Building the world’s first AI-native rare disease pharma.
End to end.
of the 10,000+ rare diseases have no approved therapy.
AI is changing that.
Agentic rare disease platform, from discovery to launch.
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Optimal disease selection
Chooses rare diseases with the greatest unmet need and highest probability of success.
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Bias-free drug discovery
Uncovers novel disease biology and small molecules to develop breakthrough rare disease treatments.
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Efficient clinical development
Designs smaller, faster trials tailored to rare disease populations to improve the probability of success.
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Lean commercial launch
Gets medicines to the patients who need them most, with a small team amplified by agentic AI.
AI-native, from discovery to launch
AI agents
Bring speed and scale to every stage of rare disease drug development, from disease selection and discovery to regulatory writing and launch.
Proprietary rare disease data
Combines multimodal data, including omics, with unique disease biology insights from Healx’s lab and proprietary Rare Disease Atlas.
A growing rare disease pipeline.
Rare & paediatric oncology
Rare neurology
The people behind Healx.
A team of 50+ combining AI and drug development expertise, dedicated to rare disease from day one.
Leadership
Christopher Moertel MDClinical Development
Mark Youssef, PharmD, MBABD & Licensing
Louisa CheungClinical Operations
Simone MansoNF1 Development
Richard Davenport PhDDrug Discovery
David Warshawsky PhDCancer Prevention
Andrea WalmsleyChemistry & Manufacturing
Miguel PignatelliTech Platform & Strategy
Trevor Bennington FCCAFinance
Nicholas StreetAgentic Operations
Board and investors
Jonathan Milner PhDChairman- Tim Guilliams PhDCo-founder & CEO
- Dave Brown PhDCo-founder
Laura ConnellBoard member
Suranga ChandratillakeBoard member
Abdul GueforBoard observer
