Abstract: The present invention relates to a novel compound and application thereof in the inhibition of HBV gene expression. The structure of the compound comprises an interfering nucleic acid for inhibiting HBV gene expression, transition points, and delivery chains of the interfering nucleic acid. By means of the delivery chains, two or three N-acetylgalactosamines can be introduced to an antisense strand 3? end of such siRNA, and two or one N-acetylgalactosamine can be correspondingly introduced to a sense strand 5? end, the total number of the introduced N-acetylgalactosamines being four. In vitro and in vivo pharmacological experiments prove that such a novel compound can continuously and efficiently inhibit HBV gene expression.
Type:
Grant
Filed:
June 20, 2022
Date of Patent:
December 12, 2023
Assignee:
Kylonova (Xiamen) Biopharma Co., Ltd.
Inventors:
Xueqin Lu, Zhuo Mu, Shengjun Wang, Yanchun Du
Abstract: The present invention relates to a novel compound and application thereof in the inhibition of HBV gene expression. The structure of the compound comprises an interfering nucleic acid for inhibiting HBV gene expression, transition points, and delivery chains of the interfering nucleic acid. By means of the delivery chains, two or three N-acetylgalactosamines can be introduced to an antisense strand 3? end of such siRNA, and two or one N-acetylgalactosamine can be correspondingly introduced to a sense strand 5? end, the total number of the introduced N-acetylgalactosamines being four. In vitro and in vivo pharmacological experiments prove that such a novel compound can continuously and efficiently inhibit HBV gene expression.
Type:
Application
Filed:
June 23, 2020
Publication date:
November 23, 2023
Applicant:
KYLONOVA (XIAMEN) BIOPHARMA CO., LTD.
Inventors:
Xueqin LU, Zhuo MU, Shengjun WANG, Yanchun DU
Abstract: Provided is a drug containing a liver targeting specific ligand and a thyroid hormone receptor agonist in its structure, which is a new drug structure formed by linking the liver targeting specific ligand with the thyroid hormone receptor agonist through a branched chain, a linker and a linking chain. Thyroid hormone receptors (TRs) are divided into two subtypes, TR-? and TR-?, wherein, TR-? is mainly expressed in liver, and TR-? is mainly expressed in heart, nervous system, etc. In certain embodiments, it is envisaged that the provided drug has the action of liver targeting, can specifically bring the thyroid hormone receptor agonist into liver, without entering heart and other issues, and may thereby avoid side effects caused by the action of the thyroid hormone receptor agonist on other issues, and maintain its therapeutic efficacy in the treatment of lipid metabolism disorders and related complications.
Abstract: The present invention belongs to the field of biomedicine, specifically relates to the field of targeting medicine. More specifically, the present invention relates to a liver-targeting compound having thyroid hormone receptor agonist characteristics and a pharmaceutical composition thereof. The compound is a compound represented by formula (1). The compound can be used for treating and/or preventing diseases caused by thyroid hormone dysregulation, and can also effectively reduce lipids in plasma and liver cells.
Abstract: The present invention belongs to the field of biomedicine, specifically relates to the field of targeting medicine. More specifically, the present invention relates to a liver-targeting compound having thyroid hormone receptor agonist characteristics and a pharmaceutical composition thereof. The compound is a compound represented by formula (1). The compound can be used for treating and/or preventing diseases caused by thyroid hormone dysregulation, and can also effectively reduce lipids in plasma and liver cells.
Abstract: Provided is a drug containing a liver targeting specific ligand and a thyroid hormone receptor agonist in its structure, which is a new drug structure formed by linking the liver targeting specific ligand with the thyroid hormone receptor agonist through a branched chain, a linker and a linking chain. Thyroid hormone receptors (TRs) are divided into two subtypes, TR-? and TR-?, wherein, TR-? is mainly expressed in liver, and TR-? is mainly expressed in heart, nervous system, etc. In certain embodiments, it is envisaged that the provided drug has the action of liver targeting, can specifically bring the thyroid hormone receptor agonist into liver, without entering heart and other issues, and may thereby avoid side effects caused by the action of the thyroid hormone receptor agonist on other issues, and maintain its therapeutic efficacy in the treatment of lipid metabolism disorders and related complications.