NTLA-2003
Glycogen Storage Disease Type Ia (GSDIa)
PreclinicalActive
Key Facts
Indication
Glycogen Storage Disease Type Ia (GSDIa)
Phase
Preclinical
Status
Active
Company
About Intellia Therapeutics
Intellia Therapeutics is a leader in translating Nobel Prize-winning CRISPR/Cas9 technology into transformative genomic medicines. The company's strategy leverages a dual-platform approach—in vivo and ex vivo editing—to advance a pipeline targeting rare genetic diseases like hereditary angioedema (HAE) and ATTR amyloidosis, with key programs in late-stage development. Backed by a strong intellectual property foundation and a strategic partnership with Regeneron, Intellia is building toward becoming a fully integrated, commercial-stage biopharmaceutical company.
View full company profileTherapeutic Areas
Other Glycogen Storage Disease Type Ia (GSDIa) Drugs
| Drug | Company | Phase |
|---|---|---|
| BEAM-301 | Beam Therapeutics | Preclinical |
| DTX401 | Ultragenyx Pharmaceutical | Phase 3 |