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Sanofi
NEWS

Sanofi Reports P-III (Baby-COMET) Trial Data on Nexviazyme for Infantile-Onset Pompe Disease (IOPD)

Shots: Sanofi has reported the P-III (Baby-COMET) trial results assessing Nexviazyme (avalglucosidase alfa; 40mg/kg, Q2W, IV) in 17 treatment-naïve pediatric pts (≤12mos.) with IOPD Trial met its 1EP, with more treatment-naïve pts (≤6mos.) alive & free of invasive ventilation at 52wks., and achieved all 2EPs, including higher survival without invasive ventilation in pts at 12…
June 30, 2026

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NEWS

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…
January 14, 2026

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NEWS

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…
December 22, 2025

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