Crispr Pioneer Launches Startup to Make Tailored Gene-Editing Treatments

Los Angeles: Last February, a baby named KJ got a special gene-editing treatment just for him. This treatment was made quickly to fix a rare genetic problem that made his body fill with harmful ammonia. Thanks to this treatment, baby KJ went home from the hospital in June.

Now, a startup called Aurora Therapeutics is here to help more patients with rare diseases. It was co-founded by Jennifer Doudna, who helped invent the Crispr gene-editing technology. She won a Nobel Prize in 2020 for her work.

Aurora wants to use a new rule from the FDA that helps approve personalized treatments for rare and serious diseases. This rule allows the FDA to approve these treatments based on how a few patients do, instead of needing big tests with lots of people. This is very helpful because it’s hard to find many patients with rare diseases.

According to FDA officials Marty Makary and Vinay Prasad, if a company shows that their treatment works for a few patients with similar issues, the FDA will give them permission to sell that treatment. This means drug companies can then use that data to make other similar drugs.

Aurora will start with a disease called phenylketonuria, or PKU, which is found in babies at birth. This disease causes high levels of a protein in the blood, making it hard for people to eat normal food. Without treatment, PKU can hurt brain growth. About 13,500 people in the U.S. live with PKU.

Edward Kaye, CEO of Aurora, says that many people need this therapy, but there are over a thousand different causes for PKU. Crispr works by using a special RNA to guide the editing to the right place in DNA, like a GPS in a car. Baby KJ’s treatment worked for him because it was made just for his specific problem.

Aurora plans to change the guide RNA to create different treatments for each version of PKU. Before, the FDA would say each version was a new drug needing its own tests. Now, Aurora can use the same technology to help many types of PKU with fewer rules to follow.

Kaye mentioned they will use base editing, which is better than regular Crispr, and will have a smoother way to create their treatments. Yurnov, another founder, said they want to make sure no mutation is left behind, showing their commitment to helping all people with PKU.