Home Online Features Apertura Gene Therapy Supports the Broad Institute in Development of Gene Therapy for Prion Disease Using Engineered AAV Capsid Targeting TfR1 for CNS Delivery

Apertura Gene Therapy Supports the Broad Institute in Development of Gene Therapy for Prion Disease Using Engineered AAV Capsid Targeting TfR1 for CNS Delivery

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March 1, 2025

Apertura Gene Therapy Supports the Broad Institute in Development of Gene Therapy for Prion Disease Using Engineered AAV Capsid Targeting TfR1 for CNS Delivery

By Apertura Gene Therapy

The prion program utilizes Apertura's preclinical development data with an AAV capsid targeting human Transferrin Receptor 1 (TfR1) to provide a roadmap for rapid advancement to the clinicUnique gene therapy

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