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Healx teams up with the Foundation for Angelman Syndrome Therapeutics to accelerate novel treatments towards the clinic

Angelman Syndrome is a rare neurogenetic disorder that primarily affects the nervous system, causing balance issues, motor impairment and developmental delays. The Foundation for Angelman Syndrome Therapeutics (FAST) is the largest non-governmental funder for Angelman Syndrome research in the world, committed to finding and progressing treatments for the condition. This exciting collaboration is the latest to come from Healx’s Rare Treatment Accelerator programme, which combines Healx’s AI-powered drug discovery technology with patient group insights and expertise to accelerate new therapies for rare diseases.
12 August 2020   |  
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Cambridge UK – 12 August 2020 – Healx, the AI-powered, patient-inspired technology company accelerating the discovery and development of rare disease treatments at scale, is proud to announce its partnership with the Foundation for Angelman Syndrome Therapeutics (FAST) today. This partnership will focus on developing therapies for Angelman syndrome, a rare neurogenetic disorder that affects approximately 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.

This 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.” 

Commenting on the collaboration, Dr Allyson Berent, Chief Science Officer at FAST, says: “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. To learn more about the RTA programme, please visit Healx’s website.



About Healx

Healx is an AI-powered, patient-inspired tech company, accelerating the discovery and development of treatments for rare diseases. There are 7,000 known rare diseases that affect 400 million people across the globe but only 5% of those conditions have approved treatments. Our mission is to discover and develop novel treatments for rare disease patients at scale through a combination of AI technology, drug discovery expertise and patient insight. Our innovative approach aims to identify existing drugs that may be repurposed and combined to treat rare diseases, meaning 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. In 2019, Healx raised $56 million in Series B funding. For more information, visit www.healx.ai or follow on Twitter and LinkedIn.

About the Foundation for Angelman Syndrome Therapeutics (FAST)

FAST (Foundation for Angelman Syndrome Therapeutics) is a Section 501(c)(3) nonprofit research organization singularly focused on funding research that holds the greatest promise of treating the symptoms of Angelman syndrome and allowing those living with the condition to realize their greatest potential and quality of life. FAST is the largest, non-governmental funder of Angelman-specific research.

For more information, please contact:

Charlotte Chorley

Head of Communications, Healx.          


Maiddy Dunigan

Chief Operating Officer, FAST



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