to genetically modify their offspring, and that every child has the right to be born free of preventable diseases.[169][170][171] For parents, genetic engineering could be seen as another child enhancement technique to add to diet, exercise, education, training, cosmetics, and plastic surgery.[172][173] Another theorist claims that moral concerns limit but do not prohibit germline engineering.[174]

A recent issue of the journal Bioethics was devoted to moral issues surrounding germline genetic engineering in people.[175]

Possible regulatory schemes include a complete ban, provision to everyone, or professional self-regulation. The American Medical Associations Council on Ethical and Judicial Affairs stated that "genetic interventions to enhance traits should be considered permissible only in severely restricted situations: (1) clear and meaningful benefits to the fetus or child; (2) no trade-off with other characteristics or traits; and (3) equal access to the genetic technology, irrespective of income or other socioeconomic characteristics."[176]

As early in the history of biotechnology as 1990, there have been scientists opposed to attempts to modify the human germline using these new tools,[177] and such concerns have continued as technology progressed.[178][179] With the advent of new techniques like CRISPR, in March 2015 a group of scientists urged a worldwide moratorium on clinical use of gene editing technologies to edit the human genome in a way that can be inherited.[132][133][134][135] In April 2015, researchers sparked controversy when they reported results of basic research to edit the DNA of non-viable human embryos using CRISPR.[180][181] A committee of the American National Academy of Sciences and National Academy of Medicine gave qualified support to human genome editing in 2017[182][183] once answers have been found to safety and efficiency problems "but only for serious conditions under stringent oversight."[184]

Regulations covering genetic modification are part of general guidelines about human-involved biomedical research. There are no international treaties which are legally binding in this area, but there are recommendations for national laws from various bodies.

The Helsinki Declaration (Ethical Principles for Medical Research Involving Human Subjects) was amended by the World Medical Association's General Assembly in 2008. This document provides principles physicians and researchers must consider when involving humans as research subjects. The Statement on Gene Therapy Research initiated by the Human Genome Organization (HUGO) in 2001 provides a legal baseline for all countries. HUGO's document emphasizes human freedom and adherence to human rights, and offers recommendations for somatic gene therapy, including the importance of recognizing public concerns about such research.[185]

No federal legislation lays out protocols or restrictions about human genetic engineering. This subject is governed by overlapping regulations from local and federal agencies, including the Department of Health and Human Services, the FDA and NIH's Recombinant DNA Advisory Committee. Researchers seeking federal funds for an investigational new drug application, (commonly the case for somatic human genetic engineering,) must obey international and federal guidelines for the protection of human subjects.[186]

NIH serves as the main gene therapy regulator for federally funded research. Privately funded research is advised to follow these regulations. NIH provides funding for research that develops or enhances genetic engineering techniques and to evaluate the ethics and quality in current research. The NIH maintains a mandatory registry of human genetic engineering research protocols that includes all federally funded projects.

An NIH advisory committee published a set of guidelines on gene manipulation.[187] The guidelines discuss lab safety as well as human test subjects and various experimental types that involve genetic changes. Several sections specifically pertain to human genetic engineering, including Section III-C-1. This section describes required review processes and other aspects when seeking approval to begin clinical research involving genetic transfer into a human patient.[188] The protocol for a gene therapy clinical trial must be approved by the NIH's Recombinant DNA Advisory Committee prior to any clinical trial beginning; this is different from any other kind of clinical trial.[187]

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