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 its Chairman and Chief Executive Officer, Rodney Varner, has participated in a second-round live interview on the Big Biz Show, an emmy-award winning nationally syndicated TV and radio show. A replay of the interview is available for viewing on the Companys website at https://bit.ly/2Esvq05.

During this interview, Mr. Varner discusses:

The Big Biz Show covers current business events, internet-related issues and other hot topics in the business world. The Big Biz Show is seen and heard in over 100 million broadcast TV homes, 150 radio stations in the U.S. and in 175 countries. The Emmy Award winning show was named by TALKERS Magazine as one of the Top 10 Most Influential Financial Shows.

The Big Biz Show can be seen and heard every weeknight on YouTOO America Television Network at 8 p.m. ET, the following morning at 7 a.m. ET, and every weekday on BizTalk Radio at 2 p.m. ET. Additionally, the show can be accessed digitally by visiting BizTVClub.com, where you can watch the Big Biz Show every weeknight at 9 p.m. ET.

To watch Mr. Varners first live appearance on the Big Biz Show in June 2020, visit Genprexs website.

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, Oncoprex, is being evaluated as a treatment for non-small cell lung cancer (NSCLC). Oncoprex 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. Oncoprex 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 Oncoprex immunogene therapy 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.

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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Genprex CEO Participates in a Second-Round Live Interview on the Big Biz Show - Business Wire

No serious adverse events reported through 24 weeks of a 52 week trial.

A single-injection intravitreal gene therapy that can durably express an intraocular anti-VEGF agent could reduce the need for repeated anti-VEGF injections and improve outcomes for patients with neovascular age-related macular degeneration (nAMD).

A team, led by Charles C. Wykoff, MD, PhD, Director of Clinical Research, Retina Consultants of Houston, presented initial findings at theAmerican Society of Retina Specialists (ASRS) 2020 Virtual Meeting on the safety and biological activity of a novel intravitreal anti-VEGF gene therapy in neovascular age-related macular degeneration.

A single-injection intravitreal gene therapy that can durably express an intraocular anti-VEGF agent could reduce the need for repeated anti-VEGF injections and improve outcomes for patients with nAMD.

In the ongoing phase 1 OPTIC study, the investigators are assessing the safety, tolerability, and efficacy of a single intravitreal injection of ADVM-022.

Included in the multicenter, open-label, multiple cohort, dose-ranging study are patients with nAMD who have demonstrated a response to anti-VEGF therapy, who were administered an intravitreal injection of ADVM-022 t 6x10^11 vg/eye for cohort 1 (n = 6) and at 2x10^11 vg/eye for cohort 2 (n = 6).

The investigators evaluated incidence and severity of adverse events, changes in best corrected visual acuity (BCVA), change in central subfield thickness (CST), and the number of aflibercept rescue injections.

Patients in the first cohort previously received frequent anti-VEGF injections, with a mean of 6.2 injections in the preceding 8 months. This patient population also had relatively good baseline BCVA (mean 65.8 ETDRS letters) and a mean baseline CST of 369.2m.

The first 24 weeks of the trial have been positive, with no serious adverse events, dose limiting toxicities or non-ocular adverse events related to ADVM-022 reported.

While ocular inflammation was seen in all patients, it occurred most frequently in the anterior segment and was generally mild and manageable with topical steroids. BCVA was also maintained with a mean change of -2.0 ETDRS letters (90% CI, -9.1-5.1). CST improved with a mean change -52.7m (90% CI, -86.5 to -18.8).

Signs of CNV disease activity on OCT improved or stabilized in the entire patient population, with no rescue injections needed. The study will continue up to 52 total weeks.

ADVM-022 is designed to provide sustained therapeutic levels of aflibercept following a single intravitreal injection, the authors wrote. Patients in cohort 1 of OPTIC treated with a single injection of ADVM-022 were able to maintain vision and improve anatomical outcomes without receiving any rescue aflibercept injections through week 24.

In a separate study presented at ASRS 2020, researchers showed new data on the treatment burden and vision acuity on anti-VEGF injection patterns for nAMD.

In the study, the investigators reviewed the Level 1 evidence of currently approved anti-VEGF agents, as well as those likely to undergo review by regulatory authorities. The researchers analyzed the anti-VEGF agents and treatment dosing regimen for each study and collected the baseline ETDRS letters, mean number of injections over a 12-month period, and change in ETDRS letters over the course of 12 months.

They analyzed a total of 23 injection regiments from studies involving 6860 eyes, including 8 (31.6%, n = 2165) involving ranibizumab every 4 weeks or pro re nata and 6 (28.6%; n = 1962) involving aflibercept dosed every 4 or 8 weeks.

The study, Intravitreal Gene Therapy for Neovascular AMD With ADVM-022: Results of the Phase 1 OPTIC Trial, was published online by ASRS 2020.

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Ongoing Trial Assesses New Anti-VEGF Gene Therapy Injection for nAMD - MD Magazine

According to the latest report published by PMR, the Cancer Gene Therapy market is anticipated to grow at a steady pace over the forecast period (2019-2029). The report sheds light on the various trends and restraining factors that are expected to shape the growth of the Cancer Gene Therapy in the upcoming years. The report ponders over the various parameters that are expected to impact revenue generation, sales, and demand for the Cancer Gene Therapy in the various regional markets.

According to the study, the Cancer Gene Therapy market is likely to attain a market value of ~US$ XX by 2019 and grow at a CAGR of ~XX% during the assessment period. The market study introspects the competition landscape of the Cancer Gene Therapy market and highlights the key developments and technological innovations witnessed in the current Cancer Gene Therapy market landscape.

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Key findings of the Cancer Gene Therapy market report:

Cancer Gene Therapy Market Segmentation

The report dissects the Cancer Gene Therapy market into different segments to provide a fair understanding of the different aspects of the Cancer Gene Therapy market.

The regional analysis of the Cancer Gene Therapy market sheds light on the growth prospects of the Cancer Gene Therapy market in different regions. The current market trends, the impact of regulatory policies, market share, size, and value of each regional market are presented in the report supported by easy-to-understand graphs and tables.

Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech

Key points covered in the report

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Key Questions Related to the Cancer Gene Therapy Market Addressed in the Report

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Increasing Government Investments and Favorable Policies to Aid the Growth of the Cancer Gene Therapy Market2020 - Owned

With reliable and impactful research methodologies, PMR provides critical information pertaining to the growth of the global Cancer Gene Therapy market. Our team of analysts monitor the ongoing developments within the Cancer Gene Therapy space and provide an unbiased assessment of the global Cancer Gene Therapy market. The data included in the report are procured from reliable and trustworthy primary and secondary sources.

According to the findings of the report, the value of the global Cancer Gene Therapy market in 2018 was ~US$ XX (Mn/Bn) and expected to attain a value of ~US$ XX (Mn/Bn) by the end of 2029. In addition, the report reveals that the global Cancer Gene Therapy market is likely to grow at a CAGR of XX% during the forecast period (2019-2029).

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Some of the leading companies profiled in the market study include:

The Cancer Gene Therapy market report provides an extensive analysis of the different product types including:

The presented market study includes a brief introduction of the Cancer Gene Therapy market to enhance the reading experience of our users. Further, a thorough quantitative and qualitative analysis of each of these segments is provided in the report along with graphs, tables, and figures to support the data.

Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech

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Key information drawn from the Cancer Gene Therapy market study

The market report aims to address the following queries:

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Consumer Demand for Eco-friendly Products Set to Boost the Prospects of the Cancer Gene Therapy Market2020 - Owned

NEW YORK, July 27, 2020 /PRNewswire/ -- Report Scope:The scope of this study encompasses an investigation of the markets cell and gene therapy tools such as GMP proteins, media, cell separation and activation reagents, viral and non-viral, cytokine release syndrome monitoring products, GMP antibodies, leukapheresis instrumentation, immunoassays (multiplex and singleplex) and bioreactors.The analyst examines each tool type, determines its current market status, examines its impact on future markets, and presents forecasts of growth over the next five years.

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Technological issues, including the latest trends, are discussed. The analyst examined the industry on a worldwide basis, from both application and demand perspectives, in the major regions of the world.

Report Includes:- 77 data tables- An overview of the global market for immunoassay-based cell and gene therapy tools, and reagents within the life sciences industry- Analyses of the global market trends, with data corresponding to market size for 2017-2019, and projections of compound annual growth rates (CAGRs) through 2024- Information about in vitro diagnostics (IVD), its background and importance of IVD testing- Discussion on prevalence of infectious disease, metabolic disorders and chronic ailments as drivers of immunoassay testing market- Description of recent technologies such as chemiluminescent immunoassays (CLIA), fluorescent immunoassays, multiplex assays and rapid diagnostic technologies- Evaluation of market potential for immunoassay-based cell and gene therapy tools and reagents, current market size and market forecast, and market share analysis of the leading players engaged- Patent review and new developments, R&D efforts, industrial changes with emphasis on recent investments, and current state of the immunoassay technology- Company profiles of the key market participants, including Bio-Rad Laboratories, Cytiva Life Sciences, Luminex Corp., Millipore Sigma, Thermo Fisher Scientific and STEMCELL Technologies

Reasons for Doing this Study:Gene and cell therapy are emerging as important tools to treat human health.Techniques such as CAR-T therapy have emerged as key ways of treating many different types of cancers.

The promise of gene therapy using technologies such as CRISPR is starting to be realized in clinical trials, and markets are scaling up to treat other diseases as well, particularly rare gene- based diseases.As these therapies are coming to the fore, a new market for tools to develop these therapies using standard methodologies is emerging.

This report will cover what those tools are, how they impact the larger life science tools market, and how they will evolve over the next five years.

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Cell and Gene Therapy Tools, and Reagents: Global Markets - cnweekly

For Immediate Release: July 24, 2020

Today, the U.S. Food and Drug Administration approved Tecartus (brexucabtagene autoleucel), a cell-based gene therapy for treatment of adult patients diagnosed with mantle cell lymphoma (MCL) who have not responded to or who have relapsed following other kinds of treatment. Tecartus, a chimeric antigen receptor (CAR) T cell therapy, is the first cell-based gene therapy approved by the FDA for the treatment of MCL.

Tremendous progress has been made in the discovery of new therapies for debilitating diseases that are difficult to treat. This approval is yet another example of customized treatments that use a patients own immune system to help fight cancer, while using a scientific advance in this promising new area of medicine, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research. Were seeing continued advances in the field of gene therapy and remain committed to supporting innovation in this promising new area of medicine.

MCL is a rare form of cancerous B-cell non-Hodgkins lymphoma that usually occurs in middle-aged or older adults. In patients with MCL, B-cells, a type of white blood cell which help the body fight infection, change into cancer cells that start to form tumors in the lymph nodes and quickly spread to other areas of the body.

Each dose of Tecartus is a customized treatment created using a patients own immune system to help fight the lymphoma. The patients T cells, a type of white blood cell, are collected and genetically modified to include a new gene that facilitates the targeting and killing of the lymphoma cells. These modified T cells are then infused back into the patient.

The safety and efficacy of Tecartus was established in a multicenter clinical trial of 60 adults with refractory or relapsed MCL who were followed for at least six months after their first objective disease response. The complete remission rate after treatment with Tecartus was 62 percent, with an objective response rate of 87 percent.

The label carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and for neurologic toxicities. Both CRS and neurologic toxicities can be fatal or life-threatening. The most common side effects of Tecartus include serious infections, low blood cell counts and a weakened immune system. Side effects from treatment usually appear within the first one to two weeks after treatment, but some side effects may occur later.

Because of the risk of CRS and neurological toxicities, Tecartus is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). The risk mitigation measures for Tecartus are identical to those of the current REMS Program for another CAR-T therapy, Yescarta.

To further evaluate the long-term safety of Tecartus, the FDA is also requiring the manufacturer to conduct a post-marketing observational study involving patients treated with Tecartus.

Tecartus was approved under the Accelerated Approval pathway and was granted Priority Review and Breakthrough Therapy designations. Tecartus also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The Tecartus application was reviewed using a cross-agency approach. The clinical review was coordinated by the FDA's Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination. The FDA remains committed to supporting safe and effective treatment options that have the potential of providing lifesaving results.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

###

07/24/2020

Link:

FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL - FDA.gov

Impact Analysis of Covid-19

The complete version of the Report will include the impact of the COVID-19, and anticipated change on the future outlook of the industry, by taking into the account the political, economic, social, and technological parameters.

Get Sample PDF Including COVID19 Impact Analysis Of Personalized Gene Therapy Treatments for Cancer, https://www.coherentmarketinsights.com/insight/request-pdf/60

The Personalized Gene Therapy Treatments for Cancer Market is expected to have a highly positive outlook for the next five years 2020-2027 according to a recently released Personalized Gene Therapy Treatments for Cancer Market research report. The report has been added to his large database by Coherent Market Insights. This report is a guide that covers key strategic developments of the market including acquisitions & mergers, new technology launch, agreements, partnerships, collaborations & joint ventures, research & development, technology, and regional expansion of major participants involved in the market on the global and regional basis.

It also going to elaborate the opportunities out there in micro niches for stakeholders to take a position, step by step investigation of the competitive landscape and even commodity professional services of famous players which include Amgen, Inc., SynerGene Therapeutics, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., Cold Genesys, Inc., Takara Bio, Inc., Bellicum Pharmaceuticals, Inc., Ziopharm Oncology, Inc., OncoSec Medical, Inc., Sevion Therapeutics, Inc., and Burzynski Clinic.

The key features of the Personalized Gene Therapy Treatments for Cancer Market report 2020-2027 are the organization, extensive amount of analysis, and data from previous and current years as well as forecast data for the next five years. Most of the report is made up of tables, charts, and figures that give our clients a clear picture of the market.

The comprehensive value chain analysis of the Personalized Gene Therapy Treatments for Cancer market will assist in attaining better product differentiation, along with a detailed understanding of the core competency of each activity involved. The market attractiveness analysis provided in the report measures the potential value of the Personalized Gene Therapy Treatments for Cancer providing business strategists with the latest growth opportunities.

Questions have been answered at Personalized Gene Therapy Treatments for Cancer Market report:

Economy Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, along with Strategies) The length of the global Personalized Gene Therapy Treatments for Cancer market opportunity? How share promote Personalized Gene Therapy Treatments for Cancer their worth from different manufacturing brands? Which will be the niches at which players profiling with thorough plans, financials, and also recent advancements should set a presence? Which will be the anticipated growth rates for your own Personalized Gene Therapy Treatments for Cancer economy altogether and also for every segment inside? Which will be the Personalized Gene Therapy Treatments for Cancer application and types and forecast accompanied closely by producers?

The research report on the Personalized Gene Therapy Treatments for Cancer market evaluates the growth trends of the industry through historical study and estimates future prospects based on comprehensive research. The report extensively provides market share, growth, trends, and forecasts for the period 2020-2027. The market size in terms of revenue (USD MN) is calculated for the study period along with the details of the factors affecting the market growth (drivers and restraints).

Key Topics Covered:

Part 01: Executive SummaryPart 02: Scope Of The ReportPart 03: Research MethodologyPart 04: Market Landscape Market Ecosystem Market Characteristics Market Segmentation Analysis

Part 05: Market Sizing Market Definition Market Sizing 2020 Market Size And Forecast 2020-2027

Part 06: Five Forces Analysis Bargaining Power of Buyers Bargaining Power of Suppliers Threat of New Entrants Threat of Substitutes Threat of Rivalry Market Condition

Part 07: Customer LandscapePart 08: Regional Landscape Geographical Segmentation Regional Comparison Americas Market Size And Forecast 2020-2027 EMEA Market Size And Forecast 2020-2027 APAC Market Size And Forecast 2020-2027

Part 09: Decision FrameworkPart 10: Drivers And Challenges Market Drivers Market Challenges

Part 11: Market TrendsPart 12: Vendor Landscape Overview Landscape Disruption Vendors Covered Vendor Classification Market Positioning Of Vendors

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Covid 19 Analysis On Personalized Gene Therapy Treatments for Cancer Market 2020 Top 10 Countries Exclusive Data, Demand, Share and Growth Analysis by...

The UKs capability to manufacture vaccines has received a substantial boost today (Thursday 23 July), as the government announces an additional 100 million to ensure that any successful COVID-19 vaccine can be produced at scale in the UK.

The investment will fund a state-of-the-art Cell and Gene Therapy Catapult Manufacturing Innovation Centre to accelerate the mass production of a successful COVID-19 vaccine in the UK. Due to open in December 2021, the Centre will have the capacity to produce millions of doses each month, ensuring the UK has the capabilities to manufacture vaccines and advanced medicines, including for emerging diseases, far into the future.

Located in Braintree, Essex, the government initiative will upgrade an existing facility to create a fully-licensed manufacturing centre. Doing so will increase the UKs ability to respond to diseases like coronavirus and to prepare for potential future pandemics while creating new, high-skilled jobs to fuel the UKs economic recovery.

The new centre will complement the Vaccines Manufacturing and Innovation Centre (VMIC), which is currently under construction in Oxfordshire thanks to a 93 million investment from the government. Once complete next year, the facility will have the capacity to produce enough vaccine doses to serve the entire UK population at scale.

While the centre is under construction, the government has invested an additional 38 million to establish a rapid deployment facility, opening later this summer, that will support efforts to ensure a successful vaccine is widely available to the public as soon as possible.

Business Secretary Alok Sharma said:

We are taking all necessary steps to ensure we can vaccinate the public as soon as a successful COVID-19 vaccine becomes available.

This new Cell and Gene Therapy Catapult Manufacturing Innovation Centre, alongside crucial investment in skills, will support our efforts to rapidly produce millions of doses of a coronavirus vaccine while ensuring the UK can respond quickly to potential future pandemics.

To support these enhanced vaccine manufacturing capabilities, the government will invest an additional 4.7 million for the Cell and Gene Therapy Catapult to ensure that the UK has the best skills and expertise through the development of virtual and physical national Centres for Advanced Therapies Training and Skills, in partnership with industry.

The facilities and online training platform will provide industry-standard skills and experience in advanced gene therapy and vaccine manufacturing, including sterile techniques for Good Manufacturing Practice which is the minimum standard that a medicines manufacturer must meet in their production processes.

Employment in the cell and gene therapy sector is predicted to reach over 6,000 jobs by 2024, with over 3,000 in manufacturing and bioprocessing.

Matthew Durdy, CEO, Cell and Gene Therapy Catapult commented:

This commitment from government through the Vaccines Taskforce will enable continued growth and productivity in the cell and gene therapy sector, as well as providing vital resource for vaccine manufacturing and economic recovery from the COVID-19 pandemic.

We are delighted to be able to deploy the specialist capabilities of the Cell and Gene Therapy Catapult in such an important initiative. Accelerating the availability of COVID-19 vaccines, increasing skills and employment, and facilitating growth of the advanced medicines industry will make a valuable contribution to the recovery of the economy.

Kate Bingham, Chair of the Vaccines Taskforce said:

Todays announcement is another important milestone for us. The work of the Vaccines Taskforce is focused on protecting the UK against COVID-19 through vaccination as quickly as possible.

In order to vaccinate our high-risk populations at the earliest opportunity, the government has agreed to proactively manufacture vaccines now, so we have millions of doses of vaccine ready if they are shown to be safe and effective. The acquisition of this state-of-the-art manufacturing centre will not only help us with this, but also ensures we are well-placed as a country to be able to cope with any pandemics or health crises in the future.

As well as addressing the immediate need to produce a COVID-19 vaccine, the new Cell and Gene Therapy Centre, developed with Innovate UK and the Cell and Gene Therapy Catapult, will be at the forefront of the growing UK cell and gene therapy industry. Scientists and researchers based in the centre will accelerate the time taken for new treatments to be delivered to patients by developing cutting-edge therapies to treat life changing diseases, such as diabetes, heart disease and cancer.

The UK is at the forefront of international efforts to research and develop a COVID-19 vaccine and has provided 131 million funding to University of Oxford and Imperial College London to accelerate their work on 2 vaccine candidates.

This follows news on Monday (20 July) that the government secured early access to 90 million vaccine doses from the BioNTech/Pfizer alliance and Valneva as part of its strategy to build a portfolio of promising new vaccines to protect the UK from COVID-19. In addition, treatments containing COVID-19-neutralising antibodies have been secured from AstraZeneca to protect those who cannot receive vaccines.

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation.

With more than 230 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies.

The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit the Cell and Gene Therapy Catapult or Innovate UK.

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Over 100 million cash boost to manufacture millions of doses of COVID-19 vaccine - GOV.UK

SEATTLE, July 27, 2020 /PRNewswire/ --AGC Biologics, a Global Biopharmaceutical Contract Development and Manufacturing Organization (CDMO), has announced that they will acquire the majority of the share capital of Molecular Medicine S.p.A. ("MolMed") on July 31, 2020. MolMed is a biotechnology company focused on research, development, production and clinical validation of cell and gene therapies for the treatment of cancer and rare diseases. AGC Biologics is now one of the very few CDMO's in the world offering both plasmid production and end-to-end cell and gene therapy services.

Cell and gene therapy is an innovative and rapidly growing therapeutic field that aims to treat diseases that do not have adequate treatments to date. Approximately 1,000 clinical trials are underway worldwide, with some products receiving market authorization in the last few years and approximately 50 new therapies expected to be authorized by 2030. Thanks to its two commercially authorized facilities, MolMed offers GMP services for the development and production of cell and gene therapies. MolMed brings deep experience and expertise in providing development and GMP manufacturing services for viral vectors and genetically modified cells, from the preclinical phase through commercial demand.

"While we work hard to take care of each other and our customers during this very challenging and uncertain time, it's also important that we ensure the continued growth of our company," says AGC Biologics CEO Patricio Massera. "AGC Biologics is committed to continuously expanding our offerings and growing our capacity to serve all the needs of current and future customers. We are very pleased to be adding MolMed and its great cell and gene therapy capabilities and track record to AGC Biologics' global CDMO service offerings."

About AGC Biologics:AGC Biologics is a leading global Contract Development and Manufacturing Organization (CDMO) with a strong commitment to deliver the highest standard of service to clients and partners. The company currently employs more than 1,000 employees worldwide. AGC Biologics' global network spans three continents, with cGMP-compliant facilities in Seattle, Washington; Boulder, Colorado; Copenhagen, Denmark; Heidelberg, Germany; and Chiba, Japan.

AGC Biologics offers deep industry expertise and unique customized services for the scale-up and cGMP manufacture of protein-based therapeutics, from pre-clinical to commercial mammalian and microbial production. Integrated service offerings include plasmid (GMP pDNA) manufacturing, cell line development, bioprocess development, formulation, analytical testing, antibody drug development and conjugation, cell banking and storage and protein expression, including the proprietary CHEF1 Expression System for mammalian production. Learn more at http://www.agcbio.com.

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AGC to Complete the Acquisition of MolMed on July 31, 2020 - NBC Right Now

When you think of a typical meal in the US South, you will likely recall the rich, hearty dishes that have immortalized themselves in the food culture of the Southern States. Fried chicken, mashed potatoes, gravy, macaroni and cheese; its all mouth-watering stuff, but of course there is a catch. Foods like these are loaded with fats and, as a result, calories. A diet heavy in such dishes can have a detrimental impact on your health and wreak havoc on your heart and circulatory system.

The CDC has highlighted the fact that, as a result of such diets, various forms of heart disease kill more Southerners than any other disease. One such example can be found in the fact that Southern Counties record a much higher death rate for diseases such as strokes than the rest of America. Strokes are fast acting and deadly, occurring every 40 seconds in the United States, claiming a life every four minutes. Thats 140,000 deaths a year, the third highest total from any disease. Globally, strokes are an epidemic and the second highest cause of death.

While a satisfying cure remains elusive, advances in biotechnology are offering new hope to the millions of people who experience strokes.

What is a stroke?

A stroke is triggered when a blood vessel in the brain gets blocked or bursts. Striking when the blood supply to part of the brain is cut off, a stroke deprives brain cells (neurons) of their critical supply of oxygen. If neurons go without oxygen for more than a few minutes, they start to die by the millions, so it is critical to act fast if you see any signs of onset. Typically a patient will exhibit distinct physical symptoms such as facial drooping, inability to raise arms and slurred speech, and the stroke usually occurs in one of two ways. During an ischemic stroke, the blood supply is stopped by a clot in a blood vessel, which accounts for roughly 85 percent of cases. A hemorrhagic stroke, in contrast, occurs when a blood vessel within the brain becomes weak and bursts.

Regardless of which scenario occurs, a stroke poses a serious threat to the health of a patient. A constant blood supply is the single most essential component in keeping the brain operational. Every cell within the body relies on oxygen from the blood in order to make energy and function, and the brain is a very oxygen-hungry organ. Despite its relatively small size, your brain requires 20 percent of your total oxygen supply. The damage can, therefore, be severe and irreversible if the blood supply to the brain is cut off for more than 10 minutes. For this reason, it is essential that current stroke therapies are administered quickly, but many stroke victims are still likely to suffer long-term consequences that can take years to correct. Consequently, scientists work tirelessly to find better therapies for stroke patients.

Treatment shortfalls and arduous recovery

The need for new and innovative therapies for stroke patients is driven by limitations in currently approved medical interventions. There are two current drug regimens used to treat strokes. Anticoagulants such as warfarin break up existing bloodclots, preventing the most common types of strokes. Antiplatelets such as clopidogrel (Plavix) can be used to help prevent blood clots from forming altogether. But neither one of these drug types repairs the brain damage often associated with strokes.

Additionally, surgery can be performed to treat brain swelling and help prevent further bleeding, but the lingering problem is that patients are still often left with long-term issues after suffering a stroke. Recovery can be a long and arduous process because of the difficulty the brain has in replacing the cellular connections that are lost when neurons die as a result of a stroke. When the cells die, the functions they are responsible for are lost. Regaining those functions often involves a long course of reablement therapies aimed at teaching the brain to build new connections and re-learn skills that were lost after a stroke.

Why gene therapy provides promise

Research indicates that in many instances, a brain can heal itself after a stroke. Cells that are damaged are not beyond repair. They can regenerate. Researchers in the field are pushing for pharmaceutical options to help speed up this regeneration of the lost network of brain cells. This is where gene therapy could make a real difference. Gene therapy offers scientists the tools required to genetically reprogram cells to help speed up the regeneration process during stroke recovery.

The use of gene therapy is still very much in its infancy, with a lot of research underway to ensure the technology can be implemented safely and effectively. Direct administration of DNA into the brain offers the advantage of producing high concentrations of therapeutic agents in a relatively localized environment. Gene transfer also provides longer duration of effect than traditional drug therapy. Recent studies have shown a lot of promise.

In one such study, researchers at Penn State University developed a gene therapy platform using a gene called neurogenic differentiation 1 (NeuroD1). The study, which was conducted on mice, investigated the potential of using a retrovirus to deliver NeuroD1 directly into the brains of mice that had suffered from a stroke. Researchers found that the therapy converted glial cells into the neuronal cells that are critical to regenerating lost brain tissue. Mice treated with this novel gene therapy not only lost less brain tissue but also showed a significant improvement in motor function. According to the leader of the research team, Prof Gong Chen:

The biggest obstacle for brain repair is that neurons cannot regenerate themselves. Many clinical trials for stroke have failed over the past several decades, largely because none of them can regenerate enough new neurons to replenish the lost neurons. I believe that turning glial cells that are already present in the brain into new neurons is the best way to replenish the lost neurons.

In another study from Florida Atlantic University, a research team explored the potential of using a protein called granulocyte-colony stimulating factor (G-CSF) to give cells in the brains of mice the genetic push they needed to trigger regeneration after stroke. The results were, once again, very positive, with treated mice showing an improvement in prognosis after stroke. The cells were more resistant to damage-induced death and this significantly improved behavioral functions in treated mice.

This therapy provides another promising avenue, but study co-author Prof Howard Prentice emphasized the need for further investigation:

Future research will need to focus on uncovering the complete mechanisms by which GCSF retains the ER and mitochondrial homeostasis.

While Prentice underscored the preliminary nature of this research, the data generated so far indicate gene therapies are extremely promising avenues for improving the lives of stroke patients. And just last month, a team at the University of San Diego Medical School released a study showing that just a single treatment that inhibits a gene known as PTB resulted in the disappearance of symptoms associated with Parkinsons disease and other neurodegenerative impairments.

Its my dream to see this through to clinical trials, to test this approach as a treatment for Parkinsons disease, but also many other diseases where neurons are lost, such as Alzheimers and Huntingtons diseases and stroke, said Xiang-Dong Fu, head of the research team. And dreaming even bigger what if we could target PTB to correct defects in other parts of the brain, to treat things like inherited brain defects?

The positive nature of Xiang-Dong Fus outlook is something we can all feel inspired by. At present, the road to recovery for stroke patients is long and their quality of life can suffer. But we have the potential to kick-start that recovery and help the brain rebuild the precious connections that enable stroke victims to lead healthier lives. For now, theres no easy answer; preventing a stroke remains a better option than treating and recovering from one. But the constant influx of advances in gene therapy could provide a quicker route to repairing the severe damage when it does occurdramatically boosting the quality of life for millions of people.

Sam Moxon has a PhD in regenerative medicine and is currently involved in dementia research. He is a freelance writer with an interest in the development of new technologies to diagnose and treat degenerative diseases. Follow him on Twitter @DrSamMoxon

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Strokes often deadly but gene therapy offers hope to rebuild critically damaged brain cells - Genetic Literacy Project

Global Gene Therapy for Age-related Macular Degeneration (COVID-19) Market Report 2020 by Key Players, Types, Applications, Countries, Market Size, Forecast to 2026 (Based on 2020 COVID-19 Worldwide Spread)

The Gene Therapy for Age-related Macular Degeneration market report provides a detailed analysis of global market size, regional and country-level market size, segmentation market growth, market share, competitive Landscape, sales analysis, the impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunities analysis, strategic market growth analysis, product launches, area marketplace expanding, and technological innovations.

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Latest research report on Gene Therapy for Age-related Macular Degeneration 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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Gene Therapy for Age-related Macular Degeneration Market 2020 | Present Scenario and Growth Prospects 2025 - Bulletin Line

TheGlobal Gene Therapy Market Is Expected To Reach Around USD 2,269 Million By 2024report covers all of the aspects required to gain a complete understanding of the pre-market conditions, current conditions as well as a well-measured forecast.This report also researches and evaluates the impact of Covid-19 outbreak on theGene Therapy Market, involving potential opportunity and challenges, drivers and risks. We present the impact assessment of Covid-19 effects onGene Therapy Market growth forecast based on different scenario (optimistic, pessimistic, very optimistic, most likely etc.).

Theresearch report published by Zion Market Researchis a comprehensive study of the globalGene Therapy Market. The subject matter experts and team of highly-skilled researchers have put in hours of work to collate an authentic research report on the globalGene Therapy Market. Analysts have studied the various products in the market and offered an unbiased opinion about the factors that likely to drive the market and restrain it. For a detailed study, researchers have used primary and secondary research methodologies. Analysts have also studied the key milestones of achieved by the globalGene Therapy Marketand compared it to the current market trends to give the readers a holistic picture of the market.

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GlobalGene Therapy Market: Drivers and Restraints

This section of the report assesses various drivers, opportunities, and restrains that lie in the market. These drivers and restraints are determined by various factors such as region, key players, innovations, and others. The report will help readers determine the key drivers and solutions for restraints. It also highlights the possible opportunities. The drivers and restraints are identified by current trends and historic milestones achieved by the market. The chapter on drivers and restraints also offers an evaluation of the investments made in production innovation through the years. The changes in environmental perspective have also been factored in to understand their impact on the growth of the globalGene Therapy Market.

The Leading Market Players Covered in this Report are:

UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics

Analysts have also highlighted the potential restraints present in the globalGene Therapy Market. With the help of market experts the report points out what changes companies can make to overcome these hurdles over the forecast years.

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GlobalGene Therapy Market: Segment Analysis

The market has various segments such as applications, end users, and products. These help in determining the growth of a particular segment of a market. The readers can assess why a certain segment is performing better than the other and then make strategic investments. The type segment includes sales value for the forecast period of 2014 to 2025. The application segment includes sales by volume and consumption for the forecast period of 2014 to 2025.

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GlobalGene Therapy Market: Regional Analysis

Different regions of the global market influence growth differently. Various factors such as economic growth, technological developments, government policies, availability of labour, and others are compared with each to determine which region will outperform other. The regions included in this report are North America, Europe, Asia Pacific, and the Middle East and Africa.

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Strategic Points Covered in TOC:

Chapter 1:Introduction, market driving force product scope, market risk, market overview, and market opportunities of the globalGene Therapy Market

Chapter 2:Evaluating the leading manufacturers of the globalGene Therapy Market which consists of its revenue, sales, and price of the products

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

Chapter 4:Presenting globalGene Therapy Market by regions, market share and with revenue and sales for the projected period

Chapter 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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The classification of the globalGene Therapy Marketis done based on the product type, segments, and end-users. The report provides an analysis of each segment together with the prediction of their development in the upcoming period. Additionally, the latest research report studies various segments of the globalGene Therapy Marketin the anticipated period.

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

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ZMR Revises Gene Therapy Market Forecast, as COVID-19 Continues to Expand Quickly Across the Globe - Market Research Posts

GW Pharmas Epidiolex made history in 2018 when the cannabis-based treatment scored an FDA nod for Dravet syndrome, a genetic disorder marked by intractable seizures. But children with Dravet face much more than just seizures. Enter Encoded Therapeutics, which just raised $135 million to advance a precision gene therapy that could treat the disease in its totality.

The series D financing will bankroll a natural history study, slated for the second half of 2020, of Dravet syndrome stemming from mutations in the SCN1A gene, as well as a phase 1 study to start in 2021. Drawn from a long list of marquee investors including GV, Matrix Capital Management and Arch Venture Partners, the capital will also support a clutch of earlier-stage gene therapies for other pediatric central nervous system (CNS) disorders.

Encoded aims to surmount hurdles in the gene therapy space, including the treatments ability to zero in on specific cells, their potency and their ability to control endogenous genes, CEO Kartik Ramamoorthi, Ph.D., told Fierce Biotech in a previous interview.

RELATED: Encoded Therapeutics bags $104M to propel 'precision gene therapy' for Dravet syndrome

Rather than "knock out" defective genes, introduce a new gene into the body or replace a faulty gene with a healthy copy, Encoded targets pieces of DNA that control gene expression. Its technology screens for and identifies sequences in the human genome dubbed regulatory elements because they control the expression of other genes.

Theyre fundamentally important as to why different types of cells are unique and operate differently, Ramamoorthi said. We can incorporate these sequences into any gene delivery vehicle of interest; were focused on adeno-associated viruses as an initial application.

Its lead program, ETX101, is designed to remedy mutations in the SCN1A genewhich causes the majority of Dravet casesby boosting expression of SCN1A in a specific cell type in the brain. This way, Encoded aims to tackle gait problems, impaired coordination and developmental delays.

Encoded hasnt disclosed its programs beyond ETX101, but Ramamoorthi said they target diseases that are similar to Dravet syndrome.

Many of these pediatric disorders result in cell type-selective dysfunction, meaning a particular type of cell in the central nervous system is not operating appropriately, usually due to a single gene dysfunction, he said.

RELATED: Taysha Gene Therapies hits the ground running with $30M, 15 programs

The latest financing comes about a year after Encodeds $104 million series C round. Since then, the company has filled out its team with gene therapy trailblazers like Chief Manufacturing OfficerAndrew Stoberand Chief Regulatory OfficerNancy Boman, M.D., Ph.D., both of whom were on the AveXis team that developed and submitted the gene therapy Zolgensma for FDA approval.

Theyve done it before in gene therapy and theyre hungry for more, Ramamoorthi said.

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Encoded Therapeutics bags $135M to push 'precision gene therapy' into the clinic - FierceBiotech

After weeks of buildup and controversial delays, Moderna has launched its pivotal study to determine whether their Covid-19 vaccine actually works. And BARDA is committing another $472 million to get them there.

The 30,000 person study will test whether two 100 g doses of their mRNA vaccine can prevent people from becoming symptomatic with Covid-19. The launch marks the beginning of a second stage in the hunt for a coronavirus vaccine, as the most well-backed and advanced efforts having been tested for safety and immunogenicity in small groups of volunteers move into studies that will determine whether they can actually prevent Covid-19 and be available in the fall or winter to stem the pandemic.

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GV's David Schenkein comes in to help mentor and bankroll a gene therapy 2.0 player, intrigued by their 'science heavy' approach to conquering...

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As more than 7,000 cell and gene therapies progress through the development pipeline and only 152 CMOs have the capabilities to manufacture them, production bottlenecks are the major obstacle to large-scale commercial manufacture once more Advanced Therapy Medicinal Products (ATMPs) are approved, experts say.

In a panel discussion called Discover/Develop: But How Will you Manufacture Your Therapeutic? at BIO Digital Conference 2020 on June 8, John Erickson, Chief Technology Officer at NIIMBL (Newark, DE, US), said the problems lie with flexibility, process control, speed, sustainability, and capital costs. Bottlenecks, especially in viral vector manufacturing, and slow processing will limit wide-scale commercial manufacture of ATMPs, he said.

Panellist Sue Behrens, Professor in Bioprocessing and Director of the Amgen Bioprocessing Center at Keck Graduate Institute (Claremont, CA, US), agreed, highlighting product stability and sterility as the big challenges, stemming from a poor understanding of how technologies interact. Process control for ATMPs is inherently difficult due to variability in the biologic starting material, but real-time biosensor readings could fix this by allowing adjustments during the process, she said.

The largest CMOs are predicting great future demand and have significantly invested in ATMP manufacturing capabilities. In May 2019, Thermo Fisher Scientific (Waltham, MA, US) acquired Brammer Bio (Cambridge, MA, US) for $1.7B, and Catalent (Somerset, NJ, US) acquired Paragon Bioservices (Baltimore, MD, US) for $1.2B in May 2019 and later MaSTherCell Global (Hainaut, Belgium) for $315M in February 2020. Despite demand being unpredictable and many problems still existing with the ATMP manufacturing process, there has been consistent and sustained spending on enhancing related capabilities (particularly for the viral vector bottleneck issue) even over recent months, as Table 1 shows.

Table 1: CMO Investments in API Biologics Cell, Gene, Vaccine, and Virus, AprilJuly 2020

Currently, there are only 34 marketed gene and cell therapies globally. It is a relatively new and high-value treatment, with Gilead Sciences Incs (Foster City, CA, US) Yescarta (axicabtagene ciloleucel), Novartis AGs (Basel, Switzerland) Zolgensma (onasemnogene abeparvovec-xioi), and Novartis Kymriah (tisagenlecleucel) being particularly lucrative in terms of 2019 revenues. However, as Figure 1 shows, there are a large number of ATMPs in the drug pipeline. In future, they will become increasingly approved and therefore improvements to the production process are required to remove manufacturing bottlenecks and enable cost-efficient manufacture at a commercial scale.

Figure 1: Advanced Therapy Medicinal Products in the Drug Pipeline

Source: GlobalData, Pharma Intelligence Center Drugs Database (Accessed: 8 July 2020) 2020 GlobalData Plc.

Notes: Pipeline drugs are in Discovery, Preclinical, IND/CTA Filed, Phase 0, Phase I, Phase II, Phase III, and Pre-Registration stages

Producing gene or cell therapies requires an inherently high level of manufacturing expertise and expensive facility requirements many pharma companies do not possess. Figure 2 assesses the current manufacturing source outlook and CMO opportunities for 12 cell or gene therapies approved in the US or EU and whose manufacturing arrangements are publicly disclosed. Many of the sponsors for these products are large-cap ($10B100B) or mega-cap (>$100B) companies, which typically choose to invest in developing their own manufacturing facilities. However, API, dose, and packaging have been outsourced for 39%, 26%, and 29% of ATMPs respectively, proving that significant opportunities exist for CMOs. Licensor/Partner agreements accounted for 56% of all manufacturing relationships.

Based on these findings, there are considerable opportunities for CMOs with the capabilities to manufacture ATMPs. However, only 152 CMOs have the capability to produce cell or gene therapy API for global markets and of these only 121 are dedicated contract CMOs, according to the Contract Service Provider database.

Figure 2: Manufacturing Sources for Gene and Cell Therapies Approved in US/EU

Source: GlobalData, Pharma Intelligence Center Drugs by Manufacturer Database (Accessed: 8 July 2020) 2020 GlobalData Plc

GlobalData is this websites parent business intelligence company.

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Few CMOs are equipped to manufacture cell and gene therapies - Pharmaceutical Technology

Thermo Fisher Scientific has launched a compliance and services portfolio, compatible with current Good Manufacturing Practice (cGMP), designed to enable rapid start-up of clinical and commercial cell and gene therapy manufacturing.

Developed to simplify and optimize the cGMP equipment installation and validation processes, the Thermo Scientific Cell Therapy Systems (CTS) Series Laboratory Equipment are comprised of a range of key lab equipment routinely used in cell and gene therapy manufacturing, including biological safety cabinets, centrifuges, CO2 incubators and cold storage systems. Each product includes a factory acceptance test documentation package comprised of operating certificates, performance specifications, user guides and technical manuals. The portfolio is also supported by field compliance services, including installation setup, IQ, OQ and other support, such as temperature mapping and calibration.

The CTS Series Laboratory Equipment are manufactured at ISO13485-certified sites, and designed to meet global mechanical and electrical safety standards.

Originally posted here:

Thermo Fisher Launches Cell And Gene Therapy Mfg. Offering - Contract Pharma

Global Gene Therapy (COVID-19) Market Report 2020 by Key Players, Types, Applications, Countries, Market Size, Forecast to 2026 (Based on 2020 COVID-19 Worldwide Spread)

The Gene Therapy market report provides a detailed analysis of global market size, regional and country-level market size, segmentation market growth, market share, competitive Landscape, sales analysis, the impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunities analysis, strategic market growth analysis, product launches, area marketplace expanding, and technological innovations.

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The detailed market intelligence report on the Global Gene Therapy Market applies the most effective of each primary and secondary analysis to weighs upon the competitive landscape and also the outstanding market players expected to dominate Global Gene Therapy Market place for the forecast 2019 2025.

Scope Of The Report:

Report evaluates the growth rate and the Market value based on Market dynamics, growth inducing factors. The complete knowledge is based on latest industry news, opportunities and trends. The report contains a comprehensive Market analysis and vendor landscape in addition to a SWOT analysis of the key vendors.

Geographically, this report split global into several key Regions, revenue (Million USD) The geography (North America, Europe, Asia-Pacific, Latin America and Middle East & Africa) focusing on key countries in each region. It also covers market drivers, restraints, opportunities, challenges, and key issues in Global Gene Therapy Market.

Key Benefits for Gene Therapy Market Reports

Global market report covers in-depth historical and forecast analysis.

Global market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

Global market report helps to identify opportunities in market place.

Global market report covers extensive analysis of emerging trends and competitive landscape.

Gene Therapy Market Segmentation:

By Disease Indication

Cancer

Genetic disorders

Cardiovascular diseases

Ophthalmology

Neurological conditions

Others

By Type of Vectors

Viral vectors

Non-viral vectors

By Type of Cells

Somatic cells

Germline cells

By Region

North America

o U.S.

o Canada

o Mexico

Europe

o UK

o France

o Germany

o Russia

o Rest of Europe

Asia-Pacific

o China

o South Korea

o India

o Japan

o Rest of Asia-Pacific

LAMEA

o Latin America

o Middle East

o Africa

Gene Therapy Market Key Players:

Pfizer Inc.

Novartis AG

Bayer AG

Sanofi

GlaxoSmithKline plc.

Amgen Inc.

Boehringer Ingelheim International GmbH

uniQure N.V.

bluebird bio, Inc.

Celgene Corporation

Others

This comprehensive report will provide:

Enhance your strategic decision making

Assist with your research, presentations and business plans

Show which emerging market opportunities to focus on

Increase your industry knowledge

Keep you up-to-date with crucial market developments

Allow you to develop informed growth strategies

Build your technical insight

Illustrate trends to exploit

Strengthen your analysis of competitors

Provide risk analysis, helping you avoid the pitfalls other companies could make

Ultimately, help you to maximize profitability for your company.

Our Market Research Solution Provides You Answer to Below Mentioned Question:

Which are the driving factors responsible for the growth of market?

Which are the roadblock factors of this market?

What are the new opportunities, by which market will grow in coming years?

What are the trends of this market?

Which are main factors responsible for new product launch?

How big is the global & regional market in terms of revenue, sales and production?

How far will the market grow in forecast period in terms of revenue, sales and production?

Which region is dominating the global market and what are the market shares of each region in the overall market in 2017?

How will each segment grow over the forecast period and how much revenue will these segment account for in 2025?

Which region has more opportunities?

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2020 Growth: Gene Therapy Market 2020 Research on Import-Export Details, Business Standards and Forecast to 2025 - Owned

EMERYVILLE, Calif.--(BUSINESS WIRE)--4D Molecular Therapeutics (4DMT), a clinical-stage leader in the development of precision-guided AAV gene medicines based on directed evolution, announced that the first patient has been dosed in the Phase 1 clinical trial of 4D-110, a Roche-licensed product candidate, delivered by a single intravitreal injection for Choroideremia. Choroideremia is a blinding and currently untreatable X-linked inherited retinal disease.

Dosing the first patient in the Phase 1 clinical trial of 4D-110 marks the first of three Therapeutic Vector Evolution pipeline candidates expected to enter the clinic in 2020, said David Kirn, MD, co-founder, chairman and chief executive officer of 4DMT. 4DMT harnesses the power of directed evolution to develop precision-guided AAV gene medicines. In the case of 4D-110, this proprietary and optimized AAV vector is designed to provide targeted delivery to the retina of a functional copy of the CHM gene by intravitreal injection, a routine clinical route of administration. Ultimately, our aim is to alter the course of this debilitating disease and to treat patients with all stages of the disease. I would like to thank the Choroideremia patient community, their families and caregivers, the Choroideremia Research Foundation, and the clinical trial physicians and staff, without whom 4DMT would not have reached this stage.

With no currently approved therapies available for patients impacted by Choroideremia, gene therapy represents a promising therapeutic approach, said David Birch, PhD, Scientific Director, Retina Foundation of the Southwest and a principal investigator for the study. Due to its optimized vector, 4D-110 is a novel gene therapy approach that shows promise in safely treating a broad region of the retina and in a broad range of patients. The potential to slow-down or halt the debilitating visual field constriction seen in this disease is an exciting opportunity for patients.

The Phase 1 open-label, dose-exploration and dose-expansion study is expected to enroll up to 15 patients with Choroideremia. The study is designed to assess the preliminary safety, tolerability and biological activity of a single intravitreal injection of 4D-110. In addition, the clinical trial will evaluate the effect of 4D-110 on the visual function and retinal degeneration.

"On behalf of the Choroideremia Research Foundation and Choroideremia patients internationally, I am extremely excited by the initiation of 4DMT's clinical trial, said Christopher Moen, MD, Chief Medical Officer of the Choroideremia Research Foundation. This pivotal milestone brings us one step closer to a transformative treatment which has the potential to end blindness from Choroideremia."

About Choroideremia and 4D-110

Affecting approximately 10,000 individuals in the United States and the European Union, Choroideremia is an X-linked, slowly-progressive, degenerative disease of the retina and choroid of the eye caused exclusively by deletions or mutations in the CHM gene, resulting in a missing or defective REP1 protein. Choroideremia initially manifests as night-blindness and peripheral visual field defects, usually starting in the first two decades of life. As the disease progresses, the visual field begins to constrict relatively early in the diseases progression, which hinders patients ability to conduct daily activities and eventually leads to vision loss.

4DMTs precision-guided gene therapy approach holds promise for the treatment of Choroideremia by using a proprietary and optimized AAV vector to deliver a functional copy of the CHM gene, resulting in the production of the REP1 protein. 4D-110, which is licensed to Roche, is comprised of a CHM transgene insert and 4DMTs proprietary vector 4D-R100, a vector designed to provide targeted delivery via intravitreal administration and to efficiently transduce all layers of the retina.

About 4DMT

4DMT is a clinical-stage precision gene medicines company harnessing the power of directed evolution to unlock the full potential of gene therapy for rare and large market diseases in lysosomal storage diseases, ophthalmology, neuromuscular diseases, and cystic fibrosis. 4DMTs proprietary Therapeutic Vector Evolution platform enables a disease first approach to product discovery and development, thereby empowering customization of AAV vectors to target specific tissue types associated with the underlying disease. These proprietary and optimized AAV vectors are designed to provide targeted delivery by routine clinical routes, efficient transduction, reduced immunogenicity, and resistance to pre-existing antibodies -- attributes that could enable the development of gene therapies that overcome known limitations of conventional AAV vectors. 4DMT vectors are designed to exhibit improved therapeutic profiles that enable the company to pursue previously untreatable patient populations and to address a broad range of rare and large market disease markets.

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4D Molecular Therapeutics Announces First Patient Dosed in Phase 1 Clinical Trial of 4D-110 by Intravitreal Injection for the Treatment of...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apic Bio, Inc. today announced the formation of its Scientific Advisory Board (SAB) which includes internationally recognized gene therapy experts.

The SAB will provide clinical and scientific expertise to help the Company advance its pipeline and leverage its proprietary silence and replace THRIVE platform.

Apics CEO, John Reilly, MS/MBA, stated, "As we continue to advance our programs, it is critical that we obtain input from individuals with diverse expertise across a broad range of relevant research and development areas. The formation of our SAB represents another important step forward for the company. We are honored to welcome such an esteemed group of clinicians and researchers who have been at the forefront of gene therapy translational medicine.

The appointments to Apics Scientific Advisory Board include:

Robert H. Brown Jr, DPhil, MD, Leo P. and Theresa M. LaChance Chair in Medical Research, Professor of Neurology, Director of the Program in Neurotherapeutics, University of Massachusetts Medical School

R. Jude Samulski, PhD, President, Chief Scientific Officer and Co-Founder at AskBio

Barry Byrne, MD, PhD, Director of the Powell Gene Therapy Center at the University of Florida and Professor of Pediatrics and Molecular Genetics & Microbiology

Rob Kotin, PhD, Adjunct Professor at University of Massachusetts Medical School

Kevin Flanigan, MD, Robert F. and Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research and Director of the Center for Gene Therapy in The Abigail Wexner Research Institute at Nationwide Children's Hospital, and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine

Mark Kay, MD, PhD, Dennis Farrey Family Professor in Pediatrics and Professor of Genetics at Stanford University School of Medicine

SAB member biographies are available at http://www.apic-bio.com/

About Apic Bio

Apic Bio is an innovative gene therapy company focused on developing first-in-class treatment options for rare, undertreated neurological and liver diseases. The Company's lead program is an adeno-associated (AAV)-based gene therapy for the treatment of the copper zinc superoxide dismutase 1 (SOD1) genetic form ALS. Preclinical studies of additional genetic forms of ALS (C9Orf72) and Alpha-1 Antitrypsin Deficiency (Alpha-1) are ongoing. The Company is also advancing discovery programs for two undisclosed CNS indications that leverage its proprietary silence and replace THRIVE platform. The Company is backed by leading and disease-centric investors, including Morningside Ventures, ALS Investment Fund, and The Alpha-1 Project (TAP). For more information please visit http://www.apic-bio.com.

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Apic Bio Announces Formation of Scientific Advisory Board - Business Wire

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