Working in collaboration with Ataxia UK and FARA, Healx will leverage its state-of-the-art AI platform and drug discovery expertise to develop novel treatments for Friedreich’s ataxia – a rare neurodegenerative condition that causes issues with balance, speech and coordination.
Friedreich’s ataxia is a progressive, inherited form of ataxia and is caused by a genetic fault in a gene that encodes a protein called frataxin. Whilst the condition is the most common type of inherited ataxia, it is still very rare, with estimates suggesting 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 five to18 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.”
This partnership is the latest to join Healx’s growing portfolio of rare disease collaborations. Last month, Healx announced that it would be working with the UK-based PKD Charity to find novel therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD) and Autosomal Recessive Polycystic Kidney Disease (ARPKD). And in January this year, the team shared the news that they would be collaborating with Mission: Cure to identify the world’s first treatments for chronic pancreatitis.
The team is always looking for other rare conditions that may benefit from its tech-driven drug repurposing approach. Patient, industry and academic groups interested in collaborating with Healx to unlock the power of repurposed drugs for rare diseases are encouraged to apply to Healx’s Rare Treatment Accelerator (RTA). The RTA is Healx’s partnering programme for researchers, universities, spinouts, patient groups and other entities with promising drug repurposing opportunities for rare diseases who are looking to licence or co-develop the drugs towards clinical trials. Working together with selected applicants, Healx will leverage its AI, drug discovery and clinical expertise to accelerate novel therapies towards clinical trials in a typical timeframe of six to 12 months. To learn more about the RTA, please go to https//healx.io/rta.
About Healx
Healx is an AI-powered, patient-inspired technology company, accelerating the discovery and development of rare disease treatments at scale. There are 7,000 known rare diseases that affect 400 million people across the globe but only 5% of those conditions have approved treatments. Healx’s mission is to identify and progress novel therapies for the 95% of rare diseases without approved treatment. To do this, Healx combines AI technology with drug discovery expertise and patient insight to identify existing drugs that may be repurposed and combined to treat rare diseases. This innovative approach means treatments can be found more quickly, efficiently and cost-effectively than traditional drug discovery methods allow. Healx was founded in 2014 in Cambridge, UK, by Dr Tim Guilliams, a Biochemical Engineer and Tech Entrepreneur, and Dr David Brown, co-inventor of Viagra and former Global Head of Drug Discovery at Roche. For more information, please visit www.healx.io or follow us on Twitter and LinkedIn.
About Ataxia UK
Ataxia UK is a patient support organisation for people affected with a range of ataxias. It is dedicated to funding and promoting research to find treatments and providing support services to everyone affected by the condition. Ataxia UK works in partnership to actively facilitate research by organising research conferences, establishing patient registries and being involved in Global and European research networks ensuring patient involvement and engagement.
About FARA
The Friedreich’s Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), nonprofit, tax-exempt organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich’s ataxia. FARA’s mission is to marshal and focus the resources and relationships needed to cure FA by raising funds for research, promoting public awareness, and aligning scientists, patients, clinicians, government agencies, pharmaceutical companies, and other organizations dedicated to curing FA and related diseases. For more information, visit www.curefa.org.