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15 April, 2020 - 09:43 By Tony Quested

Healx fights rare disorder via new global alliance

Healx, the AI-powered technology business repurposing drugs to fight rare diseases, has forged a new global alliance that could hold significant benefits for sufferers of an uncommon genetic disorder.

The Cambridge UK specialist is working with US and Europe-based patient group Children’s Tumor Foundation (CTF) to progress new AI-derived therapies for neurofibromatosis (NF).

NF is a rare genetic disorder that affects 1 in 3,000 people worldwide. Currently, no treatments exist for any of the three types of NF (NF1, NF2 and schwannomatosis). 

Healx will combine its AI technology and drug discovery expertise with CTF’s disease data, networks and patient insights to predict and progress novel therapies for NF1.

Leading the collaborative drug discovery efforts is Simone Manso who joins Healx as head of NF Strategic Partnerships. Simone also serves as a member of CTF’s board of directors and is chair of CTF Europe’s board.

Manso said: “Healx has the technology and pharmacology expertise, along with the ability to quickly move treatments towards patients, whilst CTF brings the strong NF know-how and patient, clinical and academic networks. 

“Most importantly, both teams have a shared passion for striving to improve the quality of life for NF patients. I can’t think of a better partnership and I am honoured to lead this effort, which is personally, very close to my heart. This is where the magic can happen!”

Dr Annette Bakker, President of CTF, added: “We at CTF are very excited about this partnership. It is very encouraging that Healx is committed to developing innovative treatments for NF patients in collaboration with CTF. 
CTF will facilitate the translation of Healx’s comprehensive AI-based discoveries into clinical benefit using our non-profit R&D enabling platform.”

The partnership marks the latest in a growing number of collaborative projects between Healx and patient groups worldwide – spanning multiple disease areas (including rare cancers, metabolic and neurological disorders). 

Healx’s most advanced project currently is with the US-based patient group FRAXA Research Foundation, which has progressed a number of new therapies for fragile X syndrome from the drug prediction stage, to being ready for clinical testing - in just 24 months.

Whilst Healx has recently taken on extra work to use AI and repurposing to help find treatments to target COVID-19, the company continues to move forward with its primary mission to develop new therapies for rare disease patients. This collaboration with CTF will accelerate their work in NF.

In addition to the CTF collaboration, Healx recently closed the first call for applications to its Rare Treatment Accelerator – a global partnering programme that gives patient groups and Healx the opportunity to work together to progress new rare disease treatments. 

Healx has committed $20 million in AI drug discovery resources over the next two years to this programme and will be announcing more details on the chosen collaborations in the next few months.

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