Insilico Medicine has entered a research and development (R&D) collaboration with the Mechanisms of Inherited Kidney Disorders (MIKADO) group at the University of Zurich (UZH) to discover new treatments for cystinosis using artificial intelligence (AI) platform.

Under the collaboration, the MIKADO group will use its multi-omics databank for identifying cellular and molecular pathways, which drive dangerous complications that cause cystinosis, a rare genetic disease.

Discover B2B Marketing That Performs

Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.

Find out more

The databank was obtained from cell models based on cystinosis and preclinical models using Insilico’s PandaOmics, a new target discovery AI platform.

Insilico Medicine CEO Alex Zhavoronkov said: “Cystinosis is a commonly neglected disease with large unmet needs. We are pleased to partner with MIKADO at the UZH combining the best of PandaOmics target discovery AI and human intelligence for the potential benefit of cystinosis patients worldwide.”

The MIKADO group will study disease-relevant targetable pathways and use the algorithms of Insilico for generating small molecule compound libraries.

They are expected to be assessed in preclinical models and cystinosis cell systems using the MIKADO group-developed screening technologies.

GlobalData Strategic Intelligence

US Tariffs are shifting - will you react or anticipate?

Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.

By GlobalData

MIKADO team leader and senior scientist Alessandro Luciani said: “Using innovative preclinical models and disease-relevant phenotype screening, we aim to validate novel drug targets for diseases that no cure has been found for.

“Ultimately, we want to improve the quality of life for those affected by the disease and bring tangible hope to thousands of cystinosis patients around the world.”

Caused by inactivating mutations in the CTNS gene, cystinosis slowly destroys the organs including muscles, pancreas, gonads, kidneys, eyes, the thyroid, the liver and the brain in the body.

The initial research collaboration between Insilico and UZH will be for one year.

In 2020, the company entered a collaboration with Boehringer Ingelheim to use AI technology for identifying potential therapeutic targets.

Pharmaceutical Technology Excellence Awards - Nominations Closed

Nominations are now closed for the Pharmaceutical Technology Excellence Awards. A big thanks to all the organisations that entered – your response has been outstanding, showcasing exceptional innovation, leadership, and impact.

Excellence in Action
Awarded the 2025 Pharmaceutical Technology Excellence Award for Business Expansion in Integrated Manufacturing, Upperton Pharma Solutions is rapidly expanding its UK GMP and sterile manufacturing footprint. Find out how Upperton’s integrated CDMO model helps pharma companies move from early development to clinical and niche commercial supply with fewer handovers and faster timelines.

Discover the Impact