CAP 001
Alternative Names: CAP-001Latest Information Update: 01 Jun 2022
At a glance
- Originator Capsida Biotherapeutics
- Class Gene therapies
- Mechanism of Action Gene transference; Iduronate sulfatase replacements
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Preclinical Mucopolysaccharidosis II
Most Recent Events
- 18 May 2022 Capsida Biotherapeutics has patent protection for adeno-associated virus compositions for targeted gene therapy in the US
- 18 May 2022 Preclinical trials in Mucopolysaccharidosis II in USA (IV)
- 18 May 2022 Pharmacokinetics and pharmacodynamics data from a preclinical trial in Mucopolysaccharidosis II released by Capsida Biotherapeutics