SAN DIEGO and BOSTON, July 24, 2020 /PRNewswire/ --Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") and SmartPharm Therapeutics, Inc. ("SmartPharm") announced today the signing of a letter of intent for Sorrento to acquire SmartPharm, a gene-encoded therapeutics company developing non-viral DNA and RNA gene delivery platforms for COVID-19 and rare diseases, with broad potential for application in enhancing antibody-centric therapeutics, including against COVID-19. The transaction is expected to close in August 2020. Financial terms of the deal were not disclosed.

Sorrento and SmartPharm previously announced a research and development collaboration to encode and express in vivo Sorrento's proprietary SARS-CoV-2 neutralizing monoclonal antibodies utilizing SmartPharm's Gene Mab plasmid nanoparticle platform.

Sorrento plans to accelerate the development of multiple candidates for in vivo gene-encoded expression of Sorrento's antibodies, starting with Sorrento's previously announced STI-1499, or COVI-GUARD, which is currently moving through preclinical and manufacturing requirements with an IND submission targeted for August 2020. The initial clinical trial for STI-1499 is expected to be in ICU patients to ensure safety and potentially allow a preliminary look at efficacy. In vitro results so far have demonstrated STI-1499's ability to completely neutralize SARS-CoV-2 infection at low doses, making STI-1499 Sorrento's lead candidate for potential cost-effective passive protection against COVID-19.

SmartPharm's Gene Mab platform delivers to muscles a novel low-immunogenic DNA plasmid encoded with a therapeutic antibody for long-lasting expression in vivo. If clinical trials are successful, a single administration of STI-1499dpi (DNA plasmid injection) could allow the recipient's own muscle cells to produce the antibody for a prolonged period of time after a single injection, potentially providing extended protection against COVID-19 for periods of time that might provide an alternative to vaccines. Manufacturing of DNA plasmids (in bacterial fermenters) can be done with Sorrento's in-house cGMP capabilities for a fraction of the cost associated with traditional antibody manufacturing.

"We are very encouraged by the preclinical data generated thus far by our STI-1499 neutralizing antibody against COVID-19," said Henry Ji, Ph.D., CEO of Sorrento Therapeutics. "We are excited about the prospect of leveraging SmartPharm's Gene Mab platform in combination with our G-MAB library to produce next-generation gene-encoded antibody candidates against a host of pathogens and cancer cell types. Being able to stimulate the body to produce in vivo our most potent antibodies at optimized manufacturing costs will offer an additional competitive advantage as Sorrento transitions from product development to full scale manufacturing and commercialization."

About SmartPharm Therapeutics

SmartPharm Therapeutics, Inc. is a privately held, development stage biopharmaceutical company focused on developing next-generation, non-viral gene therapies for the treatment of serious or rare diseases with the vision of creating "Biologics from Within." SmartPharm is currently developing a novel pipeline of non-viral, gene-encoded proteins for the treatment of conditions that require biologic therapy such enzyme replacement and tissue restoration. SmartPharm commenced operations in 2018 and is headquartered in Cambridge, MA, USA. For more information, please visit http://www.smartpharmtx.com.

About Sorrento Therapeutics, Inc.

Sorrento is a clinical stage, antibody-centric, biopharmaceutical company developing new therapies to treat cancers. Sorrento's multimodal, multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as fully human antibodies ("G-MAB library"), clinical stage immuno-cellular therapies ("CAR-T", "DAR-T"), antibody-drug conjugates ("ADCs"), and clinical stage oncolytic virus ("Seprehvir", "Seprehvec"). Sorrento is also developing potential antiviral therapies and vaccines against coronaviruses, including COVIDTRAP, ACE-MAB, COVI-MAB, COVI-GUARD, COVI-SHIELD and T-VIVA-19.

Sorrento's commitment to life-enhancing therapies for patients is also demonstrated by our effort to advance a first-in-class (TRPV1 agonist) non-opioid pain management small molecule, resiniferatoxin ("RTX"), and ZTlido (lidocaine topical system) 1.8% for the treatment of post-herpetic neuralgia. RTX is completing a phase IB trial for intractable pain associated with cancer and a phase 1B trial in osteoarthritis patients. ZTlido was approved by the FDA on February 28, 2018.

For more information visit http://www.sorrentotherapeutics.com

Forward-Looking Statements

This press release and any statements made for and during any presentation or meeting contain forward-looking statements related toSorrento Therapeutics, Inc., under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995 and subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding the proposed acquisition of SmartPharm; the potential effects that the acquisition of SmartPharm may have on Sorrento's business and product candidate pipeline; the expected timing for the closing of the transaction; the initiation and completion of ongoing studies for COVID-19 using antibodies and gene-encoded antibodies, and data read-outs related thereto; the potency and potential blocking capabilities of STI-1499 and STI-1499dpi and their respective impact on SARS-CoV-2; the expected length of any antiviral protection provided by STI-1499 and STI-1499dpi; the potential administration and applications of STI-1499 and STI-1499dpi, alone or in combination; the timeline and status of preclinical testing for STI-1499 and STI-1499dpi; the expected timing of an IND submission for STI-1499; expectations regarding the initial clinical trial for STI-1499; the potential safety and efficacy of STI-1499 and STI-1499dpi; the therapeutic potential of STI-1499 and STI-1499dpi for SARS-CoV-2 and COVID-19; the potential costs associated with manufacturing STI-1499dpi and other DNA plasmids; Sorrento's ability to produce antibody candidates against pathogens and cancer cells; Sorrento's ability to transition from product development to full scale manufacturing and commercialization; and Sorrento's potential position in the anti-viral immunity industry. Risks and uncertainties that could cause our actual results to differ materially and adversely from those expressed in our forward-looking statements, include, but are not limited to: risks related to Sorrento's technologies and prospects with newly acquired technologies, including the proposed acquisition of SmartPharm and the utilization of SmartPharm's Gene-Encoded Therapeutics (GET) platforms for the treatment and prevention of coronavirus infections and other pathogens and cancer cells; risks related to seeking regulatory approvals and conducting clinical trials; the clinical and commercial success of the treatment and prevention of coronavirus infections using monoclonal antibodies and gene-encoded antibodies; the viability and success of using monoclonal antibodies and gene-encoded antibodies for treatments in anti-viral therapeutic areas, including coronavirus; clinical development risks, including risks in the progress, timing, cost and results of clinical trials and product development programs; risk of difficulties or delays in obtaining regulatory approvals; risks that prior study and trial results may not be replicated in future studies and trials; risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval; risks related to seeking regulatory approvals and conducting clinical trials; risks of manufacturing drug product; risks related to leveraging the expertise of its employees, subsidiaries, affiliates and partners to assist the company in the execution of its strategies; risks related to the global impact of COVID-19 and other risks that are described in Sorrento's most recent periodic reports filed with theSecurities and Exchange Commission, including Sorrento's Annual Report on Form 10-K for the year endedDecember 31, 2019, and subsequent Quarterly Reports on Form 10-Q filed with theSecurities and Exchange Commission, including the risk factors set forth in those filings. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and we undertake no obligation to update any forward-looking statement in this press release except as required by law.

Media and Investor Relations

Contact: Dani FrankEmail: [emailprotected]

Sorrento and the Sorrento logo are registered trademarks of Sorrento Therapeutics, Inc.G-MAB, COVI-GUARD, COVI-SHIELD, COVIDTRAP, T-VIVA-19, COVI-MAB, ACE-MAB, COVI-TRACK, Saving-Life and Improving-Life are trademarks of Sorrento Therapeutics, Inc.

ZTlido is a trademark owned by Scilex Pharmaceuticals Inc.All other trademarks are the property of their respective owners. 2020 Sorrento Therapeutics, Inc. All Rights Reserved.

SOURCE Sorrento Therapeutics, Inc.

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Sorrento Enters Into Letter Of Intent To Acquire SmartPharm And Develop Pipeline Of Gene-Encoded Therapeutic Antibodies, Starting With Neutralizing...

There are good investments and there are great investments. CRISPR Therapeutics (NASDAQ:CRSP) stock certainly qualifies as the latter.

This is a company that, four years after it went public, has only been profitable for one year, yet its future is so promising that its stock is trading for 540% more than it did in 2016.

What's the big deal about CRISPR Therapeutics? The biopharmaceutical company says it is using breakthrough gene-altering therapies to treat diseases such as sickle cell disease (SCD) and beta-thalassemia, both of which are inherited blood disorders that don't have a cure and require frequent blood transfusions. The company is also working on gene therapies to treat type 1 diabetes, muscular dystrophy, cystic fibrosis, multiple myeloma, and solid tumors in pancreatic cancer and lung cancer.

Image source: Getty Images.

CRISPR Therapeutics is actually named for the technology it uses: CRISPR stands for "clusters of regularly interspaced short palindromic repeats." The gene-editing technology uses Cas9 proteins to locate a sequenceof DNA within a cell and alter it.

Though CRISPR Therapeutics has yet to bring a product to market, some of its clinical trials have had amazing results. In June, the Swiss company announced that in a joint trial with Vertex Pharmaceuticals (NASDAQ:VRTX), five patients with beta-thalassemia and two patients with sickle cell disease were treated with gene therapy CTX001.

Two of the early beta-thalassemia patients are now transfusion independent 15 months afterward. The first SCD patient in the trial is transfusion independent and free of vaso-occlusive crises (VOCs), a painful condition when blood vessels are blocked by sickled red blood cells, seven months after his dose of CTX001.

Beta-thalassemia, which reduces the body's production of hemoglobin, is extremely rare. Sickle cell anemia is common among African Americans but also can affect Latinos and people of Indian, Asian, Mediterranean backgrounds. The U.S. Department of Health and Human Services says 300,000 babies worldwide are born every year with the disease and 100,000 people in the United States are currently living with the disease.

While the results are quite promising, the study is still in its infancy. .

If you had invested $100 in CRISPR Therapeutics stock when it went public in 2016, how much would that be worth today?

By the end of the day on Oct. 19, 2016, the day CRISPR Therapeutics went public, the stock was trading at $14.09, you would have bought seven shares. Assuming you bought at that price, your original $98.63 investment would be worth $632.45 as of the close of trading Friday when the stock went for $90.35 per share. That's a gain of 541%.

CRISPR Therapeutics isn't the only biotech to use CRISPR-Cas9 technology. Others include Editas Medicine (NASDAQ:EDIT) and Intellia Therapeutics (NASDAQ:NTLA), both of which went public in 2016. Neither of those has done quite as well as CRISPR Therapeutics since its IPO.

Compared to a biotech ETF such as the iShares NASDAQ Biotechnology ETF (NASDAQ:IBB), CRISPR has shined. The ETF closed at $89.16 on the day of CRISPR's IPO. As of Friday's close, IBB was at $136.41. If you had invested $100 on Oct. 19, 2016, you would have a return of only 36%.

CRSP data by YCharts

Clinical-stage biotech stocks are inherently risky plays because there are so many hurdles these companies have to clear before they can make money. The therapies have to be approved by the Food and Drug Administration (FDA) and many therapies look promising only to fail in late-stage clinical trials.

CRISPR Therapeutics looks like a solid bet, though, both from a cash standpoint and in its strength of pipeline. The company has four candidates already in trials.It is already making money, though not a lot of it. Last year, CRISPR Therapeutics generated net income of $66.8 million, thanks to $289.5 million in collaboration revenue.

The other major question for clinical-stage biotechs is whether they have enough capital to pay for research and development until the therapies pay off. Last quarter, the company burned through $54 million in cash. However, CRISPR Therapeutics says it has more than $900 million left, so it can continue that burn rate for more than four years. The possibility that CRISPR Therapeutics' technology could actually cure diseases, not just treat them, easily makes this a risk worth taking.

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If You Invested $100 in CRISPR Therapeutics' IPO, This Is How Much Money You'd Have Now - Motley Fool

This article is an overview of the financial results of Johnson & Johnson, Novartis, Roche, Biogen and selected other healthcare companies who have reported their earnings since July 16.

Many of the top healthcare companies reporting second-quarter earnings over the past week have seen COVID-19 continue to impact revenues. But some are cautiously optimistic for the second half of the year, as they learn to better navigate the pandemic.

The biggest drug- and device-maker by market cap, Johnson & Johnson reported a 35.3% decline in its year-over-year earnings, driven in part by a 33.9% fall in medical device sales. CFO Joseph Wolk said on a July 16 earnings call that the company had previously expected to wait until the fourth quarter for a rebound in these sales, but the turnaround began earlier than expected resulting in the company raising its full-year guidance. While CEO Alex Gorsky expressed concern about a second wave of COVID-19, he pointed out that better-prepared hospitals mean the global impact is unlikely to reach levels seen in the spring.

Novartis CEO Vas NarasimhanSource: Novartis

Swiss pharmaceutical company Novartis AG reported relatively unchanged earnings year over year for the quarter, with the pandemic's effect felt most keenly on its ophthalmology and dermatology businesses, as well as therapies requiring hospital stays. This was partially offset by sales of heart disease drug Entresto, gene therapy Zolgensma and blockbuster psoriasis medicine Cosentyx.

The Basel, Switzerland-based company will not join the race to find a coronavirus vaccine, CEO Vas Narasimhan said on a July 21 earnings call. Instead, the company is focused on pivotal studies in more than 20 medicines, as well as researching inhibitors against both the current and future coronaviruses. In addition, Narasimhan expects strong uptake in Europe for nuclear therapy Lutathera and the drug's expansion in the U.S.

Roche Holding AG, another Swiss pharmaceutical giant and one of the world's biggest cancer drug manufacturers, reported a 6% decline in first-half earnings compared to the same period in 2019, with the pandemic and generic competition of its former blockbuster cancer therapies Herceptin, Avastin and MabThera/Rituxan having a combined impact on sales. However, the company expects the outlook to improve later in the year, with Roche's diagnostics unit's CEO Thomas Schinecker noting that an influx of orders for its coronavirus diagnostic tests from governments worldwide means that even after quadrupling production capacity, the company is still struggling to keep up with demand.

Cambridge, Mass.-based Biogen Inc. reported a 12% increase in second-quarter earnings, with rare disease therapy Spinraza remaining a key seller. The company announced plans to conduct a two-year phase 4 clinical study of the drug combined with Novartis' competitor therapy Zolgensma to treat infants and children with spinal muscular atrophy. GlobalData analysts said Biogen could maintain dominance in the market by demonstrating that Spinraza benefits patients who also receive the Novartis gene therapy.

Impact of reduced elective care

The reduction in elective surgeries, a key strategy to limit the initial spread of the coronavirus, took a heavier toll on healthcare equipment suppliers notably Intuitive Surgical Inc., which reported a $1.01 billion drop in revenue compared to 2019's second quarter. The surgical-robot maker's CFO, Marshall Mohr, said in a July 21 earnings call that "the recovery tail of surgery will be a long one, likely to last many quarters."

Similarly, Abbott Laboratories saw its second-quarter EPS drop 30.5% year over year, but its diabetes line remained resilient. President and CEO Robert Ford said he was "very bullish" about the opportunity for the Libre 2 blood sugar monitoring system approved in the U.S. in June and the Abbott Park, Ill.-based healthcare equipment company reissued its 2020 guidance of mid-single-digits growth.

The pause in elective procedures to slow the pandemic has impacted surgical equipment suppliers.Source: Pixel

HCA Healthcare Inc. blamed a continued drop in admissions and surgery volumes for a 12% decline in its revenue for the second quarter, but the healthcare services provider was already able to point to a rebound in May and June. The company brought in about $1.1 billion in second-quarter income although more than half came from economic relief funds.

CEO Samuel Hazen was hesitant to project into the second half of the year but said the company has gained "muscle memory" to deal with current and future COVID-19 outbreaks. RBC Capital Markets analysts expect HCA to weather the "significant challenges" of the pandemic, noting that "underlying demand in its markets remains strong, despite the likelihood for continued ebb and flow in patient census as reopenings progress and adjust to flare-ups."

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After another tough quarter, healthcare companies pin hopes on brighter H2'20 - S&P Global

QY Research has Published Latest Trending Report on Global Gene Therapy Market

Los Angeles, United State, The report titledGlobal Gene Therapy Marketis one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Gene Therapy market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Gene Therapy market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Gene Therapy market is carefully analyzed and researched about by the market analysts.

Request Sample Report and Full Report TOC:https://www.qyresearch.com/sample-form/form/1436547/global-gene-therapy-market

The Essential Content Covered in the GlobalGene Therapy Market Report:

* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

Global Gene Therapy Market is estimated to reach xxx million USD in 2020 and projected to grow at the CAGR of xx% during 2020-2026. According to the latest report added to the online repository of QY Research the Gene Therapy market has witnessed an unprecedented growth till 2020. The extrapolated future growth is expected to continue at higher rates by 2025.

Top Players of Gene Therapy Market are Studied: Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc., Advantagene

The report provides a 6-year forecast (2020-2026) assessed based on how the Gene Therapy market is predicted to grow in major regions likeUSA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others.

Segmentation by Type:Ex vivoIn Vivo

Segmentation by Application:CancerMonogenicInfectious diseaseCardiovascular diseaseOther

Reasons to Buy this Report:

Table of Contents

Table of Contents1 Report Overview

1.1 Study Scope

1.2 Key Market Segments

1.3 Players Covered: Ranking by Gene Therapy Revenue

1.4 Market Analysis by Type

1.4.1 Global Gene Therapy Market Size Growth Rate by Type: 2020 VS 2026

1.4.2 Ex vivo

1.4.3 In Vivo

1.5 Market by Application

1.5.1 Global Gene Therapy Market Share by Application: 2020 VS 2026

1.5.2 Cancer

1.5.3 Monogenic

1.5.4 Infectious disease

1.5.5 Cardiovascular disease

1.5.6 Other 1.6 Study Objectives 1.7 Years Considered2 Global Growth Trends by Regions

2.1 Gene Therapy Market Perspective (2015-2026)

2.2 Gene Therapy Growth Trends by Regions

2.2.1 Gene Therapy Market Size by Regions: 2015 VS 2020 VS 2026

2.2.2 Gene Therapy Historic Market Share by Regions (2015-2020)

2.2.3 Gene Therapy Forecasted Market Size by Regions (2021-2026) 2.3 Industry Trends and Growth Strategy 2.3.1 Market Top Trends 2.3.2 Market Drivers

2.3.3 Market Challenges

2.3.4 Porters Five Forces Analysis

2.3.5 Gene Therapy Market Growth Strategy

2.3.6 Primary Interviews with Key Gene Therapy Players (Opinion Leaders)3 Competition Landscape by Key Players

3.1 Global Top Gene Therapy Players by Market Size

3.1.1 Global Top Gene Therapy Players by Revenue (2015-2020)

3.1.2 Global Gene Therapy Revenue Market Share by Players (2015-2020)

3.1.3 Global Gene Therapy Market Share by Company Type (Tier 1, Tier 2 and Tier 3)

3.2 Global Gene Therapy Market Concentration Ratio

3.2.1 Global Gene Therapy Market Concentration Ratio (CR5 and HHI)

3.2.2 Global Top 10 and Top 5 Companies by Gene Therapy Revenue in 2019

3.3 Gene Therapy Key Players Head office and Area Served

3.4 Key Players Gene Therapy Product Solution and Service

3.5 Date of Enter into Gene Therapy Market

3.6 Mergers & Acquisitions, Expansion Plans4 Breakdown Data by Type (2015-2026)

4.1 Global Gene Therapy Historic Market Size by Type (2015-2020)

4.2 Global Gene Therapy Forecasted Market Size by Type (2021-2026)5 Gene Therapy Breakdown Data by Application (2015-2026)

5.1 Global Gene Therapy Market Size by Application (2015-2020)

5.2 Global Gene Therapy Forecasted Market Size by Application (2021-2026)6 North America

6.1 North America Gene Therapy Market Size (2015-2020)

6.2 Gene Therapy Key Players in North America (2019-2020)

6.3 North America Gene Therapy Market Size by Type (2015-2020)

6.4 North America Gene Therapy Market Size by Application (2015-2020)7 Europe

7.1 Europe Gene Therapy Market Size (2015-2020)

7.2 Gene Therapy Key Players in Europe (2019-2020)

7.3 Europe Gene Therapy Market Size by Type (2015-2020)

7.4 Europe Gene Therapy Market Size by Application (2015-2020)8 China

8.1 China Gene Therapy Market Size (2015-2020)

8.2 Gene Therapy Key Players in China (2019-2020)

8.3 China Gene Therapy Market Size by Type (2015-2020)

8.4 China Gene Therapy Market Size by Application (2015-2020)9 Japan

9.1 Japan Gene Therapy Market Size (2015-2020)

9.2 Gene Therapy Key Players in Japan (2019-2020)

9.3 Japan Gene Therapy Market Size by Type (2015-2020)

9.4 Japan Gene Therapy Market Size by Application (2015-2020)10 Southeast Asia

10.1 Southeast Asia Gene Therapy Market Size (2015-2020)

10.2 Gene Therapy Key Players in Southeast Asia (2019-2020)

10.3 Southeast Asia Gene Therapy Market Size by Type (2015-2020)

10.4 Southeast Asia Gene Therapy Market Size by Application (2015-2020)11 India

11.1 India Gene Therapy Market Size (2015-2020)

11.2 Gene Therapy Key Players in India (2019-2020)

11.3 India Gene Therapy Market Size by Type (2015-2020)

11.4 India Gene Therapy Market Size by Application (2015-2020)12 Central & South America

12.1 Central & South America Gene Therapy Market Size (2015-2020)

12.2 Gene Therapy Key Players in Central & South America (2019-2020)

12.3 Central & South America Gene Therapy Market Size by Type (2015-2020)

12.4 Central & South America Gene Therapy Market Size by Application (2015-2020)13Key Players Profiles

13.1 Bluebird Bio

13.1.1 Bluebird Bio Company Details

13.1.2 Bluebird Bio Business Overview and Its Total Revenue

13.1.3 Bluebird Bio Gene Therapy Introduction

13.1.4 Bluebird Bio Revenue in Gene Therapy Business (2015-2020))

13.1.5 Bluebird Bio Recent Development

13.2 Sangamo

13.2.1 Sangamo Company Details

13.2.2 Sangamo Business Overview and Its Total Revenue

13.2.3 Sangamo Gene Therapy Introduction

13.2.4 Sangamo Revenue in Gene Therapy Business (2015-2020)

13.2.5 Sangamo Recent Development

13.3 Spark Therapeutics

13.3.1 Spark Therapeutics Company Details

13.3.2 Spark Therapeutics Business Overview and Its Total Revenue

13.3.3 Spark Therapeutics Gene Therapy Introduction

13.3.4 Spark Therapeutics Revenue in Gene Therapy Business (2015-2020)

13.3.5 Spark Therapeutics Recent Development

13.4 Dimension Therapeutics

13.4.1 Dimension Therapeutics Company Details

Original post:

Gene Therapy Market 2020-2026: Key Vendor Landscape By Regional Output, Demand By Countries And Future Growth|Bluebird Bio, Sangamo, Spark...

Biogen is planning to trial its Spinraza spinal muscular atrophy (SMA) drug in patients who have not responded properly to Novartis gene therapy Zolgensma, combining the two ultra-expensive therapies to treat the rare muscle-wasting disease.

In the US, a single shot of Zolgensma (onasemnogene abeparvovec) costs $2.1 million, while Spinraza (nusinersen) costs $750,000 in its first year.

Biogen said it aims to begin a phase IV study as in a long-term follow-up study of Novartis drug it has been reported that some patients have been subsequently treated with Spinraza.

The phase IV RESPOND study aims to test efficacy and safety of Spinraza with a suboptimal clinical response to Zolgensma.

So far four out of 10 patients in a long-term study of Zolgensma have been subsequently treated with Spinraza, Biogen said.

Based on the planned study design, RESPOND will be a two-year, open-label study to evaluate the efficacy and safety of Spinraza in SMA patients previously treated with Zolgensma to further inform treatment decisions.

Efficacy will be assessed by change from baseline on motor function measures and additional clinical outcomes such as swallowing and caregiver burden.

Neurofilament levels, an exploratory endpoint, will also be evaluated as a marker of biological disease activity.

The primary study group aims to include 40 infants aged nine months or younger at the time of the first dose of Spinraza, who have two copies of SMN and are likely to develop type 1 disease and received Zolgensma at six months old or younger.

A second study group will include 20 children and will generate data in patients with a broader age range, up to three years old at the time of the first Spinraza dose.

After a screening period, participants will receive the approved 12 mg dose of Spinraza, which is four loading doses followed by maintenance doses every four months, over the two-year study period.

If approved by regulators the company aims to begin enroling patients in the first quarter of next year.

While Zolgensma is a gene therapy that aims to correct the genetic defect that causes the disease, Spinraza works by increasing the amount of full-length SMS protein that is critical to maintaining motor neurons.

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Biogen plans trial of Spinraza in patients not responding to SMA gene therapy - - pharmaphorum

PALM BEACH, FL, July 22, 2020 /PRNewswire/ -- The rising incidence of liver cancer and growing research activities for developing novel treatments plus the availability of newer drugs and treatments coupled with rising awareness among consumer/patients regarding liver cancer, are the factors that are driving the market. A report from Fior Markets said that the global liver cancer treatment market is expected to reach USD 2,186.63 Million by 2025, at a CAGR of 19.85% from 2018 to 2025. The report said that: "Hepatic or liver cancer is one of the most commonly prevalent types of cancer It is known for its aggressive nature and poor survival rate. Certain medical conditions such as Hepatitis B and C viral infections, type 2 diabetes, excess body weight, and liver cirrhosis are known to be the leading causes for the onset of liver cancer. Consumption of alcohol, tobacco is acknowledged to exponentially increase the risk of liver cancer. Thus, increasing research activities in the development of the novel drug and therapeutic systems are anticipated to propel the global liver cancer treatment market. Hepatocellular carcinoma, or primary liver cancer, tends to grow and expand at a fast rate if it is not detected early. This means that people who have been diagnosed with it may not survive longer than 10 to 11 months. The studies show that in the United States, hepatocellular carcinoma is the ninth leading cause of cancer-related deaths. To treat it, specialists will usually prescribe therapy with a drug called "sorafenib." But unfortunately, this drug can typically prolong survival by only 3 months, and it can have numerous adverse effects." Active biotech and pharma companies in the markets this week include: Q BioMed Inc. (OTCQB: QBIO), OPKO Health, Inc. (NASDAQ: OPK), Midatech Pharma plc (NASDAQ: MTP), Genprex, Inc. (NASDAQ: GNPX), Onconova Therapeutics, Inc. (NASDAQ: ONTX).

The report continued: "Factors such as rising incidence of liver cancer and growing research activities for developing novel treatments coupled with increasing government funding for the cancer treatment research are driving the global liver cancer treatment market. Growing use of immunotherapy drugs in the combination drug treatments is leading major drug manufacturers to develop and launch the newer medicines Growth government funding has provided a fillip for the cancer treatment research activities." It also said that the due to its increasing adoption in the combination drug treatments the immunotherapy segment is projected to grow at a CAGR of 21.63% over the forecast period."

Q BioMed Inc. (OTCQB: QBIO) BREAKING NEWS: Q BIOMED TO SCALE UP PRODUCTION OF UTTROSIDE-B, A NOVEL CHEMOTHERAPEUTIC FOR LIVER CANCER, IN PREPARATION FOR IND FILING - Q BioMed and Chemveda Life Sciences are very pleased to continue their collaboration on Uttroside-B, a chemotherapeutic that has shown remarkable potential efficacy as a treatment for liver cancer.

While focused on its commercial rollout of Strontium89, this drug development program will advance another important asset in the Company's portfolio towards monetization. The efficacy of Uttroside-B, a potent saponin, against liver cancer was demonstrated in a preclinical study published in the November 2016 issue of Scientific Reports, a Nature journal.

In the study, researchers showed that in animal models, Uttroside-B was ten times more cytotoxic to the HepG2 liver cancer cell line than sorafenib, the only drug approved by the Food and Drug Administration for liver cancer at the time, and the current first line treatment for hepatocellular carcinoma. Uttroside-B drastically shrunk tumors in mice bearing human liver cancer xenografts. In addition, in pre-clinical experiments Uttroside-B induced cytotoxicity in all liver cancer cell lines, and researchers were also able to confirm its biological safety, both by in vitro and in vivo studies.

Denis Corin, Q BioMed CEO said, "Having successfully completed a very challenging synthesis program, we are pleased to be able to advance this important asset towards the clinic and the patients we hope to treat. Liver cancer incidence rates have more than tripled since 1980, while the death rates have more than doubled during this time. More than 800,000 people are diagnosed with this cancer each year throughout the world and it accounts for more than 700,000 deaths annually. New, more effective treatments for these patients is vital and we are encouraged by the early data we have seen with our molecule."

Q BioMed and its partners in the project, the Oklahoma Medical Research Foundation and The Rajiv Gandhi Centre for Biotechnology (RGCB),will now advance the most promising candidate into preclinical testing and validation over the next few months in anticipation of an Orphan Drug application and an Investigational New Drug (IND) application for a clinical program.

The Uttroside B technology is covered by a provisional patent application. To see the full Scientific Reports study, go to:http://www.nature.com/articles/srep36318 Read this entire release and more news for QBIO at: https://www.financialnewsmedia.com/news-qbio/

Other recent developments in the biotech industry include:

OPKO Health, Inc. (NASDAQ: OPK) BioReference Laboratories, Inc., an OPKO Health company, recently announced it will accept an Indefinite Delivery Indefinite Quantity (IDIQ) contract award to provide Commercial Surge Capacity Testing for COVID-19 Emergency Response to the Centers for Disease Control and Prevention (CDC).

Under the contract with the CDC, BioReference will perform antibody testing to determine COVID-19 seroprevalence, and will provide results with key demographic information and analysis in collaboration with the CDC. The agreement's period of performance beganJuly 20, 2020, and is ongoing throughNovember 19, 2020.

Midatech Pharma plc (NASDAQ: MTP) a drug delivery technology company focused on improving the bio-delivery and bio-distribution of medicines recently announced that electronic copies of its Annual Report and Accounts for the year ended 31 December 2019 are now available from the Company's investor relations website.

Midatech Pharma PLC (dual listed on LSE AIM: MTPH; andNASDAQ: MTP) is an R&D company focused on 'Making Medicines Better' by improving delivery of drugs in the body. The Company combines existing medications with its proprietary and innovative drug delivery technologies to provide compelling oncology and rare disease products that have the potential to powerfully impact the lives of patients undergoing treatment for life threatening diseases.

Genprex, Inc. (NASDAQ: GNPX) a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, recently announced that Dr. George K. Gittes, MD of the University of Pittsburg, the lead researcher that developed the Company's potentially curative diabetes gene therapy, was awarded a grant of $2.59 million from the NIH National Institute of Diabetes and Digestive and Kidney Diseases. The grant will assist Dr. Gittes's development for his research project titled, "Alpha Cell Conversion to Beta Cells in Non-human Primates" and will build upon his accumulating groundbreaking gene therapy work toward finding a cure for diabetes. In this project, Dr. Gittes' research team will conduct important proof-of-principle studies in non-human primates as the last steps in preparation for human gene therapy clinical trials. This technology is the subject of an exclusive license agreement entered into between Genprex and the University of Pittsburgh in February of 2020.

"We are excited to receive this funding to support our research in diabetic primates as we move toward human clinical trials," said Dr. George Gittes, Co-Scientific Director and Professor of Surgery at the UPMC Children's Hospital of Pittsburgand the lead researcher behind the diabetes gene therapy. "We saw encouraging data in our preclinical mice studies, where the gene therapy reprogrammed pancreatic cells to restore normal blood glucose levelsin diabetic mice for approximately four months, which could translate to decades in humans. More recently, preliminary results in non-human primates (monkeys) has also been very promising."

Onconova Therapeutics, Inc. (NASDAQ: ONTX) recently announced the e-publication of results from a Phase 1 company-sponsored study of oral rigosertib in combination with standard dose azacitidine in the treatment of patients diagnosed with either higher-risk myelodysplastic syndrome (HR-MDS) or acute myeloid leukemia (AML) in the international hematological malignancy journalLeukemia Research.

"A key strategy emerging in the treatment of MDS is the identification of safe and effective combinations, particularly those involving oral agents. The results from this Phase 1 study represent Onconova's first efforts to explore oral rigosertib in combination with azacitidine to address the unmet medical need in patients with MDS and AML. We anticipate meeting with the FDA, in conjunction with the pivotal data readout from the INSPIRE Trial, for alignment with the agency on a registration trial for the combination of oral rigosertib plus azacitidine in HMA-nave HR-MDS," said Steven M. Fruchtman, M.D., President and CEO of Onconova.

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Orphan Drug Development to Target Increasing Incidence of Liver Cancer - PRNewswire

Global Gene Therapy for Age-related Macular Degeneration Market 2020 by Manufacturers, Countries, Type and Application, Forecast to 2025 launched by MarketsandResearch.biz aims to unveil key opportunities available in the global market to help players in securing a strong market position in the market. The report provides a detailed analysis of the market status, investment plans, production and consumption, price trends, and analysis by the market player, by region, by type, by the application. The report comprises verified and reliable market forecasts for the overall size of the global Gene Therapy for Age-related Macular Degeneration market in terms of revenue. The report discusses all major market aspects with an expert opinion on current market status along with historic data. A comprehensive analysis of the market standard, geographical regions, market key vendors, end-user applications, and products are studied in this study report.

The report embodies market outlook, modern-day methods, institution, size, revenue, and trends of the global Gene Therapy for Age-related Macular Degeneration market from 2020-2025. The report discloses various opportunities, growth and restraining factors, and challenges faced by major market players. Further, all the major probabilities relevant to market circumstances are showcased by clearly explaining segmentation analysis. The geographical research provides accurate value and volume forecasts, thereby helping market players gain profound insights into the market.

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Top Players:

Competitors analysis includes market shares for all the companies listed below, competitors analysis revenue chart, competitive dashboard, and the competitors latest strategies. Market top players can utilize this analysis to increase the upper hand over their rivals in the market. Along with, the rise and falls that resist the global Gene Therapy for Age-related Macular Degeneration market major contribution towards the rising of the key players in the leading market is discussed.

Influential players covered up in the report: RetroSense Therapeutics, REGENXBIO, AGTC,

By type, the market is segmented into: Subretinal, Intravitreal, Unspecified

By application, the market is segmented into: Monotherapy, Combination Therapy,

The analysis can be also bifurcated industry around the grounds of locations North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Colombia etc.), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa) to test the development pattern of this market at numerous geographic locations.

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Moreover, the report gives production, consumption, sales, and other market forecasts. The document contains the details associate with sales channels, suppliers, traders, dealers, research findings and conclusion, etc for the Gene Therapy for Age-related Macular Degeneration market. In-depth global market progress and outlook linked with the factors boosting the market are explained in the report.

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Global Gene Therapy for Age-related Macular Degeneration Market 2020 with COVID-19 Impact Analysis and Forecast by 2025 - NJ MMA News

Gene Therapy for Ovarian Cancer Market report involves all together a different chapter on COVID 19 Impact. The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries including Life Science, and many more. Trade barriers are further restraining the demand- supply outlook. nicolas.shaw@cognitivemarketresearch.com or call us on +1-312-376-8303.Download The report Copy form the webstie: https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-ovarian-cancer-market-report

The major players profiled in this report include: Takara Bio, VBL Therapeutics, CELSION, Targovax

Market segment by type can be split into: Intravenous, Intratumoral, Intraperitoneal

Market segment by the application can be split into: Ovarian Cancer (unspecified), Recurrent Ovarian Epithelial Cancer, Platinum Resistant Ovarian Cancer

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As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Gene Therapy for Ovarian Cancer globally. This report on Gene Therapy for Ovarian Cancer provides the analysis on impact on Covid-19 on various business segments and country markets. The report also showcases market trends and forecast to 2027, factoring the impact of COVID-19 situation.

Gene Therapy for Ovarian Cancer Market report provide an in-depth understanding of the cutting-edge competitive analysis of the emerging market trends along with the drivers, restraints, and opportunities in the market to offer worthwhile insights and current scenario for making right decision. The report covers the prominent players in the market with detailed SWOT analysis, financial overview, and key developments of last three years. Moreover, the report also offers a 360 outlook of the market through the competitive landscape of the global industry player and helps the companies to garner Gene Therapy for Ovarian Cancer Market revenue by understanding the strategic growth approaches.

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Report provides industry analysis, important insights, and a competitive and useful advantage to the pursuers. The report analyzes different segments and offers the current and future prospects of each segment. Furthermore, this research report contains an in depth analysis of the top players with data such as product specification, company profiles and product picture, sales area, and base of manufacturing in the global Gene Therapy for Ovarian Cancer market. The impact on the supply and demand of the raw materials, due to the COVID-19 is also analyzed in the global Gene Therapy for Ovarian Cancer market.

Additionally, report consists of product life cycle, which discus about the current stage of product. Further, it adds manufacturing cost analysis as well as complete manufacturing process involved. Report also adds supply chain analysis to ensure complete data of market.

Objectives of Gene Therapy for Ovarian Cancer Market Report:To justifiably share in-depth info regarding the decisive elements impacting the increase of industry (growth capacity, chances, drivers and industry specific challenge and risks)To know the Gene Therapy for Ovarian Cancer Market by pinpointing its many sub segmentsTo profile the important players and analyze their growth plansTo endeavor the amount and value of the Gene Therapy for Ovarian Cancer Market sub-markets, depending on key regions (various vital states)To analyze the Global Gene Therapy for Ovarian Cancer Market concerning growth trends, prospects and also their participation in the entire sectorTo inspect and study the Global Gene Therapy for Ovarian Cancer Market size form the company, essential regions/countries, products and applications, background information and also predictions to 2027Primary worldwide Gene Therapy for Ovarian Cancer Market manufacturing companies, to specify, clarify and analyze the product sales amount, value and market share, market rivalry landscape, SWOT analysis and development plans for the next coming yearsTo examine competitive progress such as expansions, arrangements, new product launches and acquisitions on the market

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Follow is the chapters covered in Gene Therapy for Ovarian Cancer Market:Chapter 1 Gene Therapy for Ovarian Cancer Market OverviewChapter 2 COVID 19 ImpactChapter 3 Gene Therapy for Ovarian Cancer Segment by Types (Product Technology)Chapter 4 Global Gene Therapy for Ovarian Cancer Segment by ApplicationChapter 5 Global Gene Therapy for Ovarian Cancer Market by Regions (2015-2027)Chapter 6 Global Gene Therapy for Ovarian Cancer Market Competition by ManufacturersChapter 7 Company (Top Players) Profiles and Key DataChapter 8 Global Gene Therapy for Ovarian Cancer Revenue by Regions (2015-2020)Chapter 9 Global Gene Therapy for Ovarian Cancer Revenue by TypesChapter 10 Global Gene Therapy for Ovarian Cancer Market Analysis by ApplicationChapter 11 North America Gene Therapy for Ovarian Cancer Market Development Status and OutlookChapter 12 Europe Gene Therapy for Ovarian Cancer Market Development Status and OutlookChapter 13 Asia Pacific Gene Therapy for Ovarian Cancer Market Development Status and OutlookChapter 14 South America Gene Therapy for Ovarian Cancer Market Development Status and OutlookChapter 15 Middle East & Africa Gene Therapy for Ovarian Cancer Market Development Status and OutlookChapter 16 Gene Therapy for Ovarian Cancer Manufacturing Cost AnalysisChapter 17 Marketing Strategy Analysis, Distributors/ TradersChapter 18 Global Gene Therapy for Ovarian Cancer Market Forecast (2020-2027)Chapter 19 Research Findings and ConclusionGet detailed TOC for Gene Therapy for Ovarian Cancer Market Report @ https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-ovarian-cancer-market-report#table_of_contents.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team, who will ensure that you get a report that suits your needs. You can also get in touch with our executives on to share your research requirements.nicolas.shaw@cognitivemarketresearch.com or call us on +1-312-376-8303.

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

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Drivers and Restraints of Gene Therapy for Ovarian Cancer Market Research Report 2020 Top Compnaies Include, Takara Bio, VBL Therapeutics, CELSION,...

Encoded Therapeutics, Inc., a South Francisco, Calif.-based precision gene therapy company, raised $135m in Series D financing.

The round was led by GV (formerly Google Ventures) with participation from Matrix Capital Management, ARCH Venture Partners, Illumina Ventures, RTW Investments, Boxer Capital, Nolan Capital, HBM Genomics, Menlo Ventures, Meritech Capital, Farallon Capital Management, SoftBank Vision Fund 21, and additional unnamed investors. In conjunction with the funding, David Schenkein, M.D., general partner and co-leader of GVs life sciences team, will join the Encoded Board of Directors as an observer.

Led by co-founder and chief executive officer, Kartik Ramamoorthi, Ph.D., Encoded is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. It is advancing its lead asset, ETX101, for the treatment of SCN1A+ Dravet Syndrome by leveraging a discovery engine that combines biological and computational approaches to identify and screen human DNA sequences known as regulatory elements at a high throughput scale.

The company intends to use the funds to advance its lead asset, ETX101, which was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for the treatment of SCN1A+ Dravet Syndrome.

Proceeds from the Series D will be used to conduct clinical trial activities including a natural history study to better understand the progression of SCN1A+ Dravet Syndrome as well as first-in-human trials for ETX101. Additionally, the funds will support progression of the companys pipeline of gene therapies being evaluated for additional pediatric CNS disorders.

FinSMEs

23/07/2020

Original post:

Encoded Therapeutics Raises $135M in Series D Financing - FinSMEs

XconomyNew York

A gene therapy being jointly developed by MeiraGTx and Johnson & Johnson to treat an inherited disease of the retina that leads to blindness improved patients vision after six months, data that the companies say is enough to move the potentially long-lasting treatment into pivotal testing.

The condition, called X-linked retinitis pigmentosa, is part of a group of vision disorders that collectively represent the most common cause of inherited blindness. X-linked RP, or XLRP, the most aggressive form of retinitis pigmentosa, significantly impairs patients quality of life as the disease progresses. No treatments currently exist.

The MeiraGTx (NASDAQ: MGTX) treatment is designed for patients whose XLRP is caused by mutations in the gene RPGR, which cause about 70 percent of XLRP cases and up to 20 percent of all retinitis pigmentosa cases. MeiraGTxs gene therapy is delivered via subretinal injection.

A Phase 1/2 trial, underway at five sites in the US and UK, tested three doses in 10 male patients age 5 and older for safety and tolerability. In each patient one eyethe one with worse sightwas treated; the other eye was used as a control.

Secondary goals included retinal sensitivity, which after six months had improved or stabilized in five of the seven in the low and intermediate dose groups, according to the interim data. No improvement was observed in the high dose cohort; it wont be tested in future trials. Signs of improvement were detected at the first assessment, which took place at three months, improvements that were generally sustained or increased at the next evaluation.

Most of the adverse events recorded were associated with the surgical procedure used to deliver the treatment, according to MeiraGTx; two of three patients in the high-dose cohort experienced inflammation, which was controlled with steroids.

The data were presented at the annual meeting of the American Society of Retina Specialists, which was held virtually. The interim results support advancing the treatment into a Phase 3 triala first for MeriaGTx, which was founded about five years ago, president and CEO Zandy Forbes said on a conference call Friday.

Johnson & Johnson (NYSE: [[ticker:J&J]]) made a bet on the gene therapy pipeline being developed by MeiraGTx in January 2019, when the companies inked a $100 million deal giving the pharma giant rights to the biotechs investigational XLRP treatment, plus others in its pipeline, as part of a pact to jointly develop and commercialize gene therapies to treat inherited retinal diseases. Under the terms of that deal, MeiraGTx is eligible for up to $340 million more based on the programs progression.

Including the XLRP program, MeiraGTxwhich has offices in London and New Yorkis advancing six clinical-stage programs, half of them under its collaboration with J&J. The companys initial focus is on diseases that impact the eye, central nervous system, and salivary glands.

The first-ever gene therapy approved in the US, Spark Therapeutics voretigene neparvovec (Luxturna), was for patients with an inherited retinal disease that can lead to blindness. Its 2018 approval led to the companys acquisition the following year by Roche, which splashed out $4.8 billion for the Philadelphia-based firm. Spark priced its treatment at $850,000.

Image: iStock/monkeybusinessimages

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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MeiraGTx, J&J See Phase 3 Test Ahead for Vision Loss Gene Therapy - Xconomy

Global Gene Therapy Market Research Report 2020: COVID-19 Outbreak Impact Analysis

The Gene Therapy market research report drafted by Brand Essence Market Research elucidates relevant market and competitive insights as well as regional and consumer information. In a nutshell, the research study covers every pivotal aspect of this business sphere that influences the existing trends, profitability position, market share, market size, regional valuation, and business expansion plans of key players in the Gene Therapy market.

Download Premium Sample of the Report: https://brandessenceresearch.biz/Request/Sample?ResearchPostId=165836&RequestType=Sample

Latest research report on Gene Therapy Market delivers a comprehensive study on current market trends. The outcome also includes revenue forecasts, statistics, market valuations which illustrates its growth trends and competitive landscape as well as the key players in the business.

Macular degeneration is a condition in which, macula, a part of the retina, gets damaged or deteriorated. This condition usually affects individuals who are aged 50 years and above and therefore, it is called age-related macular degeneration (AMD). AMD is the leading cause of vision loss and is directly related to the advancement of age. But smoking also plays a vital role in causing AMD. AMD is characterized by the presence of a blurred area near the center of vision that leads to distorted vision. There are two different types of AMD, including dry (atrophic) AMD (dAMD) and wet (neovascular/exudative) AMD (wAMD). The dAMD is the most common type of AMD and accounts for almost 80%-90% of the overall AMD cases.

It has been observed that age-related macular degeneration (AMD) is one of the major causes for vision loss and is characterized by the formation of a blurred area near the center of vision, a condition that mostly affects the geriatric population. According to the CDC, almost 2 million individuals in the US suffer from AMD and by 2050, this number will reach more than 5 million. This will subsequently demand the need for the development of innovative treatments for AMD, driving the markets growth.

The market research analysts have predicted that with the introduction of techniques such as fluorescein angiography, the global age-related macular degeneration market will register a CAGR of more than 7% by 2020. With the unavailability of FDA-approved treatment for dry AMD (dAMD) and the treatment of wet AMD (wAMD) involving the need of intravitreal injections for an indefinite period, gene therapy is emerging as the most-efficient approach for the treatment of age-related macular degeneration (AMD).

According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for wet AMD (wAMD). Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

This report focuses on the global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China.

The key players covered in this study RetroSense Therapeutics REGENXBIO AGTC

Market segment by Type, the product can be split into Subretinal Intravitreal Unspecified

Market segment by Application, split into Monotherapy Combination Therapy

In this study, the years considered to estimate the market size of Gene Therapy for Age-related Macular Degeneration are as follows: History Year: 2014-2018 Base Year: 2018 Estimated Year: 2019 Forecast Year 2019 to 2025

Market segment by Regions/Countries, this report covers United States Europe China Japan Southeast Asia India Central & South America

The study objectives of this report are: To analyze global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. To present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China. To strategically profile the key players and comprehensively analyze their development plan and strategies. To define, describe and forecast the market by product type, market and key regions.

For the data information by region, company, type and application, 2018 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

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Our industry professionals are working reluctantly to understand, assemble and timely deliver assessment on impact of COVID-19 disaster on many corporations and their clients to help them in taking excellent business decisions. We acknowledge everyone who is doing their part in this financial and healthcare crisis.

The Essential Content Covered in the GlobalGene Therapy Market Report:

* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

The report provides a 6-year forecast (2020-2026) assessed based on how the Gene Therapy market is predicted to grow in major regions like USA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others.

Strategic Points Covered in TOC:

Chapter 1:Introduction, market driving force product scope, market risk, market overview, and market opportunities of the global Gene Therapy market.

Chapter 2:Evaluating the leading manufacturers of the global Gene Therapy market which consists of its revenue, sales, and price of the products.

Chapter 3: the competitive nature among key manufacturers, with market share, revenue, and sales.

Chapter 4:Presenting global Gene Therapy market by regions, market share and revenue and sales for the projected period.

Chapters 5, 6, 7, 8 and 9:To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions.

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Brandessence market research publishes market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students. We have a delivery center in Pune, India and our sales office is in London.

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Investment Analysis On: Gene Therapy MARKET ? GROWING TRADE AMONG EMERGING ECONOMIES OPENING NEW OPPORTUNITIES (2020-2026) - 3rd Watch News

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (NASDAQ: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, today announced that Dr. George K. Gittes, MD of the University of Pittsburgh, the lead researcher that developed the Companys potentially curative diabetes gene therapy, was awarded a grant of $2.59 million from the National Institutes of Health (NIH) National Institute of Diabetes and Digestive and Kidney Diseases. The grant will assist Dr. Gittess development for his research project titled, Alpha Cell Conversion to Beta Cells in Non-human Primates and will build upon his accumulating groundbreaking gene therapy work toward finding a cure for diabetes. In this project, Dr. Gittes research team will conduct important proof-of-principle studies in non-human primates as the last steps in preparation for human gene therapy clinical trials. This technology is the subject of an exclusive license agreement entered into between Genprex and the University of Pittsburgh in February of 2020.

We are excited to receive this funding to support our research in diabetic primates as we move toward human clinical trials, said Dr. George Gittes, Co-Scientific Director and Professor of Surgery at the UPMC (University of Pittsburgh Medical Center) Childrens Hospital of Pittsburgh and the lead researcher behind the diabetes gene therapy. We saw encouraging data in our preclinical mice studies, where the gene therapy reprogrammed pancreatic cells to restore normal blood glucose levels in diabetic mice for approximately four months, which could translate to decades in humans. More recently, preliminary results in non-human primates (monkeys) has also been very promising.

Dr. Gittes gene therapy for diabetes, which Genprex refers to as GPX-002, uses a novel infusion process comprised of an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. The genes express proteins that transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the bodys immune system.

Diabetes affects approximately 10 percent of the U.S. population, or more than 34 million people. The diabetes gene therapy could not only become a new treatment option for millions of diabetes patients who need insulin replacement therapy, but it holds the potential to provide long-term effectiveness, or may even be a cure, for diabetic patients.

Dr. Gittes is the inventor of the gene therapy for diabetes, and he is eligible to receive royalties on this technology in the future.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to in-license and develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, GPX-001 (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). GPX-001 has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. GPX-001 has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for GPX-001 for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone. For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

The project described in this press release is being supported by the National Institute of Diabetes and Digestive And Kidney Diseases of the National Institutes of Health under Award Number R01DK120377. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of GPX-001, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including GPX-002, our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our product candidates; the timing and success at all of obtaining FDA approval of GPX-001 and our other potential product candidates including whether we receive fast track or similar regulatory designations; costs associated with developing our product candidates and whether patents will ever be issued under patent applications that are the subject of our license agreements. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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National Institutes of Health (NIH) Awards Research Grant of $2.59 Million to University of Pittsburgh for Diabetes Gene Therapy Technology Licensed...

SHANGHAI and SUZHOU, China, July 23, 2020 /PRNewswire/ --Internationally renowned clinical-stage immune cell and gene therapy company, Gracell Biotechnologies Co., Ltd. ("Gracell" or "the Company") has announced two appointments to the executive suite. Chief Medical Officer Dr. Martina Sersch, MD and Chief Financial Officer Kevin Xie, PhD, MBA both bringing extensive knowledge and leadership experience to Gracell. Along with CEO Dr. William Cao, Dr. Sersch and Dr. Xie joined Gracell in their respective functions to support the company's mission of providing novel cellular therapies for unmet medical needs.

"We are delighted that Dr. Martina Sersch and Dr. Kevin Xie joined Gracell," said Dr. William Cao, CEO of Gracell. "Gracell has achieved many significant milestones since its founding, and with the recent addition of Dr. Sersch and Dr. Xie to our executive leadership team, Gracell will be advancing faster to achieve future breakthroughs."

CMO Dr. Martina Sersch brings over 25 years of experience in academia and industry, as well as an exceptional wealth of talent and knowledge to the Company. Prior to Gracell, Martina was CMO of a Nasdaq-listed CAR-T and gene therapy company, leading the development of novel cell and gene therapies for rare diseases and oncology indications. In addition, she previously served roles of increasing responsibility in Oncology Development as well as Medical Affairs at Pfizer, Genentech/Roche and Amgen, including leading the development of CAR-T therapies, gene therapies and immuno-oncology products in hematology and solids tumors. She oversaw the development and successful filing activities of multiple early- and late-stage products regionally and globally including US, Europe and Asia. Dr. Sersch received her MD and doctorate degree from the University of Heidelberg in Germany.

"Gracell has made significant achievements in multiple projects developing FasTCAR and TruUCARTM platform which were recently presented at major conferences in the treatment for hematological malignancies and solid tumors," said Dr. Martina Sersch, "As CMO, I am excited to continue to lead an incredible team with very talented researchers in our quest in developing innovative medicines for people with high unmet medical need.

CFO Dr. Kevin Xie came to Gracell with extensive healthcare finance and investment experience in leadership positions. Prior to Gracell, Dr. Xie was most recently the President of Fosun Healthcare Holdings and Chief Representative of Fosun in New York. Before Fosun, Dr. Xie served in senior positions at multiple global healthcare investment funds for over ten years, including as the co-founder of Locust Walk Capital, healthcare sector head at Scopia Capital and a managing director at Great Point Partners. Dr. Xie holds an MBA from The Wharton School and a PhD in Chemistry from the City University of New York.

"After twenty years of investing and advising healthcare companies, I am excited to join Gracell to lead the Company's finance and business development activities, and together with R&D team to build Gracell to be a leader in cell and gene therapy industry," said Dr. Kevin Xie, "I can see Gracell is poised to translate its industry-leading research programs into the transformative treatments, which could change millions of patients' lives worldwide. By joining and leading Gracell's finance organization at this exciting stage of its development, I wish to help deliver that promise as the company progresses to the next phase of strategy."

About Gracell

Gracell Biotechnologies Co., Ltd. ("Gracell") is a clinical-stage biotechnology company, committed to developing highly efficacious and affordable cell and gene therapies for the treatment of cancer. Gracell is dedicated to resolving the remaining challenges in CAR-T, such as high production costs, lengthy manufacturing process, lack of off-the-shelf products, and inefficacy against solid tumors. Led by a group of world-class scientists, Gracell is advancing FasTCAR (overnight production and younger cell phenotype), TruUCAR (off-the-shelf), DualCAR and EnhancedCAR T cell therapies for leukemia, lymphoma, myeloma, and solid tumors.

CONTACT:

Kevin XIECFO[emailprotected]

Linc HEBusiness Development and Public Relations[emailprotected] +86-21-6403-1375

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http://www.gracellbio.com

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Gracell Expands Executive Team by Appointment of CMO Dr. Martina Sersch and CFO Dr. Kevin Xie - PRNewswire

At the Forum, GenScriptannounced the integration and upgrading of the original CDMO platform and the release of its latest brand GenScript ProBio. The new brand is dedicated to proactively providing end-to-end service from discovery to commercialization withprofessional solutions and efficientprocesses to accelerate drug development for customers.

For the past 16 years, since 2004, GenScript has gradually expanded its reach in the antibody drug discovery segment and established its capabilities in process development, helping many customers with their IND approvals during the period. In 2019, GenScript opened the Biologics Development Business Unit (BDBU), the predecessor of GenScript ProBio, and invited Dr. Brian Min, SeniorVicePresidentatSamsungBioepis, to join as CEO and form a management team with exceptional international perspectives. The core members of the team each have more than 15 years working experience at recognized international firms across the sector, including Roche, Pfizer, Henlius and Innovent, among others.

GenScript ProBio has established long-term partnerships with many industry leaders worldwide with the specific aim of advancing the development of the biopharmaceutical industry.

"The GenScript CDMO platform has established strong antibody capabilities within just 18 months since launch and has accumulated considerable experience in cell and gene therapy," said Dr. Brian Min at the launch of the brand."GenScript ProBio's one-stopantibody drug development solutions will proactively provide clients with end to end service from target to commercialization. Looking beyond China and the Asia Pacific region, many large multinational companies in the United States and Europe have also established productive partnerships with us. Operating under the principle of'Innovation through Collaboration', we fully expect to drive the development and progress of the whole industry together with our customers."

GenScript ProBio has been committed to accelerating the development of the cell therapy sector and helping firms within the sector accelerate the commercialization of cell therapy products. With the advent of the era of cell and gene therapy, GenScript ProBio plans to continue providing process optimization and production in line with international quality standards through a sound global supply chain network, empowering more partners worldwide to benefit patients anywhere at an early date.

GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO arm of the world's leading biotech company GenScript, providing end-to-end services from drug discovery to commercialization with proactive strategies, professional solutions and efficient processes to accelerate drug development for customers.

In line with the mission of "Innovation through Collaboration", GenScript ProBio is committed to helping customers shorten the time-to-market for biological drugs from discovery to commercialization, significantly lowering R&D costs and building a healthier future.

https://www.genscriptprobio.com/

SOURCE GenScript ProBio

https://www.genscriptprobio.com/

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GenScript launches CDMO segment "GenScript ProBio" at inaugural GenScript Cell and Gene Therapy Industry Development & Cooperation Forum...

The Global Hemophilia Gene Therapy Market report focuses on market size, status and forecast 2020-2027, along with this, report also focuses on market opportunities and treats, risk analysis, strategic and tactical decision-making and evaluating the market. The Hemophilia Gene Therapy market report provides data and information on changing investment structure, technological advancements, market trends and developments, capacities, and detail information about the key players of the global Maarket_Keyword market. In addition to this, report also involves development of the Hemophilia Gene Therapy market in major region across the world.

Key Players for Global Hemophilia Gene Therapy Market:

The global Hemophilia Gene Therapy market report profiles major key players of the market on the basis of business strategies, financial weaknesses and strengths and recent development.

Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline Therapeutics

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The Hemophilia Gene Therapy market report also states demand and supply figures, revenue, production, import/export consumption as well as future strategies, sales volume, gross margins, technological developments, cost and growth rate. The Global Hemophilia Gene Therapy Market report also delivers historical data from 2015 to 2020 and forecasted data from 2020 to 2027, along with SWOT analysis data of the market. This report includes information by types, by application, by region and by manufacturers or producers.

The recent outburst of the COVID-19 (Corona Virus Disease) has led the global Hemophilia Gene Therapy market to render new solutions for combatting with the rising demand for protection against the virus. Due to this outbreak, remote patient monitoring, inpatient monitoring and interactive medicine is expected to gain grip at this time.

Global Hemophilia Gene Therapy Market: Segmentation

Global Hemophilia Gene Therapy Market Segmentation: By Types

Hemophilia AHemophilia B

Global Hemophilia Gene Therapy Market segmentation: By Applications

Hemophilia A Gene TherapyHemophilia B Gene Therapy

Global Hemophilia Gene Therapy Market Segmentation: By Region

Global Hemophilia Gene Therapy market report categorized the information and data according to the major geographical regions like,

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The Global Hemophilia Gene Therapy market is displayed in 13 Chapters:

Chapter 1: Market Overview, Drivers, Restraints and OpportunitiesChapter 2: Market Competition by ManufacturersChapter 3: Production by RegionsChapter 4: Consumption by RegionsChapter 5: Production, By Types, Revenue and Market share by TypesChapter 6: Consumption, By Applications, Market share (%) and Growth Rate by ApplicationsChapter 7: Complete profiling and analysis of ManufacturersChapter 8: Manufacturing cost analysis, Raw materials analysis, Region-wise manufacturing expensesChapter 9: Industrial Chain, Sourcing Strategy and Downstream BuyersChapter 10: Marketing Strategy Analysis, Distributors/TradersChapter 11: Market Effect Factors AnalysisChapter 12: Market ForecastChapter 13: Hemophilia Gene Therapy Research Findings and Conclusion, Appendix, methodology and data source

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Global Hemophilia Gene Therapy Market 2020 Impact of COVID-19, Future Growth Analysis and Challenges | Spark Therapeutics, Ultragenyx, Shire PLC,...

Ohio-based startup Forge Biologics closed a $40 million Series A financing round that will be used to expand manufacturing capabilities and support the clinical development of a gene therapy program for rare diseases, including the typically fatal Krabbe disease.

Forge Biologics is a viral vector contract development manufacturing organization (CDMO) and therapeutics developer. The company plans to use proceeds from the funding round to expand its adeno-associated virus (AAV) manufacturing capabilities, as well as its gene therapy pipeline. Its lead program is FBX-101, a novel AAV and umbilical cord transplant combination approach to treat infantile Krabbe disease, a devastating neurodegenerative disorder. Krabbe disease has a predicted incidence of about 1 in 12,000 people in the U.S. with symptoms developing in babies and rapidly progressing to death by age two. Forge anticipates FBX-101 will enter the clinic later this year.

FBX-101 was initially developed in the laboratory of Maria Escolar, a professor of pediatrics and director of the Program for the Study of Neurodevelopment in Rare Disorders (NDRD) at the University of Pittsburgh. Escolar will serve as chief medical officer of Forge Biologics. The combination of AAV gene therapy and umbilical cord bone marrow transplant addresses both central and peripheral nerve degeneration associated with Krabbe disease, she said. Recent data showed the combination approach significantly improved efficacy compared to a single AAV administration alone in canines affected with Krabbe disease.

The companys manufacturing facility, known as The Hearth, will host end-to-end cGMP AAV manufacturing services at 500L scale, enabling biotech and pharma clients the ability to accelerate their gene therapy programs from pre-clinical development through clinical and commercial stage manufacturing.

Timothy J. Miller, president and chief executive officer of Forge Biologics, said the young company is a true gene therapy development engine. With AAV manufacturing, its pipeline and a strong leadership team, Miller said the company is ready to bring life-saving therapies for rare diseases into reality.

In addition to Miller, who previously served as CEO of Abeona Therapeutics, and Escolar, Forges leadership team includes Chief Operations Officer Jaysson Eicholtz and Chief Strategy Officer Erandi De Silva. Eicholtz was previously responsible for Nationwide Childrens Hospitals gene therapy manufacturing facility, and Silva was previously head of Program Management at Myonexus Therapeutics.

The Series A financing round was led by Perceptive Xontogeny Venture Fund with participation from Drive Capital. Chris Garabedian, CEO of Xontogeny, will join the Forge Board of Directors as its chairman.

Link:

Forge Biologics Launches with $40 Million to Manufacture and Develop Gene Therapies - BioSpace

RTW backs Encoded Therapeutics In June, RTW Venture Fund participated in a $135m Series D financing round for Encoded Therapeutics, together with other investment firms. Encoded is a US-based privately held preclinical stage biotech company developing precision gene therapies for a broad range of severe genetic disorders.

This was RTWs seventh investment following its IPO in October 2019 and its second gene therapy company.

Yesterday, Encoded announced thatits lead asset, ETX101, was granted Orphan Drug Designation (ODD) and RarePediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for the treatment of SCN1A+Dravet Syndrome. Proceeds from the Series D fundraise will be used to conduct clinical trial activities including a natural history study to better understand the progression of SCN1A+ Dravet Syndrome as well as first-in-human trials for ETX101. Additionally, the funds will support progression of the companys pipeline of gene therapies being evaluated for additional pediatric CNS (central nervous system) disorders.

Dravet syndrome is a rare, severe genetic disorder that occurs in approximately 1 in 16,000 births worldwide.The disorder is characterized by uncontrolled seizures, ataxia, significant developmental delays and anincreased risk of early mortality due to sudden unexpected death in epilepsy (SUDEP). The majority of DravetSyndrome cases are caused by loss-of-function mutations in the SCN1A gene. Current treatments reduceseizures but do not address the underlying cause of the disorder SCN1A haploinsufficiency. More informationabout Dravet Syndrome can be found at http://www.dravetfoundation.org.

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RTW backs Encoded Therapeutics - QuotedData

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Government announces funding for scale up of COVID-19 vaccine with additional investment to support upskilling in Cell and Gene Therapy and Vaccine...

Gene Therapy Market Overview 2020 2025

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

The risingtechnology in Gene Therapy Marketis also depicted in thisresearchreport. Factors that are boosting the growth of the market, and giving a positive push to thrive in the global market is explained in detail.

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Key Competitors of the Global Gene Therapy Market are: , Kite Pharma, BioVex, Novartis, Spark Therapeutics

Historical data available in the report elaborates on the development of the Gene Therapy on national, regional and international levels. Gene Therapy Market Research Report presents a detailed analysis based on the thorough research of the overall market, particularly on questions that border on the market size, growth scenario, potential opportunities, operation landscape, trend analysis, and competitive analysis.

Major Product Types covered are:Ex VivoIn Vivo

Major Applications of Gene Therapy covered are:Cancer DiseasesHematological DiseaseHereditary Disease

This study report on global Gene Therapy market throws light on the crucial trends and dynamics impacting the development of the market, including the restraints, drivers, and opportunities.

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The fundamental purpose of Gene Therapy Market report is to provide a correct and strategic analysis of the Gene Therapy industry. The report scrutinizes each segment and sub-segments presents before you a 360-degree view of the said market.

Market Scenario:

The report further highlights the development trends in the global Gene Therapy market. Factors that are driving the market growth and fueling its segments are also analyzed in the report. The report also highlights on its applications, types, deployments, components, developments of this market.

Highlights following key factors:

:-Business descriptionA detailed description of the companys operations and business divisions.:-Corporate strategyAnalysts summarization of the companys business strategy.:-SWOT AnalysisA detailed analysis of the companys strengths, weakness, opportunities and threats.:-Company historyProgression of key events associated with the company.:-Major products and servicesA list of major products, services and brands of the company.:-Key competitorsA list of key competitors to the company.:-Important locations and subsidiariesA list and contact details of key locations and subsidiaries of the company.:-Detailed financial ratios for the past five yearsThe latest financial ratios derived from the annual financial statements published by the company with 5 years history.

Our report offers:

Market share assessments for the regional and country level segments. Market share analysis of the top industry players. Strategic recommendations for the new entrants. Market forecasts for a minimum of 9 years of all the mentioned segments, sub segments and the regional markets. Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations). Strategic recommendations in key business segments based on the market estimations. Competitive landscaping mapping the key common trends. Company profiling with detailed strategies, financials, and recent developments. Supply chain trends mapping the latest technological advancements.

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According to the Latest Report: Gene Therapy Market is Growing Massively with Leading Players Kite Pharma, BioVex, Novartis, Spark Therapeutics -...

This article explains some of the terminology relating to advanced therapy medicinal products (ATMPs), including gene and cell-based therapies, tissue-engineered products, and combined ATMPs. The author explains the key EU regulations and guidance documents for each therapy type and discusses marketing authorization, accelerated regulatory pathways, and market access. He advises companies navigating this complex regulatory environment to engage with the regulatory agencies early and often during the approval process and to seek external regulatory support if in-house resources are limited.IntroductionAdvanced therapy medicinal products include cell therapies, gene therapies, and tissue-engineered products. These highly complex treatments differ from traditional medicines, both in how they are made and administered and in the type of benefits they may provide. These are products for which regulations were established relatively recently and that present challenges for regulatory agencies on how to best they should be regulated within the existing regulatory framework.TerminologyGene therapiesGene therapies treat disease by replacing, inactivating, or introducing genes into the body (see Figure 1). They deliver a corrected copy of a faulty or missing gene, typically using a vector or carrier molecule. The new gene allows cells to function correctly, alleviating all, or some, disease symptoms and potentially offering a long-term cure.Gene therapy medicinal product refers to a biologic medicinal product, which has the following characteristics:

Cell-based therapiesCell-based therapies involve transplanting substantially manipulated cells or cellular material into a patient. In some cases, the genetic material in the cells may be genetically modified. Cell therapy restores or alters certain cells to deliver therapy. Cells are first modified outside of the body.

Cells may be from the patient (autologous) or from a donor (allogeneic). Autologous therapies are manufactured using cells taken from a patient, which are then re-administered to the same patient. Therefore, each batch is unique and irreplaceable. Allogenic products are those in which batches are manufactured using material from a single donor and then administered to different patients.Somatic cell therapy medicinal product refers to a biological medicinal product, which has the following characteristics:

Guidance documentsThe European Medicines Agency (EMA) provides several guidance documents and operates a specific help desk email address (advancedtherapeis@ema.europe.com):

FIGURE 3 Marketing authorization pathways for ATMPs Reprinted with permission (Detela & Lodge)32Market accessAs of May 2020, 10 ATMPs have been granted marketing authorization in Europe seven gene therapies, one cell therapy, and two tissue-engineered products targeting several diseases in different therapeutic areas. An additional five approved ATMPs were removed from the market for commercial reasons. At the time, marketing authorizations for five gene therapies were pending.38 The high number of approved, but not marketed, products can be explained at least partially by the cost of ATMPs. It is, however, important to note the fundamental differences between conventional therapies and ATMPs (see Table 2).

OutlookNavigating this complex regulatory environment means companies need to engage with the regulatory agencies early and often throughout the approval process. Many of these therapies are developed by scientific institutions and medical research groups with typically limited regulatory resources in-house, so it is advisable to seek external regulatory support. This exciting field, with hundreds of products currently tested in clinical trials,39 should yield many new therapies with enormous benefits. Something to look forward to, particularly in a time of a global pandemic crisis.AbbreviationsATMP, advanced therapy medicinal product; CAT, Committee for Advanced Therapies; CHMP, Committee for Medicinal Products for Human Use; EMA, European Medicines Agency; EU, European Union; GMP, good manufacturing practice; SMEs, small- and medium-sized enterprises.References [All references accessed 16 July 2020]

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Regulation of advanced therapy medicinal products in the EU - Regulatory Focus