Atsena Therapeutics gains FDA incentive for gene therapy candidate

Durham-based Atsena Therapeutics, a gene therapy company focused on reversing or preventing blindness, has received an incentive from the U.S. Food and Drug Administration (FDA) for its leading product candidate.

The FDA granted Rare Pediatric Disease designation for ATSN-201, Atsena’s gene therapy product candidate for the treatment of X-linked retinoschisis (XLRS), a rare genetic disease that causes blindness.

The designation is for therapeutics intended to treat serious or life-threatening rare diseases that primarily affect patients under the age of 18. With the designation, Atsena will be eligible upon FDA approval of ATSN-201 to receive a priority review voucher (PRV) that could be used to advance another internal program or be sold to an outside company. The PRV program incentivizes companies like Atsena to invest in rare pediatric diseases with limited markets.
The Rare Pediatric Disease designation is Atsena’s second such designation received this year.

“Having both of our clinical-stage, ocular gene therapies receive this designation underscores the potential of our technology to address significant unmet needs for patients with inherited retinal diseases,” said Patrick Ritschel, chief executive officer of Atsena. “We are committed to advancing ATSN-201 in clinical trials and offering hope to patients and families affected by XLRS.”

Clinical trials underway

XLRS is caused by mutations in a gene that encodes a protein vital to photoreceptors in the eye. The condition leads to an abnormal splitting of retinal layers, impairing vision and ultimately causing blindness.

There are no approved treatments for XLRS, which is typically diagnosed in early childhood and primarily affects males. About 30,000 boys and men in the United States and Europe have the disorder.

Atsena’s ATSN-201 is a gene therapy intended to achieve therapeutic levels of gene expression in photoreceptors of the central retina.

The safety and tolerability of ATSN-201 are currently being evaluated in a Phase I/II clinical trial in male patients ages 6 and older who have XLRS. 

A second ongoing Phase I/II clinical trial is evaluating another gene therapy, ATSN-101, for Leber congenital amaurosis type 1, one of the more common causes of blindness in children. That therapy was granted Rare Pediatric Disease designation by the FDA in January.

In addition, the company is evaluating ATSN-301, a gene therapy to prevent blindness from MYO7A-associated Usher syndrome, an inherited disease that affects the retina and the inner ear. About 20,000 patients in the U.S. and Europe have the disorder.

Atsena’s product pipeline is based on novel adeno-associated virus (AAV) technology that uses altered viruses to deliver corrective genes into cells.