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February Edition 2022

Healx: AI-Powered, Patient-Inspired Treatments for Rare Diseases


There are over 7000 rare diseases. They affect 400 million people. But 95% of them have no treatment. Founded in 2014, Healx is a mission-driven technology company pioneering the next generation 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 5% 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 an unprecedented patient impact.

The Origin Story 

The Healx story began when Dr. Tim Guilliams and Dr. David Brown met a man named Nick Sireau in 2014. Nick had set up a patient group to help find treatments for an extremely rare genetic disorder that affected his sons. For a decade, he worked tirelessly, but the drug discovery process was slow and very expensive – a challenge facing nearly all patient groups today.

Tim and Dave had long been advocating drug redevelopment - the use of existing drugs for new therapeutic purposes - when used in combination with artificial intelligence. They knew that this inexpensive and accelerated approach could help others like Nick and the rare disease community overcome the challenges they were facing and find treatments faster. Nick became their inspiration – he was the living story of the therapeutic need their approach could meet.

Finding Novel Connections between Drugs and Diseases 

Traditional drug discovery is slow, expensive, and has a 5% success rate. This has led to a culture of filling pipelines with blockbuster drugs targeted to large patient populations in order to offset the cost of failures. By using AI and other frontier technologies to redevelop, combine and enhance known compounds, Healx is building a pipeline of new and effective therapies.

One disease, one target, one drug: it's an overly simple model, yet it's the one used by nearly all pharmaceutical companies. Rare diseases are often not well studied, and there is a limited understanding of many of the aspects necessary to support a drug discovery program. Healx's AI platform, Healnet, overcomes these challenges by analyzing millions of drug and disease data points to find novel connections that could be turned into new treatment opportunities. By applying frontier technologies across the discovery and development pipeline, Healx is able to run multiple stages in parallel and at scale.

Healnet is the next-generation AI platform Healx built to find novel connections between drugs and diseases. Healx uses these predictions with in-house expertise to accelerate new treatments through its pipeline to clinical trials and on to patients.

Healx is bringing treatments from prediction to patient. Artificial intelligence enables the company to rapidly identify which novel drug-disease relationships have the highest chance of success and effectively scale a drug pipeline that will deliver significant patient impact. By using artificial intelligence to de-risk the drug discovery process, Healx can run multiple programs in parallel and deliver a higher chance of treatment success.

A Part of the Rare Diseases Community 

The rare disease community is vital to the discovery of new treatments. Direct conversations and engagement with people and groups worldwide and their shared voice help steer Healx in its drug discovery and development programs.

"We continue to be a part of the rare disease community and work closely with patient groups on all of our disease projects, provide new resources for them, and support our colleagues who have a rare disease or care for family members with one," said Dr. Bruce Bloom, Chief Collaboration Officer, Healx.

Healx is always eager to increase its patient group collaborations for the diseases they are working on. Alongside its work with various patient organizations on treatment development projects, Healx engages and supports rare disease groups and their families through events, webinars, and other educational content. Advocating for the rare disease community is one of Healx's core values, and the company encourages its team to take volunteering days to help out with rare disease charities and projects.

About the CEO

Dr. Tim Guilliams, Co-founder and CEO

Tim is a tech entrepreneur from the Cambridge Cluster, passionate about using big data and AI to accelerate treatments for rare diseases. He is the Co-Founder and Chief Executive of Healx, the AI-powered, patient-inspired tech company, accelerating the discovery and development of rare disease treatments at scale. 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 5 prizes at the CogX Awards including Grand Jury Prize and Best AI Product and was listed in CB Insights Digital Health 150.

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.

"Healnet is the next-generation AI platform Healx built to find novel connections between drugs and diseases."

"By using AI and other frontier technologies to redevelop, combine and enhance known compounds, Healx is building a pipeline of new and effective therapies."