Nearly four months after Intellia Therapeutics – along with partner Regeneron Pharmaceuticals – reinvigorated the CRISPR space with the first-ever clinical evidence that CRISPR gene editing inside the body can be safe and effective, the asset in question was granted Orphan Drug Designation by the U.S. Food and Drug Administration.
Three Small CRISPR Biotechs That Could Cure 10,000 Diseases
Blood Diseases, Cancer, Collaborations, CRISPR/Cas9, Diseases, DNA, Eye Diseases, FDA, Gene Editing, Genetic Disorders, Genomics, Hematopoietic Stem Cells, Herpes Simplex Virus, Hunter Syndrome, Immune Cells, Inflammatory Disorders, J.P. Morgan Healthcare Conference, Liver Disease, Lung Disease, Muscle Disease, R&D, Sickle Cell Disease, Thalassemia, Transthyretin (TTR) Amyloidosis, Viral Infections, World Health OrganizationInvestor’s Business Daily looked at three small biotech companies that appear to be leading the CRISPR race.
Palo Alto, Calif.-based BridgeBio Pharma is launching a new spinout to develop a recently abandoned Novartis cancer treatment now in its possession.