Fyodor Urnov, the scientific director of UC Berkeley’s Innovative Genomics Institute (IGI), showed me a list of rare diseases and pointed to one carried by only 50 people. “Who’s going to work on a disease with 50 patients?” he asked. … Drug developers have little financial incentive to spend years and millions of dollars designing therapies that may need to be tailored to literally one person.
The technology is ready to treat at least some of these diseases, though. … If researchers could build one CRISPR platform for a single disease, or even several similar ones, and tweak that template to suit each patient, they could target extremely rare disorders more quickly and economically.
The new pathway opens the door to the platform approach that scientists have hoped to take. If researchers could prove they’d successfully treated a small number of patients for one rare genetic disease, they could continue customizing treatments for other mutations, and potentially also for similar conditions. That streamlined process could finally attract for-profit players ….
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