TechBio innovator spotlight: Developing new treatments for fragile X syndrome

Ahead of TechBio UK conference, we'd like to shine the spotlight on some of the companies that are working at the interface between biotechnology and technology. TechBio companies, like Healx, combine cutting-edge techniques from both sectors to draw insights from a wealth of data to inform and transform drug discovery and patient care.

Fragile X syndrome (FXS) is a rare neurodevelopmental condition that causes a range of cognitive impairments. Artificial intelligence (AI) is speeding up the discovery of new medications.

What’s the breakthrough?

Innovators at Healx have used AI and data analysis to identify potential treatments for FXS. The techniques are being used to find existing drugs that can be redeveloped. The first of these potential treatments is now entering a clinical trial with the hopes of it becoming the first combination therapy for FXS patients.

What data was used?

Healx partnered with the FRAXA Research Foundation - a FXS patient organisation - to get access to preclinical data about the condition. Publicly available gene expression data were also obtained from the National Centre for Biotechnology Information Gene Expression Omnibus (NCBI GEO).

What was done with the data?

The team at Healx analysed the genes expressed by FXS patients to create a profile. They then searched their database of drugs to find compounds with an opposite gene expression profile. This helped identify which drugs are likely to be effective therapies for the disease.

Why is this needed?

There are currently no approved therapies for FXS. The condition affects 1 in 4,000 males and 1 in 8,000 females, making it the most common genetic cause of learning difficulties in the world. Individuals affected by FXS exhibit learning, social, language, emotional, and behavioural challenges.

How was the data accessed?

Data were either in the public domain or made available through academic and patient group collaboration. Data contributors consented to their data being collected. All research proposals underwent ethical review before commencing.

About Healx

Healx was founded in 2014 and has a headcount of over 150. They are pioneering the next wave of drug discovery to bring novel, effective treatments to rare disease patients. By combining AI technology with drug discovery expertise, Healx can speed up the pace, increase the scale and improve the chance of success in drug discovery. Learn more about Healx