Companies in #Hemophilia A
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.
Regenerative medicine company developing the Cell Pouch Bio-Hybrid Organ platform — an implantable, retrievable, pre-vascularized cell containment system for type 1 diabetes, hypothyroidism, and hemophilia A.