Companies in #Fabry Disease
Found 2 companies operating in this sector.
Sangamo Therapeutics (NASDAQ: SGMO) is a genomic medicine company headquartered in Richmond, California, focused on translating ground-breaking science into genomic medicines using zinc finger nucleases (ZFNs), zinc finger protein transcription factors (ZFP-TFs), and adeno-associated virus (AAV) delivery. Sangamo's clinical-stage pipeline includes giroctocogene fitelparvovec (hemophilia A gene therapy, partnered with Pfizer) and isavufermin or SAR445136 (alpha-1 antitrypsin deficiency, partnered with Sanofi). The company is also developing preclinical in vivo genome-editing programs and ex vivo gene-edited cell therapies.
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.