Healx, the Cambridge AI technology company developing breakthrough treatments for rare diseases, has unveiled a $10 million Series A funding round led by European-leading early stage venture backer Balderton Capital.

Existing investors Jonathan Milner and  Hermann Hauser’s Amadeus Capital also participated in the round.

Healx is planning to cut medicine development time for rare diseases by 80 per cent through its repurposing model. The fundraising exercise was exclusively flagged by Business Weekly in March.

The company was founded in 2014 by Dr Tim Guilliams, a bio-chemical engineer and founder of the Cambridge Rare Disease Network, Dr David Brown, the inventor of Viagra and ex-global head of drug discovery at Roche and Dr Andreas Bender, a lecturer and researcher at Cambridge University’s Centre for Molecular Sciences Informatics.

There are 7,000 known rare diseases that affect 350 million people worldwide. One in 20 people on the planet suffer from a rare disease – in aggregate, rare diseases are not rare. Yet 95 per cent of rare diseases still do not have an approved treatment.

Rare disease patients and families have been let down by a broken traditional pharma model that is dependent on and focuses on blockbuster drugs.

Healx’s mission is to reverse this by using Artificial Intelligence, deep pharmacological expertise and extensive patient engagement to give sufferers of rare disease a better quality of life.

Healx’s massively parallel approach combines cutting edge technology with one of the largest collections of insights from rare disease patient groups.Dr Guilliams said: “Healx has a simple but profound goal: To transform the lives of rare disease patients. Our technology helps us find treatments where none currently exist.

“Our proven approach is to start from existing drugs and apply artificial intelligence to niche disease populations, working with patient groups to accelerate treatment development.

“Our success comes from our world-class multi-disciplinary team of experts, leveraging cutting-edge artificial intelligence techniques to cut the discovery-to-treatment time from years to months.” Healx’s technology has already been used successfully, including most recently in collaboration with FRAXA, the patient group for the Fragile-X Syndrome.

A drug for Fragile-X that was discovered by Healx’s platform was ready for the clinic in less than 15 months, cutting typical drug development timelines by 80% and doing so at a substantially reduced cost. At the heart of Healx, is HealNet, one of the world’s most comprehensive knowledge-bases for rare disease, mapping over one billion unique disease, patient and drug interactions.

HealNet was built and is maintained using a variety of machine learning methods applied to a wide range of data types from both publicly available and exclusive sources including scientific literature, patents, clinical trials, disease symptoms, drug targets, multi-omic data and underlying chemical structures.

HealNet facilitates highly parallelised, automated, large scale drug discovery that drastically reduces time and cost to discovery compared to traditional processes. Key to Healx’s success to date has been its treatment strategy. While the technology could be used for the development of new therapies, the company has so far focused on finding new uses for existing drugs and their application in potential combination therapies.

Applying this strategy within the context of rare disease has allowed Healx to further improve speed to market – existing drugs require fewer and less complex trials and the Orphan Drug Act was designed to encourage research and development of therapies for rare disease.

The $10m investment will be used to more than double Healx’s diverse and multi-disciplinary team of software engineers, data scientists, pharmacologists and drug development experts from the rich cross-disciplinary Cambridge ecosystem and to expand its world class artificial intelligence and machine learning technologies.

Balderton’s Suranga Chandratillake will be joining the board alongside Shaun Grady of AstraZeneca  – an involvement flagged exclusively by Business Weekly in March when we reported on the launch of the fundraising – and Dr Jonathan Milner (founder and ex-CEO of Abcam); serial tech entrepreneur Hermann Hauser, who also founded Arm, will act as an observer.

David Brown said: “The traditional drug discovery process takes 10 to 15 years at a cost of $2 billion per new drug and with a failure rate of 95 per cent – it’s broken, it’s slow, it’s high failure, and it’s not economic for rare diseases.

“However, today’s technology can change that and help 350 million under-served rare disease patients. Healx is showing that we can massively transform the rate of discovery of new medicines, reducing timelines and costs.”

Suranga Chandratillake of Balderton Capital, added: “Historically, the pharmaceutical industry has focused on what is the same about everyone, building blockbuster drugs that affect large groups of people.

“The future of medicine will focus on what is different about everyone, personalising treatment and taking the individual into context.

“The first frontier of this exciting new direction for medicine is rare disease and the only way it can be solved is by using automation to deliver a step-change in the volume of discovery that is possible.

“Healx’s HealNet platform, at the forefront of this shift, is already delivering value and Balderton is excited to have the opportunity to be part of the company’s journey.”