Affinia Therapeutics Presents New Data at American Society of Gene and Cell Therapy Annual Meeting
Data show pretreatment with IVIG can limit biodistribution of AAV9 vectors to peripheral tissues following direct CNS administration
WALTHAM, Mass., May 16, 2022 /PRNewswire/ -- 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 prevalent devastating diseases, today announced results from preclinical studies presented in an oral session at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting.
Direct administration of AAV vectors to the central nervous system (CNS) can increase transduction and expression in the CNS. However, even with direct administration, vector "escapes" the CNS and distributes to peripheral organs. In nonhuman primates, AAV9 administration to cerebrospinal fluid (CSF) is seen in peripheral organs, including the liver.
In an oral presentation later today, Affinia Therapeutics will present data showing that in mice, administration of intravenous immunoglobulin (IVIG), a widely available therapy containing antibodies to antigens on a wide spectrum of AAV serotypes, prior to AAV9 administration to the CNS, can reduce the presence of vector and vector-expression in peripheral organs, including the liver, kidney, and heart. Importantly, pretreatment with IVIG does not appear to change transduction or expression in the CNS.
"These data provide a signal that IVIG may be an additional tool to more precisely target AAV vector delivery. We believe the findings reported today will help us develop and deliver AAV gene therapies that specifically target diseased tissues while limiting off-target effects," said Laura Richman, chief development officer for Affinia Therapeutics.
Title: Pretreatment with IVIG reduces peripheral transduction of AAV9 delivered to the CNS
About Affinia Therapeutics
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SOURCE Affinia Therapeutics