Ranedis Pharmaceuticals is a private biopharmaceutical company focused on the treatment and management of rare and neglected diseases, with initial efforts focused on lysosomal storage diseases.
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
Calliditas Therapeutics ABFounded in Sweden in 2004 as Pharmalink AB, Calliditas Therapeutics develops and commercializes medicines for rare diseases, with an initial focus on kidney and liver disorders. The company pioneered the first treatment specifically designed for IgA nephropathy and markets TARPEYO in the United States, while partners commercialize related therapies in other regions.New York, NY
BioMendics, LLCBased inside the REDIzone incubator on the Northeast Ohio Medical University campus, BioMendics develops investigational medicines for rare dermatologic disorders, led by a small-molecule liquid-crystal platform aimed at repairing fragile, blistering skin. Founded by scientists, physicians, and angel investors, the company is currently advancing its topical TolaSure gel through a Phase II study for epidermolysis bullosa simplex. CEO and founder Karen M. McGuire, PhD, brings more than 15 years of drug-development experience to the research program.ROOTSTOWN, OH
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