Putting a twist on targeted therapy, Vor Biopharma heralds proof of concept for its gene-edited stem cells
Normally in a first-in-human clinical trial, researchers are looking to find the right dose of their experimental drug. Vor Biopharma set out to do something different: Not only did it want to see if its therapy, a gene-edited stem cell transplant, can engraft safely; it also hopes to help patients better tolerate another drug known for its side effects.
While early data from a single patient aren’t saying much about potential efficacy, the company believes they do offer key proof of concept for an unorthodox idea proposed by Columbia oncologist (and celebrated author) Siddhartha Mukherjee.
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