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Conjugates and methods for treating acra hypertrophy

A compound, selected technology, applied in the direction of biochemical devices and methods, other methods of inserting foreign genetic material, gene therapy, etc.

Pending Publication Date: 2022-06-10
盖纳万科学有限公司
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

However, response rates vary and often require multiple treatments and negative side effects

Method used

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  • Conjugates and methods for treating acra hypertrophy
  • Conjugates and methods for treating acra hypertrophy
  • Conjugates and methods for treating acra hypertrophy

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Experimental program
Comparison scheme
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Embodiment approach

[0162] Another aspect provides compositions comprising the double-stranded siRNA molecules described herein.

[0163] In one embodiment, the composition is a pharmaceutical composition comprising a pharmaceutically acceptable carrier.

[0164] One aspect is a compound of formula I or a salt thereof as described herein.

[0165] In one embodiment of the compound of formula I, R 1 is the targeting ligand;

[0166] L 1 is absent or is a linking group;

[0167] L 2 is absent or is a linking group;

[0168] R 2 is a double-stranded siRNA molecule selected from the double-stranded siRNAs of Table 1 and Table 2;

[0169] Ring A does not exist, and it is a 3-20 membered cycloalkyl group, a 5-20 membered aryl group, a 5-20 membered heteroaryl group or a 3-20 membered heterocycloalkyl group;

[0170] per R A independently selected from the group consisting of: hydrogen, hydroxyl, CN, F, Cl, Br, I, -C 1-2 Alkyl-OR B and C 1-8 Alkyl, the C 1-8 Alkyl is optionally selected fro...

Embodiment 1

[0680] like figure 1 As depicted in , the dose response of 24 GalNAc-conjugated siRNAs (siRNA 1-siRNA24) in PHH was evaluated. Increasing concentrations of each siRNA were incubated with primary human hepatocytes for 48 hours, where delivery was GalNAc dependent. GHR mRNA was analyzed by qPCR.

Embodiment 2

[0682] like figure 2 Depicted in , markers of liver injury were measured after a single dose of a GHR-targeting candidate. Male rats received a single subcutaneous injection of 20 mg / kg or 60 mg / kg of the indicated siRNA. Serum markers of liver injury were analyzed 14 days after dosing.

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Abstract

Provided herein are certain nucleic acids (e.g., double-stranded siRNA molecules), and conjugates comprising a targeting moiety, a double-stranded siRNA, and optionally a linking group. Certain embodiments also provide synthetic methods useful for preparing the conjugates. The conjugates are useful in the treatment of certain diseases, such as acra hypertrophy.

Description

[0001] Cross References to Related Applications [0002] This patent application claims the benefit of priority to U.S. Application Serial No. 62 / 925,659, filed October 24, 2019, which is incorporated herein by reference. Background technique [0003] Acromegaly is a disorder caused by overproduction of growth hormone (GH), which causes abnormal growth of bones, tissues, and organs. Untreated acromegaly results in reduced life expectancy, with the vast majority of the 36-60 million cases dying from cardiovascular disease. There are several treatment options for acromegaly, ranging from drug intervention to surgical removal of the pituitary tumor that triggers the disease. However, response rates vary and often require multiple treatments and negative side effects. Therefore, new therapeutic treatment options are needed. Contents of the invention [0004] Nucleic acid (eg, siRNA) therapy is an approach to treat GH hypersecretion by reducing the growth hormone receptor (GHR...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/87A61K31/7072A61K31/7076A61K31/708A61K31/7088A61K31/7115A61K31/712
CPCA61P5/00C07K9/001A61K47/549C12N15/113C12N15/1136C12N2310/14C12N2310/351A61P5/08C12N2320/30
Inventor 德鲁·康德拉托维茨詹姆斯·海斯理查德·J·霍兰德克莉丝汀·伊索凯文·麦克林托克克里斯多夫·贾斯汀·帕塞特卡马克·伍德艾伦·D·马丁玛格丽特·施瓦兹大卫·克罗史蒂文·泰勒
Owner 盖纳万科学有限公司
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