Companies in #Huntington's Disease
Found 2 companies operating in this sector.
uniQure N.V. is a gene therapy company developing a single administration of AAV-based therapies to treat serious genetic diseases, with a portfolio spanning hemophilia, Huntington's disease, ALS, Fabry disease, and refractory epilepsy. The company developed the first FDA-approved hemophilia B gene therapy (Hemgenix, partnered with CSL Behring) and is advancing AMT-130, a one-time gene therapy for Huntington's disease, as its lead wholly-owned program.
Wave Life Sciences is a clinical-stage RNA medicines company using its proprietary PRISM platform to design stereopure oligonucleotides that target genetic diseases, metabolic disorders, and neurological conditions. Lead programs include WVE-006 for Alpha-1 Antitrypsin Deficiency (AATD), WVE-003 for Huntington's disease, WVE-007 for obesity, and WVE-N531 for Duchenne muscular dystrophy, with a strategic collaboration with GSK to accelerate development of RNA-based therapeutics.