Genethon GSD1a gene therapy

Genethon is developing an investigational gene therapy for Glycogen Storage Disease Type 1a (GSD1a), a rare metabolic disorder caused by mutations in the *G6PC* gene (also known as *G6PC1*). This gene therapy aims to restore glucose-6-phosphatase (G6Pase-α or G6PC) function in the liver to prevent severe hypoglycemia and long-term complications such as liver tumors (adenomas and carcinomas). The therapy is currently in the preclinical stage, with proof of concept demonstrated in mouse models showing effectiveness in preventing hypoglycemia and reducing the occurrence of liver tumors. The development team is conducting dose-finding studies in disease models and preparing for toxicology studies to assess safety before potentially submitting a clinical trial authorization request. The therapy targets the G6PC gene product, which plays a critical role in releasing glucose into the bloodstream.

Other names

G6PC gene therapyG-6PC gene therapyG 6PC gene therapyGSD1a gene therapyGSD-1a gene therapyGSD 1a gene therapyG6PC1 gene therapyG-6PC1 gene therapyG 6PC1 gene therapy