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Vor Biopharma is pioneering engineered hematopoietic stem cells (eHSCs) to dramatically change the treatment paradigm for hematological cancers. Vor`s eHSCs are designed to generate healthy, fully functional cells with specific advantageous modifications, for example to protect the blood and bone marrow from the toxic effects of antigen-targeted therapies, protecting healthy cells from depletion but leaving tumor cells vulnerable. Vor`s platform could potentially be used to change the treatment paradigm of both hematopoietic stem cell transplants and antigen-targeted therapies such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vor`s lead program has been published in Proceedings of the National Academy of Sciences. Vor is based in Cambridge, Mass. and has a broad intellectual property base including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.
Mateon Therapeutics/Oncotelic is developing TGF-beta therapies and Apomorphine for Parkinson, ED, FSD. https://t.co/nReUVgPvhc. $OTLC
REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO`s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO`s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO`s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.
CPS Instruments is a Stuart, FL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Freeline is a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases. We are headquartered in the UK and we also have operations in Germany and the US. Freeline was founded in 2015 and builds upon the pioneering work by our Clinical and Scientific Advisor and Director, Professor Amit Nathwani, Professor of Haematology at UCL. With our liver-based investigational gene therapy technology, we aim to improve the lives of people with chronic systemic diseases and realise the potential of gene therapy treatments.