Drug Profile

CLN 301

Alternative Names: CLN-301

Latest Information Update: 04 Jul 2025

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At a glance

Originator Alcyone Therapeutics
Class Gene therapies
Mechanism of Action CLN3 protein expression stimulants; 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

Phase I/II Neuronal ceroid lipofuscinosis

Most Recent Events

24 Apr 2025 Phase-I/II clinical trials in Neuronal ceroid lipofuscinosis (Parenteral), prior to April 2025
24 Apr 2025 Efficacy and adverse event data from a phase I/II trial in Neuronal ceroid lipofuscinosis released by Alcyone Therapeutics

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