Genethon and AskBio Enter Global Licensing Agreement to Advance AAV Gene Therapy for Pompe Disease

PARIS-(BUSINESS WIRE)-January 14 2026- Genethon, a leading laboratory in gene therapy for rare diseases, has entered into an exclusive, worldwide licensing agreement with AskBio, a gene therapy company wholly owned by Bayer AG, for the use of a patented component of AB-1009 in the treatment of Pompe disease. The agreement supports the continued development of an adeno-associated virus (AAV)–based gene therapy for this severe inherited disorder.

Pompe disease is a rare genetic condition caused by deficiency of the acid alpha-glucosidase (GAA) enzyme, resulting in pathological glycogen accumulation in cells, particularly affecting skeletal muscle, cardiac muscle, and the nervous system. Despite existing therapies, significant unmet medical need remains, especially in severe infantile and late-onset forms of the disease.

The licensed technology originates from Genethon’s Immunology and Liver Diseases team, led by Giuseppe Ronzitti, which developed a novel AAV gene therapy approach using a transgene encoding a truncated form of GAA. Preclinical studies demonstrated that AAV-mediated gene delivery corrected glycogen accumulation in muscle and the central nervous system, improved cardiac hypertrophy, and ameliorated muscular and respiratory dysfunction. This AAV gene therapy strategy leverages liver-directed AAV delivery to enable systemic secretion of functional GAA.

The patented AAV gene therapy component, protected by Genethon and other French research institutions, is now licensed to AskBio for clinical development as part of its AB-1009 program. AskBio plans to advance this AAV-based gene therapy toward the clinic, with first patient recruitment anticipated in early 2026.

The collaboration highlights Genethon’s role in translating foundational AAV gene therapy research into industry-partnered clinical programs, reinforcing the growing momentum of AAV-mediated gene replacement strategies for rare neuromuscular and metabolic diseases such as Pompe disease.