Gene Therapy Research Institution Co., Ltd.

One of first Sf-RVN ® Insect Cell Line applications for clinical stage gene therapy production Merck to support safe and robust production of viral vectors Collaboration aims to bring new therapies to market faster Merck, a leading science and technology company, has signed a non-binding Memorandum of Understanding (MoU) with Gene Therapy Research Institution Co., Ltd. (GTRI), a Japanese clinical-stage biotech start-up specializing in gene therapy using adeno-associated virus (AAV) vectors. GTRI will utilize Merck’s Sf-RVN ® Insect Cell Line platform for the Good Manufacturing Practices (GMP) production of its viral vector-based gene therapy for Parkinson’s Disease. “In Japan, clinical trials of gene therapy medicines are underway in the hope of developing new treatments to fight and cure diseases,” said Isao Hatano, Head of Process Solutions, Japan, Life Science business of Merck. “With our broad portfolio of products and services, we can better equip and support GTRI in accelerating process development and manufacturing of viral vector-based gene therapy. We believe this collaboration will ultimately help save many patients suffering from serious diseases.” As part of the MoU, Merck will partner with GTRI to build a robust AAV production platform to scale up production. The collaboration will also include the use of Merck’s cell line and platform technologies for developing viral vector-based gene therapy. Additionally, Merck will provide GTRI with technical support, training and process optimization for process development at the M Lab™ Collaboration Center in Tokyo. “Since 2014, we have been working to bring our unique gene therapy techniques to refractory diseases where effective therapies have not been established, primarily focusing on Parkinson's disease and Amyotrophic Lateral Sclerosis (ALS),” said Katsuhito Asai, CEO, GTRI. “Our biggest challenge is developing robust and safe manufacturing processes for viral vectors used to introduce DNA into patients, especially on a large scale. Producing viral vectors is much more complex and larger in scale than making typical antibody drugs. Our partnership with Merck will optimize this process and help bring therapies to market faster.” Earlier this year, Merck announced a partnership with The Michael J. Fox Foundation for Parkinson's Research (MJFF) to advance research aimed at slowing progression of Parkinson’s disease. All Merck press releases are distributed by e-mail at the same time they become available on the Merck website. Please go to www.merckgroup.com/subscribe to register online, change your selection or discontinue this service. About Gene Therapy Research Institute Co., Ltd. Gene Therapy Research Institution Co., Ltd. (GTRI) is a Japanese clinical-stage biotech start-up specializing in gene therapy using adeno-associated virus (AAV) vectors. Established in 2014, GTRI aims to realize the innovative research in practice, in the fastest way from basic research to clinical studies with the goal of popularizing gene therapy. https://www.genetherapy-ri.com/en/ About Merck Merck, a leading science and technology company, operates across life science, healthcare and electronics. Around 63,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2023, Merck generated sales of € 21 billion in 65 countries. Scientific exploration and responsible entrepreneurship have been key to Merck’s technological and scientific advances. This is how Merck has thrived since its founding in 1668. The founding family remains the majority owner of the publicly listed company. Merck holds the global rights to the Merck name and brand. The only exceptions are the United States and Canada, where the business sectors of Merck operate as MilliporeSigma in life science, EMD Serono in healthcare, and EMD Electronics in electronics.

DUBLIN--( BUSINESS WIRE )--The "Global Joint Venture Partnering Terms and Agreements in Pharma, Biotech and Diagnostics 2016-2023" report has been added to ResearchAndMarkets.com's offering. The Global Joint Venture Partnering Terms and Agreements in Pharma, Biotech and Diagnostics 2016-2023 report provides comprehensive understanding and unprecedented access to the joint venture deals and agreements entered into by the worlds leading life science companies. This report provides details of the latest joint venture agreements announced in the pharmaceutical, biotechnology and diagnostic sectors. Fully up to date, the report provides details of joint venture agreements from 2016. There is an increasing willingness for parties to enter joint venture deals; such deals enable both parties to benefit from the upside of a big product win, whereas traditional licensing deals forfeit upside for near term upfront, milestone and royalty payments. Joint venture partnering allow the parties to securitize value and reduce risk, but keep a part of the potential upside should the product reach the market. Event if the licensor does not commercialize the project they can either sell those rights to the licensee partner or another partner for an amount higher than would have been achieved at an earlier stage licensing deal. Another reason for joint venture deals is the joint origin of a project. Drug development projects often require various scientific and technological novelties that stem from different companies. If both companies contributed to the origination of the project, then both have from the beginning a stake in the project. The report provides a detailed understanding and analysis of how and why companies enter joint venture deals. Understanding the flexibility of a prospective partner's negotiated deals terms provides critical insight into the negotiation process in terms of what you can expect to achieve during the negotiation of terms. Whilst many smaller companies will be seeking details of the payments clauses, the devil is in the detail in terms of how payments are triggered - contract documents provide this insight where press releases and databases do not. This report contains a comprehensive listing of all joint venture deals announced since 2016 as recorded in the Current Agreements deals and alliances database, including financial terms where available, plus links to online copies of actual joint venture contract documents as submitted to the Securities Exchange Commission by companies and their partners. Contract documents provide the answers to numerous questions about a prospective partner's flexibility on a wide range of important issues, many of which will have a significant impact on each party's ability to derive value from the deal. The initial chapters of this report provide an orientation of joint venture dealmaking and business activities. Chapter 1 provides an introduction to the report, whilst chapter 2 provides an analysis of the trends in joint venture as well as a discussion on the merits of the type of deal. Chapter 3 provides an overview of the structure of joint venture deals. The chapter includes numerous case studies to enable understanding of joint venture deals. Chapter 4 provides a review of the leading Joint venture deals since 2016. Deals are listed by headline value. Where the deal has an agreement contract published at the SEC a link provides online access to the contract via the Current Agreements deals and alliances database. Chapter 5 provides a comprehensive listing of the top 25 most active Joint venture dealmaker companies. Each deal title links via Current Agreements deals and alliances database to an online version of the full deal record, and where available, the actual contract document, providing easy access to each deal record on demand. Chapter 6 provides a comprehensive and detailed review of Joint venture deals organized by company A-Z, therapy, technology and industry type signed and announced since 2016 where a contract document is available. Contract documents provide an indepth insight into the actual deal terms agreed between the parties with respect to the Joint venture deal. The appendices to the report includes a comprehensive listing of all Joint venture deals announced since 2016. Each listing is organized as an appendix by company A-Z, stage of development at signing, therapeutic area and industry type. Each deal title links via hyperlink to an online version of the deal record including, where available, the actual contract document. The report also includes numerous table and figures that illustrate the trends and activities in Joint venture dealmaking since 2016. In conclusion, this report provides everything a prospective dealmaker needs to know about Joint venture alliances. Key benefits Global Joint Venture Partnering Terms and Agreements in Pharma, Biotech and Diagnostics 2016-2023 provides the reader with the following key benefits: In-depth understanding of joint venture partnering deal trends since 2016, with real life case studies Comprehensive listing of all joint venture deals since 2016, together with deal terms, value and press release Comprehensive access to actual joint venture contracts entered into by the world's life science companies Insight into the terms included in a joint venture agreement, together with real world clause examples Understand the key deal terms companies have agreed in previous deals Undertake due diligence to assess suitability of your proposed deal terms for partner companies Report scope Global Joint Venture Partnering Terms and Agreements in Pharma, Biotech and Diagnostics 2016-2023 is intended to provide the reader with an in-depth understanding of the joint venture trends and structure of deals entered into by leading life science companies worldwide. Global Joint Venture Partnering Terms and Agreements in Pharma, Biotech and Diagnostics 2016-2023 includes: Trends in joint venture dealmaking in the biopharma industry since 2016 Case studies of real-life joint venture deals Comprehensive listing of joint venture deals since 2016 Access to joint venture contract documents The leading joint venture deals by value since 2016 Most active joint venture dealmakers since 2016 The leading joint venture partnering resources In Global Joint Venture Partnering Terms and Agreements in Pharma, Biotech and Diagnostics 2016-2023 available deals and contracts are listed by: Company A-Z Headline value Therapeutic area Technology type Each deal title links via Weblink to an online version of the actual deal record and where available, contract document, providing easy access to each contract document on demand. The Global Joint Venture Partnering Terms and Agreements in Pharma, Biotech and Diagnostics 2016-2023 report provides comprehensive access to available contract documents for joint venture deals. Companies Mentioned 3SBio 1717 Life Science Ventures A*STAR Agency for Science Technology and Research A*STAR Institute of Microelectronics (IME) A*STAR' Institute of Molecular and Cell Biology A2A Pharmaceuticals AAP Implantate AG Abbott Laboratories Abbvie Abilita Bio ABIVAX Abzena Accellix Accuray Acnos Pharma Adept Neuro Adisseo Advanced Prenatal Therapeutics Advanced Technologies Solutions Aequus Pharmaceuticals Agilis Biotherapeutics AgonOx AirWare Labs Aitbiotech Akers Biosciences AKESOgen Aleafia Health Aleva Neurotherapeutics Allied-Bristol Life Sciences Allied Minds Ally Bridge Group Altus Formulation Alzheimer's Association Alzheimer's Research UK Amarantus BioSciences Amendia American Diabetes Association AmerisourceBergen Amunix Amyris AnaBios Anokion Antitope Antoxerene Apax Partners Aphria Arbele Arbutus ARCH Personalized Medicine Initiative Aridis Pharmaceuticals Aruvant Sciences Arvinas Ascendance Biotechnology Ascendis Pharma Asklepios Biopharmaceutical Aslan Pharma Astellas Pharma AstraZeneca ATAI Life Sciences Athenex ATL Technology Atomwise Atrapos Therapeutics Atropos Therapeutics Auramedi Farmaceutica Autobio Diagnostics AUT Roche Diagnostics Laboratory AUT University Auxly Cannabis Group Avacta Avactis Biosciences Avalon GloboCare Avivagen Avvinity Therapeutics Axis Therapeutics Bain Capital Baxter International Bayer Baylor College of Medicine Baylor Miraca Genetics Laboratories B Braun B Braun CeGaT Beckley Canopy Therapeutics Beckley Foundation Becton Dickinson BeiGene Beijing Genomics Institute (BGI) Beijing Leadman Biochemistry Berry Genomics Betaliq BevCanna BGI Tech Solutions BHB Therapeutics Bill and Melinda Gates Foundation Bio-Techne BioAmber Biodist Biodyne USA BioFields Biogen Biohealth Innovation BioHeart BioLife Solutions BioLight BiolineRX biologistex CCM BioMed X Innovation Center Biomerics BioMotiv BioPharma Services Blackstone Blockshine Technology Bloomage BioTechnology Bloom Burton BLP Management Blueberries Medical Boehringer Ingelheim Breakthrough Diagnostics Brigham and Women's Hospital Bristol-Myers Squibb Broad Institute Buchang Pharmaceutical Buck Institute for Age Research Bukwang Pharmaceuticals Button Capital Calysta Energy Cancer Research Technology Cannabics Pharmaceuticals Cannabis Biocare Cannabis Therapeutics CannAmerica Brands CannaRoyalty Canopy Health Innovations Capna Intellectual Capnia Cardio3 Biosciences Asia Holdings Casebia Therapeutics Catapult Therapy TCR CB2 Therapeutics CBDerma Technology CBDistribution Cedar Gate Technologies CeGaT Cell Therapy Catapult Celyad Centauri Therapeutics Centene Center for Genetic Engineering and Biotechnology (Cuba) Centre for Commercialization of Regenerative Medicine (CCRM) Centro de Inmunolgia Molecular Cerevel Therapeutics Cesca Therapeutics Chan Soon-Shiong Institute of Molecular Medicine ChemAxon Children's Medical Research Institute Chimera Biotec China-Israel Biological Technology China-Singapore Guangzhou Knowledge City China Isotope And Radiation Chinese Academy of Science Chinese Future Industry Investment Fund Chongqing PSK-Health Sci-tech Development Cincinnati Children's Hospital Medical Center City of Hope CJ CheilJedang ClearPath Cloaked Therapeutics Co-Diagnostics Cohen Veterans Bioscience Cold Spring Harbor Colibri Heart Valve Collagen Solutions Plc Columbus Venture Partners Concentrics Research Consortium AI Corestone Biosciences Cornerstone Animal Health Corning Covenant Animal Health Partners Cre8ive CRISPR Therapeutics CureCell Cyclica CytoBioscience Cytotheryx Cytovant Sciences Daewoong Pharmaceutical Dance Biopharm Dasman Diabetes Institute David Snow Deerfield Management DemeRx Demetra Denka Denka-KEW Genomics DepYmed DIAN Diagnostics Diavax Biosciences Dixi Dizal Pharmaceutical DolCas-Tenshi Bioceuticals DolCas Biotech Dona Blanca Dongbao Dr. Walter Bell Duke-NUS Graduate Medical School Singapore Eastar Industries Eastern Capital Easton Pharmaceuticals eCardio Diagnostics Eddingpharm Eden BioCell Elanix Biotechnologies Elevar Therapeutics Eli Lilly EMD Millipore EMD Serono Emergence Therapeutics EmoCellix Emory University Emosis Enigma Diagnostics Enova Illumination Entheogenix Biosciences ERS Genomics Evotec ExCellThera Exemplar Genetics Facebook Faes Farma Faxian Therapeutics Fedecore Fedora Pharmaceuticals FerGene Ferring Pharmaceuticals FJ Pharma Flexpoint Ford Flow Pharma Fonterra Food and Drug Administration (FDA) FORMA Therapeutics Fortress Biotech Fosun Pharmaceutical FoxBio Fox Chase Cancer Center Fraunhofer Institute for Microelectronic Circuits and Systems Frazier Healthcare Ventures Fresenius Kabi Pharmaceuticals Frontage Laboratories FSD Pharma Futura Medical Fuzionaire Diagnostics Fuzionaire Radioisotope Technologies G-treeBNT Galectin Sciences Galectin Therapeutics Galvani Bioelectronics Gauss Surgical Genentech Gene Therapy Research Institution Genetic Technologies Genevant Sciences Genome Institute of Singapore Genomix Scientific Gentag GeoVax German Research Center for Artificial Intelligence Gerresheimer GHO Capital Ginkgo BioWorks GlaxoSmithKline For more information about this report visit https://www.researchandmarkets.com/r/laae8e About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

DUBLIN--( BUSINESS WIRE )--The "AAV vectors in gene therapy - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering. AAV vectors in gene therapy: Overview Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs, and harnessing revolutionary biotechnologies have contributed extensively to the growth of the gene therapy field. AAV Vectors in Gene Therapy Emerging Drugs GS010: Gensight Biologics LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. "LUMEVOQ" was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. Valoctocogene roxaparvovec: BioMarin Pharmaceutical Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A. It is currently in preregistration stage of development. In June 2021, BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA). In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase III GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA. DTX401: Ultragenyx Pharmaceutical DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-? under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-? activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. In a Phase I/II clinical study, all nine patients showed a clinical response, with significant reductions in the need for cornstarch and improvements in glucose control and other metabolic parameters compared to baseline. The drug is currently being evaluated in Phase III clinical trial to treat patients with Glycogen storage disease type I. AAV5-RPGR: MeiraGTx AAV-RPGR is an investigational gene therapy for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase III clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15. Timrepigene emparvovec: Biogen Timrepigene emparvovec is an AAV2 vector administered by subretinal injection, which aims to provide a functioning CHM gene and expression of the REP-1 protein to restore membrane trafficking and thereby slow, stop or potentially reverse decline in vision. Data from the Phase 1/2 studies demonstrated a slower rate of decline in visual acuity in patients treated with timrepigene emparvovec compared to untreated patients in the natural history study. In addition, some patients treated with timrepigene emparvovec showed improvements in visual acuity. The studies also demonstrated that timrepigene emparvovec was generally well tolerated with an acceptable safety profile. The safety and efficacy of a single subretinal injection of timrepigene emparvovec is currently being evaluated in the ongoing Phase III STAR study. AMT 061: UniQure AMT-061 (etranacogene dezaparvovec) is an experimental gene therapy that uniQure is developing to treat hemophilia B. AMT-061 uses a modified and harmless adeno-associated virus 5 (AAV5) to deliver a highly functional copy of the F9 gene, called FIX-Padua, to patients' cells. The FIX-Padua gene version was shown to result in FIX clotting activity eight times greater than that associated with the standard F9 gene. As such, the one-time therapy - administrated directly into the bloodstream - is expected to increase FIX levels, helping to prevent and control bleeds. If approved, the therapy will be marketed globally by CSL Behring, which closed a commercialization and licensing agreement with uniQure in May 2021. UniQure and CSL Behring expect to file a regulatory application to the U.S. Food and Drug Administration (FDA) in early 2022 seeking AMT-061's approval for hemophilia B. Research and Development. RGX-314: REGENXBIO RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina. REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye. According to the company's pipeline, the drug is currently in the Phase III stage of development. SPK-8011: Spark Therapeutics Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the US, while the European Commission has granted orphan designation to SPK-8011. NFS-01: Neurophth Investigational NR082, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is a novel ophthalmic injection that is being developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND4 mutations. It is currently being evaluated in Phase II/III clinical trial. GT 005: Gyroscope Therapeutics GT005 is designed as an AAV2-based one-time investigational gene therapy for GA secondary to AMD that is delivered under the retina. GT005 aims to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein. Complement overactivation has been strongly correlated with the development and progression of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production could dampen the system's overactivity and reduce inflammation, with the goal of preserving a person's eyesight. Gyroscope is also evaluating GT005 in two Phase II clinical trials. Further product details are provided in the report AAV vectors in gene therapy: Therapeutic Assessment This segment of the report provides insights about the different AAV vectors in gene therapy drugs segregated based on following parameters that define the scope of the report, such as: Major Players in AAV vectors in gene therapy There are approx. 70+ key companies which are developing the therapies for AAV vectors in gene therapy. The companies which have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e. Preregistration include, BioMarin Pharmaceutical. Phases The publisher's report covers around 235+ products under different phases of clinical development like Late stage products (Phase III) Mid-stage products (Phase II) Early-stage product (Phase I) along with the details of Pre-clinical and Discovery stage candidates Discontinued & Inactive candidates Route of Administration Key Players BioMarin Pharmaceutical Gensight Biologics PTC therapeutics Ultragenyx Pharmaceutical MeiraGTx Pfizer Biogen uniQure Pfizer Ultragenyx Pharmaceutical REGENXBIO Biogen Spark therapeuics (Roche) Sarepta therapeutics Neurophth Therapeutics LYSOGENE Gyroscope Therapeutics Nanoscope Therapeutics Homology medicines Ultragenyx Pharmaceutical Passage Bio Freeline therapeutics Astellas Pharma Aspa therapeutics Adrenas Therapeutics ESTEVE Sio Gene Therapies Amicus therapeutics 4D Molecular therapeutics Taysha Gene Therapies Atsena Therapeutics BioMarin Pharmaceutical Abeona Therapeutics REGENXBIO uniQure Taysha Gene Therapies Asklepios BioPharmaceutical Sarepta Therapeutics Abeona Therapeutics Forge Biologics Sangamo therapeuics Bayer LogicBio therapeutics Solid Biosciences Applied Genetic Technologies Spark therapeuics (Roche) Eli lilly and company Lexeo Therapeutics Spark therapeutics Gensight Biologics Alcyone Lifesciences MeiraGTx Audentes therapeutics Vivet Therapeutics Takeda Astellas Gene Therapies Homology Medicines Adverum Biotechnologies Libella Gene Therapeutics Rocket Pharmaceuticals Brain Neurotherapy Bio, Inc. Ocugen Asklepios BioPharmaceutical Neurogene Jaguar Gene Therapy, LLC Axovia Therapeutics Coave therapeutics Novartis/ Arctos medical Novartis/Vedere Bio REGENXBIO Inc. StrideBio Abeona therapeutics Sanofi/ Sirion therapeutics Capsida Biotherapeutics Kriya therapeutics Neurophth Poseida Therapeutics, BridgeBio Pharma DiNAQOR/ Biomarin ViGeneron AskBio/Selecta Biosciences Prevail therapeutics Intas Pharmaceuticals Tenaya Therapeutics Scout Bio therapeutics Ocugen Oyster Point pharmaceuticals Cyprium Therapeutics Solid Biosciences Amicus Therapeutics Coave therapeutics AvantiBio Voyager Therapeutics/ Pfizer Aruvant Sciences Alcyone Lifesciences Gene Therapy Research Institution Co., Ltd. For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/ylu69s .