Companies in #Alpha-1 Antitrypsin Deficiency
Found 1 company 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.