Hunterian Medicine CFTR gene therapy

Hunterian Medicine is developing a CRISPR-based gene therapy for the treatment of cystic fibrosis. The program utilizes a proprietary platform featuring compact, bidirectional promoters that enable the packaging of large genetic payloads—such as the full-length cystic fibrosis transmembrane conductance regulator (CFTR) gene or CRISPR-Cas9 components—into a single adeno-associated virus (AAV) vector. This approach aims to replace, restore, or repair the CFTR gene to address the underlying cause of the disease. The project is supported by the Cystic Fibrosis Foundation's "Path to a Cure" initiative and is currently in preclinical development.