Key market opportunities lie in AAV gene therapy advancements for hereditary retinal diseases. Future prospects include combination with CRISPR for precise genome editing, novel delivery platforms, ...

Sentinel participant showed OPGx-BEST1 was well tolerated with no ocular inflammation, treatment-related adverse events, or ...

Hot on the heels of a $150 million fundraise, Atsena Therapeutics is back with early-stage data for the company’s eye disease gene therapy. The candidate improved the structure and function of the ...

FDA authorization under an IND permits first-in-human phase I/II testing of SVT-001 in familial drusen, focusing on safety ...

Opus Genetics, Inc., a clinical-stage biotechnology company focusing on gene therapies for inherited retinal diseases, announced upcoming presentations at various scientific conferences in May 2025.

"This pivotal work from the group at the University of Iowa has shown that one of the most severe degenerative diseases affecting the retina, juvenile NCL, may also be treatable with gene therapy." ...

RegenXBio reported that dosing is complete in the AFFINITY pivotal Duchenne trial, with robust enrollment in the confirmatory study and encouraging Phase 1/2 data. At 18 months, treated boys showed a ...

Ocugen, Inc., a biotechnology company focused on gene therapies for blindness diseases, announced its participation in two significant upcoming conferences: the ARVO 2025 Annual Meeting in Salt Lake ...

Coave Therapeutics unveils lead gene therapy program for retinal vascular diseases, powered by first-in-class vector for suprachoroidal delivery CoTx-101 represents a potential breakthrough for ...

Researchers are expressing optimism that gene therapy can treat a wide range of eye diseases, including some that currently have no treatments. Only one gene therapy has been approved by the US Food ...