Drug Profile

ACDN 01

Alternative Names: ACDN-01

Latest Information Update: 14 Jan 2026

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At a glance

Originator Ascidian Therapeutics
Class Eye disorder therapies; Gene therapies; RNA
Mechanism of Action ATP binding cassette transporter subfamily A member 4 expression modulators; RNA splicing modulators

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

Phase I/II Cone rod dystrophies; Stargardt disease

Most Recent Events

07 Jan 2026 Ascidian Therapeutics and Forge Biologics agree to manufacture and co-develop ACDN 01 for Stargardt disease
07 Jan 2026 Ascidian Therapeutics and Forge Biologics plan a late-stage clinical trial for Stargardt disease (Subretinal)
11 Jun 2024 Phase-I/II clinical trials in Cone-rod-dystrophies in USA (Subretinal) (NCT06467344)

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