The Food and Drug Administration has cleared Beam Therapeutics to move forward with human testing of a gene editing cancer medicine, the company said Friday.
Beam, a pioneering developer of a precise gene editing technique known as base editing, said in a short statement that the regulator is allowing it to proceed with clinical trials of the treatment, known as BEAM-201.
Beam didn’t specify when studies will begin. In the statement, CEO John Evans said the company will detail the program’s next steps in 2023.
The FDA’s decision is notable as it represents the first time the regulator has permitted human testing of a cancer cell therapy involving “multiplex editing,” in which several genes are edited simultaneously. Beam claims this approach with BEAM-201 could lead to a stronger and more durable treatment than other technologies.
“We believe the future of cell therapy involves high levels of cell engineering, enabled by multiplex base editing technology,” Evans said in the statement.
BEAM-201 was supposed to enter clinical testing this year, but the regulator paused development in August and asked for a variety of technical information from preclinical experiments before the company could proceed. Among those requests were what Beam described as “genomic rearrangement assessments,” and data on off-target edits, a safety concern with gene editing treatments.
Beam received a formal letter from the FDA in early September and responded in November. That the regulator quickly cleared Beam to start testing shows its “comfort towards multiplex editing,” wrote Stifel analyst Dae Gon Ha. That’s important as diseases caused by a single mutation “likely represent just the tip of the iceberg” for the company, he added.
Other experimental Beam treatments for sickle cell disease and hepatitis B infections also involve multiplex editing.
“Naturally, multiplex editing raises the safety bar as any safety considerations can be multiplied,” Ha wrote. Beam’s announcement “reassures us that no unwanted or unanticipated effects were observed thereby favoring base editing use in tackling genetic mutations — possibly even multiple ones simultaneously.”
The FDA has closely scrutinized genetic medicines in recent years. The regulator previously halted applications of gene editing drugs from partners Vertex Pharmaceuticals and CRISPR Therapeutics as well as from Editas Medicine, before allowing their trials to start. In November, the agency also stopped Beam partner Verve Therapeutics from beginning a U.S. study of a base editing treatment for heart disease.
Though BEAM-201 and Verve’s programs are different, the regulator’s decision “bodes well” for Verve too, Ha wrote.
Beam’s medicine is meant to be more convenient than the personalized cancer cell therapies from Gilead, Novartis and Bristol Myers Squibb that are approved for certain leukemias and lymphomas. Beam’s treatment uses cells from donors, rather than from patients, and is being developed for relapsed or refractory T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma.
Developers of these so-called off-the-shelf cell therapies have hit some setbacks and struggled to show their treatments can last as long as personalized cell-based medicines, however.