Impact to Date

$6.1 million supporting two complementary paths

$5.1M is committed to AXV-101, a targeted investigational therapy for vision loss associated with BBS1. $1M is committed to gene-agnostic approaches intended to reach patients across many inherited retinal diseases.

One commitment advances an investigational therapy created for BBS1 toward clinical study. The other expands therapeutic approaches that may help patients regardless of the gene causing their inherited retinal disease.

$5.1 million committed to AXV-101

AXV-101 is an investigational therapy aimed at treating vision loss associated with Bardet-Biedl syndrome 1. RAB's funding has supported the program as it moved toward a first clinical study.

The final $1.1 million award closed the remaining funding gap identified for planned clinical trial initiation and brought RAB's total AXV-101 commitment to $5.1 million. The program received Clinical Trial Application clearance in the third quarter of 2025. The January 2026 update reported that trial initiation was expected in early 2026.

Project reference: AXV-101 funding announcement

$1 million expanding gene-agnostic research

Gene-agnostic approaches are intended to work independently of a patient's specific disease-causing mutation. RAB's commitment supports both a portfolio of therapeutic programs and an academic research award focused on a potentially broader way to deliver retinal gene therapy.

$775,000 building a broader therapeutic portfolio

Funding directed to the RD Fund supports gene-agnostic programs across its portfolio, including neuroprotective and antioxidant strategies intended to slow retinal degeneration and vision-restoration approaches such as optogenetics.

This portion was matched dollar for dollar through the Gordon and Llura Gund Foundation Challenge, increasing the amount directed to the RD Fund to $1.55 million.

$225,000 advancing a new delivery approach

This funding completed a five-year, $375,000 Career Development Award supporting Dr. Thomas Mendel at The Ohio State University Wexner Medical Center. His preclinical work studies delivery of gene therapy onto the retinal surface, combined with insulin to accelerate uptake into retinal cells.

The gene-agnostic method may have relevance across conditions including BBS, choroideremia, Leber congenital amaurosis, Stargardt disease, Usher syndrome, and multiple forms of retinitis pigmentosa.

Project reference: Gene-agnostic research funding announcement