NEW YORK | July 9, 2026
Apertura Gene Therapy, a biotechnology company developing next-generation adeno-associated virus (AAV) capsids for genetic medicine delivery, has announced a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Human Genome Research Institute (NHGRI) to evaluate an investigational gene therapy for Niemann-Pick Disease Type C1 (NPC1). The multi-year collaboration will leverage Apertura’s proprietary TfR1 CapX™ AAV capsid, a next-generation platform engineered to cross the blood-brain barrier (BBB) through transferrin receptor 1 (TfR1) binding, enabling broad delivery of therapeutic genes throughout the central nervous system. Financial support for the research will also be provided by the Ara Parseghian Medical Research Fund, reinforcing a collaborative effort to accelerate the development of transformative therapies for one of the world’s most devastating rare neurodegenerative disorders. The agreement represents an important milestone in advancing intravenous gene therapy strategies capable of overcoming one of the greatest challenges in neurological drug delivery.
CRADA Supports Development of Innovative Gene Therapy for NPC1
The newly established Cooperative Research and Development Agreement (CRADA) will focus on evaluating the systemic intravenous delivery of an investigational AAV gene therapy using TfR1 CapX™, Apertura’s proprietary second-generation capsid technology specifically designed to achieve efficient penetration of the blood-brain barrier. Research activities will concentrate on preclinical development of a therapeutic construct targeting NPC1, with the objective of demonstrating effective gene delivery throughout the brain and spinal cord while preparing the program for future regulatory review, manufacturing, pre-Investigational New Drug (pre-IND) development, and eventual clinical evaluation.
Investigators from NICHD and NHGRI will assess whether systemic administration of the gene therapy can significantly slow neurodegeneration associated with Niemann-Pick Disease Type C1, potentially providing a safer and less invasive alternative to direct central nervous system administration. Should early findings prove encouraging, the collaboration may expand into broader preclinical studies supporting future clinical translation.
TfR1 CapX™ Platform Strengthens Next-Generation CNS Gene Delivery
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At the center of the collaboration is TfR1 CapX™, Apertura Gene Therapy’s advanced AAV capsid platform engineered to utilize the human transferrin receptor 1 (TfR1) for efficient transport across the blood-brain barrier following intravenous administration. The proprietary platform represents a significant advancement over first-generation capsid technologies by enabling broader and more efficient delivery of therapeutic genes throughout the central nervous system while reducing the complexity associated with invasive delivery approaches.
Clinical readiness for TfR1 CapX™ has already been supported through multiple preclinical development programs, regulatory engagement activities, and manufacturing initiatives involving specialized Contract Development and Manufacturing Organizations (CDMOs). Multiple commercial and non-profit organizations have already validated and licensed the technology, reflecting growing confidence in its potential to support treatments for a wide range of neurological, neurodegenerative, and genetic disorders requiring efficient central nervous system gene delivery.
Accelerating Rare Disease Innovation Through Strategic Collaboration
Niemann-Pick Disease Type C1 (NPC1) is a rare, inherited lysosomal storage disorder caused by mutations in the NPC1 gene, leading to abnormal cholesterol accumulation, progressive neurodegeneration, cerebellar ataxia, dementia, liver dysfunction, and frequently premature death during childhood or adolescence. Existing therapeutic approaches provide only modest clinical benefit, highlighting the urgent need for innovative treatment strategies capable of addressing the underlying genetic cause of the disease.
Through this strategic collaboration, Apertura Gene Therapy, NICHD, NHGRI, and the Ara Parseghian Medical Research Fund aim to accelerate development of a next-generation intravenous gene therapy that could fundamentally improve treatment outcomes for patients living with NPC1. The agreement also reinforces Apertura’s growing leadership in AAV vector engineering, blood-brain barrier-targeting technologies, and precision gene therapy, positioning the company to expand clinical applications of TfR1 CapX™ across multiple serious neurological diseases while advancing the future of genetic medicine through innovative delivery technologies and collaborative scientific research.
Source: Apertura Gene Therapy press release
