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Therapeutic Abeona and Taysha’s gene therapies have entered into agreements for ABO-202, an experimental gene therapy for childhood Batten disease.

The agreements give Taysha exclusive worldwide rights to intellectual property developed by researchers at the University of North Carolina at Chapel Hill (UNC) and Abeona, as well as access to Abeona’s expertise in the research, development and manufacture of ABO-202.

One goal is to advance gene therapy in a phase 1/2 clinical trial in patients.

“We are delighted to partner with Taysha in the development of a potential treatment for children with Batten disease,” said João Siffert, MD, CEO of Abeona, in a Press release.

Infantile Batten Disease – also called CLN1 disease, or infantile neuronal ceroid lipofuscinosis – is caused by mutations in the gene PPT1. This alters the cellular processes used to break down certain waste molecules, causing these molecules to build up in cells – especially nerve cells – which is toxic to cells.

The goal of gene therapy for childhood Batten disease is to provide an unmutated version of the PPT1 gene to a patient’s cells, effectively correcting the underlying genetic cause of the disease.

ABO-202 aims to accomplish this using a viral vector, specifically the adeno-associated virus (AAV). AAV is commonly used in gene therapy because the virus is able to introduce genetic material into cells, but is modified so as not to cause disease.

Research in animal models of childhood Batten disease have shown that ABO-202 increases survival, normalizes behavior and relieves motor defects.

Experimental therapy was originally developed in the lab of Steven Gray, PhD, associate professor at UT Southwestern Medical Center (formerly at UNC) and Chief Science Advisor to Taysha. The therapy has since been developed by Abeona.

ABO-202 has been designated an orphan drug by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA). It has also received Rare Pediatric Disease and Fast Track designations by the FDA, and the FDA has given clearance for clinical trials to begin.

Under the terms of the deal, Taysha will make an upfront payment to Abeona totaling $ 7 million, with the possibility of future payments if development milestones are met.

“We believe the ABO-202 data generated so far demonstrates great translational potential and offers hope for children suffering from this devastating disorder,” said RA Session II, President, CEO and Founder of Taysha. “We are delighted to continue to work closely with Dr. Gray to rapidly advance this promising gene therapy in the clinic. “

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