Pittsburgh-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.
Krystal Biotech’s Comprehensive Compliance Program
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Ethics And Code Of Conduct
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California Annual Declaration of Compliance
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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
PeriphaGenPeriphaGen is a Pittsburgh biopharmaceutical company developing replication-defective HSV-1 gene-therapy vectors, with a particular focus on delivering treatments to peripheral nerves and difficult-to-treat neurological disorders. The company has worked with HSV-1 vectors for more than 25 years and was the first to conduct human clinical trials using replication-defective HSV-1 vectors. Its NET platform uses the virus’s natural transport along sensory neurons to deliver high-capacity genetic payloads, while PeriphaGen also provides vector design and production technologies for research and clinical development.PITTSBURGH, PA
Vector BioMedSpun out of the nonprofit Caring Cross, Vector BioMed develops and manufactures lentiviral vectors for gene and cell therapy programs. Its LENTIVERSE™ platform combines custom vector engineering with ready-to-deploy solutions for CAR-T manufacturing, using a platform-based approach intended to shorten the path from concept to clinic. The Gaithersburg public benefit corporation pairs decades of lentiviral experience with a focus on transparent economics and expanding access to advanced therapies worldwide.GAITHERSBURG, MD
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