Brussels-based Artificial Intelligence startup Kantify and Paris-based I-Stem institute have announced a successful collaboration on using Artificial Intelligence and report the discovery of a new therapy for an uncured rare neuromuscular disease. These results, published on 27 April 2022 in Frontiers in Pharmacology, are opening a new door to the faster development of therapies against rare diseases. 

95% of rare diseases still don’t have a treatment. Identifying safe and effective drugs for a disease is a long, risky and very costly process. 

Together, Kantify and I-Stem have partnered in order to accelerate the discovery of a successful drug against a specific type of myopathy, called Limb girdle muscular dystrophy R3, which is a rare disease affecting muscles.

Thanks to the combination of Artificial Intelligence and drug screening, they have managed to identify in a reduced timeframe a molecule that can decrease the effects of this rare myopathy.

This project, started in 2016 from the collaboration of Genethon and I-Stem, was based on the original idea that this disease could be treated by limiting the degradation of misfolded forms of alpha sarcoglycan. To challenge this idea, the two institutions developed a bioassay allowing to assess in high throughput the therapeutic impact of thousands of drug candidates. 

This is when Kantify entered the project. Based on the initial results from I-Stem, Kantify used its Artificial Intelligence (AI) technology to predict what could be the possible unforeseen effects in terms of toxicity of the hit compounds and assess the potential success of their clinical development.

"Kantify’s AI technology allowed us to select very rapidly the most promising candidate. We report in this study that the combination of Givinostat and Bortezomib save from degradation the most frequent forms of misfolded alpha sarcoglycan but we also showed that it has a positive impact on cystic fibrosis. This is really exciting to see the power of this technology!” said Dr Xavier NISSAN, leading scientist of the study.

Both organizations are pioneers in their respective areas of expertise.

On the one hand, I-Stem is a known pioneer in stem cell research. I-Stem is backed by the AFM Téléthon patients’ association, the largest European patient organization for research against neuromuscular diseases.

On the other hand, Kantify has already developed a number of innovative solutions for AI and health, and announced a few weeks ago their drug discovery technology Zeptomics. Both partners have announced that they have other promising results in terms of identification of therapeutic targets thanks to AI, and are now working on new drug discovery projects. 

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