Avirmax Biopharma, Inc., a leader in developing the next generation of AAV gene therapies, today announced the commencement of Investigational New Drug (IND)-enabling studies of ABI-201, a potential first-in-class AAV vector that delivers three genes to correct the dysregulation of complement activation, anti-inflammation, to protect retinal pigment epithelia and photoreceptors as well as to block retinal neovascularization. ABI-201 is constructed to achieve durable expression of these transgenes in eye only after a single intravitreal injection, thereby preventing retinal pathogenesis and preserving vision. In vivo studies conducted across multiple animal species, including Cynomolgus monkeys, have demonstrated 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,” said Shengjiang Shawn Liu, Ph.D., Chief Executive Officer of Avirmax Biopharma. “ABI-201 has the potential to revolutionize the treatment for dry AMD and many other retinal disorders.”
Age-related Macular Degeneration is the leading cause of irreversible blindness in individuals over the age of 50, with more than 200 million people afflicted, worldwide. Dry AMD accounts for about 90% of AMD cases, yet no effective or long-lasting therapies are currently available. ABI-201 aims to address this significant unmet medical need by providing a durable solution to reduce geographic atrophy (GA) growth, block progression from dry AMD to wet AMD, and ultimately preserve patient’s vision.
ABI-201 utilizes Avirmax Biopharma‘s proprietarily engineered capsid, AAV.N54, to efficiently deliver therapeutic transgenes to the macular retina. AAV.N54 is the only engineered capsid to deliver macula-targeted gene expression via intravitreal injection. AAV.N54 is also used in ABI-110, currently in Phase 1/2a trial for wet AMD including PCV.
The ongoing IND-enabling studies are designed to evaluate the safety, tolerability, vector biodistribution, transgene expression and pharmacokinetics of ABI-201 in monkeys.
Avirmax Biopharma anticipates submitting an IND application for ABI-201 in the fourth quarter of 2025. Pending regulatory clearance, the company plans to initiate phase 1/2a trials in patients with dry AMD shortly thereafter.
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