1. Pink Sheet
  2. >>Advanced Technologies
  3. >>Cell & Gene Therapies

Gene Therapy Sponsors Risk Additional Work With Late-Stage Manufacturing Changes

 
• By Derrick Gingery

Commercial scale-up during Phase III trials could create labeling issues and force additional clinical trials, US FDA officials said.

pharmaceutical manufacturing
An OTP official said pre-IND teleconference requests may be converted to written response only because of the agency's growing workload in the space. • Source: Shutterstock

Gene therapy sponsors risk review problems and development delays if they attempt manufacturing changes during pivotal clinical studies.

Commercial production processes should be in place for the entire Phase III trial, said Graeme Price, Gene Therapy Branch 4...

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 Cell & Gene Therapies

US FDA’s July AdComms: Pediatric Vaccine Safety, Blenrep’s Return, (Maybe) Capricor’s Cell Therapy

 
• By Sue Sutter

The Pediatric Advisory Committee will review postmarketing safety for three vaccines and the oncologic drugs panel will consider the return of GSK’s belantamab mafodotin. An FDA notice about a 30 July meeting on Capricor’s deramiocel for Duchenne muscular dystrophy was withdrawn.

Elevidys Helps Sarepta To First US FDA Platform Technology Designation

 
• By Bridget Silverman

Sarepta's rAAVrh74 vector, used in the marketed Duchenne muscular dystrophy gene therapy Elevidys and across the company's limb girdle muscular dystrophy pipeline, earned a platform designation as the lead LGMD candidate prepares for BLA filing.

HHS Secretary Favors Regenerative Medicine Over Traditional Pharma: Blessing And Curse?

 
• By Sarah Karlin-Smith

Cell and gene therapy developers may not have to contend with HHS Secretary Robert F. Kennedy Jr.’s animosity like the traditional pharma industry, but his disinterest in distinguishing between the good and bad actors in the space could backfire.

To Compete With China, US FDA Must Deregulate Early Gene Therapy Studies, CAR-T Inventor Says

 
• By Sarah Karlin-Smith

FDA regulation was painted as an obstacle to US dominance in the cell and gene therapy space even as panelists at an agency event praised the Office of Therapeutic Products' track record under Nicole Verdun.

More from Advanced Technologies

To Compete With China, US FDA Must Deregulate Early Gene Therapy Studies, CAR-T Inventor Says

 
• By Sarah Karlin-Smith

FDA regulation was painted as an obstacle to US dominance in the cell and gene therapy space even as panelists at an agency event praised the Office of Therapeutic Products' track record under Nicole Verdun.

Cell/Gene Therapy Cost Recovery Options Could Include Pre-Approval Public ‘Bridge’ Funding

 
• By Kate Rawson

Expanded funding for cost recovery could dovetail with FDA Commissioner Martin Makary’s idea for a “conditional approval” pathway based on a plausible mechanism of action.

MHRA Backs Bacteriophage Innovation To Fight Antimicrobial Resistance

 
• By Eliza Slawther

The UK regulator wants to help companies to develop bacteriophages for the treatment of infections. Its first guidance on this topic offers advice to researchers and sets out the regulatory requirements they will need to meet.