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.
Opus Genetics has officially treated the first participant in its Phase 1/2 clinical trial for OPGx-001 — a new gene therapy designed for individuals with LCA5-related Leber congenital amaurosis. This milestone marks the company’s first human trial and represents another important step forward in treating inherited retinal diseases. As more early-stage therapies enter clinical testing, hope continues to grow for families affected by progressive vision loss.
Read moreThe Foundation Fighting Blindness has released four years of natural history data from its RUSH2A study, giving researchers open access to valuable insights into USH2A-related retinitis pigmentosa and Usher syndrome. This unprecedented data release is expected to accelerate clinical trial development and advance treatments for inherited retinal diseases.
Read moreFDA Fast Tracks Potential RP Treatment Aldeyra’s ADX-2191 has received Fast Track Designation from the FDA for retinitis pigmentosa. With no approved treatments currently available for most RP patients, this milestone could help speed the development of a promising new therapy.
Read moreLong-Term Results Show Promise for RP Therapy At the 2025 ASRS meeting, researchers shared 2-year results showing that MCO-010, an optogenetic therapy, provided lasting vision improvements for patients with advanced retinitis pigmentosa. Unlike traditional gene therapies, MCO-010 is gene-agnostic, meaning it could help a broad range of patients regardless of mutation.
Read moreNew Hope for XLRP Patients The SKYLINE Phase 2 trial of laruzova, a gene therapy for X-linked retinitis pigmentosa, has shown promising vision improvements at 24 months. With the pivotal VISTA Phase 3 trial now fully enrolled, this breakthrough research moves us one step closer to potential treatment for this rare inherited blindness.
Read moreFirst Patients Treated in jCell RP Trial jCyte has launched its Phase 2 JC02-88 trial testing jCell, a potential regenerative therapy for retinitis pigmentosa. This study could bring the first treatment option to the majority of RP patients, offering hope across all genetic subtypes.
Read moreScientists have discovered that blocking a protein called Prox1 may allow the eye to repair itself. This breakthrough could open the door to regenerative therapies for vision loss caused by diseases like retinitis pigmentosa.
Read moreHope for Stargardt Disease Ocugen has announced FDA clearance to begin a Phase 2/3 trial of OCU410ST, a groundbreaking modifier gene therapy for Stargardt disease. Early results show slowed disease progression and vision improvements, bringing real hope to the 1 million people worldwide affected by this inherited condition.
Read moreStem Cell–Derived Retinal Sheets Trial for RP A new clinical trial is testing DSP-3077, a one-time stem cell–derived retinal sheet implant, as a potential treatment for retinitis pigmentosa. This regenerative therapy could offer hope for patients regardless of genetic subtype. Learn how this groundbreaking study may shape the future of RP treatment.
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