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.
Hope for RP and Stargardt Disease Nanoscope Therapeutics is advancing a one-time gene therapy designed to restore vision for patients with retinitis pigmentosa and Stargardt disease. If successful, this treatment could transform care for two conditions that currently have no approved therapies.
Read moreA groundbreaking Phase 2 trial has shown the first-ever vision improvements in people with Stargardt disease — a condition with no approved treatment. Nanoscope Therapeutics’ gene-agnostic MCO-010 therapy produced meaningful gains in vision without serious side effects, paving the way for a pivotal Phase 3 trial starting in 2025.
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