Companies in #Neurology
Found 2 companies operating in this sector.
Ionis Pharmaceuticals (NASDAQ: IONS) is a Carlsbad, California-based antisense oligonucleotide (ASO) drug discovery and development company with a deep pipeline spanning neurology, cardiometabolic disease, renal disease, and rare disorders.
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.