WO2022208170A8 - The use of the splicing modulator branaplam for slowing progression of huntington's disease - Google Patents

The use of the splicing modulator branaplam for slowing progression of huntington's disease Download PDF

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Publication number
WO2022208170A8
WO2022208170A8 PCT/IB2022/000176 IB2022000176W WO2022208170A8 WO 2022208170 A8 WO2022208170 A8 WO 2022208170A8 IB 2022000176 W IB2022000176 W IB 2022000176W WO 2022208170 A8 WO2022208170 A8 WO 2022208170A8
Authority
WO
WIPO (PCT)
Prior art keywords
huntington
disease
branaplam
splicing modulator
slowing progression
Prior art date
Application number
PCT/IB2022/000176
Other languages
French (fr)
Other versions
WO2022208170A1 (en
Inventor
Martin BEIBEL
Beth Borowsky
Jang-Ho CHA
Thomas Faller
Baltazar Gomez-Mancilla
Helen GU
Caroline GUBSER KELLER
Steven KOVACS
Marc LAISNEY
Wen Lin
Thomas Peters
Nicole RENAUD
Rajeev Sivasankaran
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 WO2022208170A1 publication Critical patent/WO2022208170A1/en
Publication of WO2022208170A8 publication Critical patent/WO2022208170A8/en

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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
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Abstract

Use of a splicing modulator for a treatment slowing progression of Huntington's disease.
PCT/IB2022/000176 2021-03-29 2022-03-29 The use of the splicing modulator brataplam for slowing progression of huntington's disease WO2022208170A1 (en)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US202163167285P 2021-03-29 2021-03-29
US63/167,285 2021-03-29
US202163233538P 2021-08-16 2021-08-16
US63/233,538 2021-08-16
US202163250109P 2021-09-29 2021-09-29
US63/250,109 2021-09-29
US202163263149P 2021-10-27 2021-10-27
US63/263,149 2021-10-27

Publications (2)

Publication Number Publication Date
WO2022208170A1 WO2022208170A1 (en) 2022-10-06
WO2022208170A8 true WO2022208170A8 (en) 2022-12-29

Family

ID=81749566

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2022/000176 WO2022208170A1 (en) 2021-03-29 2022-03-29 The use of the splicing modulator brataplam for slowing progression of huntington's disease

Country Status (2)

Country Link
TW (1) TW202304446A (en)
WO (1) WO2022208170A1 (en)

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* Cited by examiner, † Cited by third party
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US8957040B2 (en) 2010-02-08 2015-02-17 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
JP6018506B2 (en) 2010-02-08 2016-11-02 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Selective reduction of allelic variants
US20130059902A1 (en) 2010-02-08 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
WO2012012467A2 (en) 2010-07-19 2012-01-26 Isis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
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KR20210107038A (en) * 2018-12-21 2021-08-31 노파르티스 아게 Oral Formulation of Branaflam

Also Published As

Publication number Publication date
TW202304446A (en) 2023-02-01
WO2022208170A1 (en) 2022-10-06

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