EP4288441A4 - Procédés de traitement de l'ataxie spinocérébelleuse de type 3 - Google Patents

Procédés de traitement de l'ataxie spinocérébelleuse de type 3 Download PDF

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Publication number
EP4288441A4
EP4288441A4 EP22750319.0A EP22750319A EP4288441A4 EP 4288441 A4 EP4288441 A4 EP 4288441A4 EP 22750319 A EP22750319 A EP 22750319A EP 4288441 A4 EP4288441 A4 EP 4288441A4
Authority
EP
European Patent Office
Prior art keywords
treatment
methods
spinocerebellar ataxia
ataxia type
type
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
EP22750319.0A
Other languages
German (de)
English (en)
Other versions
EP4288441A1 (fr
Inventor
Gayan Mirihana ARACHCHILAGE
Michael A. Arnold
Scott Barraza
Anuradha Bhattacharyya
Gary Mitchell Karp
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
PTC Therapeutics Inc
Original Assignee
PTC Therapeutics Inc
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 PTC Therapeutics Inc filed Critical PTC Therapeutics Inc
Publication of EP4288441A1 publication Critical patent/EP4288441A1/fr
Publication of EP4288441A4 publication Critical patent/EP4288441A4/fr
Pending legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
EP22750319.0A 2021-02-05 2022-02-02 Procédés de traitement de l'ataxie spinocérébelleuse de type 3 Pending EP4288441A4 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163146139P 2021-02-05 2021-02-05
PCT/US2022/014929 WO2022169864A1 (fr) 2021-02-05 2022-02-02 Procédés de traitement de l'ataxie spinocérébelleuse de type 3

Publications (2)

Publication Number Publication Date
EP4288441A1 EP4288441A1 (fr) 2023-12-13
EP4288441A4 true EP4288441A4 (fr) 2024-12-11

Family

ID=82742475

Family Applications (1)

Application Number Title Priority Date Filing Date
EP22750319.0A Pending EP4288441A4 (fr) 2021-02-05 2022-02-02 Procédés de traitement de l'ataxie spinocérébelleuse de type 3

Country Status (2)

Country Link
EP (1) EP4288441A4 (fr)
WO (1) WO2022169864A1 (fr)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12398140B2 (en) 2019-02-13 2025-08-26 Ptc Therapeutics, Inc. Substituted pyrrolo [2,3-d]pyrimidines for treating familial dysautonomia
EA202192170A1 (ru) 2019-02-13 2021-11-15 ПиТиСи ТЕРАПЬЮТИКС, ИНК. ТИЕНО[3,2-b]ПИРИДИН-7-АМИНОВЫЕ СОЕДИНЕНИЯ ДЛЯ ЛЕЧЕНИЯ СЕМЕЙНОЙ ВЕГЕТО-СОСУДИСТОЙ ДИСТОНИИ
EP4651953A1 (fr) * 2023-01-19 2025-11-26 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
EP4651950A1 (fr) * 2023-01-19 2025-11-26 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019893A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019930A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019917A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019931A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019928A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019914A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019945A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019936A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019950A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019918A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage
WO2026019946A1 (fr) * 2024-07-17 2026-01-22 Skyhawk Therapeutics, Inc. Compositions utiles pour moduler l'épissage

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015053624A2 (fr) * 2013-10-07 2015-04-16 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Élimination dirigée par un oligonucléotide antisens de sites de clivage protéolytiques, de la mutation hchwa-d, et d'expansions de répétitions de trinucléotides
WO2017053781A1 (fr) * 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions et méthodes de modulation de l'expression de l'ataxine 3
WO2020245233A1 (fr) * 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Oligonucléotides antisens ciblant l'atxn3

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2928714T3 (es) * 2015-01-16 2022-11-22 Massachusetts Gen Hospital Compuestos para mejorar el empalme del ARNm
EP3528816A4 (fr) * 2016-10-21 2020-04-08 Nimbus Lakshmi, Inc. Inhibiteurs de tyk2 et leurs utilisations
US12398140B2 (en) * 2019-02-13 2025-08-26 Ptc Therapeutics, Inc. Substituted pyrrolo [2,3-d]pyrimidines for treating familial dysautonomia

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015053624A2 (fr) * 2013-10-07 2015-04-16 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Élimination dirigée par un oligonucléotide antisens de sites de clivage protéolytiques, de la mutation hchwa-d, et d'expansions de répétitions de trinucléotides
WO2017053781A1 (fr) * 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions et méthodes de modulation de l'expression de l'ataxine 3
WO2020245233A1 (fr) * 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Oligonucléotides antisens ciblant l'atxn3

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
CRAIG S. MCINTOSH ET AL: "Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing", INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, vol. 20, no. 21, 31 October 2019 (2019-10-31), pages 5434, XP055712430, DOI: 10.3390/ijms20215434 *
EVERS MELVIN M. ET AL: "Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon", vol. 58, 6 May 2013 (2013-05-06), AMSTERDAM, NL, pages 49 - 56, XP093217684, ISSN: 0969-9961, Retrieved from the Internet <URL:https://doi.org/10.1016/j.nbd.2013.04.019> DOI: 10.1016/j.nbd.2013.04.019 *
LODEWIJK J. A. TOONEN ET AL: "Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3", SCIENTIFIC REPORTS, vol. 6, no. 1, 12 October 2016 (2016-10-12), XP055563933, DOI: 10.1038/srep35200 *
See also references of WO2022169864A1 *

Also Published As

Publication number Publication date
WO2022169864A1 (fr) 2022-08-11
EP4288441A1 (fr) 2023-12-13

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