RARE Daily

Healx and Muscular Dystrophy UK Partner to Develop New Treatments for Rare Muscle-Wasting Diseases

July 15, 2020

Rare Daily Staff

Healx, which uses artificial intelligence to accelerate the discovery and development of rare disease treatments, said it has entered a partnership with Muscular Dystrophy UK to find novel therapies for facioscapulohumeral muscular dystrophy.

Facioscapulohumeral muscular dystrophy (FSHD) is a genetic condition that causes muscle-weakening and wasting in the face, shoulder blades and upper arms. There is no cure and there is a lack of effective treatments.

The partnership, entered through Healx’ Rare Treatment Accelerator Programme, will combine MDUK’s extensive patient insights and Healx’s AI-powered drug discovery expertise to develop new therapies for the condition. The partners expect to explore other muscular dystrophies in future projects.

Healx’s innovative approach integrates the power of big data and artificial intelligence with know-how from the in-house expert pharmacology team to uncover new connections between existing drugs and diseases. This approach reduces the time, cost, and risk of traditional drug discovery methods – increasing the potential for accelerated access to more cost-effective treatments for patients.

“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,” said Bruce Bloom, chief collaboration officer at Healx.

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 1 in 3,000 people worldwide. The team is also working with U.S.-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 being ready for clinical testing – in just 24 months.

Photo:  Bruce Bloom, Chief Collaboration Officer at Healx

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