Shots:
- Genethon has entered into an exclusive, worldwide licensing agreement with AskBio for the use of a patented component of AskBio’s investigational gene therapy AB-1009 for the treatment of Pompe disease
- The licensed component is based on a transgene encoding a truncated form of GAA, developed by Genethon, which demonstrated preclinical efficacy in correcting glycogen accumulation in muscle & CNS, and improving cardiac, muscular, and respiratory function
- AskBio plans to advance clinical development of the gene therapy, with first patient enrollment anticipated in early 2026
Ref: Business Wire | Image: Genethon | Press Release
Related News: Mereo BioPharma Reports P-III (ORBIT & COSMIC) Trials Findings on Setrusumab in Osteogenesis Imperfecta
PharmaShots, your go-to media platform for customized news ranging across multiple indications. For more information, connect with us at connect@pharmashots.com
