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November Monthly Special 2021

Healx – Accelerating the Discovery and Development of Rare Disease Treatments at Scale


The amount of data collected and managed in biomedicine is ever-increasing. Thus, there is a need to rapidly and efficiently collect, analyze, and characterize all this information. Artificial intelligence (AI), with an emphasis on deep learning, holds great promise in this area and is already being successfully applied to basic research, diagnosis, drug discovery, and clinical trials. Rare diseases (RDs), which are severely underrepresented in basic and clinical research, can particularly benefit from AI technologies. Ultimately, RDs' AI-mediated knowledge could significantly boost therapy development. Presently, there are AI approaches being used in RDs and this review aims to collect and summarize these advances.

Healx is a mission-driven technology company pioneering the next wave of drug discovery in order to bring novel, effective treatments to rare disease patients around the world. There are 7,000 known rare diseases that affect 400 million people across the globe, but only five percent of those conditions have an approved treatment. By combining frontier AI technology with deep drug discovery and development expertise, Healx can accelerate the pace, increase the scale and improve the chance of success of rare disease treatment development in order to meet this huge unmet need and have unprecedented patient impact. In 2019, Healx raised $56 million in Series B funding and was listed in Tech Nation’s Future Fifty cohort. In 2020, the company won five prizes at the CogX Awards including Grand Jury Prize and Best AI Product and was listed in CB Insights Digital Health 150.

Next-Gen AI-based Drug Discovery Platform Offered

Rare Treatment Accelerator (RTA): It helps academic, patient, and industry groups advance their rare disease drug redevelopment projects. By combining its team, expertise and financial resources with your research and insights, the company can get treatments to patients faster. The RTA is open to all academic, patient and early-stage biotech groups that have a drug redevelopment opportunity for a rare disease. Although different regions have their own definitions of a rare disease, they broadly define it as any condition that affects less than 1 person in 2,000 (5 in 10,000). The company’s team is particularly keen to hear about projects with strong evidence that the redeveloped drug has therapeutic effects in preclinical disease models and is suitable to move towards human clinical trials.

Healnet: It is the world’s most efficient AI platform for rare disease drug discovery, supporting its team and partners in its mission to advance new treatments towards the clinic. The company’s AI platform uses natural language processing (NLP) to extract disease knowledge from published sources and to complement biomedical databases and proprietary, curated data. It constantly augments the depth and breadth of previously held information and fills in critical gaps in its knowledge of rare diseases. This enables greater opportunity for novel treatment discovery. Healnet’s data is integrated in the form of the largest, rare disease-focused Knowledge Graph. It shows prioritised hidden and novel connections between drugs and diseases when explored by expert pharmacologists and biologists. Healnet can present a set of pre-formed, data-driven hypotheses, which speeds up the interpretation process.

A Relentlessly Reliable Leader

Dr. Tim Guilliams is the co-founder and Chief Executive Officer of Healx. He is a tech entrepreneur from the Cambridge Cluster, passionate about using big data and AI to accelerate treatments for rare diseases. As CEO of Healx, Tim has been listed in The Sunday Times Maserati 100 list of entrepreneurs in 2019, The Telegraph’s Tech 100 in 2019, and In Vivo’s 30 Rising Leaders in the Life Sciences in 2020. He was also named as ‘One to watch’ in The Telegraph’s Top 50 Most Ambitious Business Leaders in 2020.

He is also the Co-Founder and Trustee of the Cambridge Rare Disease Network (CRDN). Prior to Healx and CRDN, he obtained his PhD at the University of Cambridge in the field of Biophysics and Neuroscience, developing nanobody technology for Parkinson's disease. Before moving to Cambridge UK, Tim obtained a MEng in Bio- and Chemical-Engineering from the University of Brussels.

“Healnet leverages the breadth of its dataset, along with disease- and/or drug-specific data, to deliver reasoned insights we would never have thought to consider.”