FDA's Marks Sees 'Two Or Three Year Window' To Transform Gene Therapy Environment

Agency has to ‘think creatively’ to make gene therapies commercially viable, CBER director says, suggesting greater use of accelerated approval endpoints and advancing manufacturing technologies. Center is focused on recruiting mid-level career people with manufacturing experience.

Gene therapy
FDA is focusing on ways to accelerate development of gene therapies • Source: Shutterstock

Peter Marks, director of the US Food and Drug Administration’s Center for Biologics Evaluation and Research, says he hears from foundations every week who are trying to develop a gene therapy but find the cost is in the $250,000 cost per dose range or higher.

“That essentially puts it out of reach,” he said. “So we’ve got to do something here

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

More from US FDA

US FDA COVID-19 Booster Strategy May Not Be As Controversial As It Seems

 

US FDA Commissioner Martin Makary’s call for placebo-controlled trials to confirm the efficacy of COVID-19 boosters is drawing a lot of criticism, but former Commissioner Robert Califf also supported the idea.

US FDA Legislative Lead Calls For User Fee Reforms As Renewal Talks Near

 

Deputy FDA Commissioner Grace Graham acknowledged the importance of user fees, while also calling for restructuring. She also said part of MAHA's mission is to reexamine uses of drugs not supported by data.

Califf In Conversation: ‘Decimated’ US FDA Teams Endanger Innovation

 

Pink Sheet sister publication In Vivo spoke to the former FDA commissioner about the recent layoffs and their impact on vaccine and other product development.

US FDA Vaccine Framework Coming In Weeks, But Details Vague

 

New CBER Director Prasad will "unleash a massive" framework on vaccines, FDA Commissioner Marty Makary said 15 May, raising questions about the impact on products nearing approval.

More from Agency Leadership