Healx

Healx, founded in 2014 in Cambridge, UK, by Dr Tim Guilliams and Dr David Brown, is an AI-driven biotech company focused on discovering and developing treatments for rare diseases. With over 7,000 known rare diseases affecting 400 million people globally, only 5% have approved treatments. Healx addresses this unmet need by combining advanced AI technology with drug discovery and development expertise to accelerate treatment development. The company has raised $70 million and built a team of over 80 experts.

Healx's AI platform, Healnet, is designed to identify potential drug combinations from the 4,000 approved drugs on the market, a process crucial in their work to combat COVID-19. The platform analyses vast biomedical data to predict successful combinations, particularly focusing on vulnerable patients with pre-existing conditions like Alström syndrome and cystic fibrosis. Healx’s efforts in this area are led by Dr Brown, a veteran in pharmaceutical innovation and co-inventor of Viagra. The company’s mission remains to advance the development of therapies for rare diseases, providing hope to millions of patients worldwide.

Healx was founded in 2014 by Dr Tim Guilliams, a PhD biophysicist, and Dr David Brown, a renowned drug discovery expert. Their journey began with an encounter that would shape their mission: they met Nick Sireau, who had established a patient group to find treatments for his sons’ rare genetic disorder. Despite a decade of tireless effort, Nick found the drug discovery process slow and prohibitively expensive—a challenge faced by many patient groups.

Tim and Dave, advocates of drug redevelopment—the repurposing of existing drugs for new therapeutic uses—saw potential in combining this approach with artificial intelligence. They believed this innovative and cost-effective method could address the challenges Nick and the broader rare disease community faced. Inspired by Nick’s story, they aimed to revolutionise the treatment landscape for rare diseases.

Healx’s mission was clear: to improve the lives of rare disease patients by accelerating the discovery and development of treatments using AI. The company’s approach centres on its next-generation AI platform, Healnet. This platform integrates data from biomedical research, scientific literature, patient insights, and Healx’s curated sources to form a comprehensive rare disease knowledge graph. By applying advanced AI models to this graph, Healnet rapidly identifies novel disease-compound relationships with the highest chances of success.

The traditional drug discovery process, predominantly focused on blockbuster drugs for common diseases, typically takes 12-15 years and costs between $2-3 billion. With a failure rate of 95%, this model is unsuitable for rare disease patients, who need timely and affordable solutions. Healx’s AI-driven approach promises to cut the time required to bring a drug to market to 6-8 years, at significantly lower costs.

Healx’s innovative methods quickly demonstrated their value. The company’s lead programme, targeting fragile X syndrome, progressed from in silico discovery to the identification of active preclinical combinations in under 24 months. This success underscored the potential of AI to accelerate drug development and improve outcomes for rare disease patients.

Partnerships play a crucial role in Healx’s strategy to expand its pipeline. The company actively seeks rare disease assets in or near human clinical trials to leverage its clinical, regulatory, and commercial infrastructure. A notable partnership is with Ono Pharmaceutical Co., Ltd., one of Japan’s largest pharmaceutical companies. This collaboration aims to identify new disease indications for Ono’s proprietary assets using Healnet’s innovative indication expansion technology.

Healx’s commitment to patient-inspired innovation extends to its values. The company prioritises the needs of rare disease patients, fostering a supportive environment for patients and their families. It also emphasises personal and professional growth for its team, promoting a culture of mutual respect, honest communication, and teamwork.
 

By 2025, Healx plans to launch around 50 programmes, aiming to bring treatments to millions of underserved rare disease patients. This ambitious goal is made possible by the company’s AI-powered drug discovery methods, focus on derisked drug candidates, and application of financial risk modelling.

Healx’s journey, from its founding to its current position at the forefront of AI-driven drug discovery, exemplifies the transformative potential of combining innovative technology with deep pharmacological expertise and patient engagement. The company continues to push the boundaries of what is possible in rare disease treatment, offering hope to millions of patients worldwide.

Healx’s mission is to improve the lives of rare disease patients by using artificial intelligence to accelerate the discovery and development of treatments. By leveraging cutting-edge AI technology, Healx aims to significantly reduce the time and cost associated with traditional drug discovery processes, providing faster access to effective treatments for those with unmet medical needs.

Healx envisions a future where every rare disease patient has access to effective and affordable treatments. The company strives to be a global leader in AI-driven drug discovery, transforming the way rare diseases are treated and making a profound impact on patient lives. By pioneering innovative solutions and fostering collaborations, Healx aims to address the vast unmet needs in the rare disease community.

Healx offers a range of innovative products and services focused on accelerating drug discovery for rare diseases. Central to its offerings is Healnet, an advanced AI-driven drug discovery platform that integrates data from biomedical research, scientific literature, patient insights, and curated sources to form a comprehensive rare disease knowledge graph. Healnet uses state-of-the-art AI models to rapidly identify novel disease-compound relationships with the highest chances of success, significantly shortening the drug development timeline.

In addition to Healnet, Healx specialises in drug redevelopment, repurposing existing drugs for new therapeutic uses. This approach, combined with AI, allows the company to discover treatments faster and at lower costs. Healx also partners with pharmaceutical companies to leverage its technology to find new indications for existing assets and enhance the efficacy of drug candidates.

With a commitment to patient-inspired innovation, Healx supports clinical trial acceleration, ensuring that promising treatments reach patients as quickly as possible. Through its comprehensive suite of services, Healx is dedicated to transforming the landscape of rare disease treatment and improving patient outcomes globally.