Breaking new grounds in the discovery of new treatments for rare diseases: Healx

Rare diseases usually have a genetic basis, often affecting patients early in childhood, and life threatening in nature. These characteristics can have a devastating psychological impact on families of children suffering from these diseases. A number of unique clinical, regulatory challenges are associated with the development of therapies for the treatment of rare diseases. In recognition of these challenges, Healx went from being an idea to a reality when its co-founders, Dr. Tim Guilliams and Dr. David Brown, met Nick Sireau.More than a decade earlier, Nick had set up a patient group to help save his sons from an extremely rare genetic disorder. He worked tirelessly, but the drug discovery process was slow and went into the millions of dollars, and this is a problem many patient groups still face today. And the truth is that most patient groups don’t find success.

Tim and Dave had long been advocating the benefits of drug repurposing, in combination with the power and speed of AI. Nick became their inspiration – he was the living story of the therapeutic need their abilities could meet. This encounter took place in 2014 and it has been growing ever since. Today, the company is a world-class team of drug discoverers, AI engineers, and bioinformaticians, all working together to accelerate the discovery of new treatments for rare diseases.

Healnet

The world’s most efficient AI platform for rare disease drug discovery, supporting its teams and partners in its mission to advance new treatments.

Predicting novel biomedical relationships: 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.

Knowledge Graph: Healnet’s data is integrated in the form of the largest, rare disease-focused Knowledge Graph. It shows prioritized 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.

Mastering biomedical complexity: The often-complex biology of rare diseases means that it is no longer effective to start with a single target in mind. Its approach is hypothesis-free: Healnet leverages the breadth of its dataset, along with disease- and/or drug-specific data, to deliver reasoned insights it would never have thought to consider.

Intelligent, Collaborative, and Efficient

Drug repurposing: Healx uses AI to accelerate drug discovery and development, but it accelerates it even more by focusing on repurposing known drugs. By saving time, it also saves money. One of the greatest benefits, however, is the safety these known drugs have already shown in late-stage trials.

Partnerships that deliver: Its collaborations with patient groups result in faster and safer new treatments for rare disease patients. But that’s not only because it shares information and expertise, or leverage the power of AI. It focuses just as much on making its partnerships long-term and mutually supportive.

The right technology: Its AI platform Healnet delivers its predictions by leveraging various public and proprietary datasets. If necessary, Healnet can also extract information from those datasets to fill in critical gaps in what it knows about certain rare diseases. The company’s experts always validate these inferences and the opportunities they bring to light.

Rare Treatment Accelerator

The Rare Treatment Accelerator is a partnering programme that gives patient groups and Healx the opportunity to work together to quickly discover and develop repurposed treatments for rare diseases using artificial intelligence (AI).

The company has committed a total of $20 million over the next two years towards finding new treatments – investing up to the value of $1 million, in AI and drug discovery resources per project. Working together, with selected applicants, it will combine its AI technology, data, disease and drug discovery expertise to develop novel therapies and take them towards clinical trials in a typical timeframe of 24 months.

Healx believes in long-term partnerships where everyone brings the determination, expertise and the resources needed to succeed in driving patient access to therapies.

Meet the Expert

Dr. Tim Guilliams, Co-Founder and CEO: Tim is a tech entrepreneur from the Cambridge Cluster (UK). He is passionate about using big data and artificial intelligence to accelerate treatments for rare diseases. He is the Co-Founder and Chief Executive of Healx Ltd, a tech company focused on treatment predictions for rare diseases. Healx was selected in the global 'Disrupt 100' list 2017, covered in the Harvard Business Review, finalist of Pitch@Palace and awarded 'Cambridge Startup of Year' 2016 and 'Cambridge Life Science Business of the Year' 2015.

Passionate about patient impact, Tim 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.

“We’re a next-generation team of drug hunters, AI engineers and bioinformaticians, all working together to accelerate the discovery of new treatments for rare diseases.”