Drug Profile

Research programme: in vivo gene editing therapies - Capsida Biotherapeutics/CRISPR Therapeutics

Latest Information Update: 28 Jul 2025

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

28 Jul 2025 No recent reports of development identified for research development in Amyotrophic-lateral-sclerosis in USA (Parenteral)
28 Jul 2025 No recent reports of development identified for research development in Friedreich's ataxia in USA (Parenteral)
24 Mar 2022 Capsida Biotherapeutics has patent protection for Adeno-associated virus compositions for targeted gene therapy in the US

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