Healx: Breaking Ground with AI-Enabled Drug Discovery for Rare Diseases


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The pace and scale of medical and scientific innovation are transforming the biopharma industry rapidly, and the need for better patient engagement and experience is spurring new tech-inspired business models.

Although there is a high level of innovation in the industry, biopharma companies are facing a complex and challenging environment due to increased competition and R&D cycle times. Bringing a new pharmaceutical drug to market takes years and can cost billions in R&D expenditures. The industry is now seeking an efficient method of approaching this process—and artificial intelligence (AI) is emerging as a faster and more accurate alternative to drug discovery for chronic and rare diseases.

Healx is a patient-inspired technology company accelerating the discovery and development of rare disease treatments with the help of its AI-powered solutions. Its advanced drug discovery platform leverages public and proprietary biomedical data in the form of a knowledge graph for rare diseases. Healx is combining this data with patient insights and cutting-edge AI models to find novel treatments, and they are bringing their first treatment to clinical trial later this year.

Maximizing the Applications of Machine Learning

Healx was Co-Founded by Dr. Tim Guilliams and Dr. David Brown in 2014. The Cambridge-based researchers are strong advocates of the benefits of drug redevelopment. Combining that with the power and speed of AI, they found a viable solution for drug discovery. Today, Healx is an 80+ strong, world-class team of drug discoverers, AI engineers, and bioinformaticians, all working together to accelerate the discovery of new treatments for rare diseases.

While the technology underpinning Healx may be complex, the premise is simple. The company finds links between existing compounds and rare diseases without treatments using a range of algorithms. Critically, these models are trained to identify connections between compounds and diseases that have never been considered before.

“I think we found a sweet spot where we can basically maximize the work by applying machine learning and finding shortcuts to this very long drug discovery process,” says Dr. Tim, Chief Executive Officer of Healx.

Collaborating to Fast-track Treatments

Healx collaborates with academic, patient, and industry groups to quickly progress new rare disease treatments through the clinics and on to the patients. The company combines its innovative AI technology and in-house drug discovery expertise with patient and scientific insight to fast-track effective redeveloped treatments towards patients. It works across a wide spectrum of rare disease areas and collaborates with various institutions from around the world to identify and progress novel therapies.

The company’s collaborative efforts can be taken up in various forms—from co-development partnerships to in-licensing of drug repurposing assets. This year alone, Healx has joined forces with Mission Cure, PKD Charity, Ataxia UK, and FARA to find treatments for rare conditions.

The Rare Treatment Accelerator

Healx is dedicated to providing rare disease treatments to those in need. It has devised a unique Rare Treatment Accelerator (RTA) program that offers necessary financial and clinical resources to other promising drug redevelopment implementers including academic groups, patient organizations, and early-stage biotech researchers.

With its AI technology, drug development, and clinical expertise—along with the necessary financial resources—Healx evaluates, enhances, and accelerates the drug development of the RTA program-aided partners. The company also aims to ensure the commencement of clinical trials within 6-12 months from the project’s start.

Dedicated Knowledge Sharing Platforms

Along with its unique AI-enabled approach to finding treatments for rare diseases, Healx’s redevelopment method identifies existing candidate drugs that can improve the quality of life of patients with rare diseases. The team deploys its AI to examine several different data types, such as genomic expressions and mutations to identify potential treatments that may reverse the disease. Such recognized candidate drugs are then tested and prepared for clinical trials.

Healx has also created a free website for knowledge sharing. RareOmics is a tool that provides information to researchers, charities, and individuals about the latest scientific literature on rare diseases. It automatically searches for hundreds of rare diseases in various journals, articles, and abstracts added to PubMed every day. Healx has equipped Rareomics with text mining tools to identify diseases, genes, species, and thousands of other scientific terms to obtain the best possible results.

Resolution to Deliver

Only a fraction of the known rare diseases in the world have approved treatments. This is largely due to the high costs and low return of rare disease drug treatments. The development costs billions of dollars yet there are a comparatively small number of people buying it. Healx aims to provide a cheaper and faster alternative to drug discovery and is rapidly scaling its team, pipeline, and technology to achieve this. Healx has recently been validated for its innovative model with the FRAXA Research Foundation. In collaboration, the company is working to find treatments for ‘Fragile X syndrome,’ which is considered to be a dominant genetic cause of autism. In the coming months, Healx and FRAXA are set to launch early trials in order to test treatments for this rare genetic disease.

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