No serious adverse events were reported. One patient had encouraging improvement in retinal sensitivity.

ARVO 2024 Highlight: PYC Reports Initial Clinical Trial Results for its RNA Therapy Targeting RP

Part B of the trial will enroll nine adult patients as well as three pediatric patients.

ARVO 2025 Highlight: Atsena’s XLRS Gene Therapy Provides Vision and Structural Improvements in Phase 1/2 Clinical Trial

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)

Despite missing the primary endpoint, the treatment improved vision for some patients.

J&J’s XLRP Gene Therapy Didn’t Meet Primary Endpoint in Phase 3 Clinical Trial

May 23, 2025. Lenore von Krusenstiern, MD, PhD, at BlueRock Therapeutics talks to host Ben Shaberman about the company's clinical trial for a photoreceptor replacement therapy for people with inherited retinal conditions such as retinitis pigmentosa and cone-rod dystrophy. The approach, which uses induced pluripotent stem cells to develop photoreceptor precursors, comes out of the lab of stem cell pioneer David Gamm, MD, PhD, University of Wisconsin-Madison.

Eye on the Cure Podcast | Episode 86: Lenore von Krusenstiern, MD, PhD

June 13, 2025. Dr. Philip Beales, a co-founder of Axovia Therapeutics, talks to host Ben Shaberman about Bardet Biedl syndrome, ciliopathies, and his company's emerging gene therapy for Bardet Biedl syndrome type 1 (BBS1) which is moving toward a clinical trial. 

Eye on the Cure Podcast | Episode 87: Dr. Philip Beales

The expanded Phase 1/2/3 shortens time to potential FDA approval.

Atsena Gets FDA Nod to Expand XLRS Gene Therapy Clinical Trial to Include Pivotal Phase 3 Cohort

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

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.