Category: HIF Activities

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 conference signage displayed at the annual meeting

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.

Spring on the Road

A woman stands behind an informational table at a RARE-X booth, with printed materials and signage displayed

May and June were busy travel months full of opportunities and learning for me as Director of Outreach and Development for Hope in Focus. Each year, I attend the Association for Research in Vision and Opthalmology (ARVO) Annual Meeting to hear the latest research updates for inherited retinal diseases (IRD). This conference brings together over 12,000 attendees and offers dozens of hours of presentations about therapy advances and provides a place for industry, advocacy, and the academic community to converge and connect on advancing research.

I then headed off to Sioux Falls, South Dakota, for my second year of training in the Professional Patient Advocates in the Life Sciences Certification Training Course. The course provides two tracks, one for those with a patient advocacy role in industry, and the other for those involved in nonprofit patient advocacy organizations. It is a small group with limited class sizes that provides an in-depth education on the roles and responsibilities of a patient advocate. We have important discussions on how to work together and best serve our communities, and I bring back so much of that learning into this role every day.

In June, I headed to Chicago with the Hope in Focus team to the 2024 VISIONS conference, which is held biennially and hosted by the Foundation Fighting Blindness. VISIONS brings together over 500 attendees from the inherited retinal disease community, many of whom are living with an IRD. We had a packed two days of listening to important updates and inspiring lectures, and our favorite was meeting families affected by Leber congenital amaurosis (LCA). Haydar, Gizem, and Toprak from this month’s lead story also attended and brought along another family from Turkey whose daughter has LCA1 GUCY2D. It was amazing to see them again! We also met a new family that has two daughters with LCA9 due to a mutation in their CRB1 gene.

Two presenters speak to an audience in a conference room, with a presentation slide displayed

I am so grateful for these opportunities to connect in person with all the facets of our community working to bring treatments to those living with inherited retinal disease.