Allogene, Notch Partner to Develop iPSC-Derived Blood Cancer Therapies

GEN | November 05, 2019

Allogene Therapeutics will partner with Notch Therapeutics to research and develop induced pluripotent stem cell (iPSC) AlloCAR™ therapies for initial blood cancer indications that include non-Hodgkin lymphoma, leukemia and multiple myeloma, the companies said today, through an exclusive worldwide collaboration and license agreement that could generate more than $300 million for Notch. Under their partnership, Allogene and Notch plan to create allogeneic cell therapy candidates from T cells or natural killer (NK) cells using Notch’s Engineered Thymic Niche (ETN) platform, created to offer potential flexibility and scalability for the production of stem cell-derived immune cell therapies. ETN is designed to enable the expansion of induced pluripotent stem cells (iPSCs) and differentiation of iPSCs from mature immune cells in scalable, fully defined, feeder-free and serum-free cultures into T cells that can be genetically tailored for any T cell-based immunotherapeutic application. The platform uses genetically tailored stem cells as a renewable source for creating allogeneic T cell therapies that are designed, according to Notch, to deliver safer, consistently manufactured and more cost-effective cell immunotherapies to patients.

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CELL AND GENE THERAPY, INDUSTRIAL IMPACT

Biocytogen Launches RenNano™ Mouse, a Fully Human Heavy Chain Antibody Platform

Biocytogen | January 05, 2023

RenNano™, a fully human heavy-chain antibody platform developed by Biocytogen Pharmaceuticals Co., Ltd., has been launched officially. RenNano™ joins RenMab™ and RenLite® as the newest member of the RenMice™ family. All three RenMice™ technologies from Biocytogen work together to speed up the search and development of entirely human monoclonal, bispecific/multispecific, and single-domain antibodies (sdAbs, or nanobodies). Camels and sharks produce heavy-chain-only antibodies (HCAb), indicating their variable domains (VHH or sdAb) are functional without pairing with the light chain. Still, humans and mice produce antibodies that require heavy and light chain coupling to be effective. Because of their molecular weight and size, sdAbs can enter solid tumor tissues and breach the blood-brain barrier. Additionally, the sdAb's larger CDR3 region allows for detecting the otherwise concealed epitopes of GPCRs and other challenging targets. Nanobodies offer their benefits, but it's challenging to use animals that naturally make HCAbs, like camels, in large numbers to create monoclonal antibodies due to their size, slow reproduction rate, and limited offspring supply. However, humanizing camelid antibody sequences for pharmaceutical use adds another layer of complexity and time to the drug development process. Accordingly, Biocytogen modified the constant area of the entirely human RenMab™ model to create the RenNano™ Mouse, which is genetically modified to manufacture HCAbs. RenNano™ Mouse is one of the world's most comprehensive human antibody platforms due to the substitution of the mouse genes with the complete human heavy chain variable genes. About Biocytogen Biocytogen, a global biotechnology company, is at the forefront of developing cutting-edge methods for studying and creating new antibody-based pharmaceuticals. Biocytogen has streamlined the entire drug development process by integrating its drug efficacy screening platforms and clinical solid development expertise with its proprietary RenMab™, RenLite®, and RenNano™ platforms for the fully human monoclonal antibody, bispecific/multispecific antibody, and nanobody develo

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CELL AND GENE THERAPY, INDUSTRIAL IMPACT

GenScript ProBio Enters into Strategic Partnership with RVAC Medicines

GenScript ProBio | February 24, 2023

GenScript ProBio, a renowned Contract Development and Manufacturing Organization (CDMO), has recently entered a strategic partnership agreement with RVAC Medicines Pte. Ltd., a biotech company specializing in the development and commercialization of mRNA vaccines and therapeutics. The collaboration is aimed at manufacturing GMP-grade plasmid DNA (pDNA) for the RVM-V001 mRNA COVID-19 vaccine candidate, as well as supporting future therapeutic pipelines. GenScript ProBio will offer GMP plasmid manufacturing service for RVAC Medicines' program, RVM-V001. By collaborating with GenScript ProBio, RVAC Medicines will be able to accelerate the clinical manufacturing of mRNA-based COVID-19 vaccines, as well as future mRNA-based vaccines designed to target other infectious diseases such as Clostriodioides difficile infection (CDI) and Respiratory syncytial virus (RSV). As RVAC proceeds with the development of its mRNA candidates, GenScript ProBio continues to offer high-quality GMP materials crucial to the objective, including plasmid DNA and the development of ancillary materials to support mRNA manufacturing processes. GenScript ProBio also provides consultative and expert guidance regarding regulatory and quality expectations of agencies worldwide. RVAC Medicines and GenScript ProBio will continue collaborating to support mRNA initiatives for both clinical and commercial stage development. Dr. Brian Min, CEO of GenScript ProBio said, "We are delighted to enter into this strategic partnership with RVAC Medicines. This collaboration will enable us to utilize our cutting-edge GMP plasmid manufacturing platform technology to support the development of mRNA-based COVID-19 vaccine and its therapeutic candidates." (Source – PR Newswire) About GenScript ProBio GenScript ProBio is a Contract Development and Manufacturing Organization (CDMO) that provides end-to-end drug discovery and commercialization services. The company's services are designed to accelerate drug development for its clients in various areas, including CGT, vaccines, biologics discovery, and antibody protein drugs. GenScript ProBio has a global presence, with subsidiaries in the Netherlands, the United States, South Korea, and China. Since October 2017, the company has helped clients in different regions, such as the United States, Europe, and Asia Pacific, to obtain over 40 IND approvals. Its goal is to drive innovation and speed up the biologics development process from target to market.

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CELL AND GENE THERAPY, INDUSTRIAL IMPACT

Rocket Pharmaceuticals' RP-A501 Receives FDA RMAT Designation

Rocket Pharmaceuticals, Inc | February 09, 2023

On February 7, 2023, Rocket Pharmaceuticals Inc., a renowned late-stage biotech firm advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet needs, announced that FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501, the company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease, a life-threatening genetic cardiac disease with no disease-altering therapies available. RMAT status was granted based on favorable safety and effectiveness findings from the Phase 1 RP-A501 clinical trial and will provide further added benefits of intensive FDA guidance and accelerated evaluation throughout the program's development. RMAT designation, the same as Breakthrough Therapy designation, offers the advantages of intensive FDA guidance on efficient drug development, such as the ability for early FDA interactions to discuss intermediate or surrogate endpoints, potential priority review of the Biologics License Application (BLA), possible ways to support accelerated approval and satisfy post-approval requirements, and other possibilities to expedite development and assessment. Rocket's RP-A501 program also has Orphan Drug (US) and Rare Pediatric designations. The Phase 2 pivotal trial is scheduled to begin in the second quarter of 2023. As disclosed previously, the company plans to conduct a single-arm, open-label trial with a biomarker-based composite endpoint and a natural history comparator. The Phase 1 program's findings provide one of the most extensive investigational gene therapy datasets for any cardiac disease. In patients with Danon Disease treated in the Phase 1 study, RP-A501 was generally well tolerated, with indications of restored expression of the deficient LAMP2 protein and durable improvement or stability of clinical parameters. Data show consistent and robust improvements in a variety of parameters, including protein expression, decreased autophagic vacuoles, troponin, brain natriuretic peptide (BNP), left ventricular (LV) mass and thickness, and enhanced NYHA class and Kansas City Cardiomyopathy Questionnaire (KCCQ/Quality of Life) measurements. Notably, these BNP and NYHA class improvements and stability contrast sharply with BNP rise and NYHA class worsening found in a representative sample of pediatric and adolescent natural history patients. Overall, the results indicate improvements and normalization in a variety of quantifiable parameters used by cardiologists in clinical practice to foster risk assessment and treatment decisions. About Rocket Pharmaceuticals, Inc Founded in 2015, Rocket Pharmaceuticals is a fully integrated clinical-stage firm developing an integrated and sustainable pipeline of investigational genetic therapeutics that have the potential to address the underlying causes of complex rare childhood diseases. Through its platform-agnostic approach, it can build the optimal therapy for each indication, possibly generating revolutionary options for patients suffering from rare genetic disorders. The company has a team of highly productive experts and is increasingly being recognized as a leading biotechnology innovator.

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