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Libra Therapeutics develops novel, disease-modifying small molecules that restore cellular balance by improving lysosomal function and autophagy, aiming to halt or slow neurodegeneration. Their lead program targets TRPML1 and plans to enter clinical trials in ALS in the near future. The company operates virtually, partnering with CROs for all R&D activities. Backed by top-tier investors, it leverages proprietary technology from Axxam S.p.A.
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