1. Pink Sheet
  2. >>Compliance

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 laying groundwork to use the next cohort of the Phase...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Manufacturing

More from Compliance

Austria To Fine Companies For Violating New Drug Stockpiling Rule

 
• By Neena Brizmohun

A new ordinance for addressing drug shortages in Austria requires drug companies distributing any of the hundreds of products listed in an accompanying annex to maintain adequate stock in the country to meet patient demand for four months.

US FDA’s Expanded Surprise Foreign Inspections: Impact And Enforcement Hoops

 
• By Anju Ghangurde

As the US FDA expands unannounced foreign inspections building on pilots in India and China, experts expect higher scrutiny of overseas sites and perhaps even an industry shakeout in the longer term, though staffing and enforcement challenges could slow things down.

Pharma Industry Argues Tax, Other Incentives Better Than Tariffs

 
• By Jessica Merrill

Pharmaceutical industry organizations offered alternatives to tariffs that could maintain a secure domestic supply chain in public comments filed in response to a federal 232 investigation.