Crispr Pioneer Launches Startup to Make Tailored Gene-Editing Treatments

"Last February, a sick infant named KJ received a gene-editing treatment made just for him. Created in just six months, it was meant to correct a rare genetic mutation that was causing toxic ammonia to build up in his small body. The treatment likely saved his life, and baby KJ was discharged from the hospital in June. Now, a new startup called Aurora Therapeutics, cofounded by gene-editing pioneer Jennifer Doudna, is aiming to scale such treatments to many more patients with rare diseases."

"Aurora plans to take advantage of a new regulatory pathway announced by Food and Drug Administration officials Marty Makary and Vinay Prasad in the fall. The new program, called the "plausible mechanism pathway," allows the FDA to approve personalized treatments for rare and fatal diseases based on data from just a handful of patients, according to Makary and Prasad in a New England Journal of Medicine article."

"Typically, new drugs must be tested in hundreds, if not thousands, of patients in order to get regulatory approval. For drug trials of rare diseases, it's difficult to recruit that many patients because so few people have the disease. The new FDA pathway provides a way for these types of drugs to be approved when a large, randomized trial isn't possible."

An infant named KJ received a bespoke CRISPR gene-editing therapy created in six months to correct a rare mutation causing toxic ammonia buildup; the treatment likely saved his life and he was discharged in June. Aurora Therapeutics, cofounded by Jennifer Doudna, aims to scale similar personalized treatments for many rare-disease patients. The FDA's new 'plausible mechanism pathway' permits approval of personalized therapies based on data from a handful of patients when large randomized trials are infeasible. Success across several consecutive bespoke therapies can lead to marketing authorization for the underlying platform. Aurora will initially target phenylketonuria (PKU).

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