Drug Profile

CAP 003

Alternative Names: CAP-003

Latest Information Update: 30 Sep 2025

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At a glance

Originator Capsida Biotherapeutics
Class Antiparkinsonians; Gene therapies
Mechanism of Action GBA protein replacements; Gene transference

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

Highest Development Phases

Suspended Parkinson's disease

Most Recent Events

30 Sep 2025 Suspended - Phase-I/II for Parkinson's disease in USA (IV) (NCT07011771)
18 Aug 2025 Phase-I/II clinical trials in Parkinson's disease in USA (IV) (NCT07011771)
12 Jun 2025 The US FDA clear IND application for the initiation of a phase I/II trial for Parkinson’s disease

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