BR112022007947A2 - Uso de modulador de splicing para tratamento que retarda a progressão da doença de huntington - Google Patents

Uso de modulador de splicing para tratamento que retarda a progressão da doença de huntington

Info

Publication number
BR112022007947A2
BR112022007947A2 BR112022007947A BR112022007947A BR112022007947A2 BR 112022007947 A2 BR112022007947 A2 BR 112022007947A2 BR 112022007947 A BR112022007947 A BR 112022007947A BR 112022007947 A BR112022007947 A BR 112022007947A BR 112022007947 A2 BR112022007947 A2 BR 112022007947A2
Authority
BR
Brazil
Prior art keywords
huntington
progression
delays
disease
treatment
Prior art date
Application number
BR112022007947A
Other languages
English (en)
Inventor
Beibel Martin
Borowsky Beth
Cha Jang-Ho
Faller Thomas
Gomez-Mancilla Baltazar
Gubser Keller Caroline
Laisney Marc
Lin Wen
Renaud Nicole
Sivasankran Rajeev
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 BR112022007947A2 publication Critical patent/BR112022007947A2/pt

Links

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

Abstract

USO DE MODULADOR DE SPLICING PARA TRATAMENTO QUE RETARDA A PROGRESSÃO DA DOENÇA DE HUNTINGTON. A presente invenção se refere ao uso de um modulador de splicing para um tratamento que retarda a progressão da doença de Huntington.
BR112022007947A 2019-11-01 2020-10-30 Uso de modulador de splicing para tratamento que retarda a progressão da doença de huntington BR112022007947A2 (pt)

Applications Claiming Priority (6)

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
PCT/IB2020/060210 WO2021084495A1 (en) 2019-11-01 2020-10-30 The use of a splicing modulator for a treatment slowing progression of huntington's disease

Publications (1)

Publication Number Publication Date
BR112022007947A2 true BR112022007947A2 (pt) 2022-07-12

Family

ID=73198372

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022007947A BR112022007947A2 (pt) 2019-11-01 2020-10-30 Uso de modulador de splicing para tratamento que retarda a progressão da doença de huntington

Country Status (12)

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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI3386511T1 (sl) 2015-12-10 2021-09-30 Ptc Therapeutics, Inc. Postopki za zdravljenje Huntingtonove bolezni
EA201992878A1 (ru) 2017-06-05 2020-05-08 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения болезни хантингтона
BR112019027717A2 (pt) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. métodos para tratar a doença de huntington
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
CA3094703A1 (en) 2018-03-27 2019-10-03 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2020005873A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating huntington's disease
CN116997548A (zh) 2020-05-13 2023-11-03 Chdi基金会股份有限公司 用于治疗亨廷顿病的htt调节剂
US20220162610A1 (en) * 2020-11-12 2022-05-26 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
AU6786594A (en) 1993-05-11 1994-12-12 University Of North Carolina At Chapel Hill, The Antisense oligonucleotides which combat aberrant splicing and methods of using the same
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
EP1416972B1 (en) 2000-11-09 2012-02-08 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
JP2009515523A (ja) 2005-11-10 2009-04-16 サンタリス ファーマ アー／エス Ｔｎｆスーパーファミリー受容体に対するスプライス切替オリゴマーならびに疾病治療における該オリゴマーの使用
WO2007089611A2 (en) 2006-01-26 2007-08-09 Isis Pharmaceuticals Inc. Compositions and their uses directed to huntingtin
EP1984499B1 (en) 2006-01-27 2015-05-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
NZ714887A (en) 2009-09-11 2019-07-26 Ionis Pharmaceuticals Inc Modulation of huntingtin expression
EP3321361B1 (en) 2010-02-08 2019-03-27 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
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
WO2011097644A2 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
WO2012012443A2 (en) 2010-07-19 2012-01-26 Bennett C Frank Modulation of dystrophia myotonica-protein kinase (dmpk) expression
EP3467109A1 (en) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
EP4269584A3 (en) 2011-08-11 2024-03-27 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US9976138B2 (en) 2011-08-29 2018-05-22 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
US20150275208A1 (en) 2012-10-12 2015-10-01 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
WO2014121287A2 (en) 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
KR20210130843A (ko) 2013-07-31 2021-11-01 노파르티스 아게 1,4-이치환된 피리다진 유사체 및 smn-결핍-관련 상태를 치료하기 위한 그의 용도
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
EP3540063A1 (en) 2014-12-24 2019-09-18 uniQure IP B.V. Rnai induced huntingtin gene suppression
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
SI3386511T1 (sl) * 2015-12-10 2021-09-30 Ptc Therapeutics, Inc. Postopki za zdravljenje Huntingtonove bolezni
EP3411080A4 (en) * 2016-02-01 2019-08-14 Arrakis Therapeutics, Inc. COMPOUNDS AND METHODS FOR TREATING RNA-MEDIATED DISEASES
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2018022473A1 (en) 2016-07-25 2018-02-01 Wave Life Sciences Ltd. Phasing
EA201992878A1 (ru) 2017-06-05 2020-05-08 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения болезни хантингтона
AU2018284853A1 (en) 2017-06-14 2019-12-19 Ptc Therapeutics, Inc. Methods for modifying RNA splicing
BR112019027717A2 (pt) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. métodos para tratar a doença de huntington
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
BR122020005073A2 (pt) 2017-08-04 2020-10-13 Skyhawk Therapeutics, Inc. Composto, composição farmacêutica e uso do composto
AU2019218987A1 (en) 2018-02-12 2020-07-23 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
CA3094703A1 (en) 2018-03-27 2019-10-03 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2020005873A1 (en) * 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating huntington's disease
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
AU2019294482B2 (en) 2018-06-27 2022-09-01 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
CN112805009A (zh) * 2018-08-07 2021-05-14 费城儿童医院 基因表达的选择性剪接调控及治疗方法
KR20210135243A (ko) 2019-02-04 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
WO2020163382A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163323A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
EP3920917A4 (en) 2019-02-04 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
CN114126613A (zh) 2019-02-05 2022-03-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3920918A4 (en) 2019-02-05 2022-11-16 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
CN113645970A (zh) 2019-02-05 2021-11-12 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3920920A4 (en) 2019-02-05 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
JP2022521467A (ja) 2019-02-05 2022-04-08 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
JP2022519323A (ja) 2019-02-06 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
EP3921311A4 (en) 2019-02-06 2022-11-09 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE

Also Published As

Publication number Publication date
JP2023500251A (ja) 2023-01-05
AU2020377204A1 (en) 2022-06-02
MX2022005254A (es) 2022-06-29
CL2022001083A1 (es) 2023-02-03
IL292129A (en) 2022-06-01
EP4051280A1 (en) 2022-09-07
CN114650822A (zh) 2022-06-21
TW202131920A (zh) 2021-09-01
WO2021084495A1 (en) 2021-05-06
CA3156848A1 (en) 2021-05-06
KR20220093335A (ko) 2022-07-05

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Legal Events

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B11A Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing
B11Y Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette]