FDA Grants Fast Track Designation to ABO-202 AAV9 Gene Therapy for Batten Disease


Abeona Therapeutics is currently planning a phase 1/2 clinical trial to evaluate ABO-202 in Batten disease.

Dr Joao Siffert, M.D

Joao Siffert, M.D., Chief Executive Officer, Abeona Therapeutics

João Siffert, MD

The FDA has granted fast track designation to Abeona Therapeutics’ ABO-202, a novel one-time AAV9 gene therapy for CLN1 disease, also known as infantile neuronal ceroid lipofuscinosis or infantile Batten disease.

In May, Abeona was cleared to begin a phase 1/2 clinical trial, evaluating its novel therapy for treatment of Batten disease, following acceptance by the FDA of its investigational new drug application. The company is expected to provide guidance as to the timing of the clinical trial later this year.

“Receiving fast track designation acknowledges the urgency for developing a therapy for children suffering from this rapidly-progressing and fatal disease and highlights the significant potential of ABO-202 to address this unmet need,” João Siffert, MD, chief executive officer, Abeona Therapeutics, said in a statement.1

Through an AAV9 gene therapy vector, ABO-202 is designed to deliver a functional copy of the Palmitoyl-Protein-Thioesterase-1 (PPT1) gene to the central nervous system and peripheral organs using a combination of intravenous and intrathecal administration.

In preclinical studies, the key findings reported that ABO-202 normalized survival and improved neurological function in mice. Additionally, the therapy showed that combination therapy utilizing both intravenous and intrathecal administrations routes improved efficacy over either delivery route alone, significantly increased survival, and improved behavior and reduced motor deficits. Early treatment was reported to significantly improve outcomes. Higher doses further improved these observations, which suggests that methods increasing total CNS exposure may be beneficial as it provided some survival and behavioral benefit to symptomatic mice.2

“ABO-202 is a promising AAV9 gene therapy that extended survival and improved neurological function in the animal model of CLN1 disease,” Steven J. Gray, PhD, Batten disease researcher and associate professor, pediatrics, UT Southwestern Medical Center, said in a statement.3 “Importantly, the combined intravenous and intrathecal administration approach showed additional benefits compared to a single route of delivery, providing a new treatment paradigm for patients with devastating neurological diseases.”

ABO-202 has previously been granted orphan drug and rare pediatric disease designations from the FDA.


1. Abeona Therapeutics Receives FDA Fast Track Designation for ABO-202 AAV9 Gene Therapy in CLN1 Disease [news release]. New York and Cleveland: Abeona Therapeutics; June 18, 2019. https://finance.yahoo.com/news/abeona-therapeutics-receives-fda-fast-122900142.html. Accessed June 18, 2019.

2. Abeona Therapeutics Receives Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease [news release]. New York and Cleveland: Abeona Therapeutics; April 20, 2018. https://www.globenewswire.com/news-release/2018/04/20/1483299/0/en/Abeona-Therapeutics-Receives-Orphan-Drug-Designation-in-the-European-Union-for-ABO-202-Gene-Therapy-Program-in-Batten-Disease.html. Accessed June 18, 2019.

3. Abeona Therapeutics Announces FDA Clearance of Investigational New Drug Application for ABO-202 Gene Therapy in CLN1 Disease [news release]. New York and Cleveland: Abeona Therapeutics; May 21, 2019. https://www.globenewswire.com/news-release/2019/05/21/1833363/0/en/Abeona-Therapeutics-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-ABO-202-Gene-Therapy-in-CLN1-Disease.html. Accessed June 18, 2019.

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