An oral, liver-restricted LXR inverse agonist for dyslipidemia: preclinical development and phase 1 trial.

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Title: An oral, liver-restricted LXR inverse agonist for dyslipidemia: preclinical development and phase 1 trial.
Authors: Li X; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland., Benegiamo G; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland., Vijayakumar A; OrsoBio, Menlo Park, CA, USA., Sroda N; OrsoBio, Menlo Park, CA, USA., Kimura M; Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Division of Developmental Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA., Huss RS; OrsoBio, Menlo Park, CA, USA., Weng S; OrsoBio, Menlo Park, CA, USA., Murakami E; OrsoBio, Menlo Park, CA, USA., Kirby BJ; OrsoBio, Menlo Park, CA, USA., von Alvensleben GVG; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland., Kremoser C; WM Therapeutics, Heidelberg, Germany., Gane EJ; New Zealand Clinical Research, University of Auckland, Auckland, New Zealand., Takebe T; Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Division of Developmental Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Premium Research Institute for Human Metaverse Medicine (WPI-PRIMe), and Division of Stem Cell and Organoid Medicine, The University of Osaka, Suita, Japan., Myers RP; OrsoBio, Menlo Park, CA, USA., Subramanian GM; OrsoBio, Menlo Park, CA, USA. mani@orsobio.com., Auwerx J; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland. admin.auwerx@epfl.ch.
Source: Nature medicine [Nat Med] 2026 Mar; Vol. 32 (3), pp. 883-893. Date of Electronic Publication: 2026 Jan 16.
Publication Type: Clinical Trial, Phase I; Journal Article; Randomized Controlled Trial
Journal Info: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE
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  Data: An oral, liver-restricted LXR inverse agonist for dyslipidemia: preclinical development and phase 1 trial.
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  Data: <searchLink fieldCode="AU" term="%22Li+X%22">Li X</searchLink>; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland.<br /><searchLink fieldCode="AU" term="%22Benegiamo+G%22">Benegiamo G</searchLink>; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland.<br /><searchLink fieldCode="AU" term="%22Vijayakumar+A%22">Vijayakumar A</searchLink>; OrsoBio, Menlo Park, CA, USA.<br /><searchLink fieldCode="AU" term="%22Sroda+N%22">Sroda N</searchLink>; OrsoBio, Menlo Park, CA, USA.<br /><searchLink fieldCode="AU" term="%22Kimura+M%22">Kimura M</searchLink>; Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Division of Developmental Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.<br /><searchLink fieldCode="AU" term="%22Huss+RS%22">Huss RS</searchLink>; OrsoBio, Menlo Park, CA, USA.<br /><searchLink fieldCode="AU" term="%22Weng+S%22">Weng S</searchLink>; OrsoBio, Menlo Park, CA, USA.<br /><searchLink fieldCode="AU" term="%22Murakami+E%22">Murakami E</searchLink>; OrsoBio, Menlo Park, CA, USA.<br /><searchLink fieldCode="AU" term="%22Kirby+BJ%22">Kirby BJ</searchLink>; OrsoBio, Menlo Park, CA, USA.<br /><searchLink fieldCode="AU" term="%22von+Alvensleben+GVG%22">von Alvensleben GVG</searchLink>; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland.<br /><searchLink fieldCode="AU" term="%22Kremoser+C%22">Kremoser C</searchLink>; WM Therapeutics, Heidelberg, Germany.<br /><searchLink fieldCode="AU" term="%22Gane+EJ%22">Gane EJ</searchLink>; New Zealand Clinical Research, University of Auckland, Auckland, New Zealand.<br /><searchLink fieldCode="AU" term="%22Takebe+T%22">Takebe T</searchLink>; Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Division of Developmental Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.; Premium Research Institute for Human Metaverse Medicine (WPI-PRIMe), and Division of Stem Cell and Organoid Medicine, The University of Osaka, Suita, Japan.<br /><searchLink fieldCode="AU" term="%22Myers+RP%22">Myers RP</searchLink>; OrsoBio, Menlo Park, CA, USA.<br /><searchLink fieldCode="AU" term="%22Subramanian+GM%22">Subramanian GM</searchLink>; OrsoBio, Menlo Park, CA, USA. mani@orsobio.com.<br /><searchLink fieldCode="AU" term="%22Auwerx+J%22">Auwerx J</searchLink>; Laboratory of Integrative Systems Physiology, Institute of Bioengineering, École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland. admin.auwerx@epfl.ch.
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  Data: <searchLink fieldCode="JN" term="%229502015%22">Nature medicine</searchLink> [Nat Med] 2026 Mar; Vol. 32 (3), pp. 883-893. <i>Date of Electronic Publication: </i>2026 Jan 16.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Nature+Publishing+Company%22">Nature Publishing Company </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>9502015 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1546-170X (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2210788956%22">10788956 </searchLink><i>NLM ISO Abbreviation: </i>Nat Med <i>Subsets: </i>MEDLINE
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