Pharma Startup Uses AI to Test Old Drugs to Treat Rare Diseases

For the past six years, Carrie Rich has been on a journey no parent wants to take. Her daughter has a form of autism caused by a rare genetic condition, and Rich is desperate to help her combat the effects of the genetic anomaly. They’ve left the girl unable to speak and blunted her ability to move and interact like her 7-year-old peers. “I’ve spoken with researchers, scientists, academics, entrepreneurs, philanthropists, investors and parents across the planet,” Rich says. “I mean, truly anyone who would speak with me. I was really hoping to find someone who could lead me in a positive direction forward.”

Rich mostly encountered bad news. She found families in similar circumstances who’d been bankrupted or torn apart by false hopes. There were painfully few signs that some kind of therapy for her daughter might one day be available. But last year a group of scientists in California who’d developed a new drug-screening program hit upon a pill they thought might help Rich’s daughter. The child began taking the pill in December, and the early indications are that it’s working.