5-[5-(PIPERIDIN-4-YL)THIENO[3,2-C]PYRAZOL-2-YL]INDAZOLE DERIVATIVES AND RELATED COMPOUNDS AS MODULATORS FOR SPLICING NUCLEIC ACIDS AND FOR THE TREATMENT OF PROLIFERATIVE DISEASES

Title:

5-[5-(PIPERIDIN-4-YL)THIENO[3,2-C]PYRAZOL-2-YL]INDAZOLE DERIVATIVES AND RELATED COMPOUNDS AS MODULATORS FOR SPLICING NUCLEIC ACIDS AND FOR THE TREATMENT OF PROLIFERATIVE DISEASES

Document Type and Number:

WIPO Patent Application WO/2023/133229

Kind Code:

A3

Abstract:

The present disclosure features compounds and related compositions that, inter alia, modulate nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as methods of use thereof.

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Inventors:

REYNOLDS DOMINIC (US)
SEILER MICHAEL (US)
AGRAWAL ANANT (US)
VAILLANCOURT FREDERIC (US)
SMITH PETER (US)
PRAJAPATI SUDEEP (US)
HOPPER ALLEN (US)
VYSKOCIL STEPAN (US)

Application Number:

PCT/US2023/010247

Publication Date:

September 28, 2023

Filing Date:

January 05, 2023

Export Citation:

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Assignee:

REMIX THERAPEUTICS INC (US)

International Classes:

C07D495/04; A61K31/381; A61K31/4155; A61K31/416; A61K31/4162; A61K31/439; A61K31/4439; A61K31/444; A61K31/4745; A61K31/5025; A61P35/00; A61P35/02; A61P35/04; C07D451/04; C07D471/04; C07D471/10; C07D487/04; C07D487/10; C07D513/04; A61K31/351; A61K31/4025; A61K31/407; A61K31/4196; A61K31/423; A61K31/428; A61K31/454; A61K31/4545; A61K31/4747; A61K31/495; A61K31/496

Domestic Patent References:

WO2019223632A1	2019-11-28
WO2020033490A1	2020-02-13
WO2019069844A1	2019-04-11
WO2020033724A1	2020-02-13
WO2020005882A1	2020-01-02
WO2011044181A1	2011-04-14
WO2019199972A1	2019-10-17
WO2022006543A1	2022-01-06
WO2022061273A1	2022-03-24

Foreign References:

CN102127096A

2011-07-20

Other References:

LEFRANC FLORENCE ET AL: "4-Bromo-2-(piperidin-1-yl)thiazol-5-yl-phenyl methanone (12b) inhibits Na+/K+-ATPase and Ras oncogene activity in cancer cells", EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY, ELSEVIER, AMSTERDAM, NL, vol. 63, 16 February 2013 (2013-02-16), pages 213 - 223, XP029233952, ISSN: 0223-5234, DOI: 10.1016/J.EJMECH.2013.01.046
ANGELBELLO ALICIA J ET AL: "Development of pharmacophore models for small molecules targeting RNA: Application to the RNA repeat expansion in myotonic dystrophy type 1", BIOORGANIC & MEDICINAL CHEMISTRY LETTERS, ELSEVIER, AMSTERDAM NL, vol. 26, no. 23, 13 October 2016 (2016-10-13), pages 5792 - 5796, XP029815426, ISSN: 0960-894X, DOI: 10.1016/J.BMCL.2016.10.037

Attorney, Agent or Firm:

LARKIN, Angelyn (US)

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