Healx
Expediting Rare Disease Treatment with the Power of AI

Unearthing appropriate treatments for rare diseases have always been a persistent challenge that the healthcare sector has typically dealt with. There are almost 7,000 rare diseases in the world, with an approved treatment for only five percent of them. This is largely due to the high costs and low return of rare disease drug treatments. Developing those treatments runs into billions of dollars, with a comparatively small number of people buying them. This means that the more than 400 million people worldwide affected by these rare diseases either have found that treatments don’t exist or their cost is inflated to unprecedented levels. But now, the time has come to put an end to their sorrow. Healx, the Cambridge, UK-based startup, is turning to the data-crunching power of artificial intelligence (AI) to rewrite the economics of drug discovery for the world’s rarest diseases.

It is widely known that the usage of AI offers a high level of precision to the complicated and time-consuming discovery phase in drug development, leading to faster development timelines and a lower failure risk down the road. By channelizing the unprecedented power of AI, Healx combines innovative AI technology with in-house drug discovery expertise and patient insight to accelerate the development of new and effective treatments for rare diseases. The AI-powered approach enables Healx to discover, develop and take novel drugs to market faster, more cost-effectively and with a probability of success three times higher than traditional drug discovery methods allow.

While the technology underpinning Healx may be complex and intricate, the premise is simple. The company uses a range of algorithms to search for links between existing diseases for which there are treatments, and rare diseases for which there are not. This process begins with a “deep data curation” phase, which sees Healx’s AI program look for gaps in existing medical data.

By applying AI technology in drug development and clinical expertise, Healx evaluates, enhances and accelerates the development of an organization’s re-purposed drugs

Once identified, the biotech startup works with patient groups and academics to help fill them. Working in tandem with the scientists and researchers, Healx follows the journey of a treatment all the way from in silico prediction and preclinical validation, through the clinic, and on to commercialisation through either the co-development or in-licensing of assets. In this manner, Healx fast-tracks the development of novel and effective rare disease treatments.

Besides, the company’s Rare Treatment Accelerator global partnering programme designed to unlock the power of repurposed drugs for rare diseases is another noteworthy inclusion in Healx’s portfolio. Working together with research institutions, patient organisations and early-stage biotech startups, the company can quickly turn its research, insights and promising repurposing therapies into novel treatments. By applying Healx’s AI technology, drug development and clinical expertise, along with the necessary financial resources, the company evaluates, enhances and accelerates the development of an organization’s repurposed drugs – with the aim of starting a clinical trial within 6-12 months from project start.

Last but not least, although Healx is highly invested in diseases that fit with their current portfolio, the company is also welcoming applications from groups working on other rare conditions where relevant assets, resources and data are available, which allows them to fast-track projects to the clinic. Thus, combining their cutting-edge technology with patient insights and drug discovery expertise, the company plans to advance 100 rare disease treatments towards the clinic by 2025.