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. Doudna is one of the inventors of the gene-editing system known as Crispr, and won a Nobel Prize in 2020 for her work on the technology.
Aurora plans