A Race Against Blindness Awards Additional $1 Million Grant to Advance Clinical Trial for Sight-Saving Gene Therapy for Bardet-Biedl Syndrome 1 (BBS1)

PHOENIX, Sept. 04, 2025 (GLOBE NEWSWIRE) -- A Race Against Blindness, the Arizona-based 501(c)(3) nonprofit founded by Dr. Stephen Johnston and Kristina Johnston, announced today that it has awarded a new $1.0 million grant to Axovia Therapeutics to support the clinical development of AXV-101, an investigational gene therapy aimed at combating childhood blindness due to retinitis pigmentosa (RP) caused by Bardet-Biedl Syndrome 1 (BBS1).

This is the organization’s third major grant in support of the program, bringing its total funding for the advancement of AXV-101 to $4.0 million since 2024. The new funding will contribute to the execution of the first-in-human open-label dose-escalation trial for AXV-101, which is expected to commence shortly in the United Kingdom, following the recent Clinical Trial Application (CTA) clearance. AXV-101, is a codon-optimized AAV9 BBS1 sub-retinal gene therapy with the goal of stopping retinal degeneration in BBS1 patients.

“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. “Every dollar given reflects a united commitment to ensuring children with BBS1 have a future where blindness is not inevitable.”

“The continued commitment of A Race Against Blindness 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,” said Prof. Phil Beales, Chief Executive Officer and Co-Founder of Axovia Therapeutics.

A Personal Mission with Global Reach

The Johnston family’s mission began after their son Luke was diagnosed with retinitis pigmentosa caused by inherited faults in the BBS1 gene — a rare genetic disorder that leads to progressive blindness and other serious health challenges. Determined to fund transformative therapies, the family has rallied thousands of supporters nationwide.

About A Race Against Blindness
A Race Against Blindness is a nonprofit organization dedicated to funding research and advancing treatments for progressive vision loss caused by rare genetic conditions such as Bardet-Biedl Syndrome (BBS) and retinitis pigmentosa (RP). Founded by Dr. Stephen Johnston and Kristina Johnston, the organization focuses on supporting clinical research, raising awareness, and engaging communities through innovative fundraising efforts. Learn more at www.araceagainstblindness.org.

Contact:
Dr. Stephen Johnston
Co-Founder & President
A Race Against Blindness
Email: press@araceagainstblindness.org

A video about the mission of A Race Against Blindness is available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/83872f4c-7247-40c9-9048-4664fa7f81bc

Legal Disclaimer:

EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.