Viagra inventor to target rare diseases with AI

Audrey Davidow knew something was wrong with her son Calvin.

(SOUNDBITE) (English) AUDREY DAVIDOW, MOTHER OF CHILD WITH RARE FORM OF AUTISM AND PITT HOPKINS FOUNDATION PRESIDENT SAYING:

"Around six months he started to miss some milestones."

He was eventually diagnosed with the condition Pitt Hopkins syndrome, a rare form of autism.

But there was barely any help out there for him.

(SOUNDBITE) (English) AUDREY DAVIDOW, MOTHER OF CHILD WITH RARE FORM OF AUTISM AND PITT HOPKINS FOUNDATION PRESIDENT SAYING:

"There was no treatment on the horizon. There was no research being done. There was no foundation for research. There was no hope."

Big pharmaceutical firms don't usually spend big on finding the solutions to the very rarest diseases

Help may come from a perhaps unlikely person: David Brown is the inventor of Viagra.

He says his start up biotech company will use AI to discover new treatments for those very type of illnesses.

And, it's already raised $56 million.

The company says AI tech will examine potential beneficial side effects of existing drugs which could also treat those rare ailments.

Viagra was originally developed as a blood pressure treatment. Its impact on erectile dysfunction was discovered by accident.

(SOUNDBITE) (English) DAVID BROWN, HEALX CHAIRMAN AND CO-FOUNDER, SAYING:

"The whole idea of repurposing drugs, rapidly and cheaply, matching it to the genome of the children with these rare diseases, it really struck a chord with me because in a way that's what we did with Viagra. So, I could see that the same opportunity was there for many other drugs as well."

Healx has worked closely with the California-based Pitt Hopkins Foundation.

Clinical trials will soon be launched to test multiple treatment combinations the company has developed for Fragile X, the leading genetic cause of autism.

Of the more than 7,000 rare diseases known today, only 5% have an approved treatment.