Usher 1F Collaborative

10/14/2026

There are many ways to , and sharing your experience can help shape the future of Usher syndrome research.

The Foundation Fighting Blindness is looking for people living with all subtypes of Usher syndrome and their caregivers to share their experiences as speakers and panelists during our virtual Patient-Focused Drug Development (PFDD) meeting on April 8, 2027.

Your voice can help researchers, drug developers, regulators, and other key stakeholders better understand the experiences and priorities of the Usher syndrome community and inform the conversation around future treatments.

Learn more and submit a nomination: www.FightingBlindness.org/usher-pfdd

Thank you to AAVantgarde, Atsena Therapeutics, Eli Lilly, The Fairfield Fund, and Sepul Bio by Théa for their generous support of this meeting.

Image Description: Dark blue background with Foundation Fighting Blindness logo in top left corner. Text below reads, "Your voice can help shape the future of Usher syndrome treatments. Call for panelists: Usher Syndrome PFDD Meeting, April 8, 2027."

10/01/2026

A Potential Usher 1F Therapy is Within Reach: Fall 2026 Newsletter
https://www.usher1f.org/file_download/inline/7159982e-711d-49e7-b877-123d8e2e9ff2

09/24/2026

This is a review article about an approach that could eventually restore vision even after retinal degeneration has become advanced.

In simple terms, diseases such as retinitis pigmentosa—including the retinal degeneration caused by Usher syndrome—destroy the eye’s photoreceptors, the light-sensing rod and cone cells. Gene therapy is generally aimed at preserving or rescuing photoreceptors that are still present. This approach is different: researchers are trying to replace photoreceptors that have already been lost, using healthy photoreceptors produced from stem cells.

The encouraging finding is that in animal models with very advanced retinal degeneration, transplanted human cone cells have been able to detect light, form new connections with surviving retinal cells, and restore some visual responses. Remarkably, other retinal cells can survive long after photoreceptors disappear and appear capable of reorganizing themselves to connect with the new cells.

The major advantage is that this could be “disease agnostic”—the treatment would not have to be designed for a particular genetic mutation. In principle, the same photoreceptor-replacement treatment might someday help people with many different inherited retinal diseases.

For Usher 1F, I would view this as potentially complementary to the mini-PCDH15 gene therapy rather than a competitor to it. Gene therapy could potentially preserve or improve the function of photoreceptors that remain, while photoreceptor replacement could someday offer an option for people whose photoreceptors have already been lost.

The important caveat: this is not yet an established treatment for patients. Researchers still need to improve how reliably the new cells connect to the existing retina and solve manufacturing, storage, safety, and regulatory challenges. Some photoreceptor-replacement programs are now moving toward or into early clinical trials, but considerable work remains.

Retinal dystrophies such as retinitis pigmentosa (RP) and age-related macular degeneration (AMD) affect millions of individuals worldwide and remain leading causes of irreversible blindness. Photoreceptor transplantation has emerged as a promising and disease-agnostic therapeutic approach for restor...

09/23/2026

SCOTTSDALE AREA FRIENDS!

We are so excited for Tiles Toward a Cure, a special evening benefiting Usher 1F Collaborative.

Whether you’re an experienced Mahjong player or brand new to the game, we’d love to have you join us for an evening of connection, fun, and impact. Beginners can learn the game in our Mahjong class, and everyone can enjoy a cocktail hour before the tiles hit the tables.

📅 Thursday, November 12
⏰ 5:30 PM
📍 Dreamhouse
🔗 www.usher1f.org/tiles

Every tile played helps fuel the research bringing hope to families living with Usher 1F.

Play. Connect. Give Hope. 💕

09/19/2026

As the days grow shorter, navigating the world becomes more difficult for those living with Usher 1F. On this Usher Syndrome Awareness Day, let’s all pause to recognize the challenges faced by those living with Usher syndrome—and the urgent need for research that can lead to treatments and a cure.

08/29/2026

"Inherited retinal diseases (IRDs) are a genetically and clinically heterogeneous group of disorders caused by pathogenic variants in more than 250 genes.1,2 Mutation-specific gene augmentation and gene editing have transformed care for a small subset of patients, yet the sheer genetic diversity and cost of developing “one therapy per gene” mean that most individuals with an IRD still lack an approved treatment.1,2 As a result, gene-agnostic strategies may broaden treatment options by targeting shared downstream pathways rather than individual mutations.3-5 These broader therapeutic approaches aimed at preserving or restoring vision are gaining increasing attention.1"

Emerging gene-agnostic strategies target shared pathways and offer options for IRD patients without mutation-specific therapies.

08/25/2026

Massive genomic newborn-screening studies are under way all over the globe. But questions about scalability, feasibility and net benefit remain.

08/23/2026

🚨 FINAL DAY! 🚨

usher1f.org/nanas-deli

Today is your last chance to win a $400 gift certificate to Nana’s Deli in Livingston, NJ!

For every $20 donation to Usher 1F Collaborative, you’ll receive one entry into the drawing. Enter once—or enter more than once to increase your chances!

And remember, this raffle is about so much more than a great meal. Every entry helps fund the research that is bringing us closer to treatments for the vision loss caused by Usher 1F.

A huge thank you to Nana’s Deli for generously donating this prize and supporting our mission!

08/22/2026

Why our researchers started developing retinal organoids early in our journey toward a cure -

A Seismic Shift From Animal to “Human-Based” Research Is Underway—Here’s What to Know About Organoids

This Medical News article discusses a push for human-centric research models, such as mini-organs grown from human cells, to complement or replace animal studies.