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ASGCT Late Breakers: Pfizer’s Duchenne Results Underwhelm As Next-Gen Approaches Emerge

Sarepta Set For Landmark Approval

 
• By Andrew McConaghie

The congress’s late breakers highlight progress across cell and gene platforms and diseases, and Duchenne is in the spotlight as Sarepta’s SRP-9001 could soon gain accelerated approval.

LA Convention Center
An aerlal view of the L.A. Convenstion Center, the venue for this year's ASGCT annual congress. • Source: Shutterstock

New data from Pfizer’s gene therapy for Duchenne muscular dystrophy suggest it may struggle to rival Sarepta’s candidate, which looks set to gain US approval within weeks.

New two-year data on children treated with PF-06939926 (fordadistrogene movaparvovec) in a Phase Ib study will be presented as a late-breaker at the American Society of Gene and

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Pipeline Watch: Eight Approvals And Nineteen Phase III Readouts

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.