Sangamo Therapeutics(SGMO) announced the official closing of the global deal withBiogen(BIIB) for gene regulation therapies in neurology, explains biotechnology sector expertJohn McCamant, editor ofThe Medical Technology Stock Letter.

More from John McCamant: Ziopharm: Catalysts in T-Cell Therapy

With roughly $650 million in cash post-deal, SGMO is trading just above its cash positive despite having the broadest pipeline in the gene therapy/editing/regulation field.

Of all things in the press release, management mentioned the cash would be used to progress the pipeline, plus the potential filing of a BLA for SB525 for hemophilia A.

Impressive Terms For Early Stage

As a reminder, Biogen paid Sangamo $350 million upfront, including a $125 million license fee and an equity investment in Sangamo stock of $225 million (@$9.21 per share).

Sangamo is also eligible to receive up to $2.37 billion in potential milestones, including $925 million on pre-approval milestones and $1.335 billion on first product sales (that does not include the royalties that are also part of the deal).

In the blockbuster markets being developed such as Alzheimers and Parkinsons diseases, SGMO will earn high-single-to-low double digit royalties on potential sales. This is a pre-IND stage deal remarkable financial terms for this early stage of development.

Hem A Update On Schedule

Pfizer(PFE) cancelled its R&D Day that was set for March due to the virus, but the Company will hold a quarterly earnings call on April 28. There is some chance that Pfizer updates investors with some of the highlights it was preparing for the R&D Day, including the SB-525 program with Sangamo.

Either way, SGMO will still be updating investors when the two longest treated patients in the ALTA study hit their 12-18 month follow-up for the duration of Factor VIII production, sometime this summer.

See also: Activision Blizzard: "Stay-at-Home" Entertainment

The 12-18 month durability data is key for SGMO given that BMRNs first 12-18 month durability data was the turning point for Wall Streets belief in their gene therapy. Sangamo is a buy under $20 with a target price of $30.

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Sangamo- Early-Stage Progress in Gene Therapy - Yahoo Finance

Worldwide Gene Therapy Market has been thoroughly elaborated in a research report put on sale by Marketresearch.biz, bearing the title Gene Therapy MarketGlobal Industry Analysis, Size, Share, Growth, Trends, and Forecast 20202029. According to the report, the market is expected to be driven by a wide range of macroeconomic and industry-related factors. The key players in this market are focusing on product strategies and developments to increase their customer base and maintain their position.

The Gene Therapy Market is an intrinsic study of the current status of this business vertical and encompasses a brief synopsis about its segmentation. This industry report is inclusive of a nearly accurate prediction of the market scenario over the forecast period 20202029 market size with respect to valuation as sales volume. The study lends focus to the top magnates comprising the competitive landscape of Gene Therapy market, as well as the geographical areas where the industry extends its horizons, in magnanimous detail.

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Operational and Emerging Players: Gene Therapy Market- Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

According to the current market situation, this report continuously observing promising growth of the global Gene Therapy market. The report further suggests market appears to progress at an accelerating rate over the forecast period. Also, the major players are elaborated on the basis of the proprietary technologies, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, report also examines R&D developments, legal policies, SWOT Analysis and strategies of Gene Therapy market players.

This Gene Therapy market research report surrounds importance on:

Manufacturing process and technology used in Gene Therapy market, key developments and trends changing in the development

Complete examination, including an evaluation of the parent market

Detailed account of market, volume and forecast, by leading key players, product type and end users applications

Environmental spread, development designs, pieces of the overall industry, key methodologies, and different financials systems of Gene Therapy market

Industrial analysis by upstream raw materials, downstream industry, current market dynamics and ensuing consumers analysis

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Gene Therapy Market Segmentation Outlook:

By Vector:Viral vectorRetrovirusesLentivirusesAdenovirusesAdeno Associated VirusHerpes Simplex VirusPoxvirusVaccinia VirusNon-viral vectorNaked/Plasmid VectorsGene GunElectroporationLipofection

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Gene Therapy Market Section by Region:

ASIA-PACIFIC MARKET: China, Southeast Asia, India, Japan, Korea, Western Asia

THE MIDDLE EAST & AFRICA MARKET: GCC, North Africa, South Africa

NORTH AMERICA MARKET: United States, Canada, Mexico

EUROPE MARKET: Germany, Netherlands, UK, France, Russia, Spain, Italy, Turkey, Switzerland

SOUTH AMERICA MARKET: Brazil, Argentina, Columbia, Chile, Peru

Our Research Methodology is based on the following main points:

i. Data Collections and Interpretation

ii. Analysis

iii. Data Validation

iv. Final Projections and Conclusion

The latest research on the Gene Therapy Market fundamentally delivers insights that can empower stakeholders, business owners, and field marketing executives to make effective investment decisions driven by facts and extremely thorough research. The study aims to provide an evaluation and deliver essential information on the competitive landscape to meet the unique requirements of the companies and individuals operating in the Gene Therapy Market for the forecast period, 20202029. To help firms comprehend the Gene Therapy industry in multiple ways, the report exhaustively assesses the share, size, and growth rate of the business worldwide.

There are 13 Sections to show the global Gene Therapy market:

Chapter 1: Market Overview, Drivers, Segmentation overview, Restraints and Opportunities

Chapter 2: Market competition by key Manufacturers

Chapter 3: Production by Regions

Chapter 4: Consumption by Regions

Chapter 5: Production By Types, Revenue and Market share by Types

Chapter 6: Market share (%) and Growth Rate by Applications, Consumption By Applications

Chapter 7: Complete profiling and analysis of leading Manufacturers

Chapter 8: Region-wise manufacturing expenses, Manufacturing cost analysis, Raw materials analysis

Chapter 9: Sourcing Strategy, Industrial Chain and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Market Effect Factors Analysis, Impact Analysis

Chapter 12: Market Forecast 2020-2029

Chapter 13: Gene Therapy Research Findings and Conclusion, methodology and data source, Appendix

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Conclusively, this report will provide you a clean view of each and every truth of the market without a need to consult another research report or a statistics supply. Our report will offer you with all of the facts about the past, present, and future of the Market.

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Inclusive Comprehension 2020: Gene Therapy Market (Trending PDF) Addressing Structure, Scope, Potential, and Growth Prospects Till 2029 - Jewish Life...

BOSTON, April 27, 2020 (GLOBE NEWSWIRE) -- Ziopharm Oncology, Inc. (ZIOP), today announced the Company will host a conference call and webcast on Thursday, May 7, at 4:30 pm EDT to provide a corporate update and discuss financial results for the first quarter ended March 31, 2020.

The call can be accessed by dialing 1-877-451-6152 (U.S. and Canada) or 1-201-389-0879 (international). The passcode for the conference call is 13701877. To access the live webcast or the subsequent archived recording, visit the Investors section of the Ziopharm website at http://www.ziopharm.com. The webcast will be recorded and available for replay on the Companys website for two weeks.

About Ziopharm Oncology, Inc.Ziopharm Oncology is an immuno-oncology company focused on developing end-to-end cost-effective solutions using its non-viral Sleeping Beauty platform for T-cell receptor (TCR) and chimeric antigen receptor (CAR) T-cell therapies and immune-stimulating gene therapy with Controlled interleukin 12 (IL-12). The Sleeping Beauty platform genetically modifies T cells with DNA plasmids to express TCRs to target neoantigens inside and outside hotspots for solid tumors and CAR to target CD19 for blood cancers using the Companys Rapid Personalized Manufacturing to produce and release CAR-T as soon as the day after gene transfer. The Sleeping Beauty platform is being advanced in collaboration with the National Cancer Institute, The University of Texas MD Anderson Cancer Center and Eden BioCell. The Company is also developing its Controlled IL-12 platform, or Ad-RTS-hIL-12 plus veledimex, as monotherapy and in combination with immune checkpoint inhibitors to treat brain cancer, including in collaboration with Regeneron Pharmaceuticals.

Ziopharm Contact:Chris TaylorVP, Investor Relations and Corporate CommunicationsT: 617.502.1881E: ctaylor@ziopharm.com

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Ziopharm Oncology to Report First Quarter 2020 Financial Results and Host a Conference Call and Webcast on May 7, 2020 - Yahoo Finance

Lysogene S.A.s (EPA:LYS): Lysogene S.A. engages in the research and clinical development of gene therapy for neurodegenerative disorders. The 64m market-cap company announced a latest loss of -4.3m on 31 December 2019 for its most recent financial year result. Many investors are wondering the rate at which LYS will turn a profit, with the big question being when will the company breakeven? Below I will provide a high-level summary of the industry analysts expectations for LYS.

Check out our latest analysis for Lysogene

According to the industry analysts covering LYS, breakeven is near. They expect the company to post a final loss in 2021, before turning a profit of 69m in 2022. LYS is therefore projected to breakeven around 2 years from now. In order to meet this breakeven date, I calculated the rate at which LYS must grow year-on-year. It turns out an average annual growth rate of 50% is expected, which is extremely buoyant. If this rate turns out to be too aggressive, LYS may become profitable much later than analysts predict.

Im not going to go through company-specific developments for LYS given that this is a high-level summary, however, keep in mind that by and large a biotech has lumpy cash flows which are contingent on the product type and stage of development the company is in. This means that a high growth rate is not unusual, especially if the company is currently in an investment period.

One thing Id like to point out is that LYS has managed its capital judiciously, with debt making up 13% of equity. This means that LYS has predominantly funded its operations from equity capital,and its low debt obligation reduces the risk around investing in the loss-making company.

There are too many aspects of LYS to cover in one brief article, but the key fundamentals for the company can all be found in one place LYSs company page on Simply Wall St. Ive also put together a list of relevant aspects you should further research:

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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Lysogene S.A. (EPA:LYS): When Will It Breakeven? - Simply Wall St

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CRISPR corrects genetic defect so cells can normalize blood sugar

Researchers at Washington University School of Medicine in St. Louis have transformed stem cells into insulin-producing cells. They used the CRISPR gene-editing tool to correct a defect that caused a form of diabetes, and implanted the cells into mice to reverse diabetes in the animals. Shown is a microscopic image of insulin-secreting beta cells (insulin is green) that were made from stem cells produced from the skin of a patient with Wolfram syndrome.

Using induced pluripotent stem cells produced from the skin of a patient with a rare, genetic form of insulin-dependent diabetes called Wolfram syndrome, researchers transformed the human stem cells into insulin-producing cells and used the gene-editing tool CRISPR-Cas9 to correct a genetic defect that had caused the syndrome. They then implanted the cells into lab mice and cured the unrelenting diabetes in those mice.

The findings, from researchers at Washington University School of Medicine in St. Louis, suggest the CRISPR-Cas9 technique may hold promise as a treatment for diabetes, particularly the forms caused by a single gene mutation, and it also may be useful one day in some patients with the more common forms of diabetes, such as type 1 and type 2.

The study is published online April 22 in the journal Science Translational Medicine.

Patients with Wolfram syndrome develop diabetes during childhood or adolescence and quickly require insulin-replacement therapy, requiring insulin injections multiple times each day. Most go on to develop problems with vision and balance, as well as other issues, and in many patients, the syndrome contributes to an early death.

This is the first time CRISPR has been used to fix a patients diabetes-causing genetic defect and successfully reverse diabetes, said co-senior investigator Jeffrey R. Millman, PhD, an assistant professor of medicine and of biomedical engineering at Washington University. For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping stone toward applying gene therapy to a broader population of patients with diabetes.

Wolfram syndrome is caused by mutations to a single gene, providing the researchers an opportunity to determine whether combining stem cell technology with CRISPR to correct the genetic error also might correct the diabetes caused by the mutation.

A few years ago, Millman and his colleagues discovered how to convert human stem cells into pancreatic beta cells. When such cells encounter blood sugar, they secrete insulin. Recently, those same researchers developed a new technique to more efficiently convert human stem cells into beta cells that are considerably better at controlling blood sugar.

In this study, they took the additional steps of deriving these cells from patients and using the CRISPR-Cas9 gene-editing tool on those cells to correct a mutation to the gene that causes Wolfram syndrome (WFS1). Then, the researchers compared the gene-edited cells to insulin-secreting beta cells from the same batch of stem cells that had not undergone editing with CRISPR.

In the test tube and in mice with a severe form of diabetes, the newly grown beta cells that were edited with CRISPR more efficiently secreted insulin in response to glucose. Diabetes disappeared quickly in mice with the CRISPR-edited cells implanted beneath the skin, and the animals blood sugar levels remained in normal range for the entire six months they were monitored. Animals receiving unedited beta cells remained diabetic. Their newly implanted beta cells could produce insulin, just not enough to reverse their diabetes.

We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation, said co-senior investigator Fumihiko Urano, MD, PhD, the Samuel E. Schechter Professor of Medicine and a professor of pathology and immunology. Its a proof of concept demonstrating that correcting gene defects that cause or contribute to diabetes in this case, in the Wolfram syndrome gene we can make beta cells that more effectively control blood sugar. Its also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration.

In the future, using CRISPR to correct certain mutations in beta cells may help patients whose diabetes is the result of multiple genetic and environmental factors, such as type 1, caused by an autoimmune process that destroys beta cells, and type 2, which is closely linked to obesity and a systemic process called insulin resistance.

Were excited about the fact that we were able to combine these two technologies growing beta cells from induced pluripotent stem cells and using CRISPR to correct genetic defects, Millman said. In fact, we found that corrected beta cells were indistinguishable from beta cells made from the stem cells of healthy people without diabetes.

Moving forward, the process of making beta cells from stem cells should get easier, the researchers said. For example, the scientists have developed less intrusive methods, making induced pluripotent stem cells from blood and they are working on developing stem cells from urine samples.

In the future, Urano said, we may be able to take a few milliliters of urine from a patient, make stem cells that we then can grow into beta cells, correct mutations in those cells with CRISPR, transplant them back into the patient, and cure their diabetes in our clinic. Genetic testing in patients with diabetes will guide us to identify genes that should be corrected, which will lead to a personalized regenerative gene therapy.

Maxwell KG, Augsornworawat P, Velazco-Cruz L, Kim MH, Asada R, Hogrebe NJ, Morikawa S, Urano F, Millman JR. Gene-edited human stem cell-derived cells from a patient with monogenic diabetes reverse pre-existing diabetes in mice. Science Translational Medicine, published online April 22, 2020.

This work was supported by the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of General Medical Sciences, the National Cancer Institute and the National Center for Advancing Translational Sciences of the National Institutes of Health (NIH). Grant numbers R01 DK114233, DK112921, TR002065, TR002345, T32 DK108742, R25 GM103757, T32 DK007120, P30 DK020579, P30 CA91842, UL1 TR000448 and UL1 TR002345. Additional assistance was provided by the Washington University Genome Engineering and iPSC Center, the Washington University Diabetes Center, and the Washington University Institute of Clnical and Translational Science, with additional funding from the JDRF, the Washington University Center of Regenerative Medicine, startup funds from the Washington University School of Medicine Department of Medicine, the Unravel Wolfram Syndrome Fund, Silberman Fund, Stowe Fund, Ellie White Foundation for Rare Genetic Disorders, Eye Hope Foundation, Snow Foundation, Feiock Fund, Childrens Discovery Institute, Manpei Suzuki Diabetes Foundation, and a JSPS Overseas Research Fellowship.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Diabetes reversed in mice with genetically edited stem cells derived from patients - Washington University School of Medicine in St. Louis

Study on the Global Hemophilia Gene Therapy Market

The report on the global Hemophilia Gene Therapy market reveals that the Hemophilia Gene Therapy market is anticipated to grow at a CAGR of ~XX% during the forecast period (2019-2029). The report provides a thorough assessment of the impact of the COVID-19 pandemic on the current and future prospects of the Hemophilia Gene Therapy market across various geographies. Further, the report provides accurate data related to the business continuity and contingency strategies adopted by leading market players operating in the Hemophilia Gene Therapy market.

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The competitive landscape section of the report tracks the business proceedings of the key players operating in the Hemophilia Gene Therapy market. The pricing, marketing, sales, and promotional strategies adopted by each market player is represented in the report. The contingency strategies of different players amidst the COVID-19 pandemic are touched upon in the presented report.

Competition AnalysisIn the competitive analysis section of the report, leading as well as prominent players of the global Hemophilia Gene Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.On the whole, the report proves to be an effective tool that players can use to gain a competitive edge over their competitors and ensure lasting success in the global Hemophilia Gene Therapy market. All of the findings, data, and information provided in the report are validated and revalidated with the help of trustworthy sources. The analysts who have authored the report took a unique and industry-best research and analysis approach for an in-depth study of the global Hemophilia Gene Therapy market.The following players are covered in this report:Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline TherapeuticsHemophilia Gene Therapy Breakdown Data by TypeHemophilia AHemophilia BHemophilia Gene Therapy Breakdown Data by ApplicationHemophilia A Gene TherapyHemophilia B Gene Therapy

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Addressing the potential impact of coronavirus disease (COVID-19) on Hemophilia Gene Therapy Market: Quantitative Analysis from 2019 to 2025 to Enable...

Analysis Report on Gene Therapy Market

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COVID-19 Impact on Gene Therapy Market

Adapting to the recent novel COVID-19 pandemic, the impact of the COVID-19 pandemic on the global Gene Therapy market is included in the present report. The influence of the novel coronavirus pandemic on the growth of the Gene Therapy market is analyzed and depicted in the report.

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Global Gene Therapy Market, by Product

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COVID-19 impact: Gene Therapy Market 2019 Business Insights,Top Companies,Growth, Market Size Global Market Share, Trends, Outlook, Opportunity and...

Spark Therapeutics Inc.

Global Gene Therapy For Inherited Genetic Disorders Market Segmentation

This market was divided into types, applications and regions. The growth of each segment provides an accurate calculation and forecast of sales by type and application in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting niche markets. Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, the Asia-Pacific region, the Middle East, and Africa and Latin America. Research analysts understand the competitive forces and provide competitive analysis for each competitor separately.

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Gene Therapy For Inherited Genetic Disorders Market Region Coverage (Regional Production, Demand & Forecast by Countries etc.):

North America (U.S., Canada, Mexico)

Europe (Germany, U.K., France, Italy, Russia, Spain etc.)

Asia-Pacific (China, India, Japan, Southeast Asia etc.)

South America (Brazil, Argentina etc.)

Middle East & Africa (Saudi Arabia, South Africa etc.)

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-> We will give you an assessment of the extent to which the market acquire commercial characteristics along with examples or instances of information that helps your assessment.

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-> We will further help you in finding any price ranges, pricing issues, and determination of price fluctuation of products in Gene Therapy For Inherited Genetic Disorders industry.

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Table of Contents:

Study Coverage: It includes study objectives, years considered for the research study, growth rate and Gene Therapy For Inherited Genetic Disorders market size of type and application segments, key manufacturers covered, product scope, and highlights of segmental analysis.

Executive Summary: In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Gene Therapy For Inherited Genetic Disorders market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Gene Therapy For Inherited Genetic Disorders Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region: It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

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Gene Therapy For Inherited Genetic Disorders Market Overview, Top Companies, Region, Application and Global Forecast by 2026 - Latest Herald

A new study led by the University of Bristol has shown a common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality of life for many patients.

Glaucoma affects over 64 million people worldwide and is a leading cause of irreversible blindness. It is usually caused by fluid building up in the front part of the eye, which increases pressure inside the eye and progressively damages the nerves responsible for sight. Current treatments include either eye drops, laser or surgery, all of which have limitations and disadvantages.

The research team led by academics at the Bristol Medical School: Translational Health Sciences tested a new approach that could provide additional treatment options and benefits. Their findings are published in the journalMolecular Therapy.

The researchers designed a gene therapy and demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue.

The treatment targeted part of the eye called the ciliary body, which produces the fluid that maintains pressure within the eye. Using the latest gene editing technology called CRISPR, a gene called Aquaporin 1 in the ciliary body was inactivated leading to reduced eye pressure.

Dr Colin Chu, Visiting Senior Research Fellow in theBristol Medical School: Translational Health Sciencesand corresponding author, said: "Currently there is no cure for glaucoma, which can lead to loss of vision if the disease is not diagnosed and treated early.

"We hope to advance towards clinical trials for this new treatment in the near future. If it's successful it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money."

The academics are currently in discussion with industry partners to support further laboratory work and rapidly progress this new treatment option towards clinical trials.

Reference: Wu et al. (2020). Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9. Molecular Therapy.DOI: 10.1016/j.ymthe.2019.12.012.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Glaucoma Could Be Successfully Treated With Gene Therapy - Technology Networks

Insulin injections cancontrol diabetes, but patients still experience serious complications such as kidney disease and skin infections. Transplanting pancreatic tissues containing functional insulin-producing beta cells is of limited use, because donors are scarce and patients must take immunosuppressant drugs afterward.

Now, scientists atWashington University in St. Louis havedeveloped a way to use gene editing system CRISPR-Cas9 to edit a mutation in human-induced pluripotent stem cells (iPSCs) and then turnthem into beta cells. When transplanted into mice, the cells reversed preexisting diabetes in a lasting way, according to results published in the journal Science Translational Medicine.

While the researchers used cells from patients with Wolfram syndromea rare childhood diabetes caused by mutations in the WFS1 genethey argue that the combination of a gene therapy with stem cells could potentially treat other forms of diabetes as well.

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

One of the biggest challenges we faced was differentiating our patient cells into beta cells. Previous approaches do not allow for this robust differentiation. We use our new differentiation protocol targeting different development and signaling pathways to generate our cells, the studys lead author, Kristina Maxwell, explained in a video statement.

Making pancreatic beta cells from patient-derived stem cells requires precise activation and repression of specific pathways, and atthe right times, to guide the development process. In a recent Nature Biotechnology study, the team described a successful method that leverages the link between a complex known as actin cytoskeleton and the expression of transcription factors that drive pancreatic cell differentiation.

This time, the researchers applied the technology to iPSCs from two patients with Wolfram syndrome. They used CRISPR to correct the mutated WFS1 gene in the cells and differentiated the edited iPSCs into fully functional beta cells.

After transplanting the corrected beta cells into diabetic mice, the animals saw their blood glucose drop quickly, suggesting the disease had been reversed. The effect lasted for the entire six-month observation period, the scientists reported. By comparison, those receiving unedited cells from patients were unable to achieve glycemic control.

RELATED:CRISPR Therapeutics, ViaCyte team up on gene-edited diabetes treatment

The idea of editing stem cells with CRISPR has already attracted interest in the biopharma industry. Back in 2018, CRISPR Therapeutics penned a deal with ViaCyte to develop off-the-shelf, gene-editing stem cell therapies for diabetes. Rather than editing iPSCs from particular patients themselves to correct a faulty gene, the pairs lead project used CRISPR to edit healthy cells so that they lackedthe B2M gene and expressed PD-L1 to protect against immune attack. The two companies unveiled positive preclinical data inSeptember.

Other research groups working on gene therapy or stem cells for diabetes include a Harvard University scientist and his startup Semma Therapeutics, whichdeveloped a method for selecting beta cells out of a mixture of cells developed from PSCs. Scientists at the University of Wisconsin-Madison recently proposed that removing the IRE1-alpha gene in beta cells could prevent immune T cells from attacking them in mice with Type 1 diabetes.

The Washington University team hopes its technology may help Type 1 diabetes patients whose disease is caused by multiple genetic and environmental factors as well as the Type 2 form linked to obesity and insulin resistance.

We can generate a virtually unlimited number of beta cells from patients with diabetes to test and discover new drugs to hopefully stop or even reverse this disease, Jeffrey Millman, the studys co-senior author, said in the video statement. Perhaps most importantly, this technology now allows for the potential use of gene therapy in combination with the patients own cells to treat their own diabetes by transplantation of lab-grown beta cells.

Link:

Reversing diabetes with CRISPR and patient-derived stem cells - FierceBiotech

Hackers leak data related to WHO, Wuhan lab and Gates Foundation

A common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality of life for many patients, say researchers.

Glaucoma affects over 64 million people worldwide and is a leading cause of irreversible blindness. It is usually caused by fluid building up in the front part of the eye, which increases pressure inside the eye and progressively damages the nerves responsible for sight.

Current treatments include either eye drops, laser or surgery, all of which have limitations and disadvantages. "At present, there is no cure for glaucoma, which can lead to loss of vision if the disease is not diagnosed and treated early," said study researcher Dr Colin Chu from the University of Bristol in the UK.

For the findings, published in the journal Molecular Therapy, the research team tested a new approach that could provide additional treatment options and benefits. The researchers designed a gene therapy and demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue.

The treatment targeted part of the eye called the ciliary body, which produces the fluid that maintains pressure within the eye. Using the latest gene-editing technology called CRISPR, a gene called Aquaporin 1 in the ciliary body was inactivated leading to reduced eye pressure.

"We hope to advance towards clinical trials for this new treatment in the near future. If it's successful it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money," Chu said. The researchers are currently in discussion with industry partners to support further laboratory work and rapidly progress this new treatment option towards clinical trials.

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Glaucoma can be successfully treated with gene therapy - International Business Times, Singapore Edition

Asklepios BioPharmaceutical, Inc. (AskBio), a prominent U.S.-based clinical-stage AAV gene therapy firm, has recently announced that it has acquired BrainVectis, a Paris-based gene therapy firm renowned for its work in neurodegenerative disorders, especially on therapies that regenerate brain cholesterol metabolism for patients suffering from Huntington's disease along with other disorders.

For the record, BrainVectis was initiated by Nathalie Cartier-Lacave, M.D., and has support and funding from Sorbonne University, the French Alternative Energies, and INSERM. Currently, Dr. Cartier-Lacave of Atomic Energy Commission (CEA) and her team are concentrating on developing gene therapies that increase the CYP46A1 enzymes expression in the brain.

Evidently, this enzyme is associated with numerous disorders where brain cholesterol metabolism dysfunction is concerned in the neurodegenerations pathology.

Sources cite that AskBio would leverage BrainVectis synthetic and capsid promotor design platforms and its capacity to advance the development of this platform and manufacturing solutions along with other BrainVectis projects.

Speaking on the move, Sheila Mikhail, CEO and Co-Founder, AskBio, said that the companys acquisition of BrainVectis provides it with a remarkable opportunity to work jointly while transforming patients lives.

Huntington's disease is one of the diseases that is irreversible and devastating and presently has no effective treatments. However, BrainVectis in-depth world-leading knowledge of neurodegenerative diseases including Huntingtons disease could allow AskBio to expand its portfolio for these diseases as well.

Prior to this news, AskBio had made headlines when it revealed that it has appointed Don Haut, Ph.D., as its new Chief Business Officer to handle business development execution and strategy.

Reportedly, Dr. Haut has over two decades of healthcare experience and was previously serving to Histogenics Inc. and Sherlock Biosciences as their Chief Business Officer.

Commenting on which, Mikhail said that Don would be a key addition to the company as he holds a wealth of business and strategy development experience within the medical technology and biotechnology fields. His expertise would help the company to effectively advance its existing platforms.

Source Credit: https://www.contractpharma.com/contents/view_breaking-news/2020-04-22/askbio-acquires-brainvectis/

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AskBio acquires BrainVectis to expand its gene therapy portfolio - News.MarketSizeForecasters.com

Ever since their son Michael was diagnosed with a rare genetic disorder, Terry and Georgia Pirovolakis have been racing to find a cure. They were getting close, and then everything came grinding to a halt.

"We had a whole bunch of researchers around the world doing different things. All of our research has basically stopped," says Terry Pirovolakis.

Michael is two-and-a-half years old and has spastic paraplegia type 50 (SPG-50), a rare neurodegenerative disease caused by a missing gene that is progressively robbing him of his ability to walk.

Over the past year, his parents have raised more than $1 million to help finance a clinical trial for custom gene therapy that promises to halt the disease. But the COVID-19 pandemic has paralyzed research around the world, and their hope along with it.

"It's hard to accept the fact that we were making so many gains," says Georgia Pirovolakis. "You know, we were hoping he would be walking by September."

Michael is just one of the many casualties of the global health crisis. From new gene therapies to help children like Michael, to the latest in cancer treatments, thousands of clinical trials deemed non-essential are on hold.

Brad Wouters is executive vice-president of Science and Research at Toronto's University Health Network, Canada's largest academic hospital. He says an example of the impact is a patient currently being treated for pancreatic cancer.

"He was part of a medical research study here that looked at the underlying genetics of that tumour, and that information revealed a potential new therapy that could be very effective for that patient in immunotherapy. But we only have access to that drug through a clinical trial. And so he won't get it."

Wouters says the only new research happening at the moment is around COVID-19. About 200 of UHN's researchers have pivoted to that, but thousands more risk losing their jobs, Wouters says, because in the past month alone UHN has lost $6 million in industry funding. It's why UHN is asking the federal government for help.

"We've been doing everything we can to try and support these jobs and support this research sector," says Wouters. "But it's over a month now, and we're really at a tipping point where we're going to see significant job losses if something can't be done."

Canada's $3-billion medical research industry relies on that brain trust, much of it made up of international students, says Martha Crago, vice-principal of Research and Innovation at McGill University.

"We wouldn't want to lose this wonderful set of brains coming into our country and helping bring solutions to the public domain," Crago says. "We need to do what we can to keep them."

The pandemic will be over at some point, Crago says, and those researchers will be needed to help regain lost scientific momentum.

Meanwhile, the Pirovolakis family fear they're running out of time.

All the therapies Michael has been doing to maintain the movement he has now, like physical and occupational therapy, have been cancelled. They're trying to improvise at home to keep him moving, and to keep their hope alive too.

The stalled research efforts means a cure for Michael may be delayed for up to a year. That could mean the difference between him ever walking or not.

"It means he's going to degrade," says Terry Pirovolakis. "The progression of the disease is going to kick in, he'll slowly become more and more paralyzed."

It's why the couple are trying to keep research into Michael's gene therapy going.

They're considering using money the family has raised so far to finance early toxicology tests on a potential treatment researchers in the U.S have already developed. They are also lobbying Canada's National Research Council to help develop a treatment or help with funding.

Georgia Pirovolakis says there are so many unknowns, and she can't bear to dwell on them.

"I just look at one day at a time. I'm not thinking about, you know, tomorrow. I'm not thinking about one month from now. I'm not thinking about the research stopping, him potentially not getting cured."

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'We're really at a tipping point': COVID-19 brings research into other medical conditions to a grinding halt - CBC.ca

RANCHO CORDOVA, Calif., April 24, 2020 /PRNewswire/ --ThermoGenesis Holdings, Inc. (Nasdaq: THMO), a market leader in automated cell processing tools and services in the cell and gene therapy field, announced today that it was featured on multiple new outlets, including a FOX40 news segment titled, "Rancho Cordova Company Says It's Working on Potential Treatment for Coronavirus," regarding the ThermoGenesis and ImmuneCyte joint venture and its research collaborator's efforts toward developing a systemic antibody approaches against COVID-19, ranging from convalescent plasma therapy and polyclonal antibody, to high-affinity monoclonal antibody drug candidates.

Using the rapid IgM/IgG antibody detection technology currently marketed by the Company, along with its own proprietary multi-component automated cell separating system, ThermoGenesis is working towards developing strategies to identify and isolate polyclonal antibodies from the blood of individuals who have recovered from the disease using convalescent whole blood or plasma, which could then be used as a potential therapeutic treatment for patients with of COVID-19.

As previously announced, the Company's joint venture partner, ImmuneCyte, has acquired from its collaborators worldwide intellectual property of four high-affinity monoclonal antibody drug candidates against COVID-19virus. These high-affinity neutralizing monoclonal antibodies were also identified from convalescent whole blood from individuals who have recently recovered from COVID-19.

The FOX40 News is accessible via the link below:

The KCRA3 News is accessible via the link below:

The in-depth local Sacramento Bee news article is accessible via the link below:

Commenting on the recent new coverage Chris Xu, PhD, an immunologist and Chief Executive Officer of ThermoGenesis, said, "We are taking a systematic approach to address several critical unmet needs in the fight against COVID-19, namely rapid tests and effective therapies. By working closely with our global collaborators, we had a head start on this critical research." Dr. Xu also noted that the U.S. Food and Drug Administration is fast tracking therapeutic products related to SARS-CoV-2, which the Company hopes may ultimately include ThermoGenesis' various antibody therapeutic approaches.

Story continues

About ThermoGenesis Holdings, Inc.

ThermoGenesis Holdings, Inc. develops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. The Company currently markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology, including its semi-automated, functionally-closed CAR-TXpressplatform, which streamlines the manufacturing process for the emerging CAR-T immunotherapy market. For more information about ThermoGenesis, please visit:www.thermogenesis.com.

About ImmuneCyte Life Sciences, Inc.

ImmuneCyte, a joint venture which is 19% owned by ThermoGenesis, develops and commercializes diagnostic and therapeutic technologies for COVID-19. The Company also provides clients the opportunity to bank their own immune cells when the cells are "healthy and unaffected" as a future resource for cellular immunotherapies, such as CAR-T. ImmuneCyte utilizes a proprietary CAR-TXpress platform, a GMP compliant close-system capable of automated separating and cryopreserving different components from blood, simultaneously. For more information about ImmuneCyte Life Sciences Inc., please visit: http://www.immunecyte.com.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements are not statements of historical facts, but rather reflect the current expectations of ThermoGenesis concerning future events and results. Forward-looking statements based on ThermoGenesis' current assumptions, expectations and beliefs are generally identifiable by use of words "may," "might," "will," "should," "expects," "plans," "anticipates," "believes," "estimates," "predicts," "potential" or "continue," or similar expressions and involve significant risks and uncertainties that could cause actual results, developments and business decisions to differ materially from those contemplated by these statements. Such forward-looking statements, including statements regarding the intended use of net proceeds from the registered direct offering as well as those factors concerning our expectations, involve risks, uncertainties and other factors, some of which are beyond our control, which may cause our actual results, performance or achievements, or industry results, to be materially different from any future results, performance, or achievements expressed or implied by such forward-looking statements. These risks, uncertainties and factors include, but are not limited to, market and other conditions, as well as those risk factors discussed in Item 1A of our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission ("SEC") as well as other documents that may be filed by ThermoGenesis from time to time with the SEC, which are available atwww.sec.gov. We undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. You are cautioned not to unduly rely on such forward-looking statements when evaluating the information presented in this press release.

Company Contact:Wendy Samford916-858-5191ir@thermogenesis.com

Investor Contact:Paula Schwartz,Rx Communications917-322-2216pschwartz@rxir.com

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SOURCE ThermoGenesis Holdings, Inc.

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ThermoGenesis and ImmuneCyte Joint Venture Developing Several Convalescent Plasma and Antibody Therapeutic Approaches Against COVID-19, Featured on...

ThisGlobalGene TherapyMarketprovides a detailed analysis of theGene Therapymarket based on segmentation such as applications, and geography and forecasts the market for 2020 to 2025. The globalGene Therapymarket is extremely fragmented. The report analyzes the markets competitive landscape and offers information on several field services manufacturers rivaling in the industry. Additionally, thisGene Therapymarket analysis report suggests strategies companies can follow and recommends key areas they should focus on, to make the most of upcoming growth opportunities.

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To help clients improve their revenue shares in the market, this research report provides an analysis of theGene Therapymarkets competitive landscape and offers information on the products offered by various leading companies. Although the market share of this segment will decrease, it will continue to dominate the market throughout the forecast years. The market is in the growth phase, as well as the competition is expected to become less intense by the end of the forecast period. It also covers accurate predictions on upcoming trends and changes in consumer behavior and the growth of the industry across the key regions.

TheGene Therapymarket segmentation is based on the regions:North America, Europe, Asia Pacific, South America, and the Middle East and Africa. These regions will witness an increase in their market share and remain the market leader for the forecast period.

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The report contains detailed information on factors that will accelerate the growth of the market during the forecast period from 2020-2025 along with the CAGR of the market during the forecast period. However, comprehensive details on factors will challenge the growth of companies rivaling in the market. It consists of a precise estimation of the globalGene Therapymarket size and its contribution to the parent market as well as a thorough analysis of the markets competitive landscape and detailed information on several vendors.

It comprehensively segments theGene Therapymarket and provides the closest approximations of the revenue numbers for the overall market and its sub segments across major regions. It would help stakeholders understand their competitors better and gain more insights to enhance their positions in theGene Therapymarket. The competitive landscape section includes a competitor ecosystem, new service developments, partnerships, and acquisitions. It would help stakeholders understand the pulse of the market and provide information on the key market drivers, restraints, challenges, and opportunities.

Contact Us:Mark StoneSales ManagerPhone: (201) 465-4211Email:sales@fiormarkets.comWeb:www.fiormarkets.com

View More Information onGene TherapyMarket :https://www.marketwatch.com/press-release/global-gene-therapy-market-2020-overview-manufacturing-analysis-development-status-competitive-analysis-to-2025-2020-04-21

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Gene Therapy Market : Global Outlook and Forecast 2020-2025 - Surfacing Magazine

Redpin Therapeutics has raised $15.5 million to bankroll chemogenetic R&D. The initial close of the series A positions Redpin to move a controllable gene therapy for the treatment of indications such as epilepsy and pain toward an IND.

New York-based Redpin is a gene therapy company with a difference. Like its peers, Redpin uses viral vectors to introduce genes into patients. Yet, while for traditional gene therapy companies that is the end of the process, for Redpin it is just the start. The genes inserted by Redpin encode for engineered ion channel proteins designed to bind to a particular drug, namely Pfizers Chantix.

When the patient takes Chantix, the molecule binds to the the ion channels now expressed by the neurons, either stimulating or silencing the cells based on what is needed to treat the disease they cause. As Redpin CEO Elma Hawkins notes, the approach differs from traditional drug development.

We sort of the do the opposite of what has been done in the pharmaceutical industry for decades and decades. Instead of making small molecules for a biologic target or receptor, we pick a drug and then we make a very, very specific receptor for it. As long as these two molecules, the receptor and the small molecule, engage, you will have an effect on the neuron, Hawkins said.

The approach could enable very direct control over cellular activities. And, as the inserted gene only has a therapeutic effect in the presence of Chantix, the approach could allow physicians to effectively turn the gene therapy on and off.

That potential has caught the attention of investors. 4BIO Capital, an advanced therapy specialist that raised a $50 million fund last year, led the round with Arkin Bio Ventures. The VC wing of Takeda also chipped in cash as did seed round investors New York Ventures and Alexandria Venture Investments.

Having spent the past year amassing preclinical data, Redpin will use the money to push toward an IND, although at this time the biotech is yet to commit to a timeline for getting into the clinic. Redpin has been more forthcoming about the broad focus of its lead program, which is designed for use in conditions such as pain and epilepsy that are characterized by hyperexcited neurons.

If you have a therapy that can 'calm that down,' that would be a terrific solution as youre acting at the site of the problem, Hawkins said. Redpin could equally use the same basic approach to activate neurons, opening up another set of indications that are potentially amenable to treatment using its Chantix-controlled gene therapies.

Whatever the goal, the basic idea is to design and introduce receptors that bind to Chantix. The oral drug, which Pfizer sells to help smokers quit, became integral to Redpins approach in part because it penetrates the brain effectively and is well tolerated at the low doses needed for the gene therapy application.

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Redpin bags A round to advance Chantix-controlled gene therapies - FierceBiotech

Home News Drugs in the Pipeline

The Food and Drug Administration (FDA) has granted Fast Track designation to PR006 (Prevail Therapeutics), an experimental gene therapy, to slow the progression of frontotemporal dementia with a GRN mutation (FTD-GRN).

FTD-GRN is a progressive neurodegenerative disease caused by mutations in the GRN gene. Patients with a GRN mutation have reduced levels of progranulin, a protein critical for lysosomal function, neuronal survival, and normal microglial activities. PR006 delivers a healthy GRN gene using an AAV9 vector to increase progranulin levels in these patients.

The Food and Drug Administration (FDA) recently accepted the Investigational New Drug application for PR006, allowing the Company to proceed with a phase 1/2 trial.

FTD-GRN progresses rapidly and there are currently no therapeutic options available, said Asa Abeliovich, MD, PhD, Founder and CEO of Prevail. We believe PR006 has the potential to fill this unmet medical need and make a significant impact for patients.

The FDA previously granted Orphan Drug designation to PR006 for the treatment of patients with frontotemporal dementia.

For more information visit prevailtherapeutics.com.

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Gene Therapy for Frontotemporal Dementia With a GRN Mutation Gets Fast Track Status - Monthly Prescribing Reference

TAMPA, Fla. & DUBLIN--(BUSINESS WIRE)-- Vycellix, Inc., an immuno-discovery cell & gene therapy company, and Avectas Limited, a cell engineering technology business, today jointly announced that the companies have entered into a collaboration agreement to develop proprietary approaches for cell-based immunotherapeutic products.

The companies will collaborate on the delivery of Vycellix's novel RNA immunomodulator VY-M using Avectas' cell engineering platform, Solupore. The collaboration will address current limitations for cell-based therapies, in particular with respect to the need to accelerate the manufacturing process, reduce the cost of manufacture, and ultimately improve patient outcomes.

"We are delighted to partner with Vycellix and join forces in the development of novel cell-based products," stated Michael Maguire, Ph.D., CEO of Avectas. "We believe Solupore will play a critical role in the manufacture of cell-based therapies and will support a path towards effective patient outcomes."

According to Vycellixs President, Douglas Calder, Solupore represents a new paradigm for delivery of transgenes, and our initial studies will evaluate Solupore to deliver our product candidate, VY-M, to T cells and natural killer (NK) cells. We expect to accelerate the expansion-time of T cells and NK cells by decreasing the non-dividing lag time, resulting in much shorter vein-to-vein delivery-time to patients. The studies will be conducted at Avectas Dublin-based facility and at Karolinska Institutet, Stockholm, Sweden.

Both Vycellix and Avectas are collaborative partners within NextGenNK, a newly established competence center for development of next-generation NK cell-based cancer immunotherapies based at Karolinska Institutet, Stockholm, Sweden. It is envisioned that Vycellix and Avectas will further expand their collaboration within the NextGenNK constellation.

We are excited to see the NextGenNK Competence Center catalyzing interactions among its industrial partners to advance NK cell-based immunotherapies, said Hans-Gustaf Ljunggren, M.D., Ph.D., Director of the NextGenNK Competence Center. The present collaboration may pave the way for similar collaborations among NextGenNK partners.

About Vycellix, Inc.: Vycellix is a private, immuno-discovery, life science company at the forefront of innovation in the development of cell & gene-based therapies targeting indications in, but not limited to, hematology/oncology, autoimmunity/chronic inflammatory diseases, and organ/tissue transplantation.

The Companys portfolio of transformational platform technologies encompass novel tools urgently sought after to enable broad global adoption of advanced therapies including: 1) the ability to generate Universal Cells (VY-UC), without the need to alter expression of any of the cellular components that control self-recognition (HLA Class I or II), obviating the need for immune-suppressive drugs and redefining the path towards off-the-shelf therapies; 2) the ability to amplify cell-potency through the upregulation of internal cytotoxic mechanisms (VY-X); 3) the ability to accelerate the expansion of cells for immunotherapy by near-elimination of non-dividing lag time to leap forward to shorter vein-to-vein time with expanded cells (VY-M); and, 4) the ability to markedly enhance gene transduction levels using viral vectors with implications for autologous and allogeneic CAR-T and CAR-NK cell development (VY-OZ).

The Companys platforms were all discovered by scientists at the world-renowned Karolinska Institutet (KI) in Stockholm, Sweden. KI is globally recognized for its Nobel Assembly, which awards the Nobel Prize in Physiology or Medicine. For more information, please visit the Companys website at: http://www.Vycellix.com and follow its Twitter feed at: @Vycellix.

About Avectas Limited: Avectas is a cell engineering technology business developing a unique delivery platform to enable the ex-vivo manufacture of our partners' gene-modified cell therapy products, which will retain high in-vivo functionality. Our vision is to be a leading non-viral cell engineering technology provider, integrated into manufacturing processes for multiple autologous and allogeneic therapies, commercialized through development and license agreements. For more information, please visit the Company's website at http://www.avectas.com.

Forward Looking Statements: This press release contains forward-looking statements. All statements other than statements of historical facts are forward-looking statements, including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as plan, expect, anticipate, may, might, will, should, project, believe, estimate, predict, potential, intend, or continue and other words or terms of similar meaning. These statements include, without limitation, statements related to the pre-clinical, regulatory, clinical and/or commercial development and all anticipated uses of VY-OZ, VY-X, VY-M and VY-UC, and the Companys plans for seeking out-licensing opportunities for these assets. These forward-looking statements are based on current plans, objectives, estimates, expectations and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with immuno-discovery product development, including risks associated with advancing products to human clinical trials and/or ultimately regulatory and commercial success which is subject to the uncertainty of regulatory approval, market adoption and other risks and uncertainties affecting Vycellix and its development programs. Other risks and uncertainties of which Vycellix is not currently aware may also affect Vycellixs forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. Vycellix undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200325005026/en/

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Vycellix and Avectas Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies - BioSpace

DUBLIN and TAMPA, Florida, March 24, 2020 /PRNewswire/ -- Avectas, a cell engineering technology business andVycellix, Inc. an immuno-discovery cell & gene therapy company, today announced that the companies have entered into a collaboration agreement to develop proprietary approaches for cell-based immunotherapeutic products.

The companies will collaborate on the delivery of Vycellix's novel RNA immunomodulator VY-M using Avectas' cell engineering platform, Solupore. The collaboration will address current limitations for cell-based therapies, in particular with respect to the need to accelerate the manufacturing process, reduce the cost of manufacture, and ultimately improve patient outcomes.

"We are delighted to partner with Vycellix and join forces in the development of novel cell-based products," says Michael Maguire, PhD, CEO of Avectas. "We believe Solupore will play a critical role in the manufacture of cell-based therapies and will support a path towards effective patient outcomes."

According to Vycellix's President, Douglas Calder, "Solupore represents a new paradigm for delivery of transgenes, and our initial studies will evaluate Solupore to deliver our new product candidate, VY-M, to T cells and NK cells. We expect to accelerate the expansion-time of T cells and NK cells by decreasing the non-dividing lag time, resulting in much shorter "vein-to-vein" delivery-time to patients." The studies will be conducted at Avectas' Dublin-based facility and at Karolinska Institutet, Stockholm, Sweden.

Both Avectas and Vycellix are collaborative partners within NextGenNK, a newly established competence center for development of next-generation NK cell-based cancer immunotherapies based at Karolinska Institutet, Stockholm, Sweden. It is envisioned that Avectas and Vycellix will further expand their collaboration within the NextGenNK constellation.

"We are excited to see the NextGenNK competence center catalysing interactions among its industrial partners to advance NK cell-based immunotherapies," says Hans-Gustaf Ljunggren, MD PhD, Director of the NextGenNK competence center. "The present collaboration may pave the way for similar collaborations among NextGenNK partners."

In February 2020, Avectas announced that it had entered an agreement with the Centre for Commercialization of Regenerative Medicine (CCRM) based in Toronto, Canada to accelerate the translation of Avectas' non-viral cell engineering platform (Solupore) into the clinic.

About Avectas:Avectas is a cell engineering technology business developing a unique delivery platform to enable the ex-vivo manufacture of our partners' gene-modified cell therapy products, which will retain high in-vivo functionality. Our vision is to be a leading non-viral cell engineering technology provider, integrated into manufacturing processes for multiple autologous and allogeneic therapies, commercialized through development and license agreements. For more information, please visit the Company's website at http://www.avectas.com

About Vycellix:Vycellix, Inc.is a private, immuno-discovery, life science company at the forefront of innovation in the development of cell & gene-based therapies targeting indications in, but not limited to, hematology/oncology, autoimmunity/chronic inflammatory diseases, and organ/tissue transplantation.

The Company's platforms were all initially discovered by scientists at the world-renowned Karolinska Institutet (KI) in Stockholm, Sweden. KI is globally recognized for its Nobel Assembly, which awards the Nobel Prize in Physiology or Medicine. For more information, please visit the Company's website at http://www.vycellix.com

View original content:http://www.prnewswire.com/news-releases/avectas-and-vycellix-announce-collaboration-to-advance-next-generation-solutions-for-the-optimized-manufacture-of-cell--gene-therapies-301028561.html

SOURCE Avectas; Vycellix

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Avectas and Vycellix Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies - BioSpace

SEATTLE, March 25, 2020 /PRNewswire/ -- According to Coherent Market Insights, the global viral vector and plasmid DNA manufacturing market is estimated to be valued at US$ 427.2 million in 2019, and is expected to exhibit a CAGR of 22.8% over the forecast period (2019-2027).

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Key Trends and Analysis of the Global Viral Vector and Plasmid DNA Manufacturing Market:

Key trends in the market include increasing incidences of cancer, rising number of product launches, and increasing collaboration and acquisition activities by key market players.

According to World Health Organization (WHO), in 2018, around 9.6 million cancer deaths occurred globally. Over the last decade, development of gene therapy for the treatment of the cancer has increased significantly. Gene therapy treatment for cancer include transfer of foreign genetic material in the targeted cancer cell in the host's body. Various types of viral vectors and plasmid DNA such as retrovirus and HGF plasmidare used in the development of gene therapy.

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Increasing product launches and approvals by regulatory authorities are expected to drive growth of the global viral vector and plasmid DNA manufacturing market over the forecast period. For instance, in December 2017, Spark Therapeutics received the U.S. FDA approval to launch the LUXTURNA in the U.S. market. It is the first FDA approved gene therapy for treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the U.S.

Furthermore, key players operating in the market are focused on adopting acquisition, agreement, and collaboration strategies, in order to expand their product offerings in markets. For instance, in December 2017, Merck KGaA entered into a commercial supply agreement with bluebird bio, Inc., a clinical-stage biopharmaceutical company. According to the agreement, Merck agreed to manufacture viral vectors for bluebird's gene therapy products targeting the rare genetic disorders.

Key Market Takeaways:

Key players operating in the global viral vector and plasmid DNA manufacturing market include

Lonza Group AG, FinVector Vision Therapies, Cobra Biologics and Pharmaceutical Services, Sigma-Aldrich Co. LLC, VGXI, Inc., VIROVEK, SIRION Biotech GmbH, FUJIFILM Diosynth Biotechnologies U.S.A., Inc., Sanofi, Cell and Gene Therapy Catapult, Brammer Bio, and MassBiologics.

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Market Segmentations:

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DNA AND RNA SAMPLE PREPARATION MARKET

DNA and RNA samples are necessary for variety of applications in drug research and development and cancer studies. High quality DNA and RNA samples are important for a wide variety of research and clinical applications. Biological studies require purified and isolated nucleic acids as the first step and in all recombinant DNA techniques. The extraction of nucleic acids from biological material requires cell lysis, inactivation of cellular nucleases, and separation of the desired nucleic acid from cellular debris.

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LIFE SCIENCE PRODUCTS MARKET

Life science products include laboratory supplies & accessories, cell culture & fermentation processes, cell therapy technologies, chromatography products, bioprocess filtration, fixed and live cell research through imaging and analysis, sample collection products, recombinant proteins, cell lines, and antibodies. These products are used for drug discovery, tissue engineering, drug screening, forensic testing, and genetic analysis.

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GLYCOBIOLOGY MARKET

Glycobiology involves study of structural aspects, biosynthesis, and biology of polysaccharides and how they function in an organism. Study of glycobiology has variety of application in areas such as drug discovery and development, diagnostic applications, therapeutic application, and industrial applications.Complex structure of glycan's and difficulty in its study, high costs of spectrometry and high performance liquid chromatography are expected to hinder growth of the market.

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View original content:http://www.prnewswire.com/news-releases/global-viral-vector-and-plasmid-dna-manufacturing-market-to-surpass-us-2-205-6-million-by-2027---cmi-301029511.html

SOURCE Coherent Market Insights

Link:

Global Viral Vector and Plasmid DNA Manufacturing Market to Surpass US$ 2,205.6 Million by 2027 - CMI - Yahoo Finance