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CLN-301

Development stage: Preclinical
Lead developer: Alcyone Therapeutics
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Intrathecal

Overview

CLN-301 is an adeno-associated viral (AAV) vector gene therapy under development for the treatment of Juvenile Batten disease (also known as Juvenile Neuronal Ceroid Lipofuscinosis or CLN3 disease). The therapy delivers a functional CLN3 coding sequence via intrathecal injection, aiming to restore the function of the defective battenin protein in affected patients. It is being developed using Falcon, a precision intra-cerebrospinal fluid drug and delivery platform technology. The primary developer is Alcyone Therapeutics.

Other names: CLN3 gene therapyCLN-3 gene therapyCLN 3 gene therapy

Targets

CLN3 (Battenin)

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