XL Therapeutics Ltd.
Research - Ram-On, North District, Israel
XL Therapeutics Ltd. Employees
XL Therapeutics Ltd. Details
XL Therapeutics is a biopharmaceutical company aiming to develop small molecule compounds (SMCs) to treat patients suffering from Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT), rare primary human immunodeficiencies that are caused by the absence or suboptimal function of the WAS protein (WASp).WAS is caused by a defect in an X-chromosome-linked gene encoding WASp. WASp, an adaptor protein expressed exclusively in hematopoietic cells, is essential for normal hematopoietic cell function, and its deficiencies lead to defects in proliferation, migration and overall activation of immune cells. The function of many hematopoietic lineages is affected by WASp deficiency. The more severe defects in WASp expression lead to WAS which includes varying degrees of thrombocytopenia, eczema, immunodeficiency, autoimmunity, and malignancy. Less severe deficiencies in protein expression can lead to XLT where the primary defects are manifested in platelet dysfunction. WAS and XLT each affect about 4 patients per million male newborns worldwide, and most WAS patients die by the age of 10 without early intervention.SCIENTIFIC APPROACHProtecting WASp from degradation in WAS/XLT patients could restore normal or near-normal protein levels and thus improve immune cell function.XL Therapeutics has identified SMCs that bind to WASp and stabilize the protein by protecting it from ubiquitylation and proteasomal degradation. These WASp-binding SMCs will undergo further development to provide a lead SMC with drugable properties and acceptable safety profile, that would present a potential novel treatment for WAS/XLT patients wherein the molecular defect in WASp affects protein stability.XL Therapeutics was established in the FutuRx incubator in May 2016.
