Healx

Healx is a leading healthtech company in the UK that uses generative artificial intelligence to find and develop treatments for rare diseases. Founded in 2014 in Cambridge by Dr Tim Guilliams and Dr David Brown, Healx aims to address one of the biggest gaps in healthcare: the fact that 95 percent of rare diseases still have no approved treatments. Healx is recognised as one of the leading UK AI companies, driving innovation in biotechnology through its advanced use of artificial intelligence to accelerate rare disease drug discovery.

What is Healx?

Healx is a clinical-stage biotechnology company that combines AI, machine learning, and deep scientific expertise to accelerate drug discovery for rare diseases. Its mission is to bring new therapies to patients faster and more efficiently by identifying new uses for existing drugs and discovering new compounds. This approach significantly reduces the time, cost, and risk involved in traditional drug development.

Founders and Leadership

Healx was founded by Dr Tim Guilliams, a social entrepreneur and co-founder of the Cambridge Rare Disease Network, and Dr David Brown, who co-invented Viagra during his time at Pfizer. Together, they built Healx to bridge the gap between AI technology and rare disease research. Today, the leadership team continues to expand the company’s reach and partnerships across the global biotech industry, positioning Healx as a major player among healthtech companies in the UK.

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Tim Guilliams, CEO, Healx

How Healx Uses AI in Drug Discovery

At the core of Healx’s success is its proprietary AI platform, Healnet. This generative AI system integrates vast biomedical datasets, patient insights, and scientific literature to create a powerful rare disease knowledge graph. Healnet analyses millions of data points to identify potential connections between diseases and drug compounds that humans alone might never detect.

The platform enables the discovery of novel drug-disease relationships at unprecedented speed. It blends traditional phenotypic and target-based methods with cutting-edge AI algorithms to find promising therapeutic candidates in months rather than years. This process helps Healx prioritise the most promising opportunities for clinical trials and collaboration.

The Healx Therapeutics Pipeline

Healx has built a strong and diverse pipeline focused on rare neurological and oncology disorders. Key programs include:

  • HLX-1502: A treatment for neurofibromatosis type 1 (NF1), which has received FDA clearance to begin Phase 2 clinical trials.
  • HLX-4310: A preclinical program targeting osteosarcoma, a rare bone cancer.
  • Fragile X Syndrome Program: An AI-discovered combination therapy that progressed from computer models to active preclinical testing in under two years.

These programs highlight how Healx therapeutics can move from AI discovery to clinical development faster than traditional research models.

Healx Funding and Strategic Partnerships

Healx has secured more than $110 million in total funding to date. In August 2024, the company announced a $47 million Series C funding round, co-led by R42 Group and Atomico, one of Europe’s largest venture capital firms. Other participants included Balderton Capital, Global Brain, and Jonathan Milner. This funding is being used to advance Healx’s clinical programs in rare oncology, renal, and neurodevelopmental disorders.

Healx’s partnerships play a vital role in expanding its scientific and commercial impact:

  • Sanofi Collaboration (2024): A partnership to identify new rare disease indications for Sanofi compounds using Healx’s AI platform.
  • SCI Ventures Alliance (2025): A joint initiative to develop AI-driven treatments for paralysis and spinal cord injuries.
  • Vuja De Sciences Partnership (2025): A US-based collaboration to combat cancer recurrence through generative AI drug discovery.

These collaborations demonstrate how Healx’s technology is being integrated into broader biopharma research to accelerate global therapeutic discovery.

The Impact of Healx on Rare Disease Research

The need for innovation in rare disease treatment is urgent. More than 400 million people worldwide are affected by rare conditions, and most lack effective therapies. Traditional drug discovery takes up to 14 years and often costs billions, making it unviable for small patient populations.

Healx is tackling this problem by combining the speed of AI with scientific expertise. Its approach reduces the time needed to move from discovery to clinical testing, often bringing therapies to patients in under eight years. This acceleration not only lowers development costs but also increases the chance of success in finding effective treatments.

Healx’s AI platform helps eliminate human bias in research by analysing diverse datasets without preconceived notions. It identifies patterns and relationships that would otherwise remain hidden, opening the door to new therapeutic possibilities.

Recent Developments and Future Outlook

2025 has been a pivotal year for Healx. In February, the company announced that the first patient had been dosed in its Phase 2 trial for HLX-1502, a major milestone in its NF1 program. This followed a successful collaboration with the US Food and Drug Administration to advance the treatment to human studies.

In July, Healx joined forces with SCI Ventures to uncover AI-driven cures for paralysis. This collaboration brings Healx’s technology into the field of neurotrauma, expanding its scope beyond rare diseases.

By September 2025, Healx announced a new alliance with Vuja De Sciences in the United States to target the recurrence of aggressive cancers. The partnership leverages Healx’s generative AI capabilities to find combination therapies that prevent tumour relapse.

Looking ahead, Healx aims to scale its Healnet platform further, expanding its therapeutic pipeline to more than 50 programs by 2027. With a strong financial foundation and growing strategic partnerships, the company is well-positioned to lead the next phase of AI-powered precision medicine.

Why Healx Stands Out

Healx’s blend of AI, biology, and human insight places it at the forefront of the third generation of drug discovery. Its model demonstrates how advanced technology can directly improve lives by creating affordable, faster, and more targeted treatments. As one of the most innovative healthtech companies in the UK, Healx represents a shift toward data-driven medical solutions that prioritise patients with unmet needs.

Frequently Asked Questions

Healx is headquartered in Cambridge, United Kingdom.

Healx was founded in 2014 by Dr Tim Guilliams and Dr David Brown.

Healx has raised more than $110 million, including $47 million in its most recent Series C funding round.

Healx’s proprietary AI platform is called Healnet.

Healx’s pipeline includes treatments for neurofibromatosis type 1, osteosarcoma, fragile X syndrome, and other rare neurological and cancer-related diseases.

Healx’s journey shows how AI can reshape drug discovery for rare diseases. By combining machine learning with expert scientific insight, the company is closing the treatment gap for conditions that have been overlooked for decades. With strong funding, growing partnerships, and an expanding clinical pipeline, Healx continues to stand out as one of the UK’s most forward-thinking biotech innovators, setting new standards in the global fight against rare diseases.

By Ann Hentin

Ann Hentin is a highly accomplished Policy & EU Affairs Adviser and a leading expert in the deployment and governance of deep tech within the European Union. Leveraging her strategic expertise in Public Affairs, Ann specializes in managing complex EU R&D Project lifecycles, with a critical focus on Ethical AI, Health Tech, and Digital Health. Her background, which includes in International Relations, Security Studies & Diplomacy, provides her with a unique perspective on bridging cutting-edge technological innovation with robust European regulatory and security frameworks, establishing her as an influential voice on responsible digital transformation across Europe.