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Sarepta Learns Why Earlier GMP Control Is Better For Promising Gene Therapies

 
• By Bowman Cox

When firms are developing highly anticipated gene therapies, it’s important to establish GMP-controlled manufacturing processes earlier in development, as Sarepta discovered when US FDA placed a clinical hold on a promising Duchenne muscular dystrophy gene therapy.

Scientific vector illustration genetic engineering and gene manipulation concept. DNA helix, DNA strand, molecule or atom, neurons. Abstract structure for Science or medical background.

Sarepta Therapeutics Inc.’s response to a clinical hold that the US FDA placed on an early-phase Duchenne muscular dystrophy trial illustrates the growing pressure on firms to begin using good manufacturing practices earlier in the development process – and more quickly overall – for promising cell and gene therapies.

As it responds to the GMP concerns that led to the hold, which was placed July 24, Sarepta also is...

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