NTLA-4001
Glycogen Storage Disease Type Ia (GSDIa)
Research/PreclinicalActive
Key Facts
Indication
Glycogen Storage Disease Type Ia (GSDIa)
Phase
Research/Preclinical
Status
Active
Company
About Intellia Therapeutics
Intellia Therapeutics is a pioneer in the development of CRISPR/Cas9-based therapeutics, aiming to cure severe genetic and other diseases through precise genome editing. The company has achieved significant milestones, including the first-ever clinical data demonstrating in vivo CRISPR genome editing in humans. Its strategy leverages a dual-platform approach, developing both in vivo (systemically administered) and ex vivo (cell-based) therapies, with key partnerships including Regeneron and Novartis to advance its pipeline.
View full company profileTherapeutic Areas
Other Glycogen Storage Disease Type Ia (GSDIa) Drugs
| Drug | Company | Phase |
|---|---|---|
| DTX401 | Ultragenyx Pharmaceutical | Phase 3 |