Vor's cancer cell therapy gets on FDA fast track ahead of 2022 data drop

The FDA has become the latest organization to validate the potential of Vor Biopharma’s approach to CRISPR-powered cell engineering, granting fast-track status to its lead asset shortly after Johnson & Johnson struck a deal to access the technology.

Vor, which was founded by Siddhartha Mukherjee, is using CRISPR to render healthy cells invisible to targeted treatments. In the case of lead candidate, VOR33, Vor is engineering cells to eliminate CD33 and thereby reduce the toxicity of Pfizer’s Mylotarg and other drugs that hit the target. Vor recently moved the lead candidate into a leukemia clinical trial. 

The FDA fast-track designation is designed to support the development and accelerate the review of drugs with the potential to treat serious unmet medical needs. As VOR33 is a novel approach for use in cancer patients, it meets the criteria for inclusion and is set to benefit from the FDA’s input.

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Vor expects to have initial clinical data from the 18-subject clinical trial in the first half of next year. The study is evaluating the engraftment of white blood cells in patients who receive an infusion of VOR33 followed by one of three doses of Mylotarg.

“We will continue to work closely with the FDA to expedite the development of VOR33, which is now actively enrolling in its phase 1/2a clinical trial for [acute myeloid leukemia] patients who currently have limited treatment options,” Robert Pietrusko, Vor’s chief regulatory and quality officer, said in a statement. 

Next year’s readout will provide an early look at whether Vor’s technology can deliver in the clinic. Investors pumped more than $200 million into the biotech in an IPO early this year, before J&J inked a deal to study one of its bispecific antibodies in combination with Vor’s cell therapies. The release lacked details of the J&J bispecific, but CD33xCD3 prospect JNJ-67571244 is a possible candidate.