A Race Against Blindness Awards $1 Million to Advance AXV-101 Gene Therapy for Childhood Blindness
Key Insights
A Race Against Blindness (search) has awarded an additional $1 million grant to Axovia Therapeutics (search), bringing total funding for AXV-101 gene therapy development to $4 million since 2024.
AXV-101 is an investigational AAV9-based gene therapy designed to combat childhood blindness caused by retinitis pigmentosa (search) in Bardet-Biedl Syndrome 1 (search) patients.
The funding will support the first-in-human open-label dose-escalation trial, which is expected to commence shortly in the United Kingdom following recent regulatory clearance.
A Race Against Blindness (search), an Arizona-based nonprofit organization, has awarded a $1 million grant to Axovia Therapeutics (search) to advance clinical development of AXV-101, an investigational gene therapy targeting childhood blindness caused by Bardet-Biedl Syndrome 1 (search) (BBS1 (search)). This marks the organization's third major grant supporting the program, bringing total funding to $4 million since 2024.
The funding will support execution of the first-in-human open-label dose-escalation trial for AXV-101, which is expected to begin shortly in the United Kingdom following recent Clinical Trial Application (CTA) clearance. AXV-101 is a codon-optimized AAV9 BBS1 (search) sub-retinal gene therapy designed to halt retinal degeneration in patients with BBS1.
Gene Therapy Approach for Rare Genetic Blindness
AXV-101 targets retinitis pigmentosa (search) (RP) caused by inherited faults in the BBS1 (search) gene, a rare genetic disorder that leads to progressive blindness and other serious health challenges. The therapy utilizes adeno-associated virus serotype 9 (AAV9) as a delivery vector for the corrected BBS1 gene, administered through sub-retinal injection with the goal of stopping retinal degeneration.
"Because of our incredible donor community and sponsors, we are making a direct and tangible impact in driving this groundbreaking program forward," said Dr. Stephen Johnston, Co-Founder and President of A Race Against Blindness (search). "Every dollar given reflects a united commitment to ensuring children with BBS1 (search) have a future where blindness is not inevitable."
Personal Mission Drives Research Funding
The Johnston family's mission began after their son Luke was diagnosed with retinitis pigmentosa (search) caused by inherited faults in the BBS1 (search) gene. Determined to fund transformative therapies, the family has rallied thousands of supporters nationwide to support research into treatments for progressive vision loss caused by rare genetic conditions.
Prof. Phil Beales, Chief Executive Officer and Co-Founder of Axovia Therapeutics (search), emphasized the importance of patient perspectives in driving the research forward. "The continued commitment of A Race Against Blindness (search) is a powerful reminder of why we do this work at Axovia. Every conversation with patients and families in the BBS community reinforces the urgency and importance of developing new therapies. As we move AXV-101 into the clinic, their perspectives continue to guide and motivate us."
Clinical Development Milestone
The upcoming first-in-human trial represents a significant milestone for AXV-101, marking the transition from preclinical development to human testing. The open-label dose-escalation design will allow researchers to evaluate safety and determine appropriate dosing for the gene therapy in BBS1 (search) patients.
A Race Against Blindness (search) focuses on funding research and advancing treatments for progressive vision loss caused by rare genetic conditions such as Bardet-Biedl Syndrome and retinitis pigmentosa (search). The organization supports clinical research, raises awareness, and engages communities through innovative fundraising efforts to advance potential treatments for these devastating conditions.