Estimates suggest that roughly one in 30,000 people in Europe and one in 50,000 people in the United States have the condition. Whilst symptoms vary, the condition is typically characterised by poor balance and coordination, difficulty speaking, a curved spine and heart problems. Some patients may also experience diabetes and chronic fatigue.
On average, symptoms first appear when an individual is around 5 - 18 years old and will worsen over time, meaning many patients have to use wheelchairs by their late 20s. Unfortunately, there is currently no cure or approved pharmacological treatment specifically for Friedreich’s ataxia but there are some treatments available to manage the symptoms. This partnership will aim to find, and accelerate to the clinic, some of the world’s first disease modifying treatments for the condition.
To do this, Healx will leverage its cutting-edge technology and innovative drug repurposing approach and combine this with
Ataxia UK and
FARA’s unrivalled patient and scientific insight. At the heart of Healx’s technology is
Healnet - their AI drug discovery platform. As one of the only AI companies in the world to focus specifically on rare diseases, Healx has developed the world’s most comprehensive biomedical knowledge graph for rare diseases which comprises millions of data points from clinical, pharmacological and scientific literature. This graph is analysed by more than 10 different AI models to rapidly uncover novel connections between drugs, diseases and other relevant medical information. These AI models have been specially trained to predict the effectiveness of repurposing and combining drugs for rare disease treatments, and in the case of this project, the team have developed a novel metabolic model that they can use to identify drug candidates.
Healx is unique in the AI drug discovery space in its creation of novel treatment opportunities through already-approved drugs. This repurposing approach shortens and de-risks the path to clinical trials - since the safety profile and mechanisms of action of the drugs are usually well characterised - meaning people in need can get effective treatments earlier and at a more reasonable cost.
Commenting on the partnership, Julie Greenfield, Head of Research at Ataxia UK, said: 'We are delighted to be partnering with Healx and FARA to help advance the discovery of much needed therapies for Friedreich's ataxia, using Healx's novel drug repurposing AI technology approach.
Jen Farmer, CEO at FARA, also said: "We believe that partnership and collaboration - sharing knowledge and resources - accelerates the pace of research and conserves valuable resources which is essential in the rare disease community. FARA is proud to partner with Healx and Ataxia UK to leverage Healx's AI technology toward identification of drug repurposing opportunities and discovery of novel treatment approaches for FA."
Bruce Bloom, Chief Collaboration Officer at Healx, says: “We are very excited to be working with Ataxia UK and FARA, who are both leaders in the field of ataxia research and support and who share our commitment of putting patients right at the heart of treatment discovery. Together, we hope this pioneering partnership will accelerate much-needed treatments for Friedreich’s ataxia from AI prediction to clinical trial and directly to patients.”