AI for Rare: Healx is Reimagining Drug Discovery with Speed, Precision, and Hope

The Silicon Review

For millions of patients suffering from rare diseases, the world of medicine has long been shaped by neglect. With more than 10,000 known rare diseases affecting an estimated 300 million people globally—of which 90% still lack approved treatment—the traditional pharmaceutical model has failed to offer solutions. But Healx, a UK-based biotech company founded in 2014, is rewriting that narrative. Powered by artificial intelligence and driven by patient-inspired urgency, Healx is bringing a radical new approach to drug discovery. At the helm of this mission is Dr. Tim Guilliams, a bioengineer and co-founder of Healx, who teamed up with Dr. David Brown, the pioneering mind behind Viagra, to tackle the inefficiencies in traditional drug development. Their inspiration was deeply personal: Nick Sireau, a father whose children were afflicted by a rare condition called Alkaptonuria (AKU), had spent years navigating a system that moved too slowly and cost too much. Tim and David believed there had to be a faster, smarter, and more equitable way—and that belief became the foundation of Healx.

Rethinking the Drug Development Paradigm

Traditional drug discovery is notoriously slow, costly, and risk-laden, often taking over a decade and more than $2 billion to bring a single drug to market—with just a 5% success rate. This economic calculus has pushed pharma giants to chase blockbuster drugs targeting large populations, leaving rare disease communities underserved. Healx flips that script by leveraging cutting-edge AI to mine massive datasets—ranging from biomedical literature and clinical studies to genetic information and chemical structures—and identify novel drug-disease relationships. Rather than developing compounds from scratch, the company specializes in redeveloping, combining, or enhancing known drugs, significantly reducing time and cost. “Our platform can simulate the equivalent of 300 years of human reading, scanning over 100 million data points to identify high-confidence links between existing compounds and rare diseases,” explains Guilliams. “This enables us to de-risk the early stages of drug discovery and run multiple programs in parallel.”

From Prediction to Patient

Healx’s process isn’t just theoretical. The company’s pipeline is expanding rapidly, featuring candidates that are already in advanced stages of development. Its lead asset, HLX-1502, is currently in Phase 2 clinical trials for Neurofibromatosis Type 1 (NF1), a genetic disorder that causes tumors to form on nerve tissue. Additional candidates are progressing through the pipeline for conditions like Fragile X Syndrome, Angelman Syndrome, and Autosomal Dominant Polycystic Kidney Disease. This rapid progress is powered by Healx’s proprietary AI platform, which brings together multiple disciplines—genetics, transcriptomics, cheminformatics, and natural language processing—to build a rich, integrated knowledge graph. These AI-generated hypotheses are then vetted by human experts before moving into preclinical and clinical validation stages. “It’s not a black box,” notes Guilliams. “Our AI doesn't replace scientists—it augments them. The platform flags the most promising leads, and then our team of domain experts reviews, validates, and advances them.”

A Model Built on Partnerships

Healx has cultivated a unique ecosystem of collaborators, ranging from academic researchers and pharmaceutical companies to patient advocacy groups. Dr. Bruce Bloom, the company’s Chief Collaboration Officer, plays a pivotal role in aligning scientific innovation with community engagement. “Patients are not just endpoints—they are collaborators in this journey,” Bloom says. “Their lived experiences provide crucial insights that guide our therapeutic strategies. We involve them at every stage, from identifying unmet needs to designing clinical trials.” This collaborative ethos extends into licensing and business development. Dr. Mark Youssef, Global Head of Business Development and Licensing, is leading efforts to partner with pharma and biotech organizations looking to expand their portfolios into rare diseases—without the burden of building from scratch.

Culture of Innovation, Grounded in Values

Behind the technology lies a team driven by a shared sense of purpose. Healx has fostered a culture that values curiosity, resilience, and humility. Employees are encouraged to take calculated risks, learn from failure, and challenge convention. The company’s four core values—Care for Rare, Grow as Individuals, Win as a Team, and Innovate and Deliver—aren’t mere slogans; they guide strategic decisions and everyday operations. “Drug discovery for rare diseases is complex and emotionally taxing,” says Guilliams. “You need a team that not only has the technical capabilities but also the emotional resilience to keep pushing forward.” That commitment to purpose is perhaps Healx’s most powerful asset. The company's story began with a single father searching for a cure, but today it’s a global movement aimed at making treatment accessible for every rare disease patient.

A Glimpse into the Future

As Healx advances its drug candidates through clinical trials, its sights are firmly set on scalability. The company believes that its model—fueled by AI and human intelligence—can revolutionize drug discovery across all therapeutic areas, not just rare diseases. But its focus will remain firmly patient-centric. “Rare diseases are our proving ground,” says Guilliams. “If we can bring effective treatments to the most underserved populations, then we’ve proven that this model can work anywhere.” With a growing pipeline, a robust AI engine, and a passionate team, Healx is not just transforming drug discovery—it’s changing lives. And for the 300 million patients still waiting for answers, that transformation can’t come soon enough.

Dr.Tim Guilliams, Co-founder and CEO