- The Food and Drug Administration is asking Beam Therapeutics for a range of technical data on an experimental gene editing cancer therapy before allowing the company to begin human testing.
- Beam detailed the FDA’s requests in a regulatory filing Tuesday after receiving a formal letter from the agency on Aug. 25. Beam earlier this month announced the FDA had put a hold on its application to start clinical trials, but offered no details then.
- The company expects to send a response to the FDA in the fourth quarter of this year, according to the filing. The therapy, called BEAM-201, is designed to treat specific forms of leukemia and lymphoma.
FDA officials are proceeding with caution as the gene editing field advances. In another recent example, the agency had issued a partial clinical hold to Editas Medicine before this July allowing that company to proceed with testing of a sickle disease treatment.
Beam is developing therapies that use base editing, a newer gene editing technology, to modify genes more precisely than the first generation of CRISPR. Instead of cutting both strands of DNA, Beam’s experimental treatments change single “letters,” an approach that may carry fewer risks.
The company already has permission to begin human testing of its lead product, BEAM-101, for sickle cell disease and plans to enroll patients in coming months. The company also intends to seek FDA permission to begin clinical trials of a second sickle cell therapy, BEAM-102, by the end of 2022.
With BEAM-201, the company is trying to advance a method called multiplex editing, which involves editing several genetic sites at the same time in the hope of creating a stronger and more durable treatment. BEAM-201 is designed to use cells from healthy donors that would then be manipulated and infused into cancer patients.
According to Beam’s filing, the FDA asked the company for information from preclinical experiments before it’ll let the company proceed. The regulator wants more information from what Beam described as "genomic rearrangement assessments," as well as data on off-target edits and from a specific type of test called a cytokine independent growth assay. It's also looking for Beam to update the brochure given to researchers with information on any new preclinical studies.
Despite the “negative optics” of a clinical hold, the FDA’s request may end up being a good thing for Beam and for the field by helping the agency establish best practices for multiplex editing, Stifel analyst Dae Gon Ha wrote in a note to clients. The company now “has the potential of becoming the guiding light for subsequent programs and setting itself as a differentiated pioneer in this arena,” he wrote.