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WO2009120700A3 - Inhibition of dcps - Google Patents

Inhibition of dcps Download PDF

Info

Publication number
WO2009120700A3
WO2009120700A3 PCT/US2009/038108 US2009038108W WO2009120700A3 WO 2009120700 A3 WO2009120700 A3 WO 2009120700A3 US 2009038108 W US2009038108 W US 2009038108W WO 2009120700 A3 WO2009120700 A3 WO 2009120700A3
Authority
WO
WIPO (PCT)
Prior art keywords
dcps
inhibition
smn
subject
methods
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.)
Ceased
Application number
PCT/US2009/038108
Other languages
French (fr)
Other versions
WO2009120700A2 (en
Inventor
Jill Jarecki
Jasbir Singh
Mark Gurney
Brian A. Pollok
Gregory Michaud
Shin-Wu Liu
Megerditch Kiledjian
Matthew Butchbach
Arthur Burghes
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.)
Rutgers State University of New Jersey
Ohio State University
Families of Spinal Muscular Atrophy
Original Assignee
Rutgers State University of New Jersey
Ohio State University
Families of Spinal Muscular Atrophy
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 Rutgers State University of New Jersey, Ohio State University, Families of Spinal Muscular Atrophy filed Critical Rutgers State University of New Jersey
Publication of WO2009120700A2 publication Critical patent/WO2009120700A2/en
Publication of WO2009120700A3 publication Critical patent/WO2009120700A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/517—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The disclosure relates to methods and compositions (e.g., compounds and pharmaceutical compositions thereof) useful for increasing expression of SMN in a cell (e.g., in vitro or in vivo). As a deficiency in SMN can result in the development of an SMA condition in a subject, the methods and compositions described herein can also be used to, e.g., treat, or prevent, an SMA condition in a subject.
PCT/US2009/038108 2008-03-26 2009-03-24 Inhibition of dcps Ceased WO2009120700A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US7204008P 2008-03-26 2008-03-26
US61/072,040 2008-03-26

Publications (2)

Publication Number Publication Date
WO2009120700A2 WO2009120700A2 (en) 2009-10-01
WO2009120700A3 true WO2009120700A3 (en) 2009-12-30

Family

ID=41114646

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2009/038108 Ceased WO2009120700A2 (en) 2008-03-26 2009-03-24 Inhibition of dcps

Country Status (1)

Country Link
WO (1) WO2009120700A2 (en)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3449926T (en) 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS OF MODULATION OF SMN2 EXCISIONS IN A SUBJECT
WO2011130515A1 (en) * 2010-04-14 2011-10-20 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Arylthiazolyl piperidines and related compounds as modulators of survival motor neuron (smn) protein production
WO2014110291A1 (en) 2013-01-09 2014-07-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
WO2015161170A2 (en) 2014-04-17 2015-10-22 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing in a subject
WO2016040748A1 (en) * 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
US10357543B2 (en) 2015-11-16 2019-07-23 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
CN115279379B (en) 2020-02-28 2025-05-09 Ionis制药公司 Compounds and methods for modulating SMN2

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050288314A1 (en) * 2004-06-08 2005-12-29 Decode Chemistry, Inc. 2,4-diaminoquinazolines for spinal muscular atrophy
US20090042900A1 (en) * 2006-08-01 2009-02-12 Decode Chemistry, Inc. 2,4-diaminoquinazolines for spinal muscular atrophy

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050288314A1 (en) * 2004-06-08 2005-12-29 Decode Chemistry, Inc. 2,4-diaminoquinazolines for spinal muscular atrophy
US20090042900A1 (en) * 2006-08-01 2009-02-12 Decode Chemistry, Inc. 2,4-diaminoquinazolines for spinal muscular atrophy

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
DAVIES ET AL.: "Spinal muscular atrophies-distinctions and therapeutic progress.", DRUGS OF THE FUTURE, vol. 31, no. 4, 2006, pages 365 - 371 *
LIU ET AL.: "The scavenger mRNA decapping enzyme DcpS is a member of the HIT family of pyrophosphatases.", EMBO J., vol. 21, no. 17, 2002, pages 4699 - 4708 *
THURMOND ET AL.: "Synthesis and biological evaluation of novel 2,4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy.", J MED CHEM., vol. 51, no. 3, 14 February 2008 (2008-02-14), pages 449 - 469 *

Also Published As

Publication number Publication date
WO2009120700A2 (en) 2009-10-01

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