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LAB282 is a small molecule therapeutic candidate being developed for the treatment of hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome. It is designed to address the underlying vascular malformations characteristic of HHT, which are caused by mutations in genes within the transforming growth factor-beta (TGF-β) signaling pathway, such as ENG or ACVRL1. While the specific molecular target has not been publicly disclosed in detail, the drug is intended to stabilize vascular structures and reduce the incidence of epistaxis (nosebleeds) and other complications associated with the disease. LAB282 has received Orphan Drug Designation from the U.S. FDA for the treatment of HHT.
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