Homology Medicines Announces Peer-Reviewed Publication on Novel Discovery of AAVHSC with Robust Distribution to the Central Nervous System and Peripheral Organs with Low Affinity for the Liver
AAVHSC16 Biodistribution Properties in Preclinical Models Demonstrated Potential for Systemic Delivery of Genetic Medicines to Brain, Heart and Muscle
Excerpt from the Press Release:
BEDFORD, Mass., July 05, 2022 (GLOBE NEWSWIRE) — Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the peer-reviewed publication of data showing that AAVHSC16, one of the capsids in its family of 15 naturally occurring AAVHSCs, demonstrated low levels of tropism to the liver while maintaining robust distribution to the central nervous system (CNS) and peripheral organs following a single I.V. administration in preclinical models. The Company believes that its unique properties, with high levels of tropism to the brain, heart and muscle, and no elevations in liver enzymes, could make AAVHSC16 an attractive capsid for new disease indications with Homology’s genetic medicines platform.
“Our ongoing efforts to fully characterize our family of 15 naturally occurring AAVHSCs as it relates to biodistribution, tissue tropism and the role different features of the capsids play, continues to reveal their unique profiles that allow us to best select capsids for different diseases,” said Albert Seymour, Ph.D., President and Chief Scientific Officer of Homology Medicines. “In the case of AAVHSC16 with its ability to reach key tissues without targeting the liver in preclinical models, we can potentially expand into additional disease areas where we want to deliver to the CNS, cardiac tissue, or muscle while avoiding exposure in the liver. By continuing to publish our discoveries about the unique structure and function of our AAVHSCs, we believe we can contribute to the field’s greater understanding and development of AAV-based therapies that will ultimately benefit more patients.”
Homology’s AAVHSC capsids differ from each other by one to four amino acids, resulting in differences in biodistribution and transduction efficiencies.
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