Mirum Pharmaceuticals is developing a compound, maralixibat, to treat Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), genetic liver disorders that affect primarily children.
CuraSen Therapeutics is focused on the discovery and development of therapies to treat neurodegenerative diseases, including Parkinson’s Disease, Alzheimer’s Disease and less common neurodegenerative disorders.
4D Molecular Therapeutics is a platform company focused on the discovery and development of novel adeno-associated virus (AAV) vectors for use in gene therapy products for the treatment of monogenetic disorders
VelosBio, Inc. is an oncology focused biotechnology company which is developing antibody drug conjugates to target solid tumors and hematological cancers.
Aura Biosciences is an innovation-driven company focused on the development of drugs using tumor targeted Viral-like Nanoparticles.
Sentien Biotechnologies, Inc. is a late preclinical stage company developing novel approaches to cell therapy.
Kezar Life Sciences is a clinical-stage biopharmaceutical company focused on the discovery and development of novel small molecule therapeutics that target protein homeostasis.
Glycomine is developing therapies for congenital disorders of glycosylation by combining replacement therapies with intracellular delivery vehicles.
MINORYX specializes in developing innovative treatments for Inborn Errors of Metabolism, a group of rare diseases of genetic origin with high unmet medical need.
Reneo Pharmaceuticals is a San Diego-based, private biotechnology company focused on the identification and development of novel treatments for orphan diseases.