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WALTHAM, Mass., May 13, 2021 (GLOBE NEWSWIRE) Affinia Therapeutics, an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and non-rare diseases, today announced results from preclinical studies presented in poster and oral sessions at the American Society of Gene and Cell Therapy (ASGCT) 24
Challenges in achieving adequate expression in the central nervous system (CNS) with conventional AAV vectors have limited the use of gene therapy to address many devastating rare and non-rare CNS diseases. Affinia Therapeutics presented data in a poster session on its proprietary AAV vector Anc80L65 showing superiority to AAV9 in various non-human primate (NHP) CNS regions after a single intrathecal injection. When delivered via lumbar puncture, Anc80L65 demonstrated up to 32-fold higher RNA expression versus AAV9 delivered via the more invasive intracisternal route of administration. An