GENV-002

GENV-002 is a gene editing therapy developed for the treatment of both infantile-onset and late-onset Pompe disease. It utilizes an adeno-associated virus (AAV) delivery system in combination with CRISPR-Cas9 gene editing to integrate a healthy donor acid-alpha-glucosidase (GAA) gene into liver cells, creating a liver depot that enables sustained enzyme production. This approach aims to overcome the limitations of traditional AAV therapies, which do not integrate into host DNA and may lose efficacy over time. By enabling early intervention—including in infants—GENV-002 offers the potential for a universal, potentially curative treatment for all forms of Pompe disease, addressing significant unmet medical need where current enzyme replacement therapies are burdensome and non-curative[1][2][3]. Developers: GeneVentiv Therapeutics, Duke University