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Gene Therapy Manufacturing Hurdle: Sponsors Unwilling To Share ‘Secret Sauce’

 
• By Brenda Sandburg

US FDA’s Peter Marks says companies are not exchanging information about adenoviral factors, which is probably slowing down the field. He describes NIH's plans for a bespoke gene therapy consortium for vector generation and continued efforts for regulatory harmonization.

Secret sauce of Gene therapy
Gene therapy companies are not exchaning manufacturing information they regard as secret sauce

The willingness of biopharma companies to share information can be crucial in advancing treatments in a new field. That's the message that the US Food and Drug Administration's Peter Marks conveyed in a meeting on the current state of gene therapy.

Marks, director of FDA's Center for Biologics Evaluation and Research, said the biggest sticking point in producing gene therapy products is manufacturing, and one of the challenges in

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Keep Talking: US FDA’s Beleagured Biologics Center Remains Vital To Regenerative Medicine

 
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Advisory committee for Capricor’s deriamocel planned, regenerative medicine advanced therapy designations proliferate for osteoarthritis, and the ranks of dual RMAT/breakthrough therapy designation holders grow.

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Potency Assay Issues Delayed US FDA Approval Of Mesoblast’s Ryoncil For Years

 
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Sponsors Like START Rare Disease Pilot, Will Prasad Maintain Its Momentum?

 
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What The EMA Can Teach HTA Bodies About Joint Clinical Assessments

 
• By Francesca Bruce

EU-level joint clinical assessments conducted under the Health Technology Assessment Regulation need to be more flexible when it comes to evidence requirements, according to experts speaking at a gene and cell therapy conference.

Canada’s HTA Agency Reveals How To Submit AI-Driven Evidence

 
• By Neena Brizmohun

Recognizing that the evidence it receives in applications for health technology assessments will increasingly be informed by artificial intelligence, the CDA-AMC has clarified its expectations for companies that use AI methods in the generation and/or reporting of evidence.