Avista Therapeutics Presents In Vivo Data for its AAV Capsid in Inherited Retinal Diseases at Association for Research in Vision and Ophthalmology 2025 Annual Meeting

Potential best-in-class intravitreal capsid demonstrates Retinoschisin expression similar to endogenous levels in X-linked retinoschisis

IND-enabling studies are ongoing to support filing expected in early 2026

PITTSBURGH, May 8, 2025 /PRNewswire/ -- Avista Therapeutics, a pre-clinical-stage biotechnology company developing innovative gene therapies for rare ophthalmic conditions, today held an oral presentation on data showcasing progress towards the development of the Company's best-in-class intravitreal capsid for inherited retinal diseases, with lead indication in X-linked retinoschisis (XLRS), at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting in Salt Lake City, Utah.

"Using our computationally guided scAAVengr-HUnT platform, we are able to identify promising AAV candidates to treat inherited retinal diseases through intravitreal delivery," said Robert Lin, Ph.D., Chief Executive Officer of Avista Therapeutics. "Our best-in-class intravitreal drug candidate AVST-101, currently being evaluated in XLRS, delivers clinically relevant low doses of RS1 that evade immune detection, underscoring its therapeutic potential to be evaluated in a wide range of indications, based on its compelling preclinical profile compared to other intravitreal delivery vectors."

Key Findings:

• ATX002 strongly outperforms 7m8 in nonhuman primate (NHP) eyes.
• ATX002 results in efficient panretinal transduction.
• ATX002 delivers endogenous levels of Retinoschisin protein from intravitreal injection.
• AVST-101, leveraging ATX002 capsid, is currently in IND (investigational new drug)-enabling studies in XLRS, with plans to submit for IND approval in early 2026.
• scAAVengr-HUnT platform generates highly efficient adeno-associated virus (AAV) vectors optimized for retinal delivery via intravitreal injection and implements innovative machine learning methods to further enhance AAV vector generation.

About Avista Therapeutics
Avista Therapeutics' mission is to develop innovative gene therapies for retinal diseases, including rare ophthalmic conditions that have a profound impact on patients' quality of life. We leverage our computationally guided scAAVengr platform to generate and validate a toolkit of proprietary AAV vectors that target specific cell types using minimally invasive intravitreal delivery with reduced dosages. Our quantitative, in vivo-based approach and clinical ophthalmology expertise allow us to rapidly translate new gene therapies to the clinic.

SOURCE Avista Therapeutics