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Your mission, our focus

Working together, we can discover and develop novel treatments for your rare disease – fast

AI-powered, patient-inspired treatments for rare diseases

We combine our innovative AI technology with patient insight and in-house drug discovery expertise to fast-track novel and effective treatments towards patients.

We work across a wide range of disease areas and collaborate with patient groups from around the world. This work takes many different forms, from co-development to the in-licensing of in-development drug redevelopment opportunities, but one constant is that patients are kept firmly at the heart of every project – they’re the real rare disease experts after all.

Together with patient groups, we’re taking a new approach to developing treatments – one that is AI-powered but, even more importantly, one that is patient-inspired. Let’s collaborate to accelerate the development of novel rare disease treatments.


Working together we’re able to

Discover and develop effective treatments for rare diseases

By combining AI technology with a focus on finding novel monotherapy or combinations of existing drugs, we are able to achieve a probability of success that’s higher than traditional drug discovery methods allow – in terms of taking a treatment from discovery phase to the market.

Healnet, our AI drug discovery platform, allows us to quickly predict which known drugs have the highest chance of success at treating rare conditions, as well as those that might work well in combination. The benefit of combining several known drugs is that we have a higher chance of being able to target more of the complex and diverse symptoms rare diseases display – and often at lower drug doses.

The added advantage of redeveloped known drugs means that we’re focusing on drugs whose safety profiles and mechanisms of action have already been established. This helps de-risk the treatment discovery process even further.

Fast-track novel treatments towards patients

Together with patient groups, we work to progress new treatments from drug prediction stage towards clinical trial phase in a time frame of around 24 months. Our AI technology and focus on drug redevelopment are key to this.

The use of AI allows us to analyse all available disease- and drug-related data to pinpoint new treatments for rare conditions at a fraction of the time it would take humans to do this. Critically, it is during this process that we often also uncover completely new disease-drug interactions.

An added benefit of this approach is that because we’re looking at known drugs that can be redeveloped, we’re often able to move directly to Phase IIa clinical trials, since the safety profile of these treatments has already been established. This further accelerates the treatment discovery process.

Keep patients at the heart of treatment discovery

From the work we’ve done to date, with patient group partners from around the globe and across multiple different disease areas, we’ve found that the best results come from a collaborative approach.

By pooling our resources to combine our AI tech and drug discovery expertise with patient insights and disease knowledge, we’re able to achieve the best results and patient outcomes.

In terms of this collaborative approach we’re keen to work in a way that benefits all involved. Take a look below for more info on how we can work together.

Ways of working together


Treatment discovery partnerships

Interested in discovering and developing treatments for your rare disease of interest? We work with patient organisations to discover and develop novel therapies – taking these treatments through clinical trials and to patients together.

To do this we typically partner with patient groups from the outset of a project. This is an approach which has proved successful when working with partners such as FRAXA Research Foundation and the Children’s Tumor Foundation.

Sharing our resources, knowledge and expertise, we’re able to fast-track the discovery and development of these treatments – with the aim of taking treatments from prediction phase to clinical trial stage within 24 months.

If you’re interested in learning more about opportunities for how we can work together, please click the button below.

Get in touch


Licensing opportunities

Do you have a redeveloped drug with evidence of efficacy in your rare disease of interest but need additional support to take these towards patients? Working together, we can quickly turn your research, insights and redevelopment assets into treatments – helping transform your research into reality.

By applying our drug development and clinical expertise, along with the necessary financial resources, we’re able to evaluate, enhance and accelerate the development of your redeveloped drugs.

In these cases, we may also be able to use our technology to validate and enhance available redevelopment assets – with the aim of starting a clinical trial within 6-12 months from project start.

This type of partnership is a particular focus of our current Rare Treatment Accelerator (RTA) programme. Click the button below to find out more.

Find out more

Hear from our patient group partners

Vector Dr Allyson Berent

Healx’s use of cutting-edge AI, combined with our scientific team’s expertise in cell lines and animal models, makes this innovative program incredibly promising - creating a unique opportunity to quickly bring therapies from bench to clinic.

Dr Allyson Berent
Chief Science Officer, Foundation for Angelman Therapeutics (FAST)
Vector Tess Harris

We are delighted to be collaborating with Healx in this pioneering partnership which has the potential to accelerate the development of much-needed treatments for both forms of PKD.

Tess Harris
CEO, PKD Charity
Vector Dr Mike Tranfaglia

The Healx partnership has been nothing short of amazing. In less than two years together, we were able to deliver decades-worth of drug discovery, and now we’re taking those discoveries to the clinic.

Dr Mike Tranfaglia
Chief Scientific Officer and Co-Founder, FRAXA Research Foundation
Vector Megan Golden

We are excited to move the needle forward for pancreatitis patients with the help of Healx. Their AI-powered drug repurposing model gives pancreatitis patients hope that they will see an effective treatment for their disease in the next five years.

Megan Golden
CEO, Mission: Cure
Vector Annette Bakker, PhD

We at Children’s Tumor Foundation (CTF) are very excited about this partnership. It is very encouraging that Healx is committed to developing innovative treatments for NF patients in collaboration with CTF.

Annette Bakker, PhD
President, Children’s Tumor Foundation
Vector Kate Adcock, PhD

It’s an exciting time for research into muscle-wasting conditions, and treatments are on the horizon. By working with world-leading partners like Healx, we can drive vital research forward and accelerate access to treatments.

Kate Adcock, PhD
Director of Research and Innovation, Muscular Dystrophy UK (MDUK)
Vector Irina Haivas

We believe that the new paradigm in drug discovery will emerge at the intersection of technology, data, and biology, and we’re confident that Healx’s team is paving the way to a new gold standard in rare disease treatment discovery.

Irina Haivas
Partner at Atomico and Board Member of Healx,
Vector Suranga Chandratillake

The future of medicine will focus on what is different about everyone, personalising treatment and taking the individual into context. Healx’s Healnet platform, at the forefront of this shift, is already delivering value and Balderton is excited to have the opportunity to be part of the company’s journey.

Suranga Chandratillake
Partner at Balderton Capital and Board Member of Healx,
Vector Trina Van Pelt

AI coupled with compute can disrupt and dramatically speed the drug discovery process, and Healx owns the most comprehensive knowledge graph of rare diseases. We’re excited to support this experienced leadership team and provide Intel technology to optimize deep learning workloads, improve performance and lower costs.

Trina Van Pelt
Vice President and Senior Managing Director at Intel Capital,
Vector Jonathan Milner

It’s a pleasure to have been part of Healx since the very beginning. The way they’ve scaled the team, the tech and the number of rare disease projects worked on over the years has been astonishing to watch. If ever proof was needed of the power of technology for societal good - this is it.

Jonathan Milner
Angel Investor and Board Member of Healx,

Helpful resources


Watch our webinar series to learn more about the tools, data and network needed to better work with industry partners.


Take a look at our sponsorship page for more information on how we support and sponsor patient organisations.

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