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PTC’s Upstaza Takes Gene Therapy Straight To Brain (And US FDA); Translarna To Return Mid-Year

 
• By Bridget Silverman

In a year full of regulatory milestones for the firm, PTC Therapeutics hopes to set some approval precedents – and basically hopes its candidates have an easier time of it than they have had before.

brain straightened out
• Source: Shutterstock

PTC Therapeutics, Inc. sees a year full of regulatory milestones ahead in 2024 following its biologics license application for Upstaza, which could become the first gene therapy delivered directly into deep gray matter of the brain.

PTC announced the submission of the Upstaza BLA for the rare genetic central nervous system disorder aromatic L-amino acid decarboxylase (AADC) deficiency on 19 March 2024. With priority review, Upstaza would hold a likely user fee goal date in November

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