The recommendations focus on using next-generation sequencing to evaluate off-target editing risks and chromosomal integrity.
The Food and Drug Administration (FDA) issued draft guidance for sponsors seeking approval of human gene therapy products that use genome editing technologies. When finalized, the guidance will provide recommendations for standardized methods for assessing the safety of genome editing therapies.
“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,” says Marty Makary, MD, MPH, FDA commissioner, in a release. “This guidance provides sponsors with clear, scientifically-grounded recommendations for evaluating off-target editing risks using state-of-the-art sequencing technologies.”
The draft guidance, issued by the Center for Biologics Evaluation and Research, supports the FDA framework for accelerating the development of individualized therapies for ultra-rare diseases. The document provides recommendations on sequencing strategies, sample selection, analysis parameters, and reporting.
The guidance builds on previous agency documents and focuses specifically on the use of next-generation sequencing-based methods to evaluate safety risks associated with off-target editing and loss of genome integrity. These recommendations are intended to support nonclinical studies submitted with investigational new drug (IND) applications and biologics license applications. The guidance applies to both ex vivo products, where cells are edited outside the body, and in vivo products, where editing occurs within the patient’s tissues.
“Next-generation sequencing not only detects off-target editing and assesses chromosomal integrity; it also requires science-based recommendations for its use. We’re giving sponsors a roadmap for comprehensive safety assessment while supporting the efficient development of these promising therapies,” says Vinay Prasad, MD, MPH, chief medical and scientific officer and Center for Biologics Evaluation and Research director, in a release.
The agency encourages sponsors to engage early in product development through Initial Targeted Engagement for Regulatory Advice on CBER/CDER Products (INTERACT) and pre-IND meetings to discuss specific development strategies. The draft guidance is available for public comment for 90 days following its publication in the Federal Register.
ID 213642156 © Kts | Dreamstime.com
Related Reading:
Watchmaker Genomics Licenses CRISPR Technology to Improve DNA Sequencing Efficiency
Baylor Genetics Adds Optical Genome Mapping and Long-Read Sequencing to Whole Genome Sequencing Test
Cirena Launches Long RNA Synthesis Platform for CRISPR Applications
