Abeona Partners With Taysha to Advance ABO-202 as Potential Gene Therapy

Abeona Therapeutics and Taysha Gene Therapies have entered into agreements concerning ABO-202, an investigative gene therapy for infantile Batten disease.

The agreements give Taysha worldwide exclusive 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 expertise on researching, developing, and manufacturing ABO-202.

A goal is to advance the gene therapy into a Phase 1/2 clinical trial in patients.

“We are excited to partner with Taysha in their further development of a potential treatment for children living with Batten disease,” João Siffert, MD, CEO of Abeona, said in a press release.

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

The intent of gene therapy for infantile Batten disease is to deliver a non-mutated 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 by using a viral vector, specifically the adeno-associated virus (AAV). AAV is commonly used in gene therapy, because the virus is able to get genetic material into cells, but is modified to not cause disease.

Research in animal models of infantile Batten disease showed that ABO-202 increased survival, normalized behavior, and eased motor defects.

The investigational therapy was originally developed in the lab of Steven Gray, PhD, an associate professor at UT Southwestern Medical Center (formerly with UNC) and Taysha’s chief scientific advisor. The therapy has since been developed by Abeona.

ABO-202 has been designated an orphan drug by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). It has also been granted rare pediatric disease and fast track designations by the FDA, and the FDA has given clearance for the start of clinical trials.

Under the agreement’s terms, Taysha will give Abeona an initial payment totaling $7 million, with the possibility of future payments if development milestones are met.

“We believe the ABO-202 data generated thus far demonstrate great translational potential and offer hope to children suffering from this devastating disorder,” said RA Session II, president, CEO, and founder of Taysha. “We are excited to continue working closely with Dr. Gray to rapidly advance this promising gene therapy into the clinic.”