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

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

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

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.

Link:

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

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.

Read the rest here:

How Gene Therapy Helped Conner Run : Short Wave - NPR

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

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.

Continue reading here:

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

Researchstore.bizhas published the latest research study onGlobal Gene Therapy in Oncology Market 2020 by Company, Type and Application, Forecast to 2025that presents a complete overview of the market with a detailed description of the global market. The report provides complete information about the advancing market trade and business data. The report highlights the dynamics of the market such as internal and external driving forces, restraining factors, risks, challenges, threats, and opportunities. The complete view linked with the progress of this globalGene Therapy in Oncologymarket by the significant players involved in this business. Analysts of this research report predict the financial attributes such as investment, pricing structures along with the profit margin.

NOTE:This report takes into account the current and future impacts of COVID-19 on this industry and offers you an in-depth analysis ofGene Therapy in Oncologymarket.

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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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Table of Contents:1 Market Overview2 Company Profiles3 Market Competition, by Players4 North America Market Size and Forecast by Countries5 Europe Market Size and Forecast by Countries6 Asia-Pacific Market Size and Forecast by Countries7 South America Market Size and Forecast by Countries8 Middle East & Africa Market Size and Forecast by Countries9 Market Size Segment by Type10 Market Size Segment by Application11 Research Findings and Conclusion12 Appendix

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

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.

Link:

Research shows promising results for LHON gene therapy - Ophthalmology Times

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

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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

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.

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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.

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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,...