- Title: Viagra inventor's startup raises $56m to target rare diseases with AI
- Date: 16th October 2019
- Summary: UNKNOWN LOCATION (FILE) (ORIGINALLY 4:3) (REUTERS) VARIOUS CLOSE-UPS OF VIAGRA BOXES
- Embargoed: 30th October 2019 14:46
- Keywords: Healx Viagra drug repurposing startup AI medicine autism rare disease
- Location: CAMBRIDGE, ENGLAND, UK / LOS ANGELES, CALIFORNIA, UNITED STATES / UNKNOWN LOCATION
- City: CAMBRIDGE, ENGLAND, UK / LOS ANGELES, CALIFORNIA, UNITED STATES / UNKNOWN LOCATION
- Country: United Kingdom
- Topics: Health/Medicine
- Reuters ID: LVA002B1CLPI3
- Aspect Ratio: 16:9
- Story Text: Big pharmaceutical firms do not usually spend big on finding the solutions to the very rarest diseases -- but now a company based in eastern England is launching an initiative to discover new treatments for exactly those conditions.
Healx, a biotechnology company in Cambridge, has raised 56 million USD to help rare disease patients by finding new uses for existing drugs.
The company uses AI to examine potential beneficial side effects of existing drugs which could also treat rare ailments.
It was co-founded in 2014 by David Brown, who 'accidentally' co-invented Viagra in the 1990s. Viagra was originally developed as a blood pressure treatment. Its impact on erectile dysfunction was discovered by accident.
Brown sees parallels in how the company works to the way in which the drug for erectile problems was developed accidentally.
"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," said David Brown, the company's co-founder.
Rare diseases currently affect 400 million people globally. Of the more than 7,000 rare diseases known today, only 5% have an approved treatment.
Audrey Davidow's son Calvin was diagnosed with the condition Pitt Hopkins syndrome, a rare form of autism, just after his first birthday.
"When he was diagnosed there was about 200 other cases in the world. And so no our geneticist didn't even know what it was. And so we just felt so alone and so hopeless. There was no treatment on the horizon. There was no research being done. There was no foundation for research. There was no hope," she said.
Healx has worked closely with the California-based Pitt Hopkins Foundation, of which Davidow is president.
Clinical trials will soon be launched to test multiple treatment combinations the company has developed for Fragile X, the leading genetic cause of autism.
The Healx model has also helped identify various FDA approved compounds that have been trialled in mice.
"It really takes what is already existing out there and makes it a possibility for a treatment to happen in a couple of years as opposed to a couple of decades," Davidow said.
Healx says its mission is to advance 100 rare disease treatments to clinical trials by 2025.
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