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20 August, 2020 - 17:21 By Tony Quested

Healx forms new alliance to fight rare neurogenetic disorder

Cambridge University spin-out Healx, an AI-powered, patient-inspired technology company accelerating the discovery and development of rare disease treatments at scale, has formed a significant partnership to fight a little-known neurogenetic disorder.

Its partnership with the Foundation for Angelman Syndrome Therapeutics (FAST) will focus on developing therapies for Angelman syndrome which affects around one in 15,000 people across the globe.

This is the latest collaboration to come from Healx’s Rare Treatment Accelerator (RTA) programme, which was launched in October 2019. 

The programme brings together Healx’s AI-powered drug discovery expertise with patient group insights and research to predict and validate new therapies from drugs that are already approved and available. This approach accelerates new treatments towards the clinic more quickly, cheaply and safely than traditional drug discovery methods allow.

The new project – the third to come from the RTA programme – will focus on quickly identifying and progressing novel treatments for Angelman syndrome. 

The condition is caused by multiple genetic mutations, the most significant of which is the loss of function in a protein-making gene called UBE3A. 

This leads to complications in the nervous system, resulting in severe issues with movement and balance as well as problems with speech. There are currently no approved treatments, which means there is high unmet clinical need for many of the symptoms associated with the condition. 

By embedding patient insights into the therapeutic development pipeline right from the start, Healx hopes to build a new, collaborative model of drug discovery. 

“Patients and their caregivers are the real experts in rare diseases,” says Dr Bruce Bloom, chief collaboration officer at Healx. “They are the ones driving forward research efforts to find new treatments and have a wealth of experience and insight about what it’s actually like to live with these conditions. 

“By integrating their expertise early on, and connecting to their well developed scientific and clinical network, we have a better chance of finding critical new therapies.

“We’re really excited about working with FAST and hope to move our AI predictions towards preclinical validation in the second of half of the year.” 

Dr Allyson Berent, chief scientific officer at FAST, adds: “FAST is very excited to partner with Healx and we welcome them to the Angelman syndrome community. 

“Healx’s use of cutting edge artificial intelligence, combined with our scientific team’s expertise in cell lines and animal models, makes this innovative program incredibly promising. This program creates a unique opportunity to quickly bring therapies from bench to clinic, which could potentially have a huge impact on all individuals living with Angelman syndrome.”

This Rare Treatment Accelerator partnership builds on a series of previous collaborations between Healx and patient groups. In June, the team announced its collaboration with Muscular Dystrophy UK, the UK’s leading charity for muscular dystrophy research, support and advocacy. 

The project will focus initially on a condition called facioscapulohumeral muscular dystrophy (FSHD), which causes muscle-weakening in the face, shoulder blades and upper arms. 

In April, Healx also 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.

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