CSL 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.
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
SERB PharmaceuticalsSERB Pharmaceuticals develops, manufactures, and supplies specialist medicines for rare diseases and medical emergencies, with a particular strength in antidotes and other critical-care treatments. Beginning in the 1950s as Société d’Études et de Recherches Biologiques, the company now supplies more than 70 medicines through direct operations and distribution partners in over 100 countries. Its fully integrated platform includes a commercial-scale polyclonal antibody manufacturing capability and four manufacturing facilities across three continents.325412CONSHOHOCKEN, 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
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