EP4288441A4 - Verfahren zur behandlung von spinocerebellarer ataxie typ 3 - Google Patents

Verfahren zur behandlung von spinocerebellarer ataxie typ 3 Download PDF

Info

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
English (en)
French (fr)
Other versions
EP4288441A1 (de
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/de
Publication of EP4288441A4 publication Critical patent/EP4288441A4/de
Pending legal-status Critical Current

Links

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 Verfahren zur behandlung von spinocerebellarer ataxie typ 3 Pending EP4288441A4 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163146139P 2021-02-05 2021-02-05
PCT/US2022/014929 WO2022169864A1 (en) 2021-02-05 2022-02-02 Methods for treating spinocerebellar ataxia type 3

Publications (2)

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

Family

ID=82742475

Family Applications (1)

Application Number Title Priority Date Filing Date
EP22750319.0A Pending EP4288441A4 (de) 2021-02-05 2022-02-02 Verfahren zur behandlung von spinocerebellarer ataxie typ 3

Country Status (2)

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

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2021009667A (es) 2019-02-13 2021-12-10 Ptc Therapeutics Inc Compuestos de tioeno[3,2-b] piridin-7-amina para tratar la disautonomía familiar.
US12398140B2 (en) 2019-02-13 2025-08-26 Ptc Therapeutics, Inc. Substituted pyrrolo [2,3-d]pyrimidines for treating familial dysautonomia
EP4651951A1 (de) * 2023-01-19 2025-11-26 Skyhawk Therapeutics, Inc. Zusammensetzungen zur modulation des spleissens
EP4651945A1 (de) * 2023-01-19 2025-11-26 Skyhawk Therapeutics, Inc. Zusammensetzungen zur modulation des spleissens

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015053624A2 (en) * 2013-10-07 2015-04-16 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions
WO2017053781A1 (en) * 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
WO2020245233A1 (en) * 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting 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 (de) * 2016-10-21 2020-04-08 Nimbus Lakshmi, Inc. Tyk2-inhibitoren und verwendungen davon
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 (en) * 2013-10-07 2015-04-16 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions
WO2017053781A1 (en) * 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
WO2020245233A1 (en) * 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting 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
EP4288441A1 (de) 2023-12-13
WO2022169864A1 (en) 2022-08-11

Similar Documents

Publication Publication Date Title
EP4288441A4 (de) Verfahren zur behandlung von spinocerebellarer ataxie typ 3
MA55209A (fr) Procédés de traitement de l&#39;amylose al
EP3781214A4 (de) Zusammensetzungen und verfahren zur behandlung von spinaler muskelatrophie
MA52219A (fr) Traitement de l&#39;hidradénite suppurée à l&#39;aide d&#39;inhibiteurs de jak
EP4288054A4 (de) Verfahren zur behandlung von spinocerebellarer ataxie typ 3
EP4030680A4 (de) Anwendungsverarbeitungsverfahren und zugehöriges produkt
EP4136254A4 (de) Ace2-gerichtete zusammensetzungen und verfahren zur behandlung von covid-19
EP3423100A4 (de) Zusammensetzungen zur behandlung von entzündungen und verfahren zur behandlung davon
EP4297871A4 (de) Verfahren und zusammensetzungen zur behandlung von agitation
EP4288059A4 (de) Verbindungen zur behandlung von spinocerebellarer ataxie typ 3
EP3761784A4 (de) Neuartiges verfahren zur aufzucht und kontrollierten freisetzung von biologischen bekämpfungsmitteln
EP3775171A4 (de) Verfahren zur behandlung von minimalem restkrebs
EP3773632A4 (de) Verfahren zur behandlung von egfrviii-exprimierenden glioblastomen
EP3927428A4 (de) Verfahren zur behandlung von atemwegserkrankungen
EP4326277A4 (de) Verfahren zur behandlung von ösophagusstrikturen
EP3856213A4 (de) Verfahren zur behandlung von infektionen unter verwendung von bakterien
EP4054589A4 (de) Verfahren zur behandlung von eosinophiler ösophagitis und reduktion von candidiasis
EP4387627A4 (de) Verfahren und zusammensetzungen zur behandlung von viralen infektionen bei tieren
EP3737365A4 (de) Zusammensetzungen und verfahren zur behandlung von krebs mit glycomimetischen peptiden
EP4274583A4 (de) Verfahren und verbindungen zur behandlung von friedreich-ataxie
MA71255A (fr) Composés pour le traitement de l&#39;ataxie spinocérébelleuse de type 3
EP4208137A4 (de) Verbindungen und verfahren zur behandlung von virusinfektionen
EP4284392A4 (de) Behandlung von astigmatismus
EP3990394A4 (de) Zusammensetzungen und verfahren zur behandlung von abwasser
EP3810156A4 (de) Bewertung und behandlung von autoimmuner ataxie

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20230803

AK Designated contracting states

Kind code of ref document: A1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)
RAP3 Party data changed (applicant data changed or rights of an application transferred)

Owner name: PTC THERAPEUTICS, INC.

REG Reference to a national code

Ref country code: DE

Ref legal event code: R079

Free format text: PREVIOUS MAIN CLASS: C07D0487040000

Ipc: A61K0031519000

A4 Supplementary search report drawn up and despatched

Effective date: 20241113

RIC1 Information provided on ipc code assigned before grant

Ipc: A61P 25/28 20060101ALI20241107BHEP

Ipc: A61P 25/14 20060101ALI20241107BHEP

Ipc: C07D 487/04 20060101ALI20241107BHEP

Ipc: A61K 31/519 20060101AFI20241107BHEP

P01 Opt-out of the competence of the unified patent court (upc) registered

Free format text: CASE NUMBER: UPC_APP_3247_4288441/2025

Effective date: 20250813

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: EXAMINATION IS IN PROGRESS

17Q First examination report despatched

Effective date: 20251103