Name: ARVO 2025 Highlight: PYC Reports Encouraging Phase 1/2 Clinical Trial Results for its RNA Therapy Targeting RP (PRPF31 Mutations) by Foundation Fighting Blindness
Duration: 4 min 53 s
Description: The company is planning a Phase 2/3 clinical trial for therapy in late 2025.

The company is planning a Phase 2/3 clinical trial for therapy in late 2025.

ARVO 2025 Highlight: PYC Reports Encouraging Phase 1/2 Clinical Trial Results for its RNA Therapy Targeting RP (PRPF31 Mutations)

The company plans to complete BLA submission in early 2026.

Nanoscope Begins Rolling BLA Submission to FDA for Seeking Approval for its Optogenetic Therapy

The therapy delivers copies of the RORA gene to address multiple disease pathways. 

Ocugen Launches Phase 2/3 Clinical Trial for Stargardt Disease Gene-Modifier Therapy

July 25, 2025. Dr. Christine Curcio, one of the world's foremost experts in age-related macular degeneration (AMD) pathogenesis and the associated risk of advanced vision loss, talks to host Ben Shaberman about her epical career and recent Proctor Medal lecture with Dr. Cynthia Owsley at the 2025 meeting of the Association for Eye and Vision Research (ARVO). Her work has led to a highly effective test for predicting who is at greatest risk of losing vision from AMD.

Eye on the Cure Podcast | Episode 89: Dr. Christine Curcio

August 8, 2025. Dr. Christina Ohnsman talks to host Ben Shaberman about Tern Therapeutics, a start-up company she co-founded to develop gene therapies for Batten disease and potentially other conditions. Dr. Ohnsman reviews early results for a Batten disease (CLN2) retinal gene therapy clinical trial. She also reflects on her previous work as a pediatric ophthalmologist and roles with Spark Therapeutics and Regenxbio. 

Eye on the Cure Podcast | Episode 90: Dr. Christina Ohnsman

Susvimo is an FDA-approved treatment that continuously delivers drug through a tiny, implanted capsule 

Wet AMD Treatment Susvimo Preserves Vision for Five Years in Phase 3 Clinical Trial

The trial is evaluating a cell dose that is 50 percent higher than the highest dose used in a previous Phase 2b trial.

jCyte Launches New Phase 2 Clinical Trial for its Cell-Based RP Therapy

The Foundation funded earlier, proof-of-concept studies for the Best1 gene therapy and later launched Opus to develop several IRD gene therapies.

Opus Genetics Receives FDA Clearance to Launch Clinical Trial for Best1 Gene Therapy

September 5, 2025. Dr. George Magrath, CEO of Opus Genetics, talks with host Ben Shaberman about the company's evolution and progress in clinically developing gene therapies for several inherited retinal diseases including those caused by mutations in the genes: LCA5, BEST1, RHO, RDH12, MERTK, NMNAT1, and CNGB1.

Eye on the Cure Podcast | Episode 91: Dr. George Magrath

NACA is a gene-agnostic, oral medication designed to preserve vision in people with retinitis pigmentosa and Usher syndrome.

Nacuity’s Antioxidative Therapy NACA Performs Encouragingly in Phase 1/2 Clinical Trial

The Foundation Fighting Blindness is the driving force in the global development of treatments and cures for blinding diseases. To learn more, go to FightingBlindness.org.

ARVO 2025 Highlight: PYC Reports Encouraging Phase 1/2 Clinical Trial Results for its RNA Therapy Targeting RP (PRPF31 Mutations)

Foundation Fighting Blindness

Related tracks