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Juneau Biosciences is a Salt Lake City, UT-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
AveXis, now a Novartis Company, is a clinical-stage gene therapy company relentlessly focused on bringing gene therapy out of the lab and into the clinical setting for patients and families devastated by rare and life-threatening neurological genetic d...
OPK Biotech is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.
Cyagra Inc is a Elizabethtown, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.