With a deep-rooted mission to help rare disease patients, social entrepreneur Tim Guilliams, co-founded health technology company, Healx and Cambridge based charity CRDN. He discusses his ambitions for the future for Healx and how a patient-centric approach and collaboration with patients and charities is key to driving research

​CEO Series: meeting the beating hearts behind the RARE brands

1.

What made you want to move into the wide world of rare disease, and then specifically Healx and what did that journey look like?

I have always been passionate about using artificial intelligence (AI) and machine learning to find treatments for rare diseases. In addition to my role at Healx, I’m also the Founding Director and Trustee of the Cambridge Rare Disease Network (CRDN), a charity that promotes awareness of rare diseases.
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I founded Healx in 2014 alongside Dr David Brown, the co-inventor of Viagra and former Global Head of Drug Discovery at Roche. Following a meeting with a rare disease parent, Nick Sireau, who was relentlessly trying to repurpose a drug called nitisinone to save his children from the ultra-rare disease, alkaptonuria (AKU), we realised how big the therapeutic unmet need was.

Rare disease patients have long been side-lined by the pharmaceutical industry who struggle to justify investment in treatments for smaller populations. At Healx, however, we believe that every disease patient deserves a treatment. This belief helps us to think big. We don’t intend to go after just one disease, we have created a platform that will help us impact 100 rare diseases by 2025.

We’ve since made it our mission to help rare disease patients access life-saving treatments. The first step towards making rare disease drug discovery a faster and more efficient process was to develop Healnet, the most comprehensive AI platform currently out there for the prediction of rare disease treatments.

2.

What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

I’d say that the biggest challenge—and also opportunity—for Healx over the next couple of years is really that of scaling up our operations to achieve our goal of translating 100 rare disease treatments towards the clinic by 2025.

We’ve had a number of successes so far in terms of showing that our drug predictions are effective at preclinical trial stage and we are very excited to be taking one such drug prediction project, for fragile X syndrome, to clinical trial stage later this year. Watch this space!

To help achieve our goal of taking 100 treatments towards the clinic, whilst keeping patients firmly at the heart of the process, we recently launched our Rare Treatment Accelerator—a global partnering programme that gives patient groups and Healx the opportunity to work together to quickly progress new rare disease treatments.

We have committed a total of $20 million in AI and drug discovery resources over the next two years towards finding new treatments. Following the close of a successful first call we’re looking forward to sharing more details of these partnerships and project progress in the coming months. In the meantime we’re inviting patient groups interested in applying for the next call to register their interest online: https://healx.io/rare-treatment-accelerator-registration/

3.

What has been your proudest moment in your career thus far?

The moment when we received positive results back from our fragile X syndrome preclinical trials showing that the treatments our AI-powered platform had predicted actually worked. That was a really special moment for all of us here at Healx.

We already knew that our technology worked and could accurately predict drugs to be repurposed to treat rare diseases, but to have them validated at preclinical stage—and for others to be able to see the efficacy of what we’re doing—was a fantastic feeling.

As an added bonus it’s great that we’ve been able to get from predication stage to clinical trial phase in such a short amount of time—just 24 months.
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The launch of our Rare Treatment Accelerator programme was also a very special moment for me. What made this all the more special though was the overwhelmingly positive response the programme sparked from rare disease patient groups worldwide—in terms of both the volume of applications and positive feedback we received.

4.

What and who are your personal and professional inspirations and why?

Ever since I can remember, I’ve had a huge passion for transforming the lives of rare disease patients—even outside my work. This is what prompted me to co-found the Cambridge Rare Disease Network (CRDN).
 
And even today, what still drives me in everything I do here at Healx is my dedication to making a difference to the lives of rare disease patients. That’s a very personal mission for me.
 
There have been a number of people along this journey that have really inspired me to push forward and achieve as much as I can for rare disease patients—and certainly too many to be able to name each and every one of them here! 

5.

What advice would you give someone considering working in the rare disease space?

The best advice I could give is to collaborate and work as closely as possible with rare disease patients and charities—they are the real rare disease experts, after all.
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When I think back across all the projects we’ve worked on at Healx, none of the successes we’ve enjoyed would have been possible without the support of exceptional patient groups, such as FRAXA Research Foundation, the Pitt Hopkins Research Foundation (PHRF) and aPODD, amongst others.
 
From the very beginning we felt that forming partnerships with patient groups was the right thing to do. As well as being experts in understanding rare disease symptomology, they are also usually the most determined partner in driving treatments forward. Involving them from the outset closes the discovery research loop earlier.

6.

Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

There’s always more that can be done to help the rare disease community—on both a national and local level. But, unfortunately, a lot of it comes down to funding.

That said, there are some great initiatives in the UK. Take the 100,000 Genomes Project as an example which was set up to sequence genomes from patients affected by rare diseases and cancer.
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The Centres of Excellence for rare diseases are also a great initiative for those patients who are lucky enough to have one. However, I feel we could do with more support to develop these Centres of Excellence. It would also be great to see more local rare disease networks set up to support families and patients across the country.

7.

What would you say are some of the biggest motivators for your employees?

​At Healx our belief that every rare disease patient deserves treatment is what drives us. Finding new treatment options for patients who currently have none is what gets us up in the morning and excited to come into work each day.
 
It’s also this patient-centric outlook that keeps us focused as a company and working together to achieve our mission—despite any setbacks. No one said finding treatments for rare diseases would be an easy task, but making sure our mission is always front of mind certainly helps put each success and failure we encounter along the way into perspective.

8.

What are the toughest parts of being a CEO, and conversely what are the most rewarding?

​By far the toughest part of being a CEO is having to turn away patients and charities that would like to partner with us. We wish we were able to work on every single rare disease, but however much we want to, we can’t. This is particularly the case for the time being as we refine our processes and drug prediction capabilities ahead of scaling up our operations.
 
Because of this, we’ve had to be very selective with which diseases we choose to work on. But having to say no to these patient groups never gets any easier.
 
The decision on which patient groups’ disease projects to prioritise initially as part of the Rare Treatment Accelerator programme was a particularly tough one for us to make. That’s why we’re now working with those groups who weren’t selected this time round to offer them the information and resources needed to help identify and address areas for additional development ahead of applying again.

9.

What would be your one wish for Healx for the year ahead?

​I am hopeful that the clinical trials we are running, in partnership with FRAXA Research Foundation, to test our predicted drug therapies are shown to have great effect. That would be amazing. More amazing still is that this would mean we were one step closer to giving fragile X patients a viable treatment option.

10.

If you weren’t CEO of Healx, what was Plan B?  What did your 10-year-old-self want to do as a job?