Companies in #Alpha-1 Antitrypsin Deficiency
Found 2 companies operating in this sector.
Intellia Therapeutics is a clinical-stage biotechnology company developing in vivo and ex vivo CRISPR/Cas9-based gene-editing therapies. Its lead program NTLA-2001 is being studied for transthyretin (ATTR) amyloidosis, with additional programs in hereditary angioedema, hemophilia A/B, and other genetic diseases. Intellia has also invested heavily in commercial-launch infrastructure, including a dedicated team working on cell-and-gene therapy readiness in advance of its first potential approvals.
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.