Healx

AI-driven drug discovery and repurposing platform focused on rare diseases.

Healx, founded in 2014 as a spin-out of the University of Cambridge, applies AI to rare-disease drug discovery and repurposing. Its technology combines a curated rare-disease knowledge graph with machine learning to identify and prioritize candidate treatments, and the company advances its own clinical-stage pipeline alongside partnerships with patient organizations and biopharma.

Headquarters: Cambridge, United Kingdom
Founded: 2014
Deployment options: cloud
Website: https://healx.ai
LinkedIn: https://www.linkedin.com/company/healx

AI Use Cases

Hit Identification & Lead Optimization
Target Identification & Validation

Vendor Scorecard

86% — Complete coverage. Coverage score — how completely Atlas documents Healx across 7 buyer-evaluation areas. Reflects available Atlas data, not solution quality.

Compliance & Validation — Not documented by Atlas

Ask the vendor: Which compliance certifications (HIPAA, 21 CFR Part 11, SOC 2, GxP) do you hold?

Deployment & Hosting — Documented

cloud

Pricing Transparency — Documented

Partnership / milestone based

Implementation & Onboarding — Documented

12+ months

Organization & Role Fit — Documented

Biotech Startup, Mid-Size Biotech, Large Pharma, Drug Discovery, R&D Leadership

Solution Scope — Documented

Drug Repurposing, Natural Language Processing, Target Identification

Company Viability — Documented

Series C, $130M raised, 70+ employees, Founded 2014

Always ask the vendor

Not captured in Atlas data — confirm directly. Excluded from the score above.

Data Rights & IP

Who owns model outputs? Is our proprietary data used to train models others can access, and where does our data reside?

Model Credibility & Explainability

Can you share validation studies or benchmarks, explain how the model reaches its conclusions, and describe how bias and drift are monitored?

Clinical Trial Activity

7 registered trials where Healx is the lead sponsor (1 currently recruiting). Source: ClinicalTrials.gov, verified 2026-07-29.

Phases: Phase 1 (1), Phase 2 (4), Phase 3 (2)

Top conditions studied: Angelman Syndrome, Fragile X Syndrome, Fragile X Syndrome (FXS), Neurofibromatosis Type 1, Primary Disease or Condition Being Studied: Angelman Syndrome (AS)

A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1 (NCT06541847, recruiting)
Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40 (NCT04823052, withdrawn)
A Study of OV101 in Individuals With Angelman Syndrome (AS) (NCT04106557, completed)
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome (NCT03882918, terminated)
A Study of OV101 in Individuals With Fragile X Syndrome (NCT03697161, completed)

Evidence & Outcomes

First patient dosed in Phase 2 trial of AI-identified HLX-1502 for neurofibromatosis type 1

Healx dosed the first patient in INSPIRE-NF1, a Phase 2 trial evaluating HLX-1502 — an oral investigational therapy advanced through its AI-driven rare-disease drug discovery and repurposing platform — for the treatment of neurofibromatosis type 1 (NF1).

press release (Healx) · 2025-02-24

Questions to Ask This Vendor

Which rare-disease areas does the knowledge graph cover most deeply?
How are AI-prioritized repurposing candidates validated before the clinic?
How do partnerships with patient organizations and biopharma typically work?
How is IP handled for repurposed compounds?