StemCell Therapy

Cancer Gene Therapy Market size is expected to exceed USD 2.5 billion by 2025. Rising cancer prevalence across the globe will positively impact the cancer gene therapy market growth.Some major findings of the cancer gene therapy market report include:

Increasing mortality rates due to cancer has created a huge demand for cancer gene therapies

Technological advancements in the biotechnology sectors has led to developments in the cancer gene therapy that are being now preferred by healthcare professionals

Regulations for cancer gene therapy are quite structured and stringent in developed economies such as the U.S. and Germany while the regulatory scenario is still ambiguous in emerging economies

Cancer gene therapy industry is still in developing phase and yet to witness numerous growth opportunities in the coming years

Some of the major industry players include: Sirion Biotech, Vigene Biosciences, bluebird bio, Ziopharm, Cellectis, Cobra, Finvector, Uniqure, Sarepta Therapeutics, Caribou among others. These industry players adopt several strategic initiatives to maintain their market position.

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Technological advancements in the biotechnology sector have led to developments and innovations in cancer gene therapy. Recently developed cancer therapies have been incorporated with genetically modified genes that blocks the growth of tumor. Moreover, efficient PCR technology and isothermal amplification technologies have been introduced that transformed the way of detecting mutations in the genes. Moreover, introduction of CRISPR gene editing tools have modified the process of developing gene therapy. Hence, advancements in technology has ensured availability of advanced cancer gene therapy that will boost the industry growth. However, high cost of cancer gene therapy may impede its demand, thereby restraining the industry growth to certain extent.

Type segment of cancer gene therapy market includes ex-vivo and in-vivo. In-vivo segment is projected to experience around 22% growth throughout the analysis timeframe owing to various advantages associated with it. In-vivo gene therapy involves direct delivery of therapeutic gene into the target cell and has shown effective results in treatment of cancer. The viral vectors that are delivered utilizing in-vivo gene therapy help in inhibiting the activity of tumor inducing genes and has also shown positive results during clinical trials. Aforementioned factors will elevate the segmental growth.

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Products of cancer gene therapy include viral vectors and non-viral vectors. Viral vectors segment was valued over USD 310 million in 2018. Viral vectors are highly preferred during gene transfer process as they have showcased high transfection efficiency. Viral vectors possess safety profile as compared to other vectors. Moreover, viral vectors expresses the desired antigen in accurate conformation enhancing the immune system.

End-users of cancer gene therapy are biopharmaceutical companies, research institutes and others. Biopharmaceutical companies segment accounted for around 48% revenue share in 2018 and is expected to experience exceptional growth in the forthcoming years. Companies such as Novartis and Roche are developing cancer gene therapies that have high adoption of viral as well as non-viral vectors, thereby proving beneficial for the segmental growth. These companies also conduct clinical trials that raises the demand for vectors, hence fostering the segmental growth.

China cancer gene therapy market will experience over 26% CAGR throughout the forecast years. Significant country growth can be attributed to the rising awareness regarding the availability of advanced therapies for treating cancer. Furthermore, increasing government initiatives and funds motivate the researchers and scientists for carrying out extensive research activities associated with cancer gene therapy that will positively influence the country growth. Above mentioned factors coupled with increasing prevalence of cancer will further stimulate the industry growth.

Cancer gene therapy industry is dominated by few major players. Cancer gene therapy industry is still in the developing phase, therefore, players involved in thie market focus on integrating advanced technology to promote developments in the therapies. The players also implement certain strategic initiatives such as merger, acquisitions and product launches for acquiring competitive advantage. For instance, in 2013, Celgene and bluebird bio collaborated to introduce innovations in gene therapies. Such collaborations will provide both the companies to gain competitive advantage over others.

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Cognizance into Cancer Gene Therapy Market and it's growth prospects - Cole of Duty

The first time Lyell CEO Rick Klausner looked at what PACT Pharma was trying to accomplish with neoantigens, non-viral T cell engineering and cancer, he felt they couldnt get it done. But in the 3 years since theyve launched, Klausner has become a believer.

Now, hes a believer and a partner.

Early Thursday morning, Klausner and PACT CEO Alex Franzusoff announced a plan to jointly pursue one of the Holy Grails of oncology R&D. Blending their technologies and bringing a wide network of leading experts to the table, the two companies are working on a personalized T cell therapy for solid tumors. And an IND is in the offing.

The collaboration joins the Lyell team, which has been concentrating on overcoming the exhaustion that afflicts the first generation of cell therapies, with a PACT group that has developed tech to identify a patients unique signature of cancer mutations and use a non-viral method to engineer their T cells into cancer therapies.

I spent some time on Wednesday talking with Klausner and Franzusoff about the deal, which comes with an undisclosed set of financials as Lyell invests in the alliance.

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Eyeing a hot IPO market, 4DMT tops up its cash reserves and preps a leap into the clinic with bespoke gene therapy vectors - Endpoints News

The first time Lyell CEO Rick Klausner looked at what PACT Pharma was trying to accomplish with neoantigens, non-viral T cell engineering and cancer, he felt they couldnt get it done. But in the 3 years since theyve launched, Klausner has become a believer.

Now, hes a believer and a partner.

Early Thursday morning, Klausner and PACT CEO Alex Franzusoff announced a plan to jointly pursue one of the Holy Grails of oncology R&D. Blending their technologies and bringing a wide network of leading experts to the table, the two companies are working on a personalized T cell therapy for solid tumors. And an IND is in the offing.

The collaboration joins the Lyell team, which has been concentrating on overcoming the exhaustion that afflicts the first generation of cell therapies, with a PACT group that has developed tech to identify a patients unique signature of cancer mutations and use a non-viral method to engineer their T cells into cancer therapies.

I spent some time on Wednesday talking with Klausner and Franzusoff about the deal, which comes with an undisclosed set of financials as Lyell invests in the alliance.

Unlock this article along with other benefits by subscribing to one of our paid plans.

SUBSCRIBE SIGN IN

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Two Bay Area upstarts out to deliver on cell therapy 2.0 join forces on a quest: pursuing a Holy Grail in oncology R&D - Endpoints News

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The report offers a systematic presentation of the existing trends, growth opportunities, market dynamics that are expected to shape the growth of the Cell and Gene Therapy market. The various research methods and tools were involved in the market analysis, to uncover crucial information about the market such as current & future trends, opportunities, business strategies and more, which in turn will aid the business decision-makers to make the right decision in future.

This Report Covers Leading Companies Associated in Worldwide Cell and Gene Therapy Market: Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

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The report begins with a brief introduction and market overview of the Cell and Gene Therapy industry followed by its market scope and size. Next, the report provides an overview of market segmentation such as type, application, and region. The drivers, limitations, and opportunities for the market are also listed along with current trends and policies in the industry.

The key players profiled in this report include: Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

Regions included:

o North America (United States, Canada, and Mexico)

o Europe (Germany, France, UK, Russia, and Italy)

o Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)

o South America (Brazil, Argentina, Colombia)

o Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

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Key Benefits:

o This study gives a detailed analysis of drivers and factors limiting the market expansion of Cell and Gene Therapy

o The micro-level analysis is conducted based on its product types, end-user applications, and geographies

o Porters five forces model gives an in-depth analysis of buyers and suppliers, threats of new entrants & substitutes and competition amongst the key market players

o By understanding the value chain analysis, the stakeholders can get a clear and detailed picture of this Cell and Gene Therapy market

The research study can answer the following Key questions:

Table of Contents

Report Overview: It includes the Cell and Gene Therapy market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary: This section of the report gives information about Cell and Gene Therapy market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players: Here, key players of the Cell and Gene Therapy market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study: All of the regions and countries analyzed in the Cell and Gene Therapy market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

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Impact of COVID-19 on Cell and Gene Therapy Market 2020 Key Players, Opportunities, Challenges, Trend and Forecast by 2027 | Amgen, Biogen, BioMarin...

18-Jun-2020

Design and Build | Pharmaceuticals

Sexton Biotechnologies has partnered with BioSpherix to create a fully-enclosed, modular fill-finish system with rapid deployment for emerging cell and gene therapy needs

Sexton Biotechnologies has announced a new collaboration with BioSpherix Medical. As a tool and technologies partners of the Cell and Gene industry, Sexton and BioSpherix both recognise the need for cost-effective and flexible automation solutions during cell and gene therapy process development. While some upstream bioprocesses have been successfully automated, the final steps of downstream bioprocess, namely Fill-Finish, are often done manually with associated risks of contamination and user error.

The inefficiencies and lack of traceable GMP controls make manual Fill-Finish processes unacceptable if companies are to scale up and scale out for late-stage clinical or commercial phases. The new collaboration will combine Sexton's off-the-shelf fill system, the Sexton Biotechnologies AF-500, with BioSpherix' exclusive Cytocentric isolator technology, allowing rapid process development and implementation of GMP Fill-Finish.

The recent global pandemic has resulted in an unprecedented acceleration of therapeutic candidates paired with significant supply chain challenges. Development of clinical and commercial stage manufacturing systems to meet this rapid expansion is likely to lead to significant delays in scale up and completion of clinical studies.

BioSpherix, having already observed that traditional GMP capacity is becoming limited, leads the field in manufacture of modular systems that reduce the time and capital burden of implementation. While constructing and operating a Class A or B environment is time and cost-prohibitive for start-up companies, the BioSpherix solution offers an attractive solution to reduce the need for high classification cleanroom, long lead times, and costs.

"The limitations of many of the available downstream processing methods and systems are inherently obvious for cell and gene therapy developers," said Sean Werner, President of Sexton Biotechnologies. "We've taken the approach with the AF-500 to have systems ready to ship so developers can immediately begin process development of small volume fill and finish with the equipment they plan to use at clinical scale. The collaboration with BioSpherix further enhances our current technology enabling it to operate within a fully closed environment along with the ability to control temperatures to limit risks during cryopreservation."

Sexton and BioSpherix recognised each other's capabilities as complimentary and that together, they could address a gap in process capabilities. Sexton's rapidly deployable Fill and Seal system, AF-500, is capable of filling and sealing up to 500 CellSeal vials in 90 minutes and is available for immediate delivery and can be installed for use within a biosafety cabinet while the BioSpherix modular isolator is under construction. The BioSpherix modular isolator can be configured for any manual or automated, large or small, simple or complex bioprocess, making it the perfect fit with Sexton's AF-500 to bring flexibility, sterility, and automation to our customers.

Link:
Sexton partners on modular fill-finish for cell and gene therapies - Cleanroom Technology

This report additionally covers the effect of COVID-19 on the worldwide market. The pandemic brought about by Coronavirus (COVID-19) has influenced each part of life all inclusive, including the business segment. This has brought along a several changes in economic situations.

A report on Gene Therapy for Ovarian Cancer market compiled by Brand Essence Market Research provides a succinct analysis regarding the values and trends existing in the current business scenario. The study also offers a brief summary of market valuation, market size, regional outlook and profit estimations of the industry. Furthermore, the report examines the competitive sphere and growth strategies of leading players in the Gene Therapy for Ovarian Cancer market. Download Premium Sample of the Report: https://industrystatsreport.com/Request/Sample?ResearchPostId=2&RequestType=Sample

TheMajorPlayersCovered in this Report:Takara Bio, VBL Therapeutics, CELSION, Targovax & More.

Product Type: Intravenous,Intratumoral,Intraperitoneal

Application: Ovarian Cancer (unspecified),Recurrent Ovarian Epithelial Cancer,Platinum-Resistant Ovarian Cancer

Results of the recent scientific undertakings towards the development of new Gene Therapy for Ovarian Cancer products have been studied. Nevertheless, the factors affecting the leading industry players to adopt synthetic sourcing of the market products have also been studied in this statistical surveying report. The conclusions provided in this report are of great value for the leading industry players. Every organization partaking in the global production of the Gene Therapy for Ovarian Cancer market products have been mentioned in this report, in order to study the insights on cost-effective manufacturing methods, competitive landscape, and new avenues for applications.

Global Gene Therapy for Ovarian CancerMarket: Regional SegmentationFor further clarification, analysts have also segmented the market on the basis of geography. This type of segmentation allows the readers to understand the volatile political scenario in varying geographies and their impact on the global Gene Therapy for Ovarian Cancermarket. On the basis of geography, the global market for Gene Therapy for Ovarian Cancerhas been segmented into:

North America(United States, Canada, and Mexico)Europe(Germany, France, UK, Russia, and Italy)Asia-Pacific(China, Japan, Korea, India, and Southeast Asia)South America(Brazil, Argentina, Colombia, etc.)Middle East and Africa(Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

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Report Methodology:

The information enclosed in this report is based upon both primary and secondary research methodologies.

Primary research methodology includes the interaction with service providers, suppliers, and industry professionals. Secondary research methodology includes a meticulous search of pertinent publications like company annual reports, financial reports, and exclusive databases.

Table of Content:

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the Global Gene Therapy for Ovarian Cancer Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the Worldwide Global Gene Therapy for Ovarian Cancer Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the Global Gene Therapy for Ovarian Cancer Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the Global Gene Therapy for Ovarian Cancer Market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the Global Gene Therapy for Ovarian Cancer Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This section of the research study shows how different end-user/application segments contribute to the Global Gene Therapy for Ovarian Cancer Market.

Market Forecast: Here, the report offers a complete forecast of the Global Gene Therapy for Ovarian Cancer Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

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Gene Therapy for Ovarian Cancer Market 2020-2025 : Regional markets, Technology, Types, and Applications - Cole of Duty

Leonard E. Post, Ph.D.

Following the uniQure Annual General Meeting of Shareholders, Leonard E. Post, Ph.D. was appointed to the Board of Directors.

~ Biopharma Executive Brings 35 Years of Research and Development Leadership ~

LEXINGTON, Mass. and AMSTERDAM, June 17, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Leonard E. Post, Ph.D. to its Board of Directors. Dr. Post, who will also chair the Companys Research & Development Committee, has extensive biotechnology industry experience including previous global R&D leadership roles at BioMarin and Onyx Pharmaceuticals.

"It is with great pleasure that we nominate Len to the uniQure board, stated Matthew Kapusta, chief executive officer of uniQure. His considerable biotechnology experience, including his important work in gene therapy, are valuable assets for uniQure as we work to advance our pipeline candidates and to prepare to file for marketing authorization of etranacogene dezaparvovec in hemophilia B.

Since 2016, Dr. Post has served as Chief Scientific Officer of Vivace Therapeutics and its sister company, Virtuoso Therapeutics, both of which are developing oncology therapeutics. From 2010 until 2016, Dr. Post served in various positions at BioMarin (NASDAQ: BMRN),including ChiefScientific Officer, during which time he oversaw the initiation of BioMarin's first gene therapy project for hemophilia A. Prior to that, Dr. Post served as Chief Scientific Officer of LEAD Therapeutics, Senior Vice President of Research & Development at Onyx Pharmaceuticals and Vice President of Discovery Research at Parke-Davis Pharmaceuticals. He is also currently an advisor to Canaan Partners.

Dr. Post is a virologist by training and did early work on engineering of herpes simplex virus as a postdoctoral fellow. He has a Bachelor of Science degree in Chemistry from the University of Michigan, and a Doctorate degree in Biochemistry from the University of Wisconsin.

About uniQure

uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.www.uniQure.com

uniQure Contacts:

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/0364d19c-b363-46bb-9277-8e349f7661fd

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uniQure Announces the Appointment of Leonard E. Post, Ph.D. to its Board of Directors - GlobeNewswire

NEW YORK and CLEVELAND, June 18, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that two abstracts related to its clinical program for recessive dystrophic epidermolysis bullosa (RDEB) will be presented at the upcoming Society for Pediatric Dermatology (SPD) 45th Annual Meeting, to be held virtually during July 10-12, 2020. RDEB is a rare connective tissue disorder without an approved treatment, in which patients suffer from severe epidermal wounds that severely impact their lives.

The first abstract features a more detailed examination than previously disclosed of data on long-term pain relief following durable healing of wounds in RDEB patients treated with EB-101 in a Phase 1/2 study. Separately, a literature review on the clinical, humanistic and economic burden of RDEB will be presented.

The posters will be presented by Jean Tang, M.D., Ph.D., Professor of Dermatology, Stanford University Medical Center and Principal Investigator of the EB-101 pivotal Phase 3 VIITALTM study, and M. Peter Marinkovich, M.D., Investigator on the VIITALTM study and Bullous Disease Clinic Director, Stanford University Medical Center. Details for the presentations are as follows:

Title: Durable Healing and Pain Reduction in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Following EB-101 Treatment of Large, Chronic WoundsPresenter: Jean Tang, M.D. Ph.D.Virtual Session Date: July 10-12, 2020

Title: The Full Burden of Recessive Dystrophic Epidermolysis Bullosa (RDEB)Presenter: M. Peter Marinkovich, M.D.Virtual Session Date: July 10-12, 2020

Following the conclusion of each virtual presentation, the posters will be available on the Events page under the Investors & Media section of Abeonas website at http://www.abeonatherapeutics.com.

More details about the programs for the SPD Annual Meeting are available at https://pedsderm.net/meetings/annual-meeting/.

About EB-101EB-101 is an autologous, gene-corrected cell therapy currently being investigated in the pivotal Phase 3 VIITALTM study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITALTM study is a multi-center, randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 30 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patients own skin cells (keratinocytes and its progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITALTM study at its fully-functional gene and cell therapy manufacturing facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101 provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. More information on the clinical trials of EB-101 can be found at https://www.abeonatherapeutics.com/clinical-trials/rdeb and ClinicalTrials.gov.

About Recessive Dystrophic Epidermolysis BullosaRecessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Companys portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeonas library of novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeonas fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITALTM study and is capable of clinical and commercial production of AAV gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Companys clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Companys goals and objectives. We have attempted to identify forward looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Companys Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

Investor Contact:Greg GinVP, Investor RelationsAbeona Therapeutics+1 (646) 813-4709ggin@abeonatherapeutics.com

Media Contact:Scott SantiamoDirector, Corporate CommunicationsAbeona Therapeutics+1 (718) 344-5843ssantiamo@abeonatherapeutics.com

Link:
Abeona Therapeutics Announces Upcoming Data Presentations at the Society for Pediatric Dermatology 45th Annual Meeting - GlobeNewswire

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, announced today that it has expanded its program for the manufacture of TUSC2 (Tumor Suppressor Candidate 2) plasmid DNA for its lead drug candidate, Oncoprex immunogene therapy, by entering into a new agreement with manufacturing partner Aldevron, LLC, (Aldevron). The new agreement provides for production of TUSC2 plasmid DNA, the active agent in Oncoprex, at full commercial scale. The Companys manufacturing at this scale should also result in significantly lower costs per unit of product manufactured due to economies of scale.

Genprexs upcoming clinical trials include a Phase I/II trial of Oncoprex combined with osimertinib (marketed by AstraZeneca as Tagrisso) for non-small cell lung cancer (NSCLC), which received Fast Track Designation in January 2020 and is expected to be initiated in early 2021. A clinical trial of Oncoprex in combination with pembrolizumab (marketed by Merck as Keytruda) in NSCLC is also planned.

We are pleased with continued progress in the scale-up of our manufacturing processes. This new agreement with Aldevron increases our manufacturing capabilities in support of our clinical trials utilizing Oncoprex immunogene therapy in combination with targeted therapies and immunotherapies against lung cancer, said Rodney Varner, Chairman and Chief Executive Officer of Genprex.

Our team is excited about the expansion of our manufacturing agreement and elevation of our long-standing relationship with Genprex, said Michelle Berg, President of GMP Nucleic Acids at Aldevron. Aldevrons GMP facility and campus buildout ensures we can meet the future manufacturing demands of companies experiencing significant growth such as Genprex; thereby enabling our ultimate goal of impacting the lives of patients.

Oncoprex consists of TUSC2 plasmid DNA encapsulated in a lipid nanoparticle. The TUSC2 gene is the active agent in Oncoprex. Data indicate that the resultant product when transfected into cancer cells both induces cell signaling that triggers programmed cell death and modulates the immune system so that the cancer cells are more susceptible to treatment.

About Aldevron

Aldevron serves the biotechnology industry offering in-stock products and custom production of nucleic acids, proteins, and antibodies. Thousands of clients use Aldevron-produced plasmids, RNA and gene editing enzymes for projects ranging from discovery research to clinical trials to commercial applications. These products are critical raw materials and key components in commercially available drugs and medical devices. Aldevron specializes in GMP manufacturing and is known for inventing the GMP-Source quality system. It is currently constructing a second manufacturing site on its headquarter campus in Fargo, North Dakota, more than doubling its production capacity. It has additional facilities in Madison, Wisconsin, and Freiburg, Germany. To learn more, visit http://www.aldevron.com.

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 Oncoprex, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our products; the timing and success of obtaining FDA approval of Oncoprex 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.

Read more:
Genprex Expands Manufacturing Program with Aldevron to Advance Oncoprex Clinical Development - Business Wire

The Gene Therapy for Age-related Macular Degeneration Market research report added by Report Ocean, is an in-depth analysis of the latest developments, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The research report compiled by Report Ocean offers the study of market opportunities and market impact that are created due to the COVID-19 pandemic. The overall market impact of COVID-19 can be witnessed in the Q1 2020 but is anticipated to be highly impacted in subsequent quarters in the whole year.

In addition to enlightens the current competitive putting and growth plans enforced with the aid of the Gene Therapy for Age-related Macular Degeneration Market players. Comprehensive secondary research was done to collect information on the market and its parent and ancillary markets. Further, primary research was performed to validate the assumptions and findings obtained from secondary research with key opinion leaders (KOL) and industry experts.

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Gene Therapy for Age-related Macular Degeneration Market Forecast Under COVID-19

Under the public background of the COVID-19 crisis, the industry is being affected by COVID-19. It is currently impossible to accurately predict the degree of control of this epidemic in various countries, and it is impossible to predict whether it will function normally for long-term economic activities.

However, historical data shows that the impact of natural disasters on the macro economy is usually short-term, and the economy often shows a V-shaped trend. Affected by the disaster and market sentiment, the economic growth rate usually drops sharply in a short period of time, but as the disaster is gradually controlled, the market sentiment returns to stability, and the temporarily suppressed consumption and investment needs will be released, resulting in Economic rebound.

Competitive Landscape:

The competitive analysis of major market players is another notable feature of the Gene Therapy for Age-related Macular Degeneration Market report; it identifies direct or indirect competitors in the market.

Key parameters which define the competitive landscape of the Gene Therapy for Age-related Macular Degeneration Market:

Revenue and Market Share by Player

Production and Share by Player

Average Price by Player

Base Distribution, Sales Area and Product Type by Player

Concentration Rate

Mergers & Acquisitions, Expansion

Manufacturing Base

Major players in the global Gene Therapy for Age-related Macular Degeneration market include:RetroSense TherapeuticsAGTCREGENXBIO

Market Segmentation:

Gene Therapy for Age-related Macular Degeneration Market is segmented on the basis of types, on the basis of applications and regions.

Geographical Analysis

Gene Therapy for Age-related Macular Degeneration Market Segment by Regions Consists:

North America (U.S. and Canada)

Europe (UK, Germany, France, Russia, Italy and Rest of Europe)

Asia-Pacific (China, Japan, India, Malaysia, Singapore, Philippines, Indonesia, Thailand, Vietnam)

South America (Brazil, Argentina, Mexico, and Rest of South America)

The Middle East and Africa (Saudi Arabia, United Arab Emirates, Turkey, Egypt, South Africa, Nigeria)

Some of the Major Highlights of TOC covers:

Gene Therapy for Age-related Macular Degeneration Market Production, Revenue (Value), Price Trend by Type

Production and Market Share by Type

Revenue and Market Share by Type

Price by Type

Gene Therapy for Age-related Macular Degeneration Market Analysis by Application

Consumption and Market Share by Application

Gene Therapy for Age-related Macular Degeneration Market Production, Consumption, Export, Import by Region

Production, Consumption, Export, Import by Region

Production, Consumption, Export, Import by Country

Production, Revenue, Price and Gross Margin

Gene Therapy for Age-related Macular Degeneration Market Manufacturing Analysis

Key Raw Materials Analysis

Market Concentration Rate of Raw Materials

Manufacturing Cost Analysis

Labor Cost Analysis

Manufacturing Cost Structure Analysis

Manufacturing Process Analysis of Gene Therapy for Age-related Macular Degeneration Market

Industrial Chain, Sourcing Strategy and Downstream Buyers

Gene Therapy for Age-related Macular Degeneration Market Chain Analysis

Raw Materials Sources of Gene Therapy for Age-related Macular Degeneration Market Major Players in 2020

Downstream Buyers

Market Dynamics

Market Drivers

Restraints

Opportunities

Increased Demand in Emerging Markets

Challenges

Porters Five Forces Analysis

Gene Therapy for Age-related Macular Degeneration Market Forecast (2020-2027)

Gene Therapy for Age-related Macular Degeneration Market Production, Revenue Forecast

Gene Therapy for Age-related Macular Degeneration Market Production, Consumption, Export and Import Forecast by Region

Gene Therapy for Age-related Macular Degeneration Market Production, Revenue and Price Forecast by Type

Gene Therapy for Age-related Macular Degeneration Market Consumption Forecast by Application

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Gene Therapy for Age-related Macular Degeneration Market by global COVID-19 impact analysis, industry trends, business strategies, opportunities and...

Credit: Robert Lisak

Exosomes are a sensational biological discovery. These minute lipid sacsamong the smallest of biological particles known as nanovesiclesare produced and then secreted by all cell types in all animal species. Bacteria produce very similar nanovesicles.

Exosomes are present in all body fluids and seem to be involved in nearly all biological processes. The main function of exosomes is to enter cells, either nearby in the tissues or systemically after transiting through the bloodstream, to deliver the genetic information that they carry. In particular, exosomes transfer microRNAs (miRNAs)small ribonucleotide polymers of about 22 bases. The extracellular miRNAs carried by exosomes can lead to alterations of DNA in the nuclei of targeted acceptor cells. The modifications to cellular DNA, in turn, alter the production of proteins and, therefore, change cell function. Exosomes are unanticipated universal nanoparticles that can mediate previously undiscovered biological processes, and alter molecular and metabolic pathways of cells and whole organisms.

These universal nanoparticles of life are likely to be of great medical importance. They might give researchers a better understanding of disease mechanisms, lead to new diagnostic tests and, perhaps most importantly, provide a means to deliver new therapies. But this will happen only if researchers study these natural entities more intensively.

Unfortunately, biomedical engineers have instead fixated on a different and less promising avenue: the development of artificial nanoparticles that imitate the function of natural exosomes for drug and small RNA delivery. Compared with naturally occurring exosomes, which have evolved an optimal composition over billions of years, engineered nanoparticles have a number of downsides. Unlike exosomeswhich can cross natural tissue barriers such as the bloodbrain barrier, can have effects for four to five days after administration and can enter the bloodstream1artificial nanoparticles cannot cross such barriers and are rapidly eliminated by mechanisms that detect foreign entities. Natural exosomes in the blood avoid physiological clearance mechanisms, but engineered nanoparticles are taken up and destroyed.

Exosome membranes are composed of unusual proportions of lipid components that give them a high surface viscosity and rigidity. This composition aids their survival in harsh conditions that kill cells. Such properties might be derived from the ancient origins of exosomes antecedent vesicles in noxious primordial seas near the beginning of biological evolutioneven before the development of bacteria.

Exosomes remarkable resistance to harsh conditions, such as the acidic and digestive-enzyme-rich environment of the stomach, means that they could be given orally as therapeutics1. Not only would this be more acceptable to and comfortable for patients, especially children, than intravenous, intraperitoneal and subcutaneous routes. But oral administration has also been shown to be a superior delivery method in mice.

Their stability and resilience are only part of what makes exosomes a natural choice for delivering genetic and anti-inflammatory molecules as therapies, both locally and systemically. They also lend themselves to therapeutic use in numerous other ways. It is likely that exosomes can be isolated from healthy individuals, and that a biologically active subpopulation can easily be enriched by a purification method called antigen or antibody affinity chromatography to promote therapy. Exosomes can also, in some instances, be used across species, without concern for immunological or genetic incompatibility, because miRNAs are often universal. Exosomes from plants might even have some medical use. And because exosomes do not contain full-length DNA, they are unlikely to cause cancer.

Exosomes also have an advantage over artificial drug carriers when it comes to targeting. Some exosomes can bind to selected antigen-specific antibody chains on their surfaces2. This gives exosomes an unrivalled ability to specifically target acceptor cells expressing particular surface antigens. Their uniquely targeted gene-altering miRNA cargo is also simple for researchers to load because activated exosomes can associate with miRNAs of choice by mere incubation3. Exosomes could therefore be used both to battle pathogens and to facilitate gene therapies for a variety of disorders.

Research indicates that exosomes might be effective therapies for diseases such as cancer, multiple sclerosis, rheumatoid arthritis, stroke, spinal-cord injury, myocardial infarction and lung fibrosis. Furthermore, investigations have begun into the use of exosome therapy for neurological conditions such as Alzheimers disease, Parkinsons disease and even autism spectrum disorder. However, much more work is needed before RNA-carrying exosomes can fulfil their therapeutic potential. One important task is to determine the nature of the surface molecules on exosomes that allow them to bind to targeting antibodies, as well as the molecular arrangements that allow them to also associate with selected therapeutic RNAs. Artificial nanoparticles do not have these capabilities. Now is the time for researchers to usher in a new era of therapeutic possibilities using RNA-delivering, natural exosome vesicles.

Philip W. Askenase is an immunologist at Yale University School of Medicine in New Haven, Connecticut.e-mail: philip.askenase@yale.edu

Here is the original post:
Artificial nanoparticles are not as good as the real thing - Scientific American

New York, June 18, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Membrane Chromatography Market by Product, Technique, Operation Mode, End User - Global Forecast to 2025" - https://www.reportlinker.com/p05339881/?utm_source=GNW 5% during the forecast period. The major factors driving the growth of this market are the benefits offered by membrane chromatography over conventional chromatography methods, increasing biopharmaceutical R&D, and increasing regulatory scrutiny on the cleaning validation of downstream purification processes.

The consumables segment commanded the largest share of the market in 2019.On the basis of product, the membrane chromatography market is segmented into consumables and accessories.The capsules, cassettes, and cartridges segment accounted for the largest share of the membrane chromatography consumables market in 2019.

The large share of this segment is primarily attributed to the increasing use of capsules and cartridges for purification applications in biomanufacturing, lab-scale production, and process development.

The affinity membrane chromatography segment is expected to register the highest growth in the global membrane adsorbers market during the forecast period.On the basis of technique, the membrane adsorbers market is segmented into ion exchange membrane chromatography, affinity membrane chromatography, and hydrophobic interaction membrane chromatography.The affinity membrane chromatography segment is estimated to grow at the highest CAGR during the forecast period.

The increasing use of protein A chromatography for antibody purification and immobilized metal affinity membrane chromatography for the purification of proteins are supporting the growth of this segment.

The pharmaceutical and biopharmaceutical companies segment commanded the largest share of the membrane chromatography market in 2019.Based on end user, the membrane chromatography market is segmented into pharmaceutical and biopharmaceutical companies, CROs, and academic and research institutes.In 2019, the pharmaceutical and biopharmaceutical companies segment accounted for the largest market share.

The large share of this segment is attributed to the increased R&D in pharmaceutical and biopharmaceutical companies and increased production of biologics.

The bind-elute segment is projected to register the highest growth in the global membrane adsorbers market during the forecast period.The bind-elute membrane chromatography segment is estimated to grow at the highest CAGR during the forecast period. The increasing demand for viruses in the production of attenuated vaccines and gene therapy and the development of high-performance bind-elute membrane chromatography solutions (with a higher binding capacity to capture larger targets such as adenovirus and lentivirus and virus-like particles) are the key factors driving the growth of this segment.

The Asia Pacific market is projected to register the highest growth in the global membrane chromatography market during the forecast period.The Asia Pacific market is estimated to grow at the highest CAGR during the forecast period. The growing biopharmaceutical industry, significant investments by key market players, increasing government support, and developing R&D infrastructure are the key factors driving the growth of this segment.

In-depth interviews were conducted with chief executive officers (CEOs), marketing directors, other directors, and executives from various key organizations operating in the membrane chromatography market. By Respondent Type: Supply Side (70%) and Demand Side (30%) By Designation: Managers (55%), CXOs (20%), and Executives (25%) By Region: North America (50%), Europe (20%), APAC (20%), and the RoW (10%)

The membrane chromatography market comprises major players such as Sartorius AG (Germany), Danaher Corporation (US), Merck Millipore (Germany), Thermo Fisher Scientific Inc. (US), and 3M Company (US). The study includes an in-depth competitive analysis of these key players in the membrane chromatography market, along with their company profiles, recent developments, and key market strategies.

Research Coverage:The market study covers the membrane chromatography market across various segments.It aims at estimating the market size and the growth potential of this market across different segments based on product, technique, end user, and region.

The study also includes an in-depth competitive analysis of the key players in the market, along with their company profiles, key observations related to their product and business offerings, recent developments, and key market strategies.

Key Benefits of Buying the Report:The report will help market leaders/new entrants in this market and provide information on the closest approximations of the revenue numbers for the overall membrane chromatography market and its subsegments.This report will help stakeholders to understand the competitive landscape, to gain more insights to better position their businesses, and to plan suitable go-to-market strategies.

The report will also help stakeholders to understand the pulse of the market and provide information on the key market drivers, restraints, opportunities, and challenges.Read the full report: https://www.reportlinker.com/p05339881/?utm_source=GNW

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

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The membrane chromatography market is estimated to grow from USD 198 million in 2020 to USD 408 million by 2025, at a CAGR of 15.5% - GlobeNewswire

bluebird on the Go with Multiple Positive Trial Data

bluebird bio (BLUE) announced data from its different trials, including Phase 1/2 clinical trial for sickle cell disease and from a Phase 3 clinical trial for transfusion-dependent beta-thalassemia patients.

Ongoing Phase 1/2 clinical trial HGB-206 aims to assess the potential of LentiGlobin in treating adolescents and adults with sickle cell disease. As on the data cutoff date, 37 patients had been treated. Group C showed promising results as all the patients included in the cohort stopped regular blood transfusions. These patients also remained transfusion-free for the time period of three months after the treatment.

Sixteen patients who were followed up for minimum six months maintained the median levels of gene therapy-derived anti-sickling hemoglobin. It also contributed to minimum 40 percent of total hemoglobin. 14 patients with previous background of acute chest syndrome (ACS) and vaso-occlusive crisis (VOC) who have been observed for a minimum time period of six months showed a 99.5 percent drop in the annualized rate of ACSs and VOCs.

Its ongoing Phase 3 clinical trials aimed to test the prospects of Zynteglo, formerly known as LentiGlobin, in treating adolescent and adult patients suffering from a range of genotypes of transfusion-dependent beta-thalassemia. Phase 3 of the Northstar-2 study involved 23 patients who were treated and observed for a median time period of 19.4 months. 89.5 percent of evaluable participants numbering 19 showed transfusion independence. Earlier, the 17 needed a median of 17.5 transfusions each year.

The Northstar-3 study involved 15 patients with genotypes of different variations. These patients were treated and then observed for a median time period of 14.4 months. 75.0% (n=6/8) reported to be transfusion independent. 84.6% (n=11/13) with a minimum of seven months' follow-up did not require a transfusion in more than seven months. Earlier, 11 of the patients needed a median of 18.5 transfusions every year.

bluebirds gene therapy involves extracting a patients stem cell and then altering it with new genetic instructions. Such altered stem cell is then infused back into the patients body. David Davidson, M.D., chief medical officer, bluebird bio, said, Vaso-occlusive crises (VOCs) are the painful, life-threatening episodes that are the primary clinical manifestation of sickle cell disease. The nearly complete elimination of VOCs that we saw in this study is impressive and demonstrates the potential of LentiGlobin for SCD as a treatment for this serious disease.

The Northstar-2 study showed three and two serious occurrences of veno-occlusive liver disease and thrombocytopenia respectively. Northstar-3 showed two serious occurrences of pyrexia. However, there was no reporting of any death, graft failure or leukemia. Zynteglo is already approved in the European Union, and the company is looking to start treating patients in Germany in the near future. It also plans to apply for the FDA approval in mid-2021.

For its Phase 1/2 trial in sickle cell anemia, an earlier report had shown significant improvement in VOCs and ACS after the gene therapy. Pursuant to these results, the company consulted with the FDA to alter the primary endpoint of the trial from improvement in hemoglobin measurements to the elimination of VOCs.

BeiGene (BGNE) announced new data from its trials aimed at assessing the potential of BTK inhibitor Brukinsa (zanubrutinib) and PD-1 inhibitor tislelizumab in treating various blood cancers. Brukinsa is a small molecule inhibitor of Brutons tyrosine kinase. It is currently being assessed as a monotherapy and in combination with other therapies of treating a wide range of B-cell malignancies.

For Phase 1/2 study of Brukinsa in patients suffering from B-cell malignancies, the overall response rate was 80 percent, while complete response rate was 15 percent. Further, the partial response rate for the trial was 65 percent. The median time to response was measured at 2.8 months. The progression-free survival rate and overall survival rate at 24 months were 59.4 percent and 83.2 percent respectively. Zanubrutinib was found to be well-tolerated in patients with R/R MZL, however, all patients experienced at least one adverse event.

The company also presented data from a Phase 2 trial of zanubrutinib with rituximab in patients suffering from R/R NHL. The trial had 41 participants, including 20 patients suffering from non-GCB DLBCL who previously received standard anthracycline rituximab-based treatment. For patients with R/R non-GCB DLBCL, the ORR was 35 percent and the CR and PR rates were 5 percent and 30 percent, respectively. For patients with R/R FL, the OR was 56.3 percent, with CR and PR at 19 percent and 38 percent, respectively.

Brukinsa is already approved in the United States for treating adult patients suffering from mantle cell lymphoma who have been given minimum one prior therapy. It was given the approval under accelerated approval pathway on the basis of its overall response rate. The drug is approved in China for treating MCL in adult patients who have received at least one prior therapy and CLL or SLL in adult patients who have received at least one prior therapy. However, the drug is not approved outside of the United States and China.

TG Therapeutics (TGTX) reported positive interim data from its Phase 1 parallel dose-escalation study of TG-1701. The drug candidate is being tested as a monotherapy and in combination with U2 for treating relapsed/refractory B-cell malignancies.

The data pertained to 82 patients suffering from the condition. Sixty-nine patients were given single agent TG-1701, out of which 25 were included in the monotherapy dose escalation cohort of the study and were given the drug candidate in 100mg to 400mg dosage once a day daily. The remaining 44 patients were included in the monotherapy dose expansion arm and were administered 200mg of the drug candidate. The TG-1701 plus U2 dose escalation portion of the study treated an additional 13 patients.

For the monotherapy dose escalation cohort with 25 patients, the drug candidate showed partial response for all the dose levels in CLL, MCL, WM and SLL. For the monotherapy dose expansion cohort, the overall response rate was recorded at 92 percent in CLL patients. MCL patients and WM patients showed 33 percent and 86 percent ORR respectively. The combination of the drug candidate with U2 showed positive clinical activity with a 77 percent ORR for all disease types. It included complete response rate in three patients. The company continues with dose escalation.

TG-1701 showed a positive preliminary safety profile for all dose levels. No patient had to discontinue the treatment, but 3 percent of the patients had to reduce the dose due to the occurrence of treatment-related adverse events. The company also provided data pertaining Phase I/Ib study aiming to assess the potential of ibrutinib in blend with umbralisib for patients with relapsed/refractory CLL or MCL.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, weve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Original post:
bluebird On The Go, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

The global Genetic Modification Therapies market report provides geographic analysis covering regions, such as North America, Europe, Asia-Pacific, and Rest of the World. The Genetic Modification Therapies market for each region is further segmented for major countries including the U.S., Canada, Germany, the U.K., France, Italy, China, India, Japan, Brazil, South Africa, and others.

The global Genetic Modification Therapies market is expected to exceed more than US$ 3.5 Billion by 2024 at a CAGR of 34% in the given forecast period.

Genetic modification therapies, significantly gene therapy and RNA therapy, have existed for many years, with very little clinical success. However, recent enhancements in these therapies, together with higher delivery systems, additional economical and sturdy gene expression constructs, precise polymer editing tools, have brought this industry to the forefront, and its currently poised for explosive growth within the coming back years.

Browse Full Report: https://www.marketresearchengine.com/genetic-modification-therapies-market

Because of the potentially curative nature of those medicines theres monumental potential in several applications, starting from cancer to neurology to rare diseases. Genetic modification therapies represent consecutive wave of medicines with monumental potential for treating and curing draining and high diseases. As a result of its wide scope, genetic modification therapy can play a vital role within the future world medical economy.

Continuing advances in key technologies like DNA editing, viral design and production, and gene expression, further as a pressing medical want in several serious and enervating disorders, are driving the expansion of the marketplace for genetic modification therapies. Developments in these multidisciplinary fields promise to advance the genetic modification therapies trade and build distinctive market opportunities.

The overall market is anticipated to witness important growth in opportunities for a spread of stakeholders within the returning decade. its necessary to spotlight that many technology suppliers, reaching to develop and / or support the event of gene therapies, with improved effectiveness and safety, have designed and already introduced advanced platforms for the engineering of vectors. Innovation during this domain has additionally semiconductor diode to the invention of novel molecular targets and strong the analysis pipelines of corporations targeted during this house. the potential to focus on numerous therapeutic areas is taken into account to be amongst the foremost outstanding growth drivers of this market.

Market Insights

The global Genetic Modification Therapies market is segregated on the basis of Platform Technology as Gene editing, Gene Therapies, Genetically Modified Cell Therapies, and RNA Therapies. Based on Delivery Technologies the global Genetic Modification Therapies market is segmented in AAV, Adenovirus, Lentivirus, Retrovirus, Other Viral, and Nonviral Based on End-User Industry the global Genetic Modification Therapies market is segmented in Hospitals, Diagnostics and Testing Laboratories, Academic and Research Organizations, and Others.

Based on Disease, the global Genetic Modification Therapies market is segmented in Cardiology, Oncology, Ophthalmology, Hematology, Musculoskeletal, Neurology, Rare Diseases, Other Indications.

Competitive Rivalry

4D Molecular Therapeutics, Abeona Therapeutics, Beam Therapeutics, Casebia Therapeutics, Editas Medicine, Fate Therapeutics, GE Healthcare, Hitachi Chemical Advanced Therapeutics, Immunocore, Jivana Biotechnology, and others are among the major players in the global Genetic Modification Therapies market. The companies are involved in several growth and expansion strategies to gain a competitive advantage. Industry participants also follow value chain integration with business operations in multiple stages of the value chain.

The Genetic Modification Therapies Market has been segmented as below:

The Genetic Modification Therapies Market is segmented on the lines of Genetic Modification Therapies Market, By Platform Technology, Genetic Modification Therapies Market, By Delivery Technologies, Genetic Modification Therapies Market, By End-User Industry, Genetic Modification Therapies Market, By Disease, Genetic Modification Therapies Market, By Region and Genetic Modification Therapies Market, By Company.

Genetic Modification Therapies Market, By Platform Technology this market is segmented on the basis of Gene editing, Gene Therapies, Genetically Modified Cell Therapies and RNA Therapies. Genetic Modification Therapies Market, By Delivery Technologies this market is segmented on the basis of AAV, Adenovirus, Lentivirus, Retrovirus, Other Viral and Nonviral. Genetic Modification Therapies Market, By End-User Industry this market is segmented on the basis of Hospitals, Diagnostics and Testing Laboratories, Academic and Research Organizations and Others. Genetic Modification Therapies Market, By Disease this market is segmented on the basis of Cardiology, Oncology, Ophthalmology, Hematology, Musculoskeletal, Neurology, Rare Diseases and Other Indications. Genetic Modification Therapies Market, By Region this market is segmented on the basis of North America, Europe, Asia-Pacific and Rest of the World. Genetic Modification Therapies Market, By Company this market is segmented on the basis of 4D Molecular Therapeutics, Abeona Therapeutics, Beam Therapeutics, Casebia Therapeutics, Editas Medicine, Fate Therapeutics, GE Healthcare, Hitachi Chemical Advanced Therapeutics, Immunocore and Jivana Biotechnology.

The report covers:

Global Genetic Modification Therapies market sizes from 2015 to 2024, along with CAGR for 2018-2024Market size comparison for 2017 vs 2024, with actual data for 2017, estimates for 2018 and forecast from 2019 to 2024Global Genetic Modification Therapies market trends, covering comprehensive range of consumer trends & manufacturer trendsValue chain analysis covering participants from raw material suppliers to the downstream buyer in the global Genetic Modification Therapies marketMajor market opportunities and challenges in forecast timeframe to be focusedCompetitive landscape with analysis on competition pattern, portfolio comparisons, development trends and strategic managementComprehensive company profiles of the key industry players

Report Scope:

The global Genetic Modification Therapies market report scope includes detailed study covering underlying factors influencing the industry trends.

The report covers analysis on regional and country level market dynamics. The scope also covers competitive overview providing company market shares along with company profiles for major revenue contributing companies.

The report scope includes detailed competitive outlook covering market shares and profiles key participants in the global Genetic Modification Therapies market share. Major industry players with significant revenue share include 4D Molecular Therapeutics, Abeona Therapeutics, Beam Therapeutics, Casebia Therapeutics, Editas Medicine, Fate Therapeutics, GE Healthcare, Hitachi Chemical Advanced Therapeutics, Immunocore, Jivana Biotechnology, and others.

Reasons to Buy this Report:

Gain detailed insights on the Genetic Modification Therapies industry trendsFind complete analysis on the market statusIdentify the Genetic Modification Therapies market opportunities and growth segmentsAnalyse competitive dynamics by evaluating business segments & product portfoliosFacilitate strategy planning and industry dynamics to enhance decision making

Request Sample Report from here: https://www.marketresearchengine.com/genetic-modification-therapies-market

Table of Contents:

IntroductionResearch MethodologyExecutive SummaryMarket Overview4.1 Introduction4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.2.4 Challenges4.2 Porters Five Force AnalysisGenetic Modification Therapies Market, By Platform TechnologyGenetic Modification Therapies Market, By Delivery TechnologiesGenetic Modification Therapies Market, By End-User IndustryGenetic Modification Therapies Market, By DiseaseGenetic Modification Therapies Market, By GeographyCompetitive InsightsCompany Profiles11.1 4D Molecular Therapeutics11.1.1 Company Overview11.1.2 Product/Service Landscape11.1.3 Financial Overview11.1.4 Recent Developments11.2 Abeona Therapeutics11.2.1 Company Overview11.2.2 Product/Service Landscape11.2.3 Financial Overview11.2.4 Recent Developments11.3 Beam Therapeutics,11.3.1 Company Overview11.3.2 Product/Service Landscape11.3.3 Financial Overview11.3.4 Recent Developments11.4 Casebia Therapeutics,11.4.1 Company Overview11.4.2 Product/Service Landscape11.4.3 Financial Overview11.4.4 Recent Developments11.5 Editas Medicine,11.5.1 Company Overview11.5.2 Product/Service Landscape11.5.3 Financial Overview11.5.4 Recent Developments11.6 Fate Therapeutics,11.6.1 Company Overview11.6.2 Product/Service Landscape11.6.3 Financial Overview11.6.4 Recent Developments11.7 GE Healthcare,11.7.1 Company Overview11.7.2 Product/Service Landscape11.7.3 Financial Overview11.7.4 Recent Developments11.8 Hitachi Chemical Advanced Therapeutics,11.8.1 Company Overview11.8.2 Product/Service Landscape11.8.3 Financial Overview11.8.4 Recent Developments11.9 Immunocore,11.9.1 Company Overview11.9.2 Product/Service Landscape11.9.3 Financial Overview11.9.4 Recent Developments11.10 Jivana Biotechnology,11.10.1 Company Overview11.10.2 Product/Service Landscape11.10.3 Financial Overview11.10.4 Recent Developments

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Genetic Modification Therapies Market 2019 | How The Industry Will Witness Substantial Growth In The Upcoming Years | Exclusive Report By MRE - Cole...

INTRODUCTION Over the last few years, the exponential growth in the pipeline of nucleic acid based therapies has led to the escalating interest of pharmaceutical industry in this domain.

New York, June 18, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Non-Viral Transfection Reagents and Systems Market, 2020-2030" - https://www.reportlinker.com/p05915358/?utm_source=GNW Presently, more than 2,000 trials evaluating different types of gene therapies are underway. Moreover, according to experts at the US FDA, around 40 new gene therapies are likely to be approved by 2022. In this context, it is worth highlighting that viral vectors are a crucial element in gene therapy development and manufacturing. Although, viral vectors have shown significant success in R&D, their applications are limited due to immunogenicity and toxicity related concerns, high development costs and the limitation on amount of genomic material that they can carry. Excessively high price tags associated with viral-based therapies, such as Zolgensma (USD 2.1 million) and Luxtruna (USD 850,000), have led to several reimbursement challenges, thereby decreasing patient access. Owing to the aforementioned concerns related to viral vectors, therapy developers are evaluating a variety of non-viral methods of gene delivery.

In the present scenario, non-viral transfection systems are not yet widely used in therapy development and clinical studies, primarily due to their relatively low efficiency compared to viruses. The applications of these methods are largely restricted to fundamental research, including protein and gene expression, and cell line development. However, there are a number of companies that have developed proprietary technologies and products to facilitate physical (electroporation, gene gun, microinjection and sonoporation), chemical (transfection reagents) and other non-viral methods of transfection (transposon based systems, piggyBac and magnetofection). We believe that, as the demand for advanced therapy medicinal products, which require genetic engineering, the opportunity for non-viral transfection system developers is also likely to grow.

SCOPE OF THE REPORTThe Non-Viral Transfection Reagents and Systems Market, 2020-2030 report features an extensive study of the various systems and technologies available for non-viral transfection, in addition to the current market landscape and future potential of product developers.

Amongst other elements, the report features: A detailed assessment of the competitive landscape of various types of non-viral transfection systems, including transfection reagents, electroporation-based transfection systems and other non-viral transfection systems, featuring product specific information, such as type of carrier used, type of molecule delivered, recommended cell type and price of the system (if available). Additionally, the chapter includes information on non-viral transfection system developers, including information on year of establishment, company size and geographical location. An insightful 2X2 representation, highlighting the competitiveness analysis of non-viral transfection system developers captured in our database, taking into consideration supplier power (based on company size) and service strength (based on strength of product portfolio, years of experience and type of molecule delivered). An analysis highlighting potential strategic partners segregated based on likelihood of entering into collaboration with non-viral transfection system developers. The analysis takes into consideration multiple relevant parameters, such as type of therapy, pipeline strength, pipeline maturity, type of vector and company size. An analysis of the big pharma players engaged in this domain, featuring a heat map based on parameters, such as type of initiative, type of therapy and target therapeutic area. An analysis of completed, ongoing and planned clinical studies related to non-viral transfection systems, featuring details on registration year, trial phase, trial status, type of sponsor, type of therapy, target therapeutic area, trial design, type of patient allocation model used, type of trial masking adopted, type of intervention, trial purpose, geographical location of trial and enrolled patient population. An in-depth analysis of close to 14,000 patents related to non-viral transfection systems that have been filed / granted since 2017, highlighting key trends associated with these patents, across type of patents, publication year, geographical location, type of applicants, issuing authority / patent offices involved, CPC symbols, emerging focus areas, leading players (in terms of number of patents granted / filed in the given time period), patent characteristics and geography. It also includes a detailed patent benchmarking and an insightful valuation analysis. A detailed publication analysis of more than 1,100 peer-reviewed, scientific articles that have been published since 2014, highlighting the key trends associated with these publications, across year of publication, focus area, type of molecule delivered, target therapeutic area, important cells and cell lines evaluated, leading players across different geographies and key journals (in terms of number of articles published in the given time period) within this domain.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future opportunity within the market. Based on several parameters, such as annual number of transfections, cost per transfection, share of non-viral systems within the transfections market and expected annual growth rate across various geographies, we have provided an informed estimate of the likely evolution of the market, in the mid to long term, for the period 2020-2030. The chapter features the likely distribution of the future opportunity across [A] non-viral transfection systems (non-viral transfection reagents, electroporation-based transfection systems and other non-viral transfection systems), [B] end-users (academic and research institutions, pharmaceutical companies and other end-users), [C] area of application (clinical application and research application) and [D] geographical regions (North America, Europe, Asia-Pacific and rest of the world).

In order to account for future uncertainties and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the markets evolution. The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. In addition, the report features detailed transcripts of interviews held with the following individuals (in alphabetical order of company names): George Eastwood (Vice-President of Business Development, Kytopen) Claudia Andretta (Global Business Development Manager, Clinical, Polyplus-transfection)

All actual figures have been sourced and analyzed from publicly available information forums. Financial figures mentioned in this report are in USD, unless otherwise specified..

RESEARCH METHODOLOGYThe research, analysis and insights presented in this report are backed by a deep understanding of insights gathered from both secondary and primary sources. For all our projects, we conduct interviews with experts in the area (academia, industry and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts views

While the focus has been on forecasting the market till 2030, the report also provides our independent view on various non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

CHAPTER OUTLINESChapter 2 is an executive summary of the insights captured in our research. It offers a high-level view on the likely evolution of the non-viral transfection systems market in the mid to long term.

Chapter 3 provides an overview of transfection and its applications, such as advanced therapy medicinal product development, gene silencing, bioproduction of therapeutic protein and stem cell engineering. It highlights different methods of transfection (such as viral and non-viral), with a brief outline of various viral vectors (AAV, adenoviral, lentiviral, retroviral and others), chemical methods (lipoplexes, polyplexes, lipoplexes and others) and physical methods (electroporation, gene gun, sonoporation, magnetofection and others) used in transfection.

Chapter 4 provides an overview of various types of non-viral transfection systems including transfection reagents, electroporation-based transfection systems and other non-viral transfection systems, featuring product specific information, such as type of carrier used, type of molecule delivered, recommended cell type and price of the system (if available). Additionally, the chapter includes information on non-viral transfection system developers, including information on year of establishment, company size and geographical location.

Chapter 5 provides a detailed competitiveness analysis of the companies offering non-viral transfection systems, taking into consideration supplier power (based on company size) and service strength (based on strength of product portfolio, years of experience and type of molecule delivered).

Chapter 6 includes detailed profiles of prominent transfection reagent developers, electroporation-based transfection system developers and other non-viral delivery system developers, featuring a brief overview of the company, its financial information (if available), recent developments and an informed future outlook.

Chapter 7 features an insightful analysis, highlighting potential strategic partners, based on likelihood of entering into collaboration with non-viral transfection system developers, taking into account several parameters, such as type of therapy, pipeline strength, pipeline maturity, company size and type of vector.

Chapter 8 highlights the activity of top ten big pharma players in this domain, featuring a heat map based on parameters, such as such as type of initiative, type of therapy and target therapeutic area.

Chapter 9 provides an analysis of completed, ongoing and planned clinical studies related to non-viral transfection systems, featuring details on registration year, trial phase, trial status, type of sponsor, type of therapy, target therapeutic area, trial design, type of patient allocation model used, type of trial masking adopted, type of intervention, trial purpose, geographical location of trial and enrolled patient population.

Chapter 10 provides an in-depth patent analysis to provide an overview of how the industry is evolving from the R&D perspective. For this analysis, we considered those patents that have been filed / granted related to non-viral transfection systems, since 2017, highlighting key trends associated with these patents, across type of patents, publication year, geographical location, type of applicants, issuing authority / patent offices involved, CPC symbols, emerging focus areas, leading players (in terms of number of patents granted / filed in the given time period), patent characteristics and geography. It also includes a detailed patent benchmarking and an insightful valuation analysis.

Chapter 11 presents a detailed publication analysis of more than 1,100 peer-reviewed, scientific articles that have been published since 2014, highlighting the key trends associated with these publications, across year of publication, focus area, type of molecule delivered, target therapeutic area, important cells and cell lines evaluated, leading players across different geographies and key journals (in terms of number of articles published in the given time period) within this domain.

Chapter 12 presents a detailed market forecast, highlighting the future potential of the non-viral transfection systems market till the year 2030. The chapter features the likely distribution of the opportunity across different [A] non-viral transfection methods (non-viral transfection reagents, electroporation-based transfection systems and other non-viral transfection systems), [B] end-users (academic and research institutions, pharmaceutical companies and other end-users), [C] area of application (clinical application and research application) and [D] geographical regions (North America, Europe, Asia-Pacific and rest of the world).

Chapter 13 is a collection of executive insights of the discussions that were held with various key stakeholders in this market. The chapter provides a brief overview of the companies and details of interviews held with George Eastwood (Vice-President of Business Development, Kytopen) and Claudia Andretta (Global Business Development Manager, Clinical, Polyplus-transfection).

Chapter 14 summarizes the entire report. It presents a list of key takeaways and offers our independent opinion on the current market scenario. Further, it summarizes the various evolutionary trends that are likely to influence the future of this market.

Chapter 15 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 16 is an appendix, which contains the list of companies and organizations mentioned in the report.Read the full report: https://www.reportlinker.com/p05915358/?utm_source=GNW

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

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Non-Viral Transfection Reagents and Systems Market, 2020-2030 - Yahoo Finance

In recent years, we have seen a trend towards the launch of new gene and cell therapies with record-breaking price tags. Such headline-grabbing launches are becoming more and more frequent, as the pipeline for advanced therapies at all stages of development continues to grow at a rapid pace[1]. We are also seeing industry and payers adopting new innovative pricing models for those products, such as outcome-based reimbursement and annuity payment models. In this article, we discuss these emerging alternative pricing models and consider the impact they may have on related licensing arrangements.

Current trends

In May 2019 AveXis, a subsidiary of pharmaceutical giant Novartis, announced that it had received approval from the US Food and Drug Administration to market its gene therapy Zolgensma for the treatment of paediatric patients with spinal muscular atrophy (SMA). Although this is the first promise of a cure for this debilitating and lethal condition, the media coverage focussed instead on Zolgensmas price tag, which at $2.1 million per patient makes it (currently) the worlds most expensive single-dose medicine.

Zolgensma is illustrative of a general trend in gene and cell therapies that have reached the market in recent years and established a new standard of pricing for single-treatment medicines. While manufacturers point to the relative cost-effectiveness of such treatments (which may offer a one-off cure for severe conditions that otherwise would require several years worth of conventional treatments and care) public and private payers are concerned about this new escalating pricing paradigm.

Health care systems may be able to absorb such high prices for rare diseases with small patient populations. However, the current reimbursement systems will be under severe pressure if (as is hoped) pipelines for advanced cell and gene therapies result in treatments for common conditions such as diabetes or heart disease. The Institute for Clinical and Economic Review in the US has estimated that if gene therapies are developed to treat only one in ten American patients with a genetic condition approximately 1% of the total population the cumulative budget impact could rise to $3 trillion[2]. For comparison, the projected total healthcare spend in the US for 2019 is $3.8 trillion[3].

Alternative Pricing Models

The pharmaceutical industry has sought to counter criticism over the high price tags for gene and cell therapies by coupling these revolutionary therapies with new and unconventional pricing and reimbursement mechanisms.

One alternative structure that has been adopted is an annuity based model which spreads the payment for an expensive treatment over several years in a pre-agreed payment plan, thus minimising the up-front cost to payers.

Another approach adopted by the industry, and perhaps an even clearer way to demonstrate value to payers, has been to tie reimbursement to patient outcomes. The industry has negotiated several of these outcomes-based reimbursement models with public and private payers for cell and gene therapies. Reimbursement payments to the drug maker under this model are conditional upon the patient reaching specific clinical outcomes by set deadlines. Depending on the model, a patients failure to meet the specified clinical outcome can result in the drug maker having to refund payments received and/or forfeit any subsequent payments.

These new models are also being blended to create payment plans which combine annuity-style payments with rebates and outcomes-dependent instalments. We expect that in the years to come other creative payment models will emerge and be adapted from other therapy areas. For example, in Australia, the government has used a subscription style model that allowed it to pay a lump sum to drug makers for unlimited access for patients to curative hepatitis C treatments such as Sovaldi for a period of time.

Example annuity and outcomes-based reimbursement models for cell and gene therapies:

Licensing challenges

Cell and gene therapies often have their roots in academic research laboratories and the main players in this field of treatments have close ties and valuable licensing agreements with academic research institutions. For example, AveXis, the biotech company that developed Zolgensma, started as a spin-out to continue research conducted at the Center for Gene Therapy at Nationwide Childrens Hospital in Columbus, Ohio. To further its spinal muscular atrophy work, the biotech also licensed a patent owned by Martine Barkats, a researcher at the Institut de Myologie, Paris. Shortly after, AveXis was bought by Novartis for $8.7 billion. Cell and gene therapies such as Zolgensma will generally have more constituent parts (such as promoters, viral vectors and cell lines) than other more conventional small molecule therapies. This means that a party commercialising a cell or gene therapy will often need to license in more third party intellectual property or materials than a manufacturer of a conventional small molecule therapy. Most cell and gene therapies reaching the market are therefore likely to be underpinned by one or more licence agreements.Licensing challenges

While much has been said about the impact of alternative pricing and reimbursement mechanisms on drug makers, payers and patients, we want to also consider the impact on licensors of the intellectual property which enables the development and manufacture of a therapy. In particular, how future pricing and reimbursement models can impact the royalties payable by licensees to their licensors. One inherent challenge is that these licences are generally negotiated many years before the commencement of discussions with payers on pricing and reimbursement mechanisms, making it very difficult to predict which scenarios will be relevant down the line. The positions of all of the stakeholders in the pricing debate are also constantly evolving, especially as data on the cost-effectiveness of annuity and outcomes-based models continues to accumulate. One factor which makes things particularly difficult for licensors in forecasting potential future royalty streams for these products is that a licensor would rarely have any involvement in negotiations regarding pricing and reimbursement so will have no control over the model adopted.

Annuity model challenges

Generally a licensor will only receive royalties once the licensee has itself received (or at least invoiced) payment from payers. An annuity payment model is therefore likely to mean that royalties will also be paid in instalments potentially spread over a number of years following treatment of a patient. While in practice this may not be a large change for licensors to adjust to (as annual payments for these high price treatments are not out of line with other orphan drug costs, most of which need to be taken over a long period of time) there are also other factors to consider.

One concern that has been raised with annuity payment models is that there may be an increased risk of non-payment as over time licensees may face difficulties in collecting payments, for example because a payer stops complying with payment schedules or becomes insolvent. This may have the knock-on effect of reducing royalties due to a licensor. Licensors may seek to reduce this non-payment risk by asking that royalties are payable on sums invoiced by a licensee, rather than sums received (although this is likely to be resisted by a licensee or perhaps only accepted with caveats). Annuity-based models are also typically more complicated and more expensive for a licensee to manage administratively and those costs are likely to be deductible from sales totals before a licensors royalties are calculated.

From a legal drafting perspective, care would also need to be taken by the licensor when defining payment terms and the royalty term (which is commonly linked to patent expiry) to ensure that the licensor continued to receive royalties in respect of patients who are treated within the royalty term, notwithstanding the fact that payment may not be received until after the patents and royalty term has expired.

Outcome-based model challenges

In relation to outcome-based models, a fundamental concern for both licensors and licensees is the uncertainty associated with a model which involves an upfront payment of the full treatment price but a refund payable some months or years down the line if the clinical outcomes are not met.

If royalties are payable on net sales of the therapy on a regular basis (e.g. quarterly or annually) then unless the licence includes a mechanism to take account of outcomes-based refunds made by the licensee to payers, the licensee could find itself out of pocket, unable to recover royalties paid to the licensor despite having had to refund the therapy price to the payer. To counter this risk, a licensee may seek to build in a royalty claw back mechanism into the licence, or to delay the point at which royalties are payable until after the relevant patient has met the required outcome. However, a licensor is unlikely to accept a significant delay in payment of royalties, particularly where the licensee has itself been paid. Academic licensors, with an obligation to invest income from technology transfer activities into research and the provision of education, are particularly unlikely to agree a royalty claw back structure which could force them to refund royalties or milestones a year or more after having received them.

One alternative option may be to agree that the licensee can make deductions against future royalty payments. A further alternative could be for some portion of the royalties paid to be retained in escrow for a period of time, to be released to the licensor upon achievement of a positive clinical outcome or expiry of a set period of time. However, escrow arrangements necessarily increase the complexity of agreements and are difficult to negotiate upfront when payment and reimbursement models and the associated outcome triggers have not yet been set.

A compromise?

As we have outlined in this article, although there are some things each party can consider at the outset of negotiating a licence, getting into protracted negotiations about hypothetical scenarios is unlikely to be attractive to either party.

The parties may wish to adopt an alternative approach of including robust governance provisions in the licence to deal specifically with this issue. For example, establishing a committee comprised of representatives of both parties to oversee and review issues relating to pricing and reimbursement. This may give the licensor a clearer oversight (and potentially input) into decisions which may impact future royalty streams and may present the licensee with an opportunity to propose alternative payment structures to support its desired pricing model. This could be combined with a mechanism for proposing and agreeing amendments to payment provisions in the licence if necessary to accommodate pricing and reimbursement issues which were unforeseen at the outset. Of course the success of such mechanisms will depend on the strength of the relationship between the parties and a combined willingness to work together and potentially compromise. It would also be important to ensure it is clear what happens where the parties cannot agree (e.g. escalation? expert determination? preservation of the status quo?). However, in a future where pricing and reimbursement issues are only likely to become more complex and of key importance to the success of complex treatments such as cell and gene therapies, it will be interesting to see whether this is a route industry explores.

Conclusion

The launch in recent years of a number of advanced cell and gene therapies with blockbuster price tags has heralded a new era for drug pricing and associated payment and reimbursement issues. It is a trend that looks likely to continue if current pipelines can also deliver much anticipated advanced therapies for common conditions. The high prices associated with these products present a myriad of issues however, not only for patients, payers and healthcare providers, but also for the licensors of the underlying intellectual property underpinning such treatments as industry adopts innovative new payment and reimbursement models which may impact on royalty streams.

When negotiating a licence to technology underpinning a cell or gene therapy the parties should consider how less conventional pricing mechanisms may impact the royalty structure. However, while there are some issues licensees and licensors may be able to consider upfront, it is difficult to anticipate the issues that may become relevant at a stage where pricing models have not been set, particularly as there is no one-size-fits-all pricing approach.

We have proposed an increased use of robust governance processes in a licensing relationship as one option to consider. It will also be interesting to see whether any trends emerge in relation to upfront and milestone payments in response to the challenges outlined above. In particular, licensees may push for more back-loaded or performance-related milestone payments to reflect the risks associated with pricing models which take a longer term view of the cost benefits of these types of therapies. We look forward to seeing what innovative approaches licensors and licensees adopt to adapt to these challenges in the years to come.

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Cell and gene therapies - Lexology

RESEARCH TRIANGLE PARK Theres a new biotech company setting up shop in the Triangle, and its flush with cash and headed up by some big names in the industry.

MeetKriya Therapeutics the brainchild of Dr. Shankar Ramaswamy, former chief business officer for Axovant Gene Therapies; Fraser Wright, co-founder of Sparks Therapeutics; and Roger Jeffs, the former United Therapeutics CEO who has deep rootsinNorth Carolina.

Launched in 2019, the biotech startup has dual headquarters in Durham and Palo Alto, California, and is billing itself as a next-generation gene therapy company focused on designing and developing treatments for highly prevalent and severe chronic conditions, like diabetes and obesity.

Earlier this month, it arrived in a big way after securing $80.5 million in Series A financing during a pandemic.

Its never easy. But itsa really significant pool of capital for us so were thankful to have been able to get it done,Ramaswamy, Kriyas CEO, told NC Biotech in a video interview this week.[Our] investors have a very long term vision of what a next generation gene therapy company could look like, and were very supportive building towards that vision.

Fraser Wright, PhDScientific Co-Founder and Chief Scientific Advisor; Shankar Ramaswamy, MDCo-Founder, Chairman, and CEO; and Nachi Gupta, MD, PhDChief of Staff.

Among the investors: QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures (associated with Sutter Hill Ventures), Narya Capital, Amplo,Paul Manning, andAsia Alpha. The round followed an initial seed financing led by Transhuman Capital late last year.

Itsis amilestone for the company andsets us up for success to goout and execute on the things that we really want to get done.

Ramaswamy says the company is now ready to scale, and is focused on building out its teams on both coasts.

We expect to grow very quickly both here in the Bay Area and in North Carolina, he said, emphasizing the Triangles importance as its manufacturing hub. That could be dozens of employees [here] in the not so distant future, if not larger over time.

How it will work: co-founders Ramaswamy and Wright will be based in the Bay area along with finance operations and early-stage research.

Meanwhile, in Durham, co-founder Jeffs will lead a team focused on development and manufacturing. It will include Britt Petty, AveXis former head of global manufacturing and Melissa Rhodes, former chief development officer at Altavant Sciences; and Mitch Lower, another Avexis veteran.

I dont view North Carolina as a satellite office.Thats where well be building our internal manufacturing infrastructure to solve for one of the key bottlenecks in gene therapy,which is manufacturing capacity and quality, saidRamaswamy.

Theres a very strong pool of talent in North Carolina, especially in biologics manufacturing. And [our team] has a very strong track record and history of success with biologics manufacturing, and strong experience there as well. So we think its a great place to be, given the past couple of decades, where there have been so many successful products actually manufactured in North Carolina.

Already, Kriyahas a number of gene therapies in the pipeline.

Among them: KT-A112, an investigational gene therapy administered by intramuscular injection that delivers the genes to produce insulin and glucokinase for type 1 and type 2 diabetes;KT-A522, an investigational gene therapy administered by salivary gland injection that delivers the gene to produce a glucagon-like peptide 1 (GLP-1) receptor agonist for type 2 diabetes and severe obesity; andKT-A83, an investigational gene therapy administered by intrapancreatic injection that delivers the gene to produce modified insulin growth factor 1 (IGF-1) for type 1 diabetes.

The team is currently set up in a temporary office in Durham, but plans to move intoamore permanent space somewhere in the Research Triangle in the near future.

Kriya is building a leading team and cutting-edge infrastructure to engineer best-in-class gene therapies for severe chronic conditions and accelerate their advancement into human clinical trials, saidJeffs, its vice chairman. Through its R&D laboratory capabilities in the Bay Area and in-house process development and manufacturing infrastructure inResearch Triangle Park, I believe that Kriya will be uniquely positioned to become a leader in the gene therapy field.

(c) North Carolina Biotechnology Center

Durhams Kriya Therapuetics lands $80M to advance gene therapies for diabetes, severe obesity

Originally posted here:
Bicoastal startup Kriya Therapeutics to grow gene therapy manufacturing in NC - WRAL Tech Wire

Gene therapy does more than treat genetic diseases it can cure them. A one-time dose of a non-replicative viral vector, such as commonly used recombinant adeno-associated virus (AAV), delivers a functional gene to replace or compensate for a dysfunctional version that is causing a patients disease (Figure 1). As a cutting-edge biopharmaceutical technology, there are multiple gene therapies now FDA approved; with hundreds more in clinical trials, were likely to see many more of these therapies on the market soon.1 However, to keep up with the rapid pace of clinical research, developers are working to streamline the manufacturing and quality control process to improve quality and lower the cost of bringing these important drugs to market.Developers use a multitude of analytical tests to develop gene therapies and optimize their manufacturing process. When developers get aberrant test results, they must be able to interpret where the problem lies. Did the manufacturing process produce an undesirable product, or is the analytical testing method unreliable? Analytical testing companies that have the infrastructure, personnel, and experience often partner with developers to tighten up analytical variability so that results of tests clearly indicate where there are opportunities to increase efficiency and product quality.

Figure 1. Gene delivery by recombinant viral vector.During gene therapy, viral capsids containing the therapeutic gene are taken up by the patients cells and the genetic material is delivered to the nucleus. There, the gene gets expressed as a protein necessary for the patients health. Credit: Avomeen.

Figure 2. A full AAV capsid and associated capsid impurities. Complete viral capsids have AAV are assembled from 60 capsid proteins, with a defined stoichiometry and shape and contain a therapeutic gene. AAV vector impurities include capsids that contain too many copies of the gene (overfilled), those that contain lower copy numbers or truncations of the gene (partially full), or empty capsids that contain no genetic material. Credit:Avomeen.

There are several ways to measure the empty/full capsid ratio, and as developers are establishing their chemistry, manufacturing and control (CMC) protocol, it is important that they choose an optimized method, as they must use that method for effective quality control from early process development to lot release and stability.3 Gene therapy developers may choose analytical ultracentrifugation to evaluate capsids, but while highly effective, this method is not as quantitative, robust or efficient as some newer methods. High-performance liquid chromatography (HPLC) using AAV full/empty analytical columns have been demonstrated to be highly effective at separating full, empty, and improperly filled capsids for robust quantification. Additionally, this method is higher throughput than ultracentrifugation, and requires less precious AAV sample to run.

Cellular potency is evaluated by transducing cells with the AAV product and then measuring a phenotypic or functional outcome due to the transduction. Developing these tests can be challenging because there is no one-size-fits-all test that will give developers the answers they need. Developers often draw on the experience of analytical labs to determine how to best evaluate their AAV products transduction efficiency.A gene therapy in development must also be tested to ensure that it is free of residual, process-related impurities such as polyethylenimine, iodixanol, poloxamer, and other excipients that must be removed in the final product to ensure safety. Few research and manufacturing facilities have the equipment and expertise necessary to perform this kind of testing, and it is advisable to find one that has experience testing polymers, extractables and leachables to examine if components of the manufacturing equipment or drugs packaging are not contaminating the final product.

As fast-paced as the gene therapy field is now, it stands to become a true race to the finish line to bring new gene therapies to market in the near future. Regulatory bodies are becoming more familiar with reviewing gene therapies, and the road to commercialization will move more quickly. There is no denying that gene therapies will bring incredible benefits to patients, but it will be crucial to improve manufacturing efficiency and lower costs to make gene therapies more accessible to the patients who need them.References

1. Colasante, W., Diesel, P., and Gerlovin, Lev. (2018). New Approaches To Market Access And Reimbursement For Gene And Cell Therapies. Cell & Gene. Retrieved from: https://www.cellandgene.com/doc/new-approaches-to-market-access-and-reimbursement-for-gene-and-cell-therapies-0001

2. Fraser Wright, J. (2014). Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment. Biomedicines, 2, 80-97; doi:10.3390/biomedicines2010080

3. U.S. Food & Drug Administration (2019). Guidance for Human Somatic Cell Therapy and Gene Therapy. Retrieved from: https://www.fda.gov/animal-veterinary/guidance-industry/chemistry-manufacturing-and-controls-cmc-guidances-industry-gfis

4. Stein, R. (2019). At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever. NPR. Retrieved from: https://www.npr.org/sections/health-shots/2019/05/24/725404168/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever

5. Cohen, J.T, Chambers, J. D., Silver, M. C., Lin, P., Neumann, P.J. (2019). Putting The Costs And Benefits Of New Gene Therapies Into Perspective. Health Affairs. Retrieved from: https://www.healthaffairs.org/do/10.1377/hblog20190827.553404/full/

6. ATCC (accessed May, 2020) ATCC Virus Reference Materials. Retrieved from: https://www.atcc.org/en/Standards/Standards_Programs/ATCC_Virus_Reference_Materials.aspx#

7. U.S. FDA (2020). FDA Details Policies on Gene Therapies in Seven Guidances. Retrieved from: https://www.fdanews.com/articles/195767-fda-details-policies-on-gene-therapies-in-seven-guidances

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Troubleshooting the Development of New Gene Therapies - Technology Networks

ATLANTA, May 26, 2020 /PRNewswire/ -- Expression Therapeutics has announced that it has received clearance by the United States Food and Drug Administration (FDA) to proceed following review of its Investigational New Drug Application (IND) for clinical testing of its novel lentiviral vector-based gene therapy ET3 for hemophilia A. Hemophilia A is the most common severe congenital bleeding disorder and afflicts approximately 1 in 8,000 people. Without treatment, severe hemophilia is crippling and fatal by late adolescence to early adulthood.

The ET3 gene therapy developed by Expression Therapeutics combines innovative platform technologies in protein bioengineering and tissue-directed expression. ET3 consists of autologous mobilized peripheral blood stem and progenitor cells transduced with a recombinant lentiviral vector, encoding a bioengineered coagulation factor VIII transgene designed for highlevel expression at low vector copy number. In the ET3 trial, subjects will be preconditioned with low-dose stem and immune cell suppressing agents prior to receiving a single infusion of ET3. The high-expression factor VIII can correct the bleeding tendency in hemophilia A. The duration of ET3 activity is expected to be the normal lifetime of the patient. Expression Therapeutics expects to initiate a Phase 1 clinical trial titled ET3-201 at Emory University and enroll patients shortly.

"We are extremely pleased that the FDA has granted permission to proceed with this clinical study," said Trent Spencer, Ph.D., President of Expression Therapeutics and Director of the Cell and Gene Therapy Program in the Aflac Cancer and Blood Disorders Center at Emory University.

Hematopoietic stem and progenitor cell lentiviral gene therapy is currently the only approach that offers the possibility of permanent cure of hemophilia A and provides an opportunity to reach both pediatric and adult populations.

"We are very excited to get the hemophilia A clinical trial underway, the first of six gene therapy products currently under development at Expression Therapeutics," said Mohan Rao, Ph.D., CEO of Expression Therapeutics.

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

For inquiries, please contact:

Ashley WalshDirector of Corporate DevelopmentExpression Therapeutics 1860 Montreal RoadTucker, Georgia 30084[emailprotected]+1 312.637.2975

SOURCE Expression Therapeutics

http://www.expressiontherapeutics.com

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Expression Therapeutics Announces IND Approval by the FDA for Hemophilia A Gene Therapy - PRNewswire

This agreement covers the manufacturing of the Adeno-Associated Virus (AAV)

LYON, France and PARIS, May 26, 2020 /PRNewswire/ -- Novasep, a leading supplier of services and technologies for the life sciences industry, and Lysogene (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced the signature of an agreement for the development and manufacturing of LYS-GM101, an AAVrh10-based gene therapy drug candidate for the treatment of GM1 Gangliosidosis, a rare neuronopathic lysosomal storage disorder.

With this collaboration, the two companies consolidate their long-lasting partnership initiated with the development and manufacturing of Lysogene's lead gene therapy product, LYS-SAF302, currently in clinical phase 2/3.

Mark Plavsic, Lysogene's Chief Technical Officer, said: "Following the successful relationship developed during the past 4 years, I am very pleased to continue working with Novasep, which is emerging as a true leader in gene therapy development and manufacturing. By extending our collaboration, we secure the clinical production of our experimental treatment for GM1 gangliosidosis and take an option for a smooth and effective technical transfer to a future commercial process."

Cedric Volanti, Novasep's President of Biopharma Solutions, said: "We, at Novasep, are delighted to pursue and extend our partnership with Lysogene. Novasep will bring its expertise and mobilize its production capacities to first help Lysogene in the clinical development of its innovative gene therapy treatment for GM1 gangliosidosis; and secondly, to shorten the transition to a commercial product manufacturing by ensuring a smooth process transfer to our commercial manufacturing facility."

About Lysogene

Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/3 clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. http://www.lysogene.com.

About Novasep

Novasep provides cost-effective and sustainable manufacturing solutions for the life sciences industries.

With 20 years' experience in the development and manufacturing of biomolecules, Novasep offers a full range of CDMO services for:

- Viral vectors (AAV, Adenovirus, Lentivirus, HSV, VSV, VEEV) for cell and gene therapy, immunotherapy and vaccination, from process development to cGMP production

- Fill & Finish services for viral vectors, attenuated and live viruses, mAbs, plasmids and other biologics, from formulation to packaging

As part of its growth strategy Rise-2, Novasep recently unveiled a new facility, Senrise-IV, dedicated to the commercial production of viral vectors which has been completed last year by Senefill, a new Fill & Finish commercial facility for aseptic operations. Both facilities located in Seneffe, Belgium, will contribute to the success of biopharmaceuticals' projects.

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SOURCE Novasep

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NOVASEP and LYSOGENE Announce Their New Collaboration for Development and Production of GM1 Gangliosidosis Gene Therapy Product - BioSpace