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Nekkar Lab is an Italian biotechnology company specializing in the discovery and development of small molecule therapeutics for rare genetic diseases, specifically Myotonic Dystrophy Type 1 (DM1). As a spin-off from the University of Bari Aldo Moro, the company leverages deep expertise in medicinal chemistry to target toxic RNA-protein interactions. Their primary approach involves the development of compounds designed to displace Muscleblind-like (MBNL) proteins from sequestering CUG repeat expansions, thereby restoring normal RNA splicing. Nekkar Lab's lead candidate is currently in the pre-clinical stage, aiming to provide a disease-modifying treatment for patients with limited therapeutic options. The company is supported by venture capital and academic partnerships to advance its pipeline of RNA-targeting agents.
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