Avirmax Biopharma prepares for IND-enabling study of ABI-201

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Avirmax Biopharma recently announced the beginning of Investigational New Drug (IND)-enabling studies of ABI-201, a potential AAV vector that delivers 3 genes to correct the dysregulation of complement activation, anti-inflammation, to protect retinal pigment epithelia and photoreceptors in patients. The potential new drug also aims to block retinal neovascularization.

ABI-201 is made to complete long-lasting expression of these transgenes in the eye only after one intravitreal injection, preventing retinal pathogenesis and preserving vision. In previous in vivo studies conducted in multiple animal species, such as Cynomolgus monkeys, results showed the anticipated biological functions of the therapeutic genes encoded by the ABI-201 vector.

“We are excited to reach this critical milestone in ABI-201 development,” Shengjiang Shawn Liu, chief executive officer of Avirmax Biopharma, said. “ABI-201 has the potential to revolutionize the treatment for dry AMD and many other retinal disorders.”

The vector uses Avirmax Biopharma proprietarily engineered capsid, AAV.N54, to deliver therapeutic transgenes to the macular retina. AAV.N54 is currently the only engineered capsid to transport macula-targeted gene expression through an intravitreal injection. It is also used in ABI-110, which is presently in Phase 1/2a trial for wet AMD.

Right now, the IND-enabling studies are made to test the safety, tolerability, vector biodistribution, transgene expression, and pharmacokinetics of ABI-201 in monkeys. Avirmax Biopharma expects to submit an IND application for ABI-201 in the fourth quarter of 2025.

Reference:

1. Avirmax Biopharma Initiates IND-enabling Studies of ABI-201; a Breakthrough Gene Therapy for Dry Age-related Macular Degeneration. BioSpace. Accessed March 3, 2025. https://www.biospace.com/press-releases/avirmax-biopharma-initiates-ind-enabling-studies-of-abi-201-a-breakthrough-gene-therapy-for-dry-age-related-macular-degeneration