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This week’s blog was written by our 2020-2021 Corporate Partner, Healx! This is the first of a recurring blog series, which will explore a variety of themes in rare disease research.

Healx is hosting a “Kickstart Drug Repurposing: Finding Treatments for your Rare Disease through Drug Redevelopment” workshop at our virtual Drug Repurposing for Rare Diseases 2021 Conference. Get to know their team and claim your free ticket now to join us on the 15th – 16th June!

At Healx, we believe the best way to find new rare disease treatments is to start with known drugs. By combining our artificial intelligence (AI) technology with patient insights and the expertise of our drug discovery team, we redevelop known compounds to create novel and effective treatments for rare diseases.

This AI-powered redevelopment approach allows us to quickly and efficiently discover and develop new treatments with a high probability of success. It also allows us to increase the number of diseases – and patients – that we’re able to find treatments for.

From drug prediction to pre-clinical validation, clinical trials to commercialisation, this focus on redeveloping drugs is beneficial in a number of different ways. But who better to explore the reasons why than our very own team members! Read on to discover how our team believes this approach benefits the development of new rare disease treatments.

Justin Grace
Machine Learning Engineer
Team: Technology

Our team is responsible for developing the machine learning (ML) and natural language processing (NLP) tools that power our AI drug-discovery platform – Healnet. Healnet integrates data from multiple sources (everything from disease ontologies to clinical trial data and scientific literature) into a detailed knowledge graph of rare disease information. This graph is then analysed by multiple state-of-the-art AI models to uncover previously unknown relationships between existing compounds and rare diseases.

From a technical perspective, the benefit of focusing on known drugs is that it increases the speed with which we’re able to predict therapies. Traditional drug discovery is often focused on the development of new chemical entities (NCEs) and this process can take many years, carrying with it a significant failure rate.

By concentrating on known chemical entities – the 4,000 approved drugs already out in the world – we’re able to narrow down the search for potential treatments, because our AI technology is trained to read all the available information out there and quickly find connections between those compounds and diseases. So rather than starting from scratch, we already have a good idea of how compounds act in certain biological environments and our technology can pinpoint and rank the drug predictions with the highest probability of success. This information is then shared with our pre-clinical and clinical teams for expert validation. Our approach is very much a collaboration between human expertise and AI tools.

Emma Davies, PhD
Principal Scientist
Team: Pre-clinical

As part of the pre-clinical team, I analyse the drug predictions provided by our technology to decide which compounds – and in which combinations, formulations and dosing – we should progress through pre-clinical trials.

The focus on known drugs helps increase the speed and efficiency with which we’re able to progress through the in vivo and in vitro stages of the pre-clinical testing phase, since known drugs often have well-characterised profiles featuring information on other drug-drug interactions along with associated dosing and safety data. Not only does knowing more about these drugs to start with give us a better idea of how best to combine, administer and dose any treatments we progress through the clinic, the known safety and efficacy data also means we may be able to progress more quickly to Phase 2 clinical trials after pre-clinical validation.

Clara Tang, PhD
Business Manager – Alliance Strategy
Team: Partnerships

My role sees me working closely with patient organisations across every phase of our rare disease programmes – from discovery and development right through to taking these treatments to the market. This patient group input informs everything we do, such as deciding which symptoms to prioritise as targets for our treatments, helping with clinical trial planning and eventually, patient access.

From my viewpoint, by prioritising drugs that already have known safety profiles, it increases our probability of success. This means we’re able to increase the chances that these drugs will progress through clinical trials, in as short a time frame as possible, and on to patients where they can really begin to have a positive impact.


David King, PhD
Senior Director of Regulatory Affairs
Team: Clinical

I’m responsible for the regulatory aspects of clinical trial planning, patient participation and clinical trial execution here at Healx. This means working closely with all the functions at Healx to develop and submit the information required by regulatory agencies (e.g. EMA, FDA) to start a clinical trial and ultimately get approval to put the treatment on the market. Thanks to our focus on known drugs, whose safety profiles have already been established, this allows us to decrease the amount of new information that we need to generate at Healx. As a result, we can initiate a clinical trial sooner than when developing a new compound.

An added benefit of working with already-approved compounds is that we can reference the safety data provided by the originator company to support the registration of the product for approval to market the new treatment. This allows us to apply sooner for marketing approval to make these new treatments available to patients.

More information

We hope you enjoyed reading this post on the role of drug redevelopment for rare diseases. If you’re interested in learning more, please take a look at Healx’s website or get in touch with them online.

And if you are already in the process of redeveloping a compound to treat your rare disease of interest, we recommend taking a look at Healx’s Rare Treatment Accelerator (RTA) partnering programme which is currently accepting applications.

The benefits of working with known drugs in the search for rare disease treatments

by Guest Contributer time to read: 7 min