Abeona 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.
Forge BiologicsFounded by gene therapy developers for developers, Forge Biologics is a Columbus, Ohio-based contract development and manufacturing organization focused on AAV gene therapy development and production. Its 200,000-plus-square-foot Hearth facility has 20 cGMP suites and supports programs from research through clinical and commercial manufacturing. Forge is part of Ajinomoto Bio-Pharma Services and brings more than 450 experts to its work with gene therapy developers.GROVE CITY, OH
Andelyn BiosciencesBorn from decades of gene-therapy research at Nationwide Children’s Hospital, Columbus-based Andelyn Biosciences is a contract development and manufacturing organization that develops and produces viral vectors and plasmids for cell and gene therapies. Spun out as an independent company in January 2020, Andelyn carries a patient-centered origin story into work spanning early development through clinical and commercial manufacturing. Its two Ohio facilities support a record that includes more than 500 cGMP clinical batches and the production experience behind an FDA-approved systemic gene therapy.COLUMBUS, OH
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
Rocket Pharmaceuticals, IncRocket 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.NEW YORK, NY