Companies in #Hemophilia A and B
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.
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.