Rare Disease Report

Gene Therapy for Juvenile Batten Disease Gets Orphan Drug Designation

JUNE 29, 2017
James Radke
The FDA has granted Abeona Therapeutics Inc. Orphan Drug Designation (ODD) for ABO-201 (AAV-CLN3), its gene therapy program for juvenile Batten disease, (juvenile neuronal ceroid lipofuscinosis, JNCL).
The AAV-based gene therapy is being designed as a single intravenous infusion to treat children with the currently fatal rare disease. Simply put, the gene therapy is being developed not to treat these children, but to cure them.
This is the 4th gene therapy the company has in development that has obtained an Orphan Drug Designation. The other 3 are being developed to treat recessive dystrophic epidermolysis bullosa and both sanfilippo syndrome type A and type B. The company also has a variety of other gene therapies in development for other rare diseases.

There are several forms of Batten disease based on which gene mutation involved. Juvenile Batten disease is due to mutations on the CLN3 gene, and is the target patient population for ABO-201.
These children develop normally for the first few years of life, but between the ages of 4 and 7 years, the first signs and symptoms begin to appear. The first noticeable sign is typically gradual vision impairment. The children may also begin to show signs of intellectual impairment and by the age of 10, seizures may start and intensify over time.

As patients become teenagers, speech and motor control may become compromised and behavioral problems may also develop. There is a large variance in symptoms but with no treatment currently available, the buildup of fat in the cells of this lysosomal storage disorder will eventually take a patient’s life. Death commonly occurs between the ages of 15 and 35 years.

Per Abeona; “Receiving FDA orphan drug designation for ABO-201 augments Abeona’s suite of regulatory achievements, providing certain benefits and incentives, including marketing exclusivity, that are strategically important from a regulatory and commercial perspective,” stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. “The published ABO-201 preclinical data from Dr. Tammy Kielian’s lab support the clinical translation for patients with juvenile Batten disease, and demonstrated the importance of selecting the right vector and delivery route for potential CNS benefit and to remove the underlying pathology associated with the disease.  This designation helps advance the ABO-201 program and we look forward to initiating human clinical trials later this year.

In animal studies, a single injection of ABO-201 improved motor function, glial activation, and lysosomal pathology in a mouse model for Juvenile Batten disease. 

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