MUMBAI, India, Nov. 2 -- Intellectual Property India has published a patent application (202617094925 A) filed by The Broad Institute, Inc. on August 05, 2026, for Aav Capsid Modifications That Enable Improved Cns-Wide Gene Delivery Through Interactions With The Transferrin Receptor.
Inventors include Deverman, Benjamin; Chan, Ken; Huang, Qin; Barry, Andrew; and Eid, Fatmaelzahraa Sobhy Abdelmouty.
The application for the patent was published on October 30, 2026, under issue no. 44/2026.
Abstract: Developing vehicles that efficiently deliver genes throughout the human central nervous system (CNS) will broaden the range of treatable genetic diseases. Applicants engineered an AAV capsid, BI-hTFRl, that binds human Transferrin Receptor (TfRl), a protein expressed on the blood-brain barrier (BBB). BI-hTFRl was actively transported across a human brain endothelial cell layer and, relative to AAV9, provided 40-50 times greater reporter expression in the CNS of human TFRC knock-in mice. The enhanced tropism was CNS-specific and absent in wild type mice. When used to deliver GBA1, mutations of which cause Gaucher disease and are linked to Parkinson's disease, BI-hTFRl substantially increased brain and cerebrospinal fluid glucocerebrosidase activity compared to AAV9.
Disclaimer: Curated by HT Syndication.
