July 23, 2026 —
Apertura Gene Therapy announced its participation in a consortium selected to receive funding from the ARPA-H THRIVE program to develop gene therapies for rare childhood diseases.
The consortium is led by the Broad Institute of MIT and Harvard’s Center for Therapeutic Genetics and will focus on building a shared platform for rare central nervous system diseases. Apertura will contribute TfR1 CapX™, its intravenously administered, blood-brain barrier-crossing AAV capsid, for use in base and prime gene editing programs.
The consortium will work to create the Pediatric Epilepsies and Rare CNS Gene Editing Platform, or PERC, designed to share manufacturing, regulatory precedent, and clinical infrastructure across multiple disease programs. The goal is to develop genetic medicines faster than would be possible using traditional one-disease-at-a-time approaches.
Developmental and epileptic encephalopathies affect more than three million children worldwide and can be caused by mutations across more than 400 genes. This genetic diversity makes traditional drug development difficult, especially for ultra-rare CNS conditions with small patient populations.
TfR1 CapX is designed to target human transferrin receptor 1, or hTfR1, cross the blood-brain barrier after intravenous administration, and enable broad delivery to the brain and spinal cord. The capsid was initially engineered by Ben Deverman, PhD, and colleagues.
Within the consortium, TfR1 CapX will be paired with base editing and prime editing technologies developed in the laboratory of David Liu, PhD, at the Broad Institute. These programmable editing technologies may enable mutation-specific approaches for rare pediatric neurogenetic diseases.
The consortium includes academic researchers, clinicians, patient advocacy organizations, and biotechnology companies. By working directly with advocacy groups and sharing development infrastructure across programs, the initiative aims to close a major gap in therapeutic development for rare childhood CNS diseases.
Apertura said the project deepens its commitment to making TfR1 CapX broadly available across the field. The company has signed multiple licensing agreements for TfR1 CapX programs, several of which are expected to enter clinical trials within the next 12 months.
TfR1 CapX is a proprietary second-generation capsid designed to improve CNS delivery compared with Apertura’s first-generation BI-hTFR1 capsid. The first-generation capsid was previously described in Science. As BBB-crossing AAV technologies advance, programs such as PERC may help define how systemic CNS gene editing platforms can be developed for highly heterogeneous rare diseases.
