StemCell Therapy

Global Cancer Gene Therapy Market: Overview

Cancer could be defined as uncontrolled cell growth in the body leading to organ malfunction. If untreated, it can lead to death. Uncontrolled growth of cell is managed by the body in several ways, one of them is by deploying white blood cells to detect and eradicate these cancerous cells. It has been discovered that the immune system could be manipulated to influence cancerous cells to destroy itself.

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Radiation and chemotherapy therapy have consistent and reliable effects to decrease cancerous cells in the body. Recently, immunotherapy for hematological cancers has experienced a recognition and is of interest for many researchers Scientists have developed methods to isolate, replicate, and develop cancer-destroying cells from the patients blood cancer and injecting those cells back for the destruction of their cancers, with durable remissions.

New options for the treatment is needed to be developed if order to achieve elimination of cancer suffering and death by 2020. According to NCI, 5-year survival rate for cancers such as lung (15%), glioblastoma (5%), pancreatic (4%), and liver (7%) remains very low. Current available treatments have several side effects, the systemic toxicity due to chemotherapy results in nausea, mild cognitive impairments, and mouth ulcerations, in addition to long-term side effects such as increasing risk of developing other types of cancers. Therefore, new and innovative treatment methods are required to reduce the suffering of cancer patients.

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GlobalCancer Gene Therapy Market: Drivers and Restraints

The emerging field of cancer Gene Therapy offers varied potential treatments. Gene therapy involves a range of treatment types, which use genetic material to alter cells (either in vivo or in vitro) to help cure the disease. Cancer Gene Therapy shown efficacy in various in vitro and preclinical testing. Preclinical testing for cancer gene therapy has been performed on glioma, pancreatic cancer, liver cancer, and many other cancers.

Increase in prevalence of cancer, rise in government funding and initiatives, growth in pipeline of cancer gene therapy products, and collaborations to develop and launch gene-therapy products are some factors driving the market. According to NCBI researchers, development of genetically-modified T-cell therapies for treatment of cancer has had maximum clinical impact among other gene therapies. However, high treatment cost is a major limitation in the cancer gene therapy market. The reason behind the huge cost for cancer gene therapy is the necessity of rigorous, exhaustive clinical trials; also treatment by cancer gene therapy differs from person to person depending upon the genetic acceptance of every patient, unlike other drugs thereby limiting the market growth.

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GlobalCancer Gene Therapy Market: Key Segments

Based on type, the cancer gene therapy market is segmented into gene transfer immunotherapy and oncolytic virotherapy. Immunotherapy uses genetically modified cells and viral particles to stimulate the immune system to destroy cancer cells. Immunotherapy include treatment with either cytokine gene delivery or tumor antigen gene delivery. Oncolytic virotherapy uses viral particles, which replicate within the cancer cell causing the death of the cell. It is an emerging treatment modality that is expected to shows great promise, particularly in metastatic cancer treatment.

It includes treatment with adenovirus, retrovirus, lentivirus, herpes simplex virus, adeno-associated virus, simian virus, alphavirus, and vaccinia virus. Gene transfer is the newest treatment modality that is expected to introduce new modified genes into cancerous cell or associated tissue for destruction of cell or to slow down cancer growth. This technique is flexible as a wide variety of vectors and genes are used for clinical trials with positive outcomes. As gene therapy advance, they could be used alone or in combination with other treatments to control the disease. Gene transfer or gene replacement is performed using naked/plasmid vectors, electroporation, sonoporation, magnetofection, and gene gun.

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Based on region, the global cancer gene therapy market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa. North America is anticipated to hold the largest market share. The U.S. dominates the cancer gene therapy market owing to its increase in funding for research & development and other government initiatives. Key players in the biotech industry are engaging in research & development of gene therapy products. Moreover, rising demand for DNA vaccines and growing interest of venture capitalists to investment in commercialization of gene-based cancer therapies are likely to propel the market. The cancer gene therapy market in Asia Pacific is anticipated to expand at a rapid pace as in China cancer gene therapy is anticipated to attribute for largest revenue, due to the recent launch of Gendicine and rising healthcare expenditure with strong R&D facilities.

GlobalCancer Gene Therapy Market: Key Players

Key players operating in the global cancer gene therapy market are Adaptimmune, ZioPharm Oncology Altor Bioscience, MolMed, bluebird bio, Shanghai Sunway Biotech company limited , MultiVir, Shenzhen SiBiono GeneTech, Corporation.

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Cancer Gene Therapy Market Outlook for Major Applications/end Users, Consumption, Share and Growth Rate 2025 - Cole of Duty

A group of patient advocacy groups has sent a letter to the Centers for Medicare and Medicaid Services urging the agency to establish the new Medicare Severity-Diagnosis Related Group for Chimeric Antigen Receptor T-cell Immunotherapy.

In the letter, the stakeholders argued that CAR-T therapies offer improved outcomes for patients with relapsed/refractory diffuse large B-cell lymphoma and B-cell acute lymphoblastic leukemia and provides hope for many more with other hard-to-treat cancers

The new MS-DRG would also give hospitals and providers a new course of treatment for those cancers.

The letter backed up its praise with a study in which lymphoma patients had significant improvements in their physical, social and emotional wellbeing following CAR-T therapy.

The organizations signing the letter include the American Cancer Society Cancer Action Network, BMT Infonet, Cancer Support Community, CLL Society, CrowdCare Foundation/Myeloma Crowd, International Myeloma Foundation, Leukemia & Lymphoma Society, Lymphoma Research Foundation, Society for Immunotherapy of Cancer and The Pink Fund.

WHY THIS MATTERS

This letter is a comment to CMS' proposed inpatient prospective payment system rule issued in May.

Under the current rule, CAR-T hospital cases are paid at the same rate as bone marrow transplants and qualify for additional payments through the temporary new technology add-on payment for high-cost cases that are set to expire this year.

The rule would create a separate hospital payment category for CAR-T therapy.

The new MS-DRG for CAR-T would provide predictable payment rates for hospitals administering the therapy.

CAR-T is a gene therapy that uses a patient's own genetically modified immune cells as a treatment for certain types of cancer. This is instead of additional chemotherapy or other types of treatment paid for under the inpatient prospective payment system.

The open comment period for the proposed rule ends today, Friday, July 10.

THE LARGER TREND

For years, organizations such as the American Society for Blood and Marrow Transplantation have been asking for an MS-DRG to be created for CAR-T therapies.

ON THE RECORD

"Our organizations commend CMS for its action and appreciate its forward-leaning policy proposal that will optimize patient access for CAR-T therapy," the stakeholders wrote. "We support finalization of the proposed rule and look forward to working with CMS to further support novel treatments for unmet medical needs among immunotherapy patients."

Twitter:@HackettMalloryEmail the writer:mhackett@himss.org

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Stakeholder urge CMS to finalize new CAR-T therapy payment rule - Healthcare Finance News

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimers drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agencys decision-making time to a mere 6 months.

The news drove a 10% spike in Biogens stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Flagship execs take a lesson from nature to master 'gene writing,' launching a star-studded biotech with big ambitions to cure disease - Endpoints...

NEW YORK and CLEVELAND, July 10, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that two poster presentations related to its clinical program for recessive dystrophic epidermolysis bullosa (RDEB) were featured at the Society for Pediatric Dermatology (SPD) 45th Annual Meeting. The first poster includes a detailed analysis of patients with RDEB in the EB-101 Phase 1/2a trial showing that wound healing following EB-101 treatment was associated with improved long-term pain relief. A separate poster provides insights on the significant disease burden associated with RDEB, highlighting data from a literature review on the clinical characteristics, humanistic consequences and economic impact of living with RDEB on patients and their families.

The large wounds of RDEB cause substantial pain, and only palliative treatments are currently available, said Joo Siffert, M.D., Chief Executive Officer of Abeona. The data presented at SPD showed that EB-101 treatment of large, chronic wounds resulted in considerable and durable reduction in wound burden, which was associated with long-term pain relief for up to five years. The second poster at SPD helps to characterize the disease burden and management of RDEB, providing an important reminder of the extraordinary toll RDEB takes on quality of life, and underscores the need for therapies that reduce wound burden and the associated humanistic and economic impact.

EB-101 Treatment of Large, Chronic Wounds Is Associated with Durable Healing and Pain Reduction in Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB)

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, presented long-term outcomes following EB-101 treatment for large, chronic wounds in patients with RDEB. EB-101 treatment resulted in considerable and durable reduction in wound burden in the range of three to five years in a Phase 1/2a study. Wound healing of 50% or greater following EB-101 treatment was associated with no pain at treated sites at three years, four years and five years post-treatment, compared with presence of pain in 53% of wound sites at baseline. The ongoing VIITALTM study will further characterize the relationship between reduction of wound burden and pain relief following EB-101 treatment.

The Full Burden of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

M. Peter Marinkovich, M.D., Bullous Disease Clinic Director, Stanford University Medical Center, and Investigator in the VIITALTM study, presented findings from a literature review of 65 studies that provide new insights on the disease burden from the perspective of patients with RDEB and their families. Key observations of the clinical, humanistic and economic burden of RDEB include:

Abeonas posters from the SPD 45th Annual Meeting are available on the News/Events page under the Investors & Media section of Abeonas website at http://www.abeonatherapeutics.com.

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 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 their 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 (Identifier: NCT04227106).

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 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-based 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

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Abeona Therapeutics Announces Two Presentations Related to Its RDEB Clinical Program at the Society for Pediatric Dermatology 45th Annual Meeting -...

Global Cancer Gene Therapy Market Report 2019-Market Size, Share, Price, Trend and Forecast report is based on comprehensive analysis conducted by experienced and professional experts. The report mentions, factors that are influencing growth such as drivers, restrains of the market. The report offers in-depth analysis of trends and opportunities in the Cancer Gene Therapy Market. The report offers figurative estimations and predicts future for upcoming years on the basis of the recent developments and historic data. For the gathering information and estimating revenue for all segments, researchers have used top-down and bottom-up approach. On the basis of data collected from primary and secondary research and trusted data sources the report offers future predictions of revenue and market share.

The Leading Market Players Covered in this Report are : Adaptimmune,GlaxoSmithKline,Bluebird bio,Merck,Celgene,Shanghai Sunway Biotech .

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Impact of Covid-19 in Cancer Gene Therapy Market:The utility-owned segment is mainly being driven by increasing financial incentives and regulatory supports from the governments globally. The current utility-owned Cancer Gene Therapy are affected primarily by the COVID-19 pandemic. Most of the projects in China, the US, Germany, and South Korea are delayed, and the companies are facing short-term operational issues due to supply chain constraints and lack of site access due to the COVID-19 outbreak. Asia-Pacific is anticipated to get highly affected by the spread of the COVID-19 due to the effect of the pandemic in China, Japan, and India. China is the epic center of this lethal disease. China is a major country in terms of the chemical industry.

Key Businesses Segmentation of Cancer Gene Therapy MarketOn the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, sales volume, Cancer Gene Therapy market share and growth rate of Cancer Gene Therapy foreach application, including-

On the basis of product,this report displays the sales volume, revenue (Million USD), product price, Cancer Gene Therapy market share and growth rate ofeach type, primarily split into-

Cancer Gene Therapy Market Regional Analysis Includes: Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia) Europe(Turkey, Germany, Russia UK, Italy, France, etc.) North America(the United States, Mexico, and Canada.) South America(Brazil etc.) The Middle East and Africa(GCC Countries and Egypt.)

Key Highlights of the Table of Contents:Cancer Gene Therapy Market Study Coverage:It includes key market segments, key manufacturers covered, the scope of products offered in the years considered, global Cancer Gene Therapy market and study objectives. Additionally, it touches the segmentation study provided in the report on the basis of the type of product and applications.Cancer Gene Therapy Market Executive summary:This section emphasizes the key studies, market growth rate, competitive landscape, market drivers, trends, and issues in addition to the macroscopic indicators.Cancer Gene Therapy Market Production by Region:The report delivers data related to import and export, revenue, production, and key players of all regional markets studied are covered in this section.Cancer Gene Therapy Market Profile of Manufacturers:Analysis of each market player profiled is detailed in this section. This segment also provides SWOT analysis, products, production, value, capacity, and other vital factors of the individual player.

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Key Questions Answered: To gain insightful analyses of the Cancer Gene Therapy market and have a comprehensive understanding of the global market and its commercial landscape. Assess the production processes, major issues, and solutions to mitigate the development risk. To understand the most affecting driving and restraining forces in the market and its impact on the global Cancer Gene Therapy market. Learn about the Cancer Gene Therapy market strategies that are being adopted by leading respective organizations. To understand the future outlook and prospects for the market. Besides the standard structure reports, we also provide custom research according to specific requirements.

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Cancer Gene Therapy Market Present Scenario, the Growth Prospects with Forecast 2024 - 3rd Watch News

The global Hemophilia Gene Therapy Market analysis provides pioneering landscape of market along with market augmentation history and key development involved in the industry. The report also features comprehensive research study for high growth potential industries professional survey with market analysis. Hemophilia Gene Therapy Market report helps the companies to understand the market trends and future market prospective,opportunities and articulate the critical business strategies.

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Major Players Are:BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC, Sangamo Therapeutics, Inc., and Freeline Therapeutics

Regions & Countries Mentioned In The Hemophilia Gene Therapy Market Report:

Key Highlights of the Table of Contents:

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Various analytical tools such as SWOT, Feasibility analysis, Porters Five Forces analysis, Value Chain analysis, and Capacity utilization analysis are implemented while evaluating the Hemophilia Gene Therapy Market which certainly helps a reader to get a deeper perception of the market and its participants. Additionally, it covers a cardinal evaluation of market history, patterns, changing dynamics, market and manufacturing trends, demand and supply activities, and technological development.

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Hemophilia Gene Therapy Market to Etch New Growth Ratios with Adoption in Developing Economies - 3rd Watch News

BOSTON and LONDON and MILAN, Italy, July 09, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and MolMed S.p.A (MLMD.MI), one of the companys principal contract development and manufacturing partners, today announced that they have extended their collaboration initiated in April 2018 for a period of five years through June 2025.

With the extension of the collaboration, MolMed will continue to support activities related to the development and manufacturing of vectors and drug products for several of Orchards investigational ex vivo hematopoietic stem cell (HSC) gene therapies in the upcoming years, including OTL-200 for metachromatic leukodystrophy (MLD) and OTL-103 for Wiskott Aldrich syndrome (WAS), as well as for additional pipeline programs including OTL-203 for mucopolysaccharidosis type I (MPS-I). MolMed is the first company to have obtained good manufacturing practice (GMP) authorization for the gene and cell therapy markets in Europe and is the manufacturer for Strimvelis, Orchards ex vivo HSC gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) and the first such treatment approved by the European Medicines Agency (EMA).

We are looking forward to continuing to build and expand upon our partnership with MolMed, who have supported the progression of many of our programs since their earliest clinical development stages, said Frank Thomas, president and chief operating officer of Orchard. Their expertise in gene therapy manufacturing, coupled with their deep knowledge of our programs, will be invaluable as our therapies for MLD and WAS approach anticipated approval and commercialization in Europe and across the globe.

Luca Alberici, MolMed's chief business officer, added, "We are pleased to have strengthened our collaboration with Orchard to support them in their mission of bringing potentially transformative therapies to those suffering from severe rare diseases. After being Orchards exclusive manufacturer for Strimvelis, we are looking forward to supporting their manufacturing needs for additional programs both in clinical trials and in potential commercial applications following the anticipated approval of OTL-200 for MLD in Europe later this year.

OTL-200 for MLD is currently under review by the EMA with a decision expected later this year.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

About MolMedMolMed S.p.A. is a biotechnology company focused on research, development, manufacturing and clinical validation of novel cell and gene therapies. MolMed, established in 1996, has been listed since March 2008 on the Italian Stock Exchange managed by Borsa Italiana, and has its registered office in Milan, at the Biotechnology Department of Ospedale San Raffaele and an operating site at Bresso, at the OpenZone campus.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for or marketing approval of its product candidates, and Orchards expectations concerning its partnership with MolMed. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development and commercial programs; the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the risk of delays in Orchards ability to commercialize its product candidates, if approved; and the risk that Orchard may not receive the expected benefits from its collaboration with MolMed or that Orchard or MolMed will not fully perform under the terms of their collaboration agreement. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter ended March 31, 2020, as filed with the U.S. Securities and Exchange Commission (SEC) on May 7, 2020, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Orchard Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

MolMed Contacts

Investor Relations & Communications DepartmentMolMed S.p.A.+39 02 21277.205investor.relations@molmed.com

Tommasina CazzatoPress OfficeCommunity Group+39 345 7357751tommasina.cazzato@communitygroup.it

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Orchard Therapeutics and MolMed Announce Extension of Gene Therapy Manufacturing Collaboration - BioSpace

Mutations in more than 270 genes have been implicated in inherited eye diseases like retinitis pigmentosa. Now, Biogen has formed a research pact with Harvards Massachusetts Eye and Ear thats aimed at developing a gene therapy to help some patients with these blinding diseases.

The gene at the center of the new agreement, PRPF31, has been linked to autosomal dominant retinitis pigmentosa. PRPF31 mutations are believed to cause an estimated 25% of all retinitis pigmentosa cases. The partners did not disclose the financial terms of the deal.

The tie-up comes eight months after a Mass Eye and Ear team published preclinical research demonstrating a gene therapy technique for repairing cells withmutated PRPF31 genes. The technique partially restored the structure and function of retinal pigment epithelium cells, the team reported in the journal Molecular Therapy Methods & Clinical Development. The research was led by Eric Pierce, M.D., Ph.D., professor at Harvard Medical School and director of the inherited retinal disorders service at Mass Eye and Ear.

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Pierces team developed the technique, called adeno-associated virus (AAV)-mediated gene augmentation therapy, with the goal of preserving and possibly bringing back some vision in patients with PRPF31 mutations, he said in a statement. Biogen now has an exclusive license to develop the therapy worldwide and will fund the studies necessary to seek FDA approval.

Biogen has been working to build its expertise in gene therapy. In March 2019, it acquired Nightstar Therapeutics, which is in early development of a treatment for X-linked retinitis pigmentosa, for $877 million. Biogen fought off three other bidders to consummate that deala clear sign of the growing interest in gene therapy.

RELATED: Biogen-Nightstar deal sheds light on gene therapy feeding frenzy

Not all of Biogens forays into gene therapy for ocular diseases have succeeded, though. In 2018, the company pulled out of a research collaboration with Applied Genetic Technologies to develop several gene therapies, including one to treat the inherited retinal disorder X-linked retinoschisis. That therapy was shelved after it was ineffective in a phase 1/2 trial.

Several other gene therapies are being developed to treat retinitis pigmentosa. They include Allergans RST-001, which the company picked up when it acquired RetroSense Therapeutics for $60 million in 2016. RST-001 targets channelrhodopsin, a photosensitivity gene, and is designed to restore light sensitivity to retinal cells. It is currently enrolling patients for a phase 2a trial.

Mass Eye and Ear was the first center to administer Luxturna, Spark Therapeutics gene therapy for retinal degeneration caused by mutations in the gene RPE65, after the product was approved in 2017. One of the exciting aspects of our collaboration with Biogen is that mutations in the PRPF31 gene affect approximately 10 to 20 times more people than mutations in the RPE65 gene, Pierce said in the statement. Success with PRPF31 gene therapy could provide visual benefit to more patients, which is our ultimate goal.

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Biogen boosts gene therapy strategy with Harvard pact focused on inherited eye disease - FierceBiotech

In experiments in rats and mice, two Johns Hopkins scientists an engineer and an ophthalmologist report the successful use of nanoparticles to deliver gene therapy for blinding eye disease. A uniquely engineered large molecule allows researchers to compact large bundles of therapeutic DNA to be delivered into the cells of the eye.The research, described in Science Advances, provides evidence of the potential value of nanoparticle-delivered gene therapy to treat wet age-related macular degeneration an eye disease characterized by abnormal blood vessel growth that damages the light-sensitive tissue in the back of the eye as well as more rare, inherited blinding diseases of the retina.

Many gene therapy approaches depend on viral vectors, which use a viruss natural ability to carry genetic material into cells. However, viruses create an immune response, which prevents repeat dosing, and the most commonly used one for ocular gene therapy cannot carry large genes.

Some of the most prevalent inherited retinal degenerations are due to mutations in large genes that simply cannot fit into the most commonly used viral vector, says Peter Campochiaro, M.D., the Eccles Professor of Ophthalmology at the Johns Hopkins University School of Medicine, and a member of the Johns Hopkins Medicine Wilmer Eye Institute.

To overcome such limitations, Campochiaro and Jordan Green, Ph.D., developed a new approach involving a biodegradable polymer that surrounds and compacts long stretches of DNA, creating nanoparticles that can enter the cells. This technology allows the researchers to convert the cells of the eye into minifactories for a therapeutic protein.

To first test whether the nanoparticles could reach their target cells, the researchers loaded the nanoparticles with a gene for a florescent protein that causes cells to light up like a glow stick.

This glowing molecule allowed the researchers to determine the location, amount and duration of gene expression achievable with the nanoparticles.

They found that even eight months after treatment, the majority of the light-sensitive cells in the rats eyes glowed, showing that the nanoparticles effectively deposited the florescent gene into the cells.

Next, the researchers set up a similar experiment, this time using the nanoparticles to shuttle a biologically relevant gene into the eye. They loaded the nanoparticles with a gene for vascular endothelial growth factor (VEGF), which is responsible for the growth of abnormal blood vessels in people with wet macular degeneration.

The researchers injected the eyes of 30 rats with the nanoparticles carrying the VEGF gene and determined the effects in the retina one, two and five months after injection. One month after injection, each rat tested had developed abnormal blood vessels under and within the retina, like those seen in patients with wet macular degeneration. The abnormal blood vessels were more extensive at two and five months after injection, and there was associated scarring under the retina similar to that seen in chronic untreated wet macular degeneration.

These results show that the genes delivered by nanoparticles stayed active within the cells for several months, says Campochiaro.

Finally, to test a nanoparticles ability to deliver a therapeutic gene for the disease, the researchers used mice genetically engineered to develop a form of wet macular degeneration similar to that in humans. The researchers loaded nanoparticles with a gene that produces a protein that neutralizes VEGF.

Currently, physicians inject such proteins that block VEGF proteins into the eyes of people with macular degeneration, a treatment that helps control the overgrowth of abnormal, leaky blood vessels. But this procedure must be repeated frequently and is burdensome for patients and their caretakers.

Three weeks after injecting nanoparticles containing the gene for the anti-VEGF protein, the mice had a 60% reduction in abnormal blood vessels when compared to control mice. The same effect was seen 35 days later.

These results are extremely promising, says Jordan Green, Ph.D., professor of biomedical engineering at the Johns Hopkins University School of Medicine. We have the ability to reach the cells most significantly affected by degenerative eye disease with nonviral treatments that can allow the eye to create its own sustained therapies.

An estimated 1.6 million people in the U.S. with macular degeneration receive injected drugs to the eye every four to six weeks. A gene therapy treatment could provide a way for the eyes tissue to prevent further vision deterioration with as little as a few initial treatments. Genetic diseases that cause blindness could be treated in a similar way, by introducing functional versions of genes that inherited mutations have disabled.

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

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Success In Use of Nanoparticles To Deliver Gene Therapy for Eye Disease In Rodents - Technology Networks

NEW YORK, July 9, 2020 /PRNewswire/ --

Global Gene Therapy Market to Reach US$4.2 Billion by the Year 2027 Amid the COVID-19 crisis, the global market for Gene Therapy estimated at US$701.2 Million in the year 2020, is projected to reach a revised size of US$4.2 Billion by 2027, growing at a CAGR of 29.3% over the analysis period 2020-2027.Lentivirus, one of the segments analyzed in the report, is projected to grow at a 21.7% CAGR to reach US$130.1 Million by the end of the analysis period.After an early analysis of the business implications of the pandemic and its induced economic crisis, growth in the AAV segment is readjusted to a revised 24.9% CAGR for the next 7-year period. This segment currently accounts for a 13.5% share of the global Gene Therapy market.

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=PRN

The U.S. Accounts for Over 26.8% of Global Market Size in 2020, While China is Forecast to Grow at a 36.3% CAGR for the Period of 2020-2027 The Gene Therapy market in the U.S. is estimated at US$188.2 Million in the year 2020. The country currently accounts for a 26.84% share in the global market. China, the world second largest economy, is forecast to reach an estimated market size of US$1.1 Billion in the year 2027 trailing a CAGR of 36.3% through 2027. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 23.2% and 26.7% respectively over the 2020-2027 period. Within Europe, Germany is forecast to grow at approximately 25.1% CAGR while Rest of European market (as defined in the study) will reach US$1.1 Billion by the year 2027.

RetroVirus & Gamma RetroVirus Segment Corners a 55.5% Share in 2020 In the global RetroVirus & Gamma RetroVirus segment, USA, Canada, Japan, China and Europe will drive the 28.6% CAGR estimated for this segment. These regional markets accounting for a combined market size of US$293.4 Million in the year 2020 will reach a projected size of US$1.7 Billion by the close of the analysis period. China will remain among the fastest growing in this cluster of regional markets. Led by countries such as Australia, India, and South Korea, the market in Asia-Pacific is forecast to reach US$662.2 Million by the year 2027, while Latin America will expand at a 30.1% CAGR through the analysis period. We bring years of research experience to this 16th edition of our report. The 248-page report presents concise insights into how the pandemic has impacted production and the buy side for 2020 and 2021. A short-term phased recovery by key geography is also addressed.

- Competitors identified in this market include, among others,

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=PRN

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE

II. EXECUTIVE SUMMARY

1. MARKET OVERVIEW Global Competitor Market Shares Gene Therapy Competitor Market Share Scenario Worldwide (in %): 2019 & 2028 Impact of Covid-19 and a Looming Global Recession

2. FOCUS ON SELECT PLAYERS

3. MARKET TRENDS & DRIVERS

4. GLOBAL MARKET PERSPECTIVE Table 1: Gene Therapy Global Market Estimates and Forecasts in US$ Thousand by Region/Country: 2020-2027

Table 2: Gene Therapy Global Retrospective Market Scenario in US$ Thousand by Region/Country: 2012-2019

Table 3: Gene Therapy Market Share Shift across Key Geographies Worldwide: 2012 VS 2020 VS 2027

Table 4: Lentivirus (Vector) World Market by Region/Country in US$ Thousand: 2020 to 2027

Table 5: Lentivirus (Vector) Historic Market Analysis by Region/Country in US$ Thousand: 2012 to 2019

Table 6: Lentivirus (Vector) Market Share Breakdown of Worldwide Sales by Region/Country: 2012 VS 2020 VS 2027

Table 7: AAV (Vector) Potential Growth Markets Worldwide in US$ Thousand: 2020 to 2027

Table 8: AAV (Vector) Historic Market Perspective by Region/Country in US$ Thousand: 2012 to 2019

Table 9: AAV (Vector) Market Sales Breakdown by Region/Countryin Percentage: 2012 VS 2020 VS 2027

Table 10: RetroVirus & Gamma RetroVirus (Vector) Geographic Market Spread Worldwide in US$ Thousand: 2020 to 2027

Table 11: RetroVirus & Gamma RetroVirus (Vector) Region Wise Breakdown of Global Historic Demand in US$ Thousand: 2012 to 2019

Table 12: RetroVirus & Gamma RetroVirus (Vector) Market Share Distribution in Percentage by Region/Country: 2012 VS 2020 VS 2027

Table 13: Modified Herpes Simplex Virus (Vector) World Market Estimates and Forecasts by Region/Country in US$ Thousand: 2020to 2027

Table 14: Modified Herpes Simplex Virus (Vector) Market Historic Review by Region/Country in US$ Thousand: 2012 to 2019

Table 15: Modified Herpes Simplex Virus (Vector) Market Share Breakdown by Region/Country: 2012 VS 2020 VS 2027

Table 16: Adenovirus (Vector) World Market by Region/Country in US$ Thousand: 2020 to 2027

Table 17: Adenovirus (Vector) Historic Market Analysis byRegion/Country in US$ Thousand: 2012 to 2019

Table 18: Adenovirus (Vector) Market Share Distribution in Percentage by Region/Country: 2012 VS 2020 VS 2027

Table 19: Other Applications (Vector) World Market Estimates and Forecasts in US$ Thousand by Region/Country: 2020 to 2027

Table 20: Other Applications (Vector) Market Worldwide Historic Review by Region/Country in US$ Thousand: 2012 to 2019

Table 21: Other Applications (Vector) Market Percentage Share Distribution by Region/Country: 2012 VS 2020 VS 2027

III. MARKET ANALYSIS

GEOGRAPHIC MARKET ANALYSIS

UNITED STATES Market Facts & Figures US Gene Therapy Market Share (in %) by Company: 2019 & 2025 Market Analytics Table 22: United States Gene Therapy Market Estimates and Projections in US$ Thousand by Vector: 2020 to 2027

Table 23: Gene Therapy Market in the United States by Vector: A Historic Review in US$ Thousand for 2012-2019

Table 24: United States Gene Therapy Market Share Breakdown by Vector: 2012 VS 2020 VS 2027

CANADA Table 25: Canadian Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020 to 2027

Table 26: Canadian Gene Therapy Historic Market Review by Vector in US$ Thousand: 2012-2019

Table 27: Gene Therapy Market in Canada: Percentage Share Breakdown of Sales by Vector for 2012, 2020, and 2027

JAPAN Table 28: Japanese Market for Gene Therapy: Annual Sales Estimates and Projections in US$ Thousand by Vector for the Period 2020-2027

Table 29: Gene Therapy Market in Japan: Historic Sales Analysisin US$ Thousand by Vector for the Period 2012-2019

Table 30: Japanese Gene Therapy Market Share Analysis by Vector: 2012 VS 2020 VS 2027

CHINA Table 31: Chinese Gene Therapy Market Growth Prospects in US$Thousand by Vector for the Period 2020-2027

Table 32: Gene Therapy Historic Market Analysis in China in US$ Thousand by Vector: 2012-2019

Table 33: Chinese Gene Therapy Market by Vector: Percentage Breakdown of Sales for 2012, 2020, and 2027

EUROPE Market Facts & Figures European Gene Therapy Market: Competitor Market Share Scenario (in %) for 2019 & 2025 Market Analytics Table 34: European Gene Therapy Market Demand Scenario in US$ Thousand by Region/Country: 2020-2027

Table 35: Gene Therapy Market in Europe: A Historic Market Perspective in US$ Thousand by Region/Country for the Period2012-2019

Table 36: European Gene Therapy Market Share Shift by Region/Country: 2012 VS 2020 VS 2027

Table 37: European Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020-2027

Table 38: Gene Therapy Market in Europe in US$ Thousand by Vector: A Historic Review for the Period 2012-2019

Table 39: European Gene Therapy Market Share Breakdown byVector: 2012 VS 2020 VS 2027

FRANCE Table 40: Gene Therapy Market in France by Vector: Estimates and Projections in US$ Thousand for the Period 2020-2027

Table 41: French Gene Therapy Historic Market Scenario in US$ Thousand by Vector: 2012-2019

Table 42: French Gene Therapy Market Share Analysis by Vector: 2012 VS 2020 VS 2027

GERMANYTable 43: Gene Therapy Market in Germany: Recent Past, Current and Future Analysis in US$ Thousand by Vector for the Period2020-2027

Table 44: German Gene Therapy Historic Market Analysis in US$ Thousand by Vector: 2012-2019

Table 45: German Gene Therapy Market Share Breakdown by Vector: 2012 VS 2020 VS 2027

ITALY Table 46: Italian Gene Therapy Market Growth Prospects in US$ Thousand by Vector for the Period 2020-2027

Table 47: Gene Therapy Historic Market Analysis in Italy in US$ Thousand by Vector: 2012-2019

Table 48: Italian Gene Therapy Market by Vector: Percentage Breakdown of Sales for 2012, 2020, and 2027

UNITED KINGDOM Table 49: United Kingdom Market for Gene Therapy: Annual Sales Estimates and Projections in US$ Thousand by Vector for thePeriod 2020-2027

Table 50: Gene Therapy Market in the United Kingdom: Historic Sales Analysis in US$ Thousand by Vector for the Period 2012-2019

Table 51: United Kingdom Gene Therapy Market Share Analysis byVector: 2012 VS 2020 VS 2027

SPAIN Table 52: Spanish Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020 to 2027

Table 53: Spanish Gene Therapy Historic Market Review by Vector in US$ Thousand: 2012-2019

Table 54: Gene Therapy Market in Spain: Percentage Share Breakdown of Sales by Vector for 2012, 2020, and 2027

RUSSIATable 55: Russian Gene Therapy Market Estimates and Projections in US$ Thousand by Vector: 2020 to 2027

Table 56: Gene Therapy Market in Russia by Vector: A Historic Review in US$ Thousand for 2012-2019

Table 57: Russian Gene Therapy Market Share Breakdown byVector: 2012 VS 2020 VS 2027

REST OF EUROPE Table 58: Rest of Europe Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020-2027

Table 59: Gene Therapy Market in Rest of Europe in US$ Thousand by Vector: A Historic Review for the Period 2012-2019

Table 60: Rest of Europe Gene Therapy Market Share Breakdown by Vector: 2012 VS 2020 VS 2027

ASIA-PACIFIC Table 61: Asia-Pacific Gene Therapy Market Estimates and Forecasts in US$ Thousand by Region/Country: 2020-2027

Table 62: Gene Therapy Market in Asia-Pacific: Historic Market Analysis in US$ Thousand by Region/Country for the Period 2012-2019

Table 63: Asia-Pacific Gene Therapy Market Share Analysis by Region/Country: 2012 VS 2020 VS 2027

Table 64: Gene Therapy Market in Asia-Pacific by Vector: Estimates and Projections in US$ Thousand for the Period 2020-2027

Table 65: Asia-Pacific Gene Therapy Historic Market Scenario in US$ Thousand by Vector: 2012-2019

Table 66: Asia-Pacific Gene Therapy Market Share Analysis by Vector: 2012 VS 2020 VS 2027

AUSTRALIA Table 67: Gene Therapy Market in Australia: Recent Past, Current and Future Analysis in US$ Thousand by Vector for the Period 2020-2027

Table 68: Australian Gene Therapy Historic Market Analysis in US$ Thousand by Vector: 2012-2019

Table 69: Australian Gene Therapy Market Share Breakdown byVector: 2012 VS 2020 VS 2027

INDIA Table 70: Indian Gene Therapy Market Estimates and Forecasts in US$ Thousand by Vector: 2020 to 2027

Table 71: Indian Gene Therapy Historic Market Review by Vectorin US$ Thousand: 2012-2019

Table 72: Gene Therapy Market in India: Percentage Share Breakdown of Sales by Vector for 2012, 2020, and 2027

SOUTH KOREA Table 73: Gene Therapy Market in South Korea: Recent Past, Current and Future Analysis in US$ Thousand by Vector for thePeriod 2020-2027

Table 74: South Korean Gene Therapy Historic Market Analysis in US$ Thousand by Vector: 2012-2019

Table 75: Gene Therapy Market Share Distribution in South Korea by Vector: 2012 VS 2020 VS 2027

REST OF ASIA-PACIFIC Table 76: Rest of Asia-Pacific Market for Gene Therapy: Annual Sales Estimates and Projections in US$ Thousand by Vector for the Period 2020-2027

Table 77: Gene Therapy Market in Rest of Asia-Pacific: Historic Sales Analysis in US$ Thousand by Vector for the Period2012-2019

Table 78: Rest of Asia-Pacific Gene Therapy Market Share Analysis by Vector: 2012 VS 2020 VS 2027

LATIN AMERICA Table 79: Latin American Gene Therapy Market Trends by Region/Country in US$ Thousand: 2020-2027

Table 80: Gene Therapy Market in Latin America in US$ Thousand by Region/Country: A Historic Perspective for the Period 2012-2019

Table 81: Latin American Gene Therapy Market PercentageBreakdown of Sales by Region/Country: 2012, 2020, and 2027

Table 82: Latin American Gene Therapy Market Growth Prospects in US$ Thousand by Vector for the Period 2020-2027

Table 83: Gene Therapy Historic Market Analysis in Latin America in US$ Thousand by Vector: 2012-2019

Table 84: Latin American Gene Therapy Market by Vector: Percentage Breakdown of Sales for 2012, 2020, and 2027

ARGENTINA Table 85: Argentinean Gene Therapy Market Estimates andForecasts in US$ Thousand by Vector: 2020-2027

Table 86: Gene Therapy Market in Argentina in US$ Thousand by Vector: A Historic Review for the Period 2012-2019

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Global Gene Therapy Industry - PRNewswire

Gilead Sciences is ditching a new avenue of treating hepatitis B as it cuts ties with Precision Biosciences less than two years into their research pact.

Back in September 2018, Gilead laid up to $445 million on the table in biobucks that saw the pair collaborate on gene therapies aimed at eliminating viral infections in vivo by using Precisions genome editing platform.

Under the deal, Gilead was on tap to fully fund the effort and run clinical trials while Precision was on the hook for early development, formulation and preclinical work.

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Current HBV treatments may suppress viral replication, but they do not completely clear out the virus; the infections covalently closed circular DNA, or cccDNA, enables HBV replication to restart if treatment is stopped.

Precisions ARCUS editing platform is derived from a natural enzyme called a homing endonuclease that can target long sequences and is used to insert or delete DNA. The Durham, North Carolina-based companys fully synthetic version can be designed to locate and disrupt particular sequences and minimize off-target effects.

Gileads preliminary, in vitro studies using ARCUS nucleases had shown significant activity against cccDNA and integrated HBV DNA in human liver cells. But, two years down the line, Gilead no longer wants in.

Precision, which is predominately working on next-gen cancer therapies, will regain the license to its hep B program. Its not clear what prompted the decision, but the biotech could be on the lookout for a new partner.

This was a highly productive and well-aligned collaboration, and we deeply value the opportunity to advance our ARCUS genome editing technology and a potential cure for HBV alongside a global leader in infectious disease, said Derek Jantz, Ph.D., co-founder and chief scientific officer of Precision.

Key learnings from this program and how to develop liver-directed gene editing therapeutic candidates are directly applicable to our in vivo pipeline. While we consider new partnership opportunities for HBV, we are focused on progressing our internal lead proprietary gene correction program for primary hyperoxaluria type 1 for which we expect to nominate a clinical candidate later this year.

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Gilead axes $445M Precision Biosciences gene therapy hep B pact - FierceBiotech

Madison researchers have developed a safer and more efficient way to deliver a promising new method for treating cancer and liver disorders and for vaccination including a COVID-19 vaccine from Moderna Therapeutics that has advanced to clinical trials with humans.

The technology relies on inserting into cells pieces of carefully designed messenger RNA (mRNA), a strip of genetic material that human cells typically transcribe from a persons DNA in order to make useful proteins and go about their business. Problems delivering mRNA safely and intact without running afoul of the immune system have held back mRNA-based therapy, but UWMadison researchers are making tiny balls of minerals that appear to do the trick in mice.

These microparticles have pores on their surface that are on the nanometer scale that allow them to pick up and carry molecules like proteins or messenger RNA, saysWilliam Murphy, a UWMadison professor of biomedical engineering and orthopedics. They mimic something commonly seen in archaeology, when we find intact protein or DNA on a bone sample or an eggshell from thousands of years ago. The mineral components helped to stabilize those molecules for all that time.

Murphy and UWMadison collaborators used the mineral-coated microparticles (MCMs) which are 5 to 10 micrometers in diameter, about the size of a human cell in a series of experiments to deliver mRNA to cells surrounding wounds in diabetic mice. Wounds healed faster in MCM-treated mice, and cells in related experiments showed much more efficient pickup of the mRNA molecules than other delivery methods.

The researchers described their findings today in the journal Science Advances.In a healthy cell, DNA is transcribed into mRNA, and mRNA serves as the instructions the cells machinery uses to make proteins. A strip of mRNA created in a lab can be substituted into the process to tell a cell to make something new. If that something is a certain kind of antigen, a molecule that alerts the immune system to the presence of a potentially harmful virus, the mRNA has done the job of a vaccine.

The UWMadison researchers coded mRNA with instructions directing cell ribosomes to pump out a growth factor, a protein that prompts healing processes that are otherwise slow to unfold or nonexistent in the diabetic mice (and many severely diabetic people).

mRNA is short-lived in the body, though, so to deliver enough to cells typically means administering large and frequent doses in which the mRNA strands are carried by containers made of molecules called cationic polymers.

Oftentimes the cationic component is toxic. The more mRNA you deliver, the more therapeutic effect you get, but the more likely it is that youre going to see toxic effect, too. So, its a trade-off, Murphy says. What we found is when we deliver from the MCMs, we dont see that toxicity. And because MCM delivery protects the mRNA from degrading, you can get more mRNA where you want it while mitigating the toxic effects.

The new study also paired mRNA with an immune-system-inhibiting protein, to make sure the target cells didnt pick the mRNA out as a foreign object and destroy or eject it.

Successful mRNA delivery usually keeps a cell working on new instructions for about 24 hours, and the molecules they produce disperse throughout the body. Thats enough for vaccines and the antigens they produce. To keep lengthy processes like growing replacement tissue to heal skin or organs, the proteins or growth factors produced by the cells need to hang around for much longer.

What weve seen with the MCMs is, once the cells take up the mRNA and start making protein, that protein will bind right back within the MCM particle, Murphy says. Then it gets released over the course of weeks. Were basically taking something that would normally last maybe hours or even a day, and were making it last for a long time.

And because the MCMs are large enough that they dont enter the bloodstream and float away, they stay right where they are needed to keep releasing helpful therapy. In the mice, that therapeutic activity kept going for more than 20 days.

They are made of minerals similar to tooth enamel and bone, but designed to be reabsorbed by the body when theyre not useful anymore, says Murphy, whose work is supported by the Environmental Protection Agency, the National Institutes of Health and the National Science Foundation and a donation from UWMadison alums Michael and Mary Sue Shannon.

We can control their lifespan by adjusting the way theyre made, so they dissolve harmlessly when we want.

The technology behind the microparticles was patented with the help of the Wisconsin Alumni Research Foundation and is licensed to Dianomi Therapeutics, a company Murphy co-founded.

The researchers are now working on growing bone and cartilage and repairing spinal cord injuries with mRNA delivered by MCMs.

Reference: Khalil et al. (2020).Single-dose mRNA therapy via biomaterial-mediated sequestration of overexpressed proteins. Science Advances.DOI: 10.1126/sciadv.aba2422.

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

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Safer and More Efficient Method To Deliver Gene Therapy - Technology Networks

Written by AZoNanoJul 8 2020

Two researchers from Johns Hopkins Universityan ophthalmologist and an engineerhave successfully used nanoparticles to deliver gene therapy for blinding eye disease. They achieved this by performing experiments in mice and rats.

Image Credit: Johns Hopkins Medicine.

Using an exclusively designed large molecule, the researchers could compact huge bundles of therapeutic DNA to be delivered into the cells of the eye.

Reported in the Science Advances journal on July 3rd, 2020, the study offers evidence of the prospective value of nanoparticle-delivered gene therapy for the treatment of wet age-related macular degeneration.

Macular degeneration is an eye disease in which blood vessel growth is abnormal, causing damage to the light-sensitive tissue at the back of the eye, together with rarer, inherited blinding diseases of the retina.

Several gene therapy techniques rely on viral vectors, which tap the natural ability of a virus to carry genetic material into cells. But viruses tend to create an immune response that prevents repeat dosing, and the one most often used for ocular gene therapy is not capable of carrying large genes.

Some of the most prevalent inherited retinal degenerations are due to mutations in large genes that simply cannot fit into the most commonly used viral vector.

Peter Campochiaro, MD, Eccles Professor of Ophthalmology, Johns Hopkins University School of Medicine

Campochiaro is also a member of the Johns Hopkins Medicine Wilmer Eye Institute.

Campochiaro and Jordan Green, PhD, created a new technique to overcome such drawbacks, which involves using a biodegradable polymer that surrounds and compacts long DNA stretches. This helps create nanoparticles with the potential to enterthe cells. This technology enables the conversion of the cells of the eye into mini factories for a therapeutic protein.

The researchers first tested whether the nanoparticles enter their target cells by loading the nanoparticles with a gene for a fluorescent protein that makes cells glow similar to a glow stick.

Using the glowing molecule, the researchers were able to find the location, duration, and amount of gene expression that can be achieved using the nanoparticles.

Even eight months following treatment, it was found that most of the light-sensitive cells in the eyes of the rats glowed, demonstrating that the nanoparticles effectively loaded the fluorescent gene into the cells.

A similar experiment was also performed using the nanoparticles to shuttle a biologically relevant gene into the eye. A gene for vascular endothelial growth factor (VEGF) was loaded into the nanoparticles, where the gene takes part in the growth of abnormal blood vessels in people suffering from wet macular degeneration.

The eyes of 30 rats were injected with the nanoparticles that carried the VEGF gene, and the effects in the retina were determined one, two, and five months post-injection. One month post-injection, abnormal blood vessels developed in each tested rat under and inside the retina, similar to those seen in patients suffering from wet macular degeneration.

The abnormal blood vessels were found to be more widespread at two and five months post-injection, and there was related scarring under the retina such as those observed in chronic untreated wet macular degeneration.

These results show that the genes delivered by nanoparticles stayed active within the cells for several months.

Peter Campochiaro, MD, Eccles Professor of Ophthalmology, School of Medicine, Johns Hopkins University

Lastly, the researchers tested the ability of a nanoparticle to deliver a therapeutic gene for the disease by using mice that had been genetically engineered to develop a kind of wet macular degeneration such as those seen in humans. Nanoparticles were loaded with a gene that synthesizes a protein that neutralizes VEGF.

At present, such proteins that block VEGF proteins are injected by physicians into the eyes of people suffering from macular degeneration. This treatment helps control the overgrowth of abnormal, leaky blood vessels. However, this process must be repeated often and is cumbersome for patients and their caretakers.

Three weeks post-injection of nanoparticles with the gene for the anti-VEGF protein, a 60% decrease in abnormal blood vessels was observed in the mice.

The same effect was observed 35 days later.

These results are extremely promising. We have the ability to reach the cells most significantly affected by degenerative eye disease with nonviral treatments that can allow the eye to create its own sustained therapies.

Jordan Green, PhD, Professor of Biomedical Engineering, School of Medicine, Johns Hopkins University

In the United States, approximately 1.6 million people suffering from macular degeneration are administered injected drugs to the eye every four to six weeks. A gene therapy treatment could offer a means for the tissue of the eye to prevent further deterioration of vision with only a few initial treatments.

Genetic diseases that lead to blindness could be similarly treated by introducing functional versions of genes disabled by inherited mutations.

Jikui Shen, Jayoung Kim, Stephany Tzeng, Kun Ding, Zibran Hafiz, Da Long, and Jiangxia Wang from the Johns Hopkins University School of Medicine are the other researchers involved in this study.

This study was financially supported by the National Eye Institute (01EY031097, R21EY026148, R01EY028996, EY01765), the National Institute of Biomedical Imaging and Bioengineering (R01EB022148) Research to Prevent Blindness (the Dr H. James and Carole Free Catalyst Award and an unrestricted grant), the Louis B. Thalheimer Fund for Translational Research; the Barth Syndrome Foundation, Samsung, Conrad and Lois Aschenbach, Per Bang-Jensen, Andrew and Yvette Marriott, and Jean Lake.

Shen, J., et al. (2020) Suprachoroidal gene transfer with nonviral nanoparticles. Science Advances. doi.org/10.1126/sciadv.aba1606.

Source: https://www.hopkinsmedicine.org/

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Nanoparticles Used to Deliver Gene Therapy for Macular Degeneration - AZoNano

Scientists report promising results from a study that aims to develop a new approach to treating the most common inherited eye condition

Pixabay/Arek Socha

The study, which was published in Stem Cell Reports, involved using a viral vector to carry a replacement gene to malfunctioning cells in retinal organoids.

Following treatment, analysis revealed that the mini retinas had begun to produce a protein that is essential for vision.

Professor Mike Cheetham, from UCL, helped to develop the mini retinas that were used to test the gene therapy.

He highlighted that the mini retinas allow researchers to reproduce many different elements of inherited disease.

It makes it possible for us to study in detail why people go blind and try to find ways to prevent blindness. Its exciting that the gene therapy seems to be so effective for this form of retinitis pigmentosa, Professor Cheetham said.

The research was part-funded by Fight for Sight. The charitys chief executive, Sherine Krause, described the findings as incredibly promising.

Professor Cheetham and his teams work in collaboration with Trinity College Dublin represents a significant breakthrough for eye research and shows the importance of science to find new treatments for the prevailing causes of sight loss, she said.

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UK researchers use mini retinas to test gene therapy for RP - AOP

NEW YORK and CLEVELAND, July 09, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced the appointment of Michael Amoroso as Senior Vice President and Chief Commercial Officer (CCO), effective immediately. Mr. Amoroso brings to Abeona over 20 years of product commercialization experience in the biotechnology and pharmaceutical industries, most recently as Senior Vice President and Head of Worldwide Commercial, Cell Therapy at Kite, a Gilead Company.

Mr. Amoroso will have overall responsibility for building the Companys commercial organization, developing the commercialization strategy for EB-101, its autologous, gene-corrected cell therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) and its lead product candidate, as well as leading pre-commercial planning for its investigational adeno-associated virus vector (AAV)-based gene therapies.

Michael is a highly accomplished commercial leader with a focus on cell and gene therapies and a proven track record of launching innovative drugs for rare diseases, said Joo Siffert, M.D., Chief Executive Officer of Abeona. As we advance our pivotal Phase 3 VIITAL study of EB-101 in RDEB, Michaels proven track record in commercialization, supply chain management for personalized, autologous cell therapies, experience in developing novel launch plans, working closely with governments around the world to ensure patients have access, and ability for building commercial and organizational capabilities will lay the groundwork for our potential go-to-market strategy for EB-101. Furthermore, his history of integrating commercial perspective into pipeline programs will be instrumental in positioning our investigational AAV gene therapies to shape the treatment paradigm for patients with MPS IIIA, MPS IIIB, and other rare genetic diseases.

Prior to joining Abeona, Mr. Amoroso held various senior level commercial positions at leading biopharmaceutical companies, including Kite, Eisai Inc., Celgene Corporation (now a subsidiary of Bristol-Myers Squibb Company), and Sanofi. At Kite, he was responsible for the companys worldwide commercial organization leading the commercialization efforts for the autologous CAR T-cell therapy, YESCARTA, and the future cell therapy pipeline. Before Kite, Mr. Amoroso was Senior Vice President, Americas for Eisais Commercial Oncology Business Group, where he was accountable for teams charged with creating and driving commercial strategy and implementation for the companys approved products and earlier-stage assets. Previously, Mr. Amoroso worked at Celgene for six years in several commercial roles before serving as the organizations Commercial Lead for CAR T-cell therapy programs. In this capacity, he helped Celgene develop an organizational model to commercialize cell therapies including specialized manufacturing and customer services for patients with lymphoma and myeloma. Before joining Celgene, Mr. Amoroso held various marketing and sales leadership positions over his 10-plus year tenure at Sanofi. Mr. Amoroso earned his M.B.A. in Management from the Stern School of Business, New York University, and his B.A. in Biological Sciences, summa cum laude, from Rider University.

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-based 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

Continued here:
Abeona Therapeutics Announces Appointment of Michael Amoroso as Chief Commercial OfficerTo lead Abeona's commercial organization, EB-101...

Biogen has picked up a new, preclinical gene therapy program from Massachusetts Eye and Ear for inherited retinal degeneration due to mutations in the PRPF31 gene, among the most common causes for autosomal dominant retinitis pigmentosa. Theyre building on the work of Harvards Eric Pierce. The treatment of IRDs with highly effective AAV-based gene therapies is core to Biogens ophthalmology strategy, said Chris Henderson, the research head at Biogen. This agreement underscores our commitment to that strategy and builds off of our acquisition of Nightstar Therapeutics in 2019 and our active clinical trials of gene therapies for different genetic forms of IRD.

Sarepta has inked a collaboration with Hansa to develop their experimental drug imlifidase as a pre-treatment for their gene therapies. The drug is intended for use in patients who have neutralizing antibodies that would prevent gene therapies for Duchenne muscular dystrophy and Limb-girdle muscular dystrophy from working. Hansa gets a $10 million upfront and up to $397.5 million in milestones.

Its been raining money on Wall Street at least when it comes to drug developers. CRISPR Therapeutics $CRSP and Acceleron each raised a whopping $450 million this week after pricing follow-on offerings. CRISPR priced 6,428,572 common shares at a public offering price of $70.00 per share, while Acceleron $XLRN auctioned off 4,864,864 shares of common stock at a price to the public of $92.50 per share.

The transatlantic biotech player Immatics has completed its flip onto Nasdaq through the Arya Sciences Acquisition Corp. The cancer drug biotech will trade as $IMTX after it raised $253 million in the process. The SPAC was set up by Perceptive Advisors.

Seattle-based Neoleukin Therapeutics, meanwhile, raised $76.2 million $NLTX for its work on protein therapeutics.

Link:
Biogen bags an AAV gene therapy program from Massachusetts Eye and Ear; Biotechs raised $1B-plus in latest round of follow-ons - Endpoints News

Hemophilia Gene Therapy Market

IndustryGrowthInsights, 09-07-2020: The research report on the Hemophilia Gene Therapy Market is a deep analysis of the market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. Experts have studied the historical data and compared it with the changing market situations. The report covers all the necessary information required by new entrants as well as the existing players to gain deeper insight.

Furthermore, the statistical survey in the report focuses on product specifications, costs, production capacities, marketing channels, and market players. Upstream raw materials, downstream demand analysis, and a list of end-user industries have been studied systematically, along with the suppliers in this market. The product flow and distribution channel have also been presented in this research report.

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The Major Manufacturers Covered in this Report:Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline TherapeuticsHemophilia Gene Therap

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By Types:Hemophilia AHemophilia BHemophilia Gene Therap

By Applications:Hemophilia A Gene TherapyHemophilia B Gene Therapy

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In conclusion, the Hemophilia Gene Therapy Market report is a reliable source for accessing the research data that is projected to exponentially accelerate your business. The report provides information such as economic scenarios, benefits, limits, trends, market growth rate, and figures. SWOT analysis is also incorporated in the report along with speculation attainability investigation and venture return investigation.

About IndustryGrowthInsights:IndustryGrowthInsights has set its benchmark in the market research industry by providing syndicated and customized research report to the clients. The database of the company is updated on a daily basis to prompt the clients with the latest trends and in-depth analysis of the industry. Our pool of database contains various industry verticals that include: IT & Telecom, Food Beverage, Automotive, Healthcare, Chemicals and Energy, Consumer foods, Food and beverages, and many more. Each and every report goes through the proper research methodology, validated from the professionals and analysts to ensure the eminent quality reports.

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Hemophilia Gene Therapy Market 2020 Global analysis, opportunities and forecast to 2026 | By Top Leading Vendors like Spark Therapeutics, Ultragenyx,...

Gene Therapy Market Forecast 2020-2026

The Global Gene Therapy Market research report provides and in-depth analysis on industry- and economy-wide database for business management that could potentially offer development and profitability for players in this market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. It offers critical information pertaining to the current and future growth of the market. It focuses on technologies, volume, and materials in, and in-depth analysis of the market. The study has a section dedicated for profiling key companies in the market along with the market shares they hold.

The report consists of trends that are anticipated to impact the growth of the Gene Therapy Market during the forecast period between 2020 and 2026. Evaluation of these trends is included in the report, along with their product innovations.

Get a PDF Copy of the Sample Report for free @ https://industrygrowthinsights.com/request-sample/?reportId=168032

The Report Covers the Following Companies:Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.AdvantageneGene Therap

By Types:Ex vivoIn VivoGene Therap

By Applications:CancerMonogenicInfectious diseaseCardiovascular diseaseOther

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Years Considered to Estimate the Market Size:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year: 2020-2026

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Make an Inquiry of This Report @ https://industrygrowthinsights.com/enquiry-before-buying/?reportId=168032

About Industrygrowthinsights:Industrygrowthinsights has set its benchmark in the market research industry by providing syndicated and customized research report to the clients. The database of the company is updated on a daily basis to prompt the clients with the latest trends and in-depth analysis of the industry. Our pool of database contains various industry verticals that include: IT & Telecom, Food Beverage, Automotive, Healthcare, Chemicals and Energy, Consumer foods, Food and beverages, and many more. Each and every report goes through the proper research methodology, validated from the professionals and analysts to ensure the eminent quality reports.

Contact Info:Name: Alex MathewsAddress: 500 East E Street, Ontario,CA 91764, United States.Phone No: USA: +1 909 545 6473 | IND: +91-7000061386Email: [emailprotected]Website: https://Industrygrowthinsights.com

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How Gene Therapy Market Will Dominate In Coming Years? Report Covering Products, Financial Information, Developments, Swot Analysis And Strategies |...

Orphan Drug Designation granted to Neurogenes adeno-associated virus vector with engineered transgene encoding the human CLN5 gene

Neurogene Inc., a company founded with a mission to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to adeno-associated virus vector with engineered transgene encoding the human CLN5 gene for patients with CLN5, a form of Batten disease. Batten disease, a common name for a rare class of diseases called neuronal ceroid lipofuscinoses (NCL), affects an estimated 2-4 out of every 100,000 children in the United States.

"CLN5 is a devastating neurodegenerative disease with no FDA approved treatment options," said Rachel McMinn, Ph.D., Neurogenes Founder and Chief Executive Officer. "Receiving Orphan Drug Designation from the FDA is an important regulatory milestone, and we look forward to advancing our gene therapy program into the clinic."

The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

About CLN5Batten disease, also called neuronal ceroid lipofuscinoses (NCLs), is a family of rare and fatal neurodegenerative diseases caused by pathogenic changes in one of a series of genes that result in the accumulation of abnormal storage material across multiple organ systems, including the brain, eye, skin and other tissues. The most prominent effects occur in the brain, where the progressive and inevitable loss of neurons lead to devastating declines in cognitive and motor function in those with Batten disease. The subtype CLN5 is a rare, pediatric-onset and rapidly progressive disease caused by defects in the CLN5 gene. CLN5 disease is characterized by progressive deterioration in intellectual and motor capabilities and vision loss, as well as seizures and death in childhood or adolescence. Diagnosis of the disease is confirmed through genetic testing. Currently, there are no approved disease-modifying therapies available.

About Genetic TestingNeurogene is committed to lowering the barriers of obtaining a genetic diagnosis for patients and has partnered with Invitae to co-sponsor two genetic testing programs. Healthcare providers can order, at no charge, an Invitae Epilepsy panel for any child under the age of eight who has had an unprovoked seizure, or the Detect Lysosomal Storage Diseases panel for patients suspected of having a lysosomal storage disease. Visit https://www.invitae.com/en/sponsored-testing/ for more details.

About Neurogene Inc.Neurogene Inc. is focused on developing life-changing genetic medicines for patients and their families affected by rare, devastating neurological diseases. We partner with leading academic researchers, patient advocacy organizations and caregivers to bring therapies to patients that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options exist today. Our lead programs are designed to use AAV-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also investing in novel technology to develop treatments for diseases not well served by gene therapy. For more information, visit http://www.neurogene.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200707005106/en/

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FDA Grants Orphan Drug Designation to Neurogenes Gene Therapy for the Treatment of CLN5 Batten Disease - Yahoo Finance

Carmine Therapeutics and Takeda Collaborate to Discover and Develop Rare Disease Gene Therapies Using Novel Red Blood Cell Extracellular Vesicles Platform

Singapore based startup Carmine Therapeutics has announced that it has signed a research collaboration agreement with Japanese firm Takeda Pharmaceutical Company Limited o discover, develop and commercialize transformative non-viral gene therapies for two rare disease targets using Carmines REGENT(TM) technology, based on red blood cell extracellular vesicles. In addition, Takeda has committed a $5M convertible loan in support of the development of Carmines novel REGENT platform.

Under the terms of the agreement, Carmine will receive an upfront payment, research funding support, and is eligible for over $900M in total milestone payments plus tiered royalties.

Takeda has an option to license the programs following the completion of pre-clinical proof of concept studies and would be responsible for clinical development and commercialization.

Carmine Therapeutics is pioneering a novel class of therapeutics based on its REGENT(TM) technology which leverages red blood cell extracellular vesicles (RBCEVs), first published in Nature Communications in 2018.

An initial focus is non-viral gene therapies. Compared to adeno-associated virus (AAV)-based gene therapy, RBCEV-based gene therapy has the potential for repeat dosing, a significantly larger transgene payload capacity in excess of 11KB, and enhanced bio-distribution in selected tissues through RBCEV surface modification.

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Carmine Therapeutics teams up with Takeda for non-viral gene therapies - BSA bureau