Rare disease and diagnostic specialists Centogene will partner with artificial intelligence (AI)-specialist Insilico Medicine to develop new drugs for a rare inherited neurometabolic disease called Niemann-Pick Disease Type C (NPC), which currently has no approved treatment options.
NPC is a rare debilitating lyosomal storage disease normally caused by mutations in the genes NPC1 and NPC2. It affects around 1 in 150,000 people and at least half of those with the condition show symptoms, which are neurodegenerative in nature, before the age of 10 years. Prognosis depends largely on the age of onset, with children often dying fairly quickly while adults who develop the condition can live with it for a long time.
U.S.-based Insilico Medicine hit the news in December 2021 with the announcement that it had brought the first AI-designed and discovered drug candidate to clinical trials in less than three years. Centogene is now hoping to benefit from this experience to help target NPC.
Centogene, which has its headquarters in Rostock, Germany, has a focus on diagnosing and treating rare diseases. While its focus has been mostly diagnostic in the past, it started an AI initiative in 2017 specifically to drive drug development for orphan diseases.
It has accumulated a large database of clinical, genetic, metabolomics, and proteomic information on 450,000 rare disease cases with two billion data points in total.
“Centogene’s rare disease-centric Bio/Databank is unique in the richness of the data it holds. By leveraging Insilico’s powerful proprietary AI platform, PandaOmics, with the multiomic research data from Centogene, I hope our collaboration will result in the discovery of a therapeutic target for NPC,” said Alex Zhavoronkov, founder and CEO of Insilico Medicine. “Ultimately we want to improve the quality of life for those affected by the disease and bring tangible hope to thousands of NPC patients around the world.”
The collaboration will initially last 20 weeks and will involve use of Centogene’s database and NPC cell lines, as well as Insilico’s AI-based drug target discovery platform. After initial target identification, both companies will look at the data and decide how to proceed. Any intellectual property generated by the collaboration will belong to Centogene.
“We are excited to be working together with Insilico as we seek to identify novel therapeutic targets for NPC,” added Carsten Ullrich, Senior Director of Artificial Intelligence at Centogene. “Combining both Centogene’s expertise in multiomics and unique global insights from the world’s largest NPC cohort with Insilico’s next-generation AI platform has the potential to enable an accelerated cure for this rare and often rapidly progressing disease.”
