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Allena Pharmaceuticals is dedicated to developing and commercializing first-in-class, oral enzyme therapeutics to treat patients with rare and severe metabolic disorders that affect the kidney. The company is focused on metabolic disorders that result in excess accumulation of certain metabolites, such as oxalate and urate, that can cause kidney stones, damage the kidney, and potentially lead to chronic kidney disease, or CKD, and end-stage renal disease. Allena`s proprietary technological approach enables the design, formulation and delivery of non-absorbed and stable enzymes orally and in sufficient doses for activity in the GI tract. This approach utilizes the GI tract to degrade metabolites, such as oxalate and urate, reducing plasma and urine levels, and in turn, reducing their disease burden on the kidney over time.
LGC Group, formerly the Laboratory of the Government Chemist, is an international life sciences measurement and testing company, which also provides the role and duties of the UK Government Chemist, a statutory role and adviser to the government.
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.
Enzyvant is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 in the first half of 2018. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.
Most patients who are prescribed blockbuster therapies today don`t respond to the treatment, costing the health care industry billions in wasted drugs while patients continue to suffer. Scipher`s platform identifies which drug will work based on the patient`s fundamental disease biology, and not based on symptoms, disease classification, or medical bias. A simple test predicts which drug he/she will respond to, ensuring that most optimal treatment is prescribed from day one. The molecular data generated by our tests is then used to fuel novel target discovery to address a clear unmet medical need in patients who do not respond to any existing therapy.