Apertura Gene Therapy Named to Consortium Receiving Funding from the Advanced Research Projects Agency for Health (ARPA-H) THRIVE Program to Develop Gene Therapies for Rare Childhood Diseases

—Consortium led by the Broad Institute's Center for Therapeutic Genetics to use Apertura's novel CNS-targeted AAV capsid, TfR1 CapX™, for base and prime gene editing programs—

NEW YORK, July 23, 2026 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, today announced participation in a consortium selected to receive funding from the Advanced Research Projects Agency for Health (ARPA-H) THRIVE program to develop gene therapies for rare childhood diseases. THRIVE is led by ARPA-H Program Manager Daria Fedyukina, Ph.D. The consortium, led by the Broad Institute of MIT and Harvard's Center for Therapeutic Genetics, will use Apertura's novel central nervous system-targeted AAV capsid, TfR1 CapX™, for base and prime gene editing programs.

Composed of academic researchers, clinicians, patient advocates, and biotechnology companies, the consortium will work to create a Pediatric Epilepsies and Rare CNS (PERC) Gene Editing Platform that shares manufacturing, regulatory precedent, and clinical infrastructure across diseases. The goal of the consortium is to address a gap in development efforts and develop therapies faster than is currently possible for patients with a wide range of rare central nervous system diseases.

"More than three million children worldwide are affected by developmental and epileptic encephalopathies that are caused by mutations across more than 400 different genes, making it difficult to develop therapies for these diseases with traditional approaches," said Andrew Steinsapir, Acting Chief Technology Officer at Apertura and Gene Therapy Program Lead at Deerfield Management. "By working directly with patient advocacy organizations and sharing drug development infrastructure across programs, the consortium is taking an innovative approach to advancing treatments for a wide range of neurogenetic disorders."

TfR1 CapX was initially engineered by Ben Deverman, Ph.D., Senior Director, Vector Engineering and an Institute Scientist at the Broad Institute, and colleagues. The capsid is administered intravenously and designed to target human transferrin receptor 1 (hTfR1), cross the blood-brain barrier, and enable broad distribution to the brain and spinal cord.

TfR1 CapX will be paired with base and prime gene editing technologies developed in the lab of David Liu, Ph.D., Richard Merkin Professor and Director of the Merkin Institute for Transformative Technologies in Healthcare, Core Institute Member, and Director of the Chemical Biology and Therapeutic Sciences Program at the Broad Institute.

Manufacturing support for the consortium will be provided by Viralgen, a leading contract development and manufacturing organization (CDMO) specializing in recombinant adeno-associated virus (rAAV) gene therapies. "Viralgen has extensive experience working with partners to manufacture AAV gene therapies, and we look forward to supporting the consortium as it advances therapies for rare CNS diseases," said Andy Holt, Chief Commercial Officer at Viralgen.

This project deepens Apertura's commitment to providing TfR1 CapX broadly across the industry. Apertura has signed multiple licensing agreements for TfR1 CapX programs, several of which are expected to enter clinical trials within the next 12 months.

About TfR1 CapX™

TfR1 CapX™ is a leading IV-administered, BBB-crossing capsid. Multiple for-profit and non-profit organizations have validated and licensed TfR1 CapX, and other groups are in discussions to license the technology. Clinical readiness has been supported by several preclinical development programs, including regulatory engagement and manufacturing by contract development and manufacturing organizations (CDMOs). TfR1 CapX is a proprietary, second-generation capsid that demonstrates superior CNS delivery compared to Apertura's first-generation capsid, BI-hTFR1. Research on the first-generation BI-hTFR1 capsid was published in Science.

About Apertura Gene Therapy

Apertura Gene Therapy develops genetic medicines and next-generation AAV capsids that engage human-relevant receptors, aiming to enable more effective and selective gene delivery. The company's lead capsid, TfR1 CapX™, leverages human transferrin receptor 1 to enable intravenous delivery to the brain and spinal cord. This established transport mechanism has a strong clinical track record in pediatric and geriatric populations, expanding its potential to treat serious neurological and genetic diseases. Apertura has licensed its next-generation capsids to multiple partners, with several programs expected to enter clinical trials over the next 12 months. Founded in 2021 on technology from the Broad Institute and supported by Deerfield Management, Apertura Gene Therapy is headquartered in New York City. Learn more at aperturagtx.com and follow us on LinkedIn.

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SOURCE Apertura Gene Therapy