CL2022001083A1 - El uso de un modulador de empalme para un tratamiento que retrasa la progresión de la enfermedad de huntington - Google Patents

El uso de un modulador de empalme para un tratamiento que retrasa la progresión de la enfermedad de huntington

Info

Publication number
CL2022001083A1
CL2022001083A1 CL2022001083A CL2022001083A CL2022001083A1 CL 2022001083 A1 CL2022001083 A1 CL 2022001083A1 CL 2022001083 A CL2022001083 A CL 2022001083A CL 2022001083 A CL2022001083 A CL 2022001083A CL 2022001083 A1 CL2022001083 A1 CL 2022001083A1
Authority
CL
Chile
Prior art keywords
huntington
progression
delays
disease
treatment
Prior art date
Application number
CL2022001083A
Other languages
English (en)
Inventor
Thomas Faller
Martin Beibel
Beth Borowsky
Jang-Ho Cha
Baltazar Gomez-Mancilla
Keller Caroline Gubser
Marc Laisney
Wen Lin
Nicole Renaud
Rajeev Vasankran
Original Assignee
Novartis Ag
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Novartis Ag filed Critical Novartis Ag
Publication of CL2022001083A1 publication Critical patent/CL2022001083A1/es

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia

Landscapes

  • Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Supply And Installment Of Electrical Components (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Controlling Rewinding, Feeding, Winding, Or Abnormalities Of Webs (AREA)

Abstract

Uso de un modulador de empalme para un tratamiento que retrasa la progresión de la enfermedad de Huntington.
CL2022001083A 2019-11-01 2022-04-28 El uso de un modulador de empalme para un tratamiento que retrasa la progresión de la enfermedad de huntington CL2022001083A1 (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962929582P 2019-11-01 2019-11-01
US201962930776P 2019-11-05 2019-11-05
US201962949698P 2019-12-18 2019-12-18
US202062963836P 2020-01-21 2020-01-21
US202063027124P 2020-05-19 2020-05-19

Publications (1)

Publication Number Publication Date
CL2022001083A1 true CL2022001083A1 (es) 2023-02-03

Family

ID=73198372

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2022001083A CL2022001083A1 (es) 2019-11-01 2022-04-28 El uso de un modulador de empalme para un tratamiento que retrasa la progresión de la enfermedad de huntington

Country Status (12)

Country Link
EP (1) EP4051280A1 (es)
JP (1) JP2023500251A (es)
KR (1) KR20220093335A (es)
CN (1) CN114650822A (es)
AU (1) AU2020377204A1 (es)
BR (1) BR112022007947A2 (es)
CA (1) CA3156848A1 (es)
CL (1) CL2022001083A1 (es)
IL (1) IL292129A (es)
MX (1) MX2022005254A (es)
TW (1) TW202131920A (es)
WO (1) WO2021084495A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2021001091A (es) 2015-12-10 2022-04-26 Ptc Therapeutics Inc Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington.
US11407753B2 (en) 2017-06-05 2022-08-09 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
US11382918B2 (en) 2017-06-28 2022-07-12 Ptc Therapeutics, Inc. Methods for treating Huntington's Disease
BR112019027719A2 (pt) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. métodos para tratar a doença de huntington
JP7399870B2 (ja) 2018-03-27 2023-12-18 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するための化合物
JP7421507B2 (ja) 2018-06-27 2024-01-24 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するためのヘテロ環式およびヘテロアリール化合物
TW202208358A (zh) 2020-05-13 2022-03-01 美商Ｃｈｄｉ基金會股份有限公司 用於治療亨汀頓舞蹈症之htt調節劑
CA3199442A1 (en) * 2020-11-12 2022-05-19 Ptc Therapeutics Inc. Novel rna transcript

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH08510130A (ja) 1993-05-11 1996-10-29 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
US20050054836A1 (en) 2000-11-09 2005-03-10 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
JP4223027B2 (ja) 2005-06-30 2009-02-12 シャープ株式会社 画像形成装置及び秘匿データ送信方法
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
AU2006315758A1 (en) 2005-11-10 2007-05-24 Ercole Biotech, Inc. Splice switching oligomers for TNF superfamily receptors and their use in treatment of disease
US7951934B2 (en) 2006-01-26 2011-05-31 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
JP5213723B2 (ja) 2006-01-27 2013-06-19 アイシス ファーマシューティカルズ， インコーポレーテッド マイクロｒｎａの調節に使用するためのオリゴマー化合物及び組成物
SI2475675T1 (sl) 2009-09-11 2017-03-31 Ionis Pharmaceuticals, Inc. Modulacija ekspresije huntingtina
WO2011097641A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
EP3628750A1 (en) 2010-02-08 2020-04-01 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
AU2011213562B2 (en) 2010-02-08 2016-07-07 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP2595664B1 (en) 2010-07-19 2018-10-17 Ionis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
US10017764B2 (en) 2011-02-08 2018-07-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
EP2742135B2 (en) 2011-08-11 2020-06-10 Ionis Pharmaceuticals, Inc. Linkage modified gapped oligomeric compounds and uses thereof
EP2751269B1 (en) 2011-08-29 2016-03-23 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
MY174339A (en) 2012-08-13 2020-04-09 Novartis Ag 1,4-disubstituted pyridazine analogs and methods for treating smn-deficiency-related conditions
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
CA2887884A1 (en) 2012-10-12 2014-04-17 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
ES2817050T3 (es) 2013-02-04 2021-04-06 Ionis Pharmaceuticals Inc Compuestos antisentido selectivos y usos de los mismos
AU2014296255B2 (en) 2013-07-31 2017-08-03 Novartis Ag 1,4-disubstituted pyridazine derivatives and their use for treating SMN-deficiency-related conditions
KR102423317B1 (ko) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 키랄 디자인
JP6839094B2 (ja) 2014-12-24 2021-03-03 ユニクア・アイピー・ベーフェー ＲＮＡｉで誘発されるハンチンチン遺伝子抑制
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MX2021001091A (es) 2015-12-10 2022-04-26 Ptc Therapeutics Inc Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington.
AU2017215201B2 (en) * 2016-02-01 2023-10-19 Arrakis Therapeutics, Inc. Compounds and methods of treating RNA-mediated diseases
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
US11407753B2 (en) 2017-06-05 2022-08-09 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
CA3065547A1 (en) 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing
BR112019027719A2 (pt) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. métodos para tratar a doença de huntington
US11382918B2 (en) 2017-06-28 2022-07-12 Ptc Therapeutics, Inc. Methods for treating Huntington's Disease
EP3661509A4 (en) 2017-08-04 2021-01-13 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING THE SPLICE
EP3752612A4 (en) 2018-02-12 2021-11-10 Ionis Pharmaceuticals, Inc. MODIFIED COMPOUNDS AND USES THEREOF
JP7399870B2 (ja) 2018-03-27 2023-12-18 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するための化合物
EP3814360A1 (en) 2018-06-27 2021-05-05 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
CN112805280A (zh) 2018-06-27 2021-05-14 Ptc医疗公司 用于治疗亨廷顿氏病的杂芳基化合物
JP7421507B2 (ja) 2018-06-27 2024-01-24 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するためのヘテロ環式およびヘテロアリール化合物
EP3833357A4 (en) * 2018-08-07 2022-06-08 The Children's Hospital of Philadelphia REGULATION OF GENE EXPRESSION BY ALTERNATIVE SPLICING, AND THERAPEUTIC METHODS
JP2022519294A (ja) 2019-02-04 2022-03-22 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
KR20210135244A (ko) 2019-02-04 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
KR20210135243A (ko) 2019-02-04 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
CN114007612A (zh) 2019-02-05 2022-02-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
KR20210135239A (ko) 2019-02-05 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
EP3920926A4 (en) 2019-02-05 2022-10-19 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
EP3920919A4 (en) 2019-02-05 2023-03-29 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
KR20210135241A (ko) 2019-02-05 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
CN113677344A (zh) 2019-02-06 2021-11-19 斯基霍克疗法公司 用于调节剪接的方法和组合物
JP2022523154A (ja) 2019-02-06 2022-04-21 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物

Also Published As

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KR20220093335A (ko) 2022-07-05
CN114650822A (zh) 2022-06-21
AU2020377204A1 (en) 2022-06-02
WO2021084495A1 (en) 2021-05-06
IL292129A (en) 2022-06-01
JP2023500251A (ja) 2023-01-05
CA3156848A1 (en) 2021-05-06
TW202131920A (zh) 2021-09-01
MX2022005254A (es) 2022-06-29
EP4051280A1 (en) 2022-09-07
BR112022007947A2 (pt) 2022-07-12

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