CAMBRIDGE U.K.—July 15 saw Healx, an artificial intelligence (AI)-powered technology company looking to accelerate the discovery and development of rare disease treatments at scale, announce its Rare Treatment Accelerator Programme (RTA) partnership with Muscular Dystrophy UK (MDUK). The RTA collaboration will initially focus on finding novel therapies for facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle-weakening and wasting in the face, shoulder blades and upper arms.
The framework for the RTA partnership aims to explore other muscular dystrophies in future projects.
FSHD is one of the more common forms of muscular dystrophy, with an estimated three to five people in every 100,000 affected globally. In the United Kingdom alone, there are between 2,000 and 2,500 people living with the condition, but there is no cure---and a lack also of effective treatments means new therapeutic options are urgently needed.
By combining MDUK’s extensive patient insights and Healx’s AI-powered drug discovery expertise, the partnership aims to develop new therapies for the condition.
“We’re really excited about this partnership, and look forward to working with Healx to identify potentially promising therapies for FSHD,” said Dr. Kate Adcock, director of research and innovation at Muscular Dystrophy UK. “It’s an exciting time for research into muscle-wasting conditions, and treatments are on the horizon. By working with world-leading partners like Healx, we can drive vital research forward and accelerate access to treatments.”
Added Dr. Bruce Bloom, chief collaboration officer at Healx: “Having successfully carried out drug matching projects for similar conditions, the team is looking forward to applying their expertise in neurological and neuromuscular diseases to find a treatment for this life-altering condition. We are looking forward to sharing more updates about this project in the coming months.”
Healx’s says that its approach “integrates the power of Big Data and AI with expertise from the in-house expert pharmacology team to uncover new connections between existing drugs and diseases.” This approach is intended to reduce the time, cost and risk of traditional drug discovery methods and increase the potential for accelerated access to more cost-effective treatments for patients.
This Rare Treatment Accelerator Programme partnership is the latest in a series of RTA collaborations between Healx and patient groups. In April, the company announced that it would be working with the Children’s Tumor Foundation to develop new therapies for neurofibromatosis, a rare genetic disorder that affects one in 3,000 people worldwide. The team is also working with US-based patient group FRAXA Research Foundation to find new treatments for fragile X syndrome, the most common inherited cause of autism and intellectual disabilities in the world. Working in collaboration, they have progressed a number of new therapies from the drug prediction stage to clinical trial readiness in just 24 months.