The draft guidance supports the agency’s new pathway designed to speed up the development of custom gene therapies.
The FDA has unveiled new draft guidance for gene editors, specifically those using next-generation sequencing techniques to pinpoint unintentional consequences associated with certain gene therapies.
The safety guidance is intended to help standardize approaches that evaluate the safety of genome editing therapies, with the ultimate goal of getting novel treatments to patients faster, according to the agency—including individualized therapies for ultra-rare diseases.
Issued by the FDA’s Center for Biologics Evaluation and Research, the draft builds on guidance from early 2024 for developing therapeutics with human genome editing tools and encompasses both ex vivo and in vivo products.
The new guidance recommendations are designed to support nonclinical studies accompanying investigational new drug and biologics license applications, and include suggestions for sequencing strategies, sample selection and analysis parameters.
“We’re giving sponsors a roadmap for comprehensive safety assessment while supporting the efficient development of these promising therapies,” outgoing CBER Director Vinay Prasad said in a statement Tuesday.
“Our goal is to work collaboratively with the scientific community to bring safe and effective genome editing therapies to patients who need them most,” said Prasad, who will be departing the agency at the end of the month after a controversial—and intermittent—tenure with the regulator.
The FDA is asking for public comment on the draft guidance.
The guidelines follow the FDA’s February announcement regarding a new pathway designed to accelerate the development of bespoke therapies for ultra-rare diseases. The approach formalizes the approach used to create the first custom CRISPR therapy for baby KJ, a young boy born with a rare metabolic condition. The framework focuses on gene editing and RNA-based treatments.
“Genome editing holds extraordinary promise for treating previously incurable genetic diseases, and today’s announcement represents the FDA’s forward approach to drive innovation and advance the development of genome editing therapies,” FDA Commissioner Marty Makary said in Tuesday’s statement.
“This guidance provides sponsors with clear, scientifically-grounded recommendations for evaluating off-target editing risks using state-of-the-art sequencing technologies,” Makary added. “We are serious about moving this ball forward.”
While the agency unveiled a new approach for certain individualized therapies, the regulator has been under fire in recent days for several rare disease decisions. Critics have pointed to surprise regulatory decisions that went against earlier feedback from the FDA and a lack of advisory committee meetings.
