AI drug discovery collaboration for rare disease cystinosis

pharmafile | March 10, 2022 | News story | Business Services  

Insilico Medicine has entered a research and development collaboration with the Mechanisms of Inherited Kidney Disorders (MIKADO) group at the University of Zurich (UZH), to apply Insilico’s generative Artificial Intelligence platform, for the discovery of potential therapeutics for cystinosis, a rare genetic disease.

Under the collaboration, the MIKADO group will use its multi-omics databank for identifying the cellular and molecular pathways which drive dangerous complications causing cystinosis. Multi-omics is a biological analysis approach.

Cystinosis is a multisystem genetic disorder affecting 1 in 100,000-200,000 live births, for which there are currently no curative treatments. The disease poses a lifelong risk to those affected, slowly destroying the body’s organs, such as the eyes, thyroid, pancreas, gonads, and brain. It is an inherited disease, caused by an abnormal buildup of a certain amino acid called cystine.

“I am thrilled by the collaboration between MIKADO and Insilico. With the power of artificial intelligence-driven, systems biology-based drug discovery,” commented Olivier Devuyst, MD, PhD, head of MIKADO group at the UZH. “We intend to accelerate the analysis and discovery of drug targets, with the goal of bringing novel breakthrough medicines to cystinosis patients while decreasing costs and increasing probabilities of success.”

The MIKADO group and Insilico will be working together to identify, rank, and annotate novel drug targets to identify potential therapeutics for cystinosis. The duration of the initial research collaboration will be one year.

Alex Zhavoronkov PhD, CEO of Insilico Medicine shared: “Cystinosis is a commonly neglected disease with large unmet need. 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 databank was obtained from cell models based on cystinosis, and preclinical models using Insilico’s PandaOmics, a new target discovery AI platform.

“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,” MIKADO team leader and senior scientist Alessandro Luciani said. “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.”

Ana Ovey

Related Content

No items found

Latest content