Genethon Signs Worldwide Licensing Agreement with AskBio (Bayer AG) to Advance Gene Therapy for Pompe Disease

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…

ByPrince GiriJanuary 14, 2026

BioMarin to Acquire Amicus Therapeutics for ~$4.8B

Shots: BioMarin has entered into a definitive agreement to acquire Amicus, strengthening BioMarin's commercial portfolio with Galafold (migalastat) for Fabry Disease & Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) for Pompe Disease As per the deal, BioMarin will acquire Amicus for $14.5/share in an all-cash transaction for a total equity value of ~$4.8B; closing is expected in Q2’26 Amicus also…

ByRidhi RastogiDecember 22, 2025

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

Shots: With the healthcare sector moving ahead with its technological advances, there is still an unexplored section in therapeutics that requires much more attention. Through innovations, life science companies have evolved to make breakthrough discoveries in many focus areas, but when it comes to the rare disease segment, all our endeavors seem to be bleak…

BySaurabh ChaubeyMarch 14, 2023

Genethon Signs Worldwide Licensing Agreement with AskBio (Bayer AG) to Advance Gene Therapy for Pompe Disease

BioMarin to Acquire Amicus Therapeutics for ~$4.8B

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

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