Atsena lands another FDA incentive for gene therapy to fight blindness

Atsena Therapeutics, a Durham company developing gene therapies to treat genetic causes of blindness, has received another incentive from the U.S. Food and Drug Administration (FDA) to help boost the development of its leading product candidate.

The FDA has granted Atsena an Orphan Drug Designation for ATSN-201, the company’s product candidate targeting X-linked retinoschisis (XLRS), a rare genetic disease that causes blindness, mostly among men.

In late August, the FDA also granted Rare Pediatric Disease (RPD) designation for ATSN-201. That designation, the second Atsena has received this year, is for therapeutics intended to treat serious or life-threatening rare diseases primarily affecting patients under 18. That designation means if ATSN-201 gets marketing approval, Atsena will be eligible for a priority review voucher. The company could use the voucher to advance another internal program or sell it to another company. The voucher program incentivizes companies to invest in rare pediatric diseases with limited markets.

Designations mark ATSN-201 ‘inflection point’

“These designations mark a significant inflection point for the potential of this ocular gene therapy in an inherited retinal disease that currently has no available treatments,” said Atsena CEO Patrick Ritschel. “We look forward to bringing hope to patients affected by this rare disease and are confident these designations will expedite our path forward.”

The FDA grants Orphan Drug Designation to drugs and biologics intended for safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. The designation provides certain incentives, such as tax credits toward the cost of clinical trials upon approval and prescription drug user fee waivers. A designated Orphan Drug is entitled to seven years of market exclusivity for treating the designated disease, which is independent from the drug’s intellectual property protection.

XLRS is an inherited retinal disease with no treatment yet

There are no approved treatments for XLRS, which is typically diagnosed in early childhood and affects approximately 30,000 males in the U.S. and EU. 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.

Atsena is evaluating the safety and tolerability of ATSN-201 in a study called LIGHTHOUSE, a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial in male patients ages 6 and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. More information is available at ClinicalTrials.gov. Search for the identifier NCT05878860.

ATSN-201 leverages AAV.SPR, Atsena’s novel spreading capsid using the harmless AAV virus as a “delivery vehicle.” 

AAV technology has Triangle roots

The AAV technology was developed by gene therapy pioneer R. Jude Samulski, Ph.D., co-founder of Research Triangle Park-based Asklepios BioPharmaceutical (AskBio) and co-founder of Columbus Children’s Foundation, a Chapel Hill nonprofit biotech organization that helps children with ultra-rare genetic diseases.

The North Carolina Biotechnology Center provided a $250,000 grant to the University of North Carolina at Chapel Hill to help recruit Samulski from the University of Pittsburgh in 1993. In total, NCBiotech provided about $1 million in grants and loans to help support Samulski’s work, ultimately resulting in North Carolina becoming a global leader in gene and cell therapy development and production.