Rocket Pharmaceuticals is a Cranbury, New Jersey biotechnology company developing and manufacturing genetic therapies intended to address the root causes of rare diseases. Its integrated research and manufacturing operation includes roughly 30,000 square feet of laboratories and a 103,720-square-foot facility built in 2022, with programs spanning inherited cardiovascular and blood disorders. Rocket’s first FDA-approved gene therapy, KRESLADI, treats severe leukocyte adhesion deficiency-I.
Krystal Biotech, IncPittsburgh-based Krystal Biotech develops and commercializes redosable genetic medicines for rare diseases, using an engineered HSV-1 vector platform designed for focal, minimally invasive gene delivery. Its first commercial product, VYJUVEK®, is the first and only redosable gene therapy for dystrophic epidermolysis bullosa, while two U.S. GMP facilities—including a roughly 155,000-square-foot ASTRA site—support manufacturing and pipeline development. Krystal is fully integrated across vector design, gene therapy manufacturing and commercialization.325412PITTSBURGH, PA
Leadiant Biosciences, IncFrom its Rockville base, Leadiant Biosciences develops and distributes prescription therapies for people living with rare diseases. The private U.S. pharmaceutical company draws on a longstanding heritage in orphan-drug development, becoming the fourth company in the world to receive a U.S. Orphan Drug Designation in 1984. Its research teams work with physicians, researchers, academic institutions and biotechnology organizations, while patient-support programs provide education and resources beyond the medicine itself.ROCKVILLE, MD
Abeona Therapeutics IncAbeona Therapeutics is a commercial-stage biopharmaceutical company developing and delivering cell and gene therapies for serious diseases, with its Cleveland, Ohio facility serving as the commercial manufacturing site for ZEVASKYN®, approved in April 2025. The company combines ex vivo gene therapy and viral-vector expertise with a developing PSMA-targeted synthetic immune receptor T-cell therapy for advanced prostate cancer. Its fully integrated manufacturing model keeps critical production steps in-house, supporting supply and consistency for patients with rare disease.CLEVELAND, OH
CSL BehringCSL Behring develops and manufactures biotherapies for people living with rare and serious diseases, with a particular focus on bleeding disorders, immune deficiencies and hereditary angioedema. Its work combines plasma-derived and recombinant therapies with genetic medicine, and the company operates CSL Plasma as a vertically integrated collection network whose donated plasma is used to make therapies distributed in more than 100 countries. CSL Behring is part of CSL, a biotechnology company headquartered in Melbourne that employs more than 32,000 people.KING OF PRUSSIA, PA