NEW YORK, N.Y., July 23, 2026
Apertura Gene Therapy has announced its participation in a consortium selected to receive funding through the Advanced Research Projects Agency for Health (ARPA-H) THRIVE Program, a major U.S. initiative designed to accelerate the development of gene therapies for rare childhood diseases. Led by the Broad Institute of MIT and Harvard’s Center for Therapeutic Genetics, the consortium will leverage Apertura’s TfR1 CapX™, a next-generation central nervous system (CNS)-targeted adeno-associated virus (AAV) capsid, to support advanced base editing and prime editing programs for rare neurological disorders. The collaboration aims to establish a shared development platform that streamlines manufacturing, regulatory pathways, and clinical infrastructure, enabling researchers to accelerate innovative treatments for children affected by devastating rare CNS diseases and developmental epileptic encephalopathies. The initiative highlights the growing role of collaborative gene therapy ecosystems in advancing precision medicine for underserved patient populations.
ARPA-H Consortium Targets Rare Childhood CNS Disorders
The ARPA-H THRIVE Program has assembled a multidisciplinary consortium consisting of academic researchers, biotechnology companies, clinicians, and patient advocacy organizations to establish the Pediatric Epilepsies and Rare CNS (PERC) Gene Editing Platform. Rather than developing therapies individually for hundreds of rare disorders, the consortium will create a shared infrastructure that enables multiple disease programs to utilize common manufacturing capabilities, regulatory expertise, and clinical development resources. This collaborative approach is expected to accelerate the delivery of gene therapies for developmental and epileptic encephalopathies, a group of severe neurological disorders affecting more than three million children worldwide and associated with mutations across more than 400 different genes. By reducing duplication across development programs, the initiative seeks to overcome one of the most significant challenges in rare disease research while improving access to advanced genetic medicines for patients with limited treatment options.
TfR1 CapX Advances Next-Generation Gene Editing Delivery
At the center of Apertura’s contribution is TfR1 CapX™, a proprietary second-generation AAV capsid engineered to target human transferrin receptor 1 (hTfR1), enabling intravenous delivery across the blood-brain barrier and broad distribution throughout the brain and spinal cord. Originally engineered by researchers at the Broad Institute, the capsid is designed to improve delivery efficiency for advanced base editing and prime editing technologies developed by the laboratory of David Liu, Ph.D. The consortium will combine this innovative delivery platform with cutting-edge gene editing tools to develop treatments for a broad range of rare central nervous system disorders. Manufacturing support will be provided by Viralgen, a specialized contract development and manufacturing organization (CDMO) with extensive expertise in recombinant AAV gene therapy production, helping ensure scalable manufacturing capabilities as candidate therapies advance toward clinical development.
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Partnership Strengthens Apertura’s Gene Therapy Pipeline
Participation in the ARPA-H THRIVE consortium further strengthens Apertura Gene Therapy’s strategy of expanding access to TfR1 CapX™ across the gene therapy industry. The company has already established multiple licensing agreements for its proprietary CNS-targeted capsid technology, with several partner programs expected to enter clinical trials within the next 12 months. By supporting collaborative research alongside leading academic institutions, biotechnology innovators, manufacturing specialists, and patient advocacy organizations, Apertura is helping advance a new generation of precision gene therapies capable of treating previously inaccessible neurological disorders. As investment in gene editing, AAV vector engineering, and rare disease therapeutics continues to accelerate, the consortium represents an important milestone in building scalable platforms that can rapidly translate breakthrough scientific discoveries into life-changing treatments for children with rare genetic diseases.
Source: Apertura Gene Therapy press release
