Axovia Therapeutics receives grant from A Race Against Blindness to support upcoming clinical trial

(Image credit: AdobeStock/Sergey)

In December 2024, a $2.0 million grant was given to Axovia Therapeutics by A Race Against Blindness to further fund a planned clinical trial for the company’s product candidate, AXV101, focused on combating childhood blindness due to retinitis pigmentosa (RP) caused by Bardet-Biedl Syndrome (BBS) type 1. With the grant, A Race Against Blindness contributed $3.0 million in total funding during 2024 to help AXV101 move forward in its development.

“Because of our incredible supporters, we’ve taken another huge step forward in the race against blindness,” Stephen Johnston, co-founder of A Race Against Blindness, said. “But much work remains, and continuing to advance this therapy into and through a clinical trial remains our top priority. The potential of this gene therapy to address vision loss brings hope to thousands of children facing blindness due to BBS1 worldwide. However, additional funding is needed to support this program through clinical trials.”

Since the donation from A Race Against Blindness, Axovia Therapeutics has made significant progress in nearing the anticipated Clinical Trial Application (CTA) submission. If cleared, it would allow the company to start a clinical trial in the U.K. in mid-2025.

Recently, it was announced that Axovia Therapeutics and Viralgen will be partnering to advance the development and manufacture of an AAV9-based investigational gene therapy aimed at treating retinal dystrophy in patients with Bardet-Biedl Syndrome (BBS).

The grant also supported advancing manufacturing, GLP toxicology studies, and interactions with the U.K. regulatory agency for the candidate and will most likely support the planned clinical trial.

Reference:

1. Viralgen and Axovia Therapeutics partner to manufacture gene therapy for Bardet-Biedl Syndrome. Ophthalmology Times. Accessed February 10, 2025. Viralgen and Axovia Therapeutics partner to manufacture gene therapy for Bardet-Biedl Syndrome

2. A Race Against Blindness Announces $2 Million Grant to Support Sight-Saving Clinical Trial. A Race Against Blindness. Published February 5, 2025. Accessed February 10, 2025. https://www.globenewswire.com/news-release/2025/02/05/3021169/0/en/A-Race-Against-Blindness-Announces-2-Million-Grant-to-Support-Sight-Saving-Clinical-Trial.html#:~:text=05%2C%202025%20(GLOBE%20NEWSWIRE),the%20company%27s%20product%20candidate%2C%20AXV101%2C