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NIH grant 1R43HD121137-01: Development of a PET Imaging Platform to De-Risk AAV Gene Therapy Clinical Trials
Institute: {'code': 'HD', 'abbreviation': 'NICHD', 'name': 'Eunice Kennedy Shriver National Institute of Child Health and Human Development', 'admin_org_id': '12500030', 'admin_funding_url': ''}. PI: Yaniv Tivon. Award: $306395.
Start: 2026-09-25T00:00:00.
Abstract
Genetic diseases affect 1% of live births. Although each individual disorder is rare, collectively they affect millions
of Americans. Adeno-associated virus (AAV) gene therapies treat genetic disorders by hijacking a virus’
machinery to deliver a therapeutic transgene to patients. The transgene expresses an essential protein that may
be missing or defective in patients inflicted by 1 of the 6,000 known genetic disorders. Despite this exciting
breakthrough, limited knowledge of AAV pharmacokinetics leads to a critical barrier—high clinical development
risk, hindering the development of new therapies.
We propose to monitor AAV gene therapy pharmacokinetics using a common imaging modality called positron
emission tomography (PET) to provide early signs of success/failure in large animal models and early clinical
trials, thereby mitigating risks of resource-intensive late-stage clinical trials. To achieve this, a novel PET reporter
gene will be developed and co-delivered with the therapeutic transgene by AAVs, enabling non-invasive
transgene biodistribution and persistence tracking over time.
Specific aims include: 1) PET reporter optimization and 2) PET reporter evaluation in vivo. Successful completion
will pave the way for the development of a clinical platform that improves AAV gene therapy clinical trial success
rates.document fetched 2026-10-04 02:44