Healx joins forces with Boehringer Ingelheim to discover new treatment approaches for rare neurological diseases

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Healx, the AI-powered biotechnology company focused on developing breakthrough treatments for rare diseases, today announces it has entered into an agreement with Boehringer Ingelheim to identify indications related to rare neurological diseases.

The project will leverage Healx’s comprehensive AI-based drug discovery platform, Healnet, and expertise in rare diseases and pharmacology, to identify potential new indications for assets from Boehringer Ingelheim’s pipeline.

Healx will use their data analysis and technological capabilities to assist Boehringer Ingelheim’s efforts in prioritising selected indications for further research. Their AI platform, which draws upon the most comprehensive knowledge graph of rare disease data to intelligently predict and match treatments to rare diseases, will play a key part in the collaborative effort.

In addition, Healx has significant experience in the area of rare neurological disorders, such as fragile X syndrome as well as Pitt-Hopkins syndrome. Healx's AI-based drug discovery platform (Healnet) has proven success in that it identified eight repurposing drug candidates with potential benefits for fragile X syndrome, which were not considered for the condition prior to Healx’s involvement. As a result, several candidates have demonstrated efficacy in vivo and one candidate is now progressing toward a Phase 2a trial.

In another project, carried out in collaboration with the Pitt Hopkins Research Foundation, Healnet was successfully used to investigate, predict and progress novel treatment options for the ultra-rare neurodevelopmental disorder Pitt-Hopkins syndrome.

Dr Tim Guilliams, Founder and CEO of Healx, commented: “Healx is dedicated to transforming the lives of rare disease patients by intelligently matching and developing potential drug treatments to their conditions. We believe that sophisticated technologies, such as AI and machine learning methods, will be fundamental to any drug discovery and development project in the future.

The project with Boehringer Ingelheim presents an opportunity for further innovation in identifying rare disease candidates for patients whose medical needs are not currently met. Harnessing BI’s expertise for drug development, especially in rare neurological conditions, in combination with our AI-powered drug discovery platform, will enable faster identification of potential new treatments.”

More than 7,000 rare diseases have been identified to date (diseases affecting fewer than 1 in 2,000 people), with 250 new rare diseases classified annually. Collectively, this impacts over 400 million individuals worldwide with less than 5% of these rare diseases having approved treatments. There is therefore a substantial unmet medical need1.

Healx’s mission is to use its AI platform, deep pharmacological expertise and extensive patient engagement to improve the quality of life and care for patients with rare diseases. Its business model is to develop its own pipeline of proprietary drug candidates, as well as to work in partnership with a range of organisations to explore novel pharmacological routes and indication expansion opportunities.

The project with Boehringer Ingelheim adds to Healx’s portfolio of agreements and collaborations with pharmaceutical and biotechnology companies, academia, clinical institutes and rare disease charities since its foundation in 2014.

1 Rare Neurological Disease Special Report, Neurology Reviews, March 2019.


About Healx

Healx is an AI-powered and patient-inspired technology company, accelerating the discovery and development of rare disease treatments. Their AI drug discovery platform leverages public and proprietary biomedical data and features the world’s leading knowledge graph for rare diseases. Combining their technology with patient insights and drug discovery expertise, Healx’s mission is to advance 100 rare disease treatments towards the clinic by 2025.


Healx is an AI-powered and patient-inspired technology company, accelerating the discovery and development of rare disease treatments.