Non-profit funding sight-saving research for children
Stories of Hope
Follow A Race Against Blindness research funding, inherited retinal disease developments, community stories, grant announcements, and updates from families racing toward sight-saving treatments.
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University College London named Dr. Victor Hernandez, co-founder of Axovia Therapeutics, to its 200 Faces. In thanking the people behind his gene therapy for Bardet-Biedl syndrome, he singled out A Race Against Blindness.
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Every dollar below has been paid out to research, made possible by the generosity of our sponsors and donors. Real people making an impact on saving children's eyesight.
total research funding, fully paid out · as of October 2026
$3M awarded
To fund the preclinical development and regulatory preparation of AXV-101, a gene therapy for Bardet-Biedl Syndrome type 1 (BBS1), advancing the program toward its first human clinical trial.
$1M awarded
To support clinical development of the first-in-human trial, including trial preparation and regulatory progress following clinical trial clearance.
$2.1M awarded
To close the remaining funding gap required to launch the AXV-101 clinical trial, enabling the study to begin in early 2026; and, through a grant to the Foundation Fighting Blindness, to fund early-stage gene therapy research with Dr. Thomas Mendel and gene-agnostic therapy (NACA) aimed at slowing vision loss in RP. A matched donor brought in an additional $775,000, bringing the total impact of that grant round to $1.55M.
University College London named Dr. Victor Hernandez, co-founder of Axovia Therapeutics, to its 200 Faces. In thanking the people behind his gene therapy for Bardet-Biedl syndrome, he singled out A Race Against Blindness.
Read moreNewly added funding closes critical gap and enables planned trial initiation in early 2026
Read morePatient advocacy is now one of the most powerful drivers of progress in retinal gene therapy. Learn how nonprofit involvement, family voices, awareness, and fundraising are accelerating research for Bardet-Biedl Syndrome and inherited retinal diseases — and why this work matters now more than ever.
Read moreWe recently attended the Gene Therapy for Ophthalmic Disorders Summit, where leading researchers and clinicians shared promising advances in retinal gene therapy. From new vision endpoints to breakthroughs in delivery methods and patient advocacy, the conference highlighted real progress toward treatments for BBS, RP, and other inherited retinal diseases. Here’s what we learned — and why it matters for families urgently waiting for hope.
Read moreThe latest innovations in gene therapy — from optogenetics to engineered AAV capsids to AI-driven breakthroughs — are rapidly accelerating progress toward treatments for Bardet-Biedl Syndrome and other inherited retinal diseases. Here’s what these advances mean for families urgently awaiting hope.
Read moreThe FDA has approved the first-ever gene therapy for Wiskott-Aldrich syndrome, marking a major milestone for the rare disease community. While this treatment is not for vision loss, it represents important progress for gene therapy as a whole — and brings renewed hope for families awaiting treatments for inherited retinal diseases like BBS and retinitis pigmentosa.
Read moreBeacon Therapeutics has treated the first patient in a new clinical trial evaluating bilateral gene therapy for X-linked retinitis pigmentosa. This milestone builds on encouraging earlier data and represents continued momentum in retinal gene therapy research — bringing renewed hope to families affected by inherited retinal diseases.
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