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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.
GrayBug® is a platform drug delivery company that is developing proprietary controlled release technologies for ophthalmic pharmaceutical indications. The company`s lead product is a polymer-drug biomolecular conjugate for the treatment of neovascular diseases, including age-related macular degeneration (AMD). GrayBug also has a proprietary platform technology that allows sustained drug delivery into various compartments of the eye while minimizing inflammation common with current controlled release technologies applied to the eye.
Arcellx, Inc. is a clinical-stage biotechnology company reimagining cell therapy by engineering innovative immunotherapies for patients with cancer and other incurable diseases. Arcellx believes that cell therapies are one of the forward pillars of medicine and Arcellx`s mission is to advance humanity by developing cell therapies that are safer, more effective, and more broadly accessible. Arcellx`s lead product candidate, CART-ddBCMA, is being developed for the treatment of relapsed or refractory multiple myeloma (r/r MM) in an ongoing Phase 1 study. CART-ddBCMA has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations by the U.S. Food and Drug Administration. Arcellx is also advancing its dosable and controllable CAR-T therapy, ARC-SparX, through two programs: a Phase 1 study of ACLX-001 for r/r MM, initiated in the second quarter of 2022; and ACLX-002 in relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndrome, expected to enter the clinic in the second half of 2022.
PreciThera, Inc. is a biotechnology company committed to the development of therapies for rare bone diseases using the combined application of computational technology and a deep understanding of disease pathology.
Bayer and CRISPR Therapeutics have entered into an agreement to create Casebia to discover, develop and commercialize new breakthrough therapeutics to cure blood disorders, blindness, and congenital heart disease. CRISPR Therapeutics will contribute its proprietary CRISPR-Cas9 gene-editing technology and intellectual property, while Bayer will make available its protein engineering expertise and relevant disease know-how. It is the first long-term strategic partnership of its kind to make a substantial investment in the development of target delivery systems in an effort to bring systemic in vivo CRISPR-Cas9 gene editing technology applications to patients.