This Project Grant awarded by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) provides $818,734 to The Johns Hopkins University to evaluate gene therapy approaches for treating cystic fibrosis (CF). The key objectives are to assess the safety and efficacy of delivering a truncated CFTR gene via an adeno-associated viral (AAV) vector in non-human primate and ferret models, as well as conduct a Phase I clinical trial in patients with the F508 CFTR mutation. The proposed work aims to address limitations in current treatments by developing a gene therapy to treat all mutations in the CF gene, rather than just the common F508 variant. The multi-year project from July 2024 to April 2029 will generate critical data on the pharmacology, toxicology, and therapeutic potential of this AAV-based CF gene therapy approach.
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