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ABO-201

Development stage: Preclinical
Lead developer: Abeona Therapeutics
Modality: Gene Therapies
Administration: Intravenous, Intrathecal

Overview

ABO-201 is an adeno-associated virus (AAV) gene therapy designed for the treatment of CLN3 disease, also known as juvenile Batten disease. It utilizes a self-complementary AAV9 (scAAV9) vector to deliver a functional copy of the CLN3 gene, aiming to address the underlying genetic cause of this rare lysosomal storage disorder. The program originated from research at the University of Nebraska Medical Center and the Ohio State Innovation Foundation. ABO-201 has completed IND-enabling studies in preclinical models, demonstrating proof-of-concept for restoring CLN3 function and positioning the program for clinical development.

Other names: scAAV9 CLN3scAAV-9 CLN3scAAV 9 CLN3

Targets

CLN3 (Battenin)

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