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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.
AVROBIO`s vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
Regional Clinical Research is a Endicott, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Atsena Therapeutics is a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness.
Founded by Longwood Fund, Pyxis Oncology is building a differentiated portfolio of biologics, including antibody-drug conjugates (ADCs) and immunotherapies, to improve the lives of patients with difficult-to-treat cancers. Pyxis is employing site-specific conjugation technology to develop highly stable ADCs with enhanced therapeutic indexes. Pyxis is also advancing a diverse portfolio of immunotherapies that target broad immune regulators as well as novel immune checkpoints identified through its immuno-oncology and cold tumor discovery efforts.