Affinia Therapeutics Presents Preclinical Data for Genetic Cardiomyopathy and Sporadic ALS Programs with Novel Cardiotropic and BBB-Penetrant AAV Capsids at the American Society of Gene & Cell Therapy 2024 Annual Meeting

Novel cardiotropic capsids transduce more than 90% of cardiomyocytes in non-human primates (NHPs) and detarget the liver and dorsal root ganglia (DRG); show safety and efficacy in a mouse model of cardiac dysfunction

Novel BBB-penetrant capsid transduces more than 50% of neurons in NHPs across spinal cord, cortex, and deep-brain regions and detarget the liver and DRG; shows greater than 50% reduction of Ataxin-2, a gene believed to be involved in ALS

Excerpt from the Press Release:

WALTHAM, Mass., May 10, 2024 /PRNewswire/ — Affinia Therapeutics (“Affinia”), an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and prevalent devastating diseases, today announced the presentation of new preclinical data on its novel AAV capsids for genetic cardiomyopathies and diseases of the central nervous system (CNS) such as amyotrophic lateral sclerosis (ALS), as well as the Company’s high-yield manufacturing process. This research will be presented in oral sessions today at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting, being held May 7-11, 2024 in Baltimore, MD and virtually.

Affinia has leveraged its proprietary platform to rationally design capsids with increased tropism to cardiac muscle, skeletal muscle, or CNS with more uniform tissue distribution than AAV9. This improved biodistribution is attained while detargeting the liver and dorsal root ganglia (DRG), both potential sites of toxicity. Affinia’s novel capsids have favorable manufacturing yields and levels of preexisting population immunity. Preclinical efficacy and safety results with a cardiotropic vector encoding for the BAG3 protein provide proof-of-concept data to support the expansion to multiple cardiomyopathies. In addition, preclinical efficacy and safety results with a blood-brain barrier (BBB)-penetrant vector encoding for the knockdown of Ataxin-2 (Atxn2), a gene believed to be involved in sporadic ALS, provide proof of concept data to support the expansion to multiple CNS diseases.

“Compared with AAV9, our next-generation bespoke capsids demonstrate superior cardiac transduction while detargeting the liver and DRG in single-clone NHP studies. Similarly, our BBB-penetrant capsid exhibits widespread distribution across the brain with more than 50% neurons transduced, while also detargeting the liver and DRG,” said Charles Albright, Ph.D., Affinia’s Chief Scientific Officer. “The Affinia capsids are highly differentiated from conventional AAVs being used in cardiac and CNS programs currently in clinical trials, and we look forward to advancing programs with our capsids toward the clinic.”

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