Hope in Focus Attends Foundation Fighting Blindness Retinal Therapeutics Innovation Summit and Association for Research in Vision and Ophthalmology Annual Meeting

Hope in Focus Director of Outreach and Development Courtney Coates attended the Retinal Therapeutics Innovation Summit on May 2, 2025, hosted by the Foundation Fighting Blindness. This annual summit showcases over 25 presentations on advancing therapies, lessons learned, and progress on natural history studies and clinical trial endpoints.

Eric Hartman, Choroideremia Research Foundation with Courtney Coates

Highlights for the LCA community included:
Twelve-month clinical trial data for LCA5 showing a clear safety profile, clinically meaningful improvement in vision, and durability.
Lessons learned from the Editas gene editing trial for CEP290
The Road Ahead for SepulBio’s Antisense Oligonucleotide Therapy for LCA10 CEP290

Additionally, several presentations provided updates on programs for IRDs in various treatment modalities. Clinical trial endpoints continue to be an important topic of discussion as the field works to develop outcomes that are easy to measure, reflect a clinically meaningful improvement in vision, and can be validated for regulators.

ARVO 2025

The summit was followed by the ARVO Annual Meeting, an essential resource for research updates, early pre-clinical research posters, and networking with the entire ecosystem of ophthalmic drug development. We continue to monitor early research for ultra-rare gene mutations, consistently engage with industry to incorporate the patient voice into clinical trials, and promote policy positions that will benefit the rare disease and LCA community.

For additional questions, please feel free to reach out to Courtney at courtney@hopeinfocus.org.