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Genethon Enters Licensing Agreement with AskBio to Develop Gene Therapy for Pompe Disease.
Jan 14, 2026
Contract Type
agreement
RomeBiotechnologyHealth CareGeneticsTherapeutics
Description
Genethon has signed an exclusive worldwide licensing agreement with AskBio for the use of a patented component of AB-1009 to treat Pompe disease. This collaboration aims to leverage Genethon's research capabilities to develop innovative therapeutic solutions for patients suffering from rare diseases.
Company Information
Company
Genethon
Location
Rome, Lazio, Italy
About
Généthon is a pioneer in the discovery and development of gene therapies for rare diseases, established by the AFM-Téléthon. The organization employs over 200 scientists and professionals dedicated to developing innovative therapies that improve the lives of patients with rare genetic diseases. It is known for its contributions to the field, including the first gene therapy for spinal muscular atrophy. Currently, Genethon has multiple products in clinical trials targeting various conditions.