WO2013169793A3 - Methods and compositions for tdp-43 proteinopathies - Google Patents

Methods and compositions for tdp-43 proteinopathies Download PDF

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Publication number
WO2013169793A3
WO2013169793A3 PCT/US2013/039968 US2013039968W WO2013169793A3 WO 2013169793 A3 WO2013169793 A3 WO 2013169793A3 US 2013039968 W US2013039968 W US 2013039968W WO 2013169793 A3 WO2013169793 A3 WO 2013169793A3
Authority
WO
WIPO (PCT)
Prior art keywords
inhibitor
tdp
neurons
neural progenitors
methods
Prior art date
Application number
PCT/US2013/039968
Other languages
French (fr)
Other versions
WO2013169793A2 (en
Inventor
Ashkan Javaherian
Original Assignee
Ipierian, 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 Ipierian, Inc. filed Critical Ipierian, Inc.
Publication of WO2013169793A2 publication Critical patent/WO2013169793A2/en
Publication of WO2013169793A3 publication Critical patent/WO2013169793A3/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
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365—Lactones
    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47—Quinolines; Isoquinolines
    • A61K31/473—Quinolines; Isoquinolines ortho- or peri-condensed with carbocyclic ring systems, e.g. acridines, phenanthridines
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • 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
    • A61K31/63—Compounds containing para-N-benzenesulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonyl hydrazide
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7048—Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin, digitoxin or digoxin

Abstract

Provided herein are methods for treating a subject suffering from a TDP-43 proteinopathy, e.g., sporadic ALS or FTD by administering to the subject a composition comprising a therapeutically effective amount of a JNK inhibitor, a MAPK inhibitor, a proteasome inhibitor, a Topoisomerase I inhibitor, a Topoisomerase II inhibitor, a HSP-90 inhibitor, a 5-HT antagonist, a CDK inhibitor, or a transcription inhibitor. The provided methods can also be used to reduce TDP-43 aggregation in human neural progenitors or neurons in neural progenitors or neurons that exhibit TDP-43 aggregates. In addition, methods are provided to identify agents that modulate (decrease or increase) TDP-43 aggregation in human neural progenitors or neurons that exhibit TDP-43 aggregates. Also provided are human induced pluripotent stem cell (hiPSC) lines generated from sporadic ALS patients, where the hiPSC lines may be differentiated into neural progenitors or neurons (e.g., motor progenitors and motor neurons) that exhibit a TDP-43 aggregate. Further provided are isolated populations of cells containing neural progenitors or neurons derived from the aforementioned hiPSC lines.
PCT/US2013/039968 2012-05-09 2013-05-07 Methods and compositions for tdp-43 proteinopathies WO2013169793A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261644937P 2012-05-09 2012-05-09
US61/644,937 2012-05-09

Publications (2)

Publication Number Publication Date
WO2013169793A2 WO2013169793A2 (en) 2013-11-14
WO2013169793A3 true WO2013169793A3 (en) 2014-02-06

Family

ID=49551438

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2013/039968 WO2013169793A2 (en) 2012-05-09 2013-05-07 Methods and compositions for tdp-43 proteinopathies

Country Status (1)

Country Link
WO (1) WO2013169793A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11566003B2 (en) 2017-03-30 2023-01-31 Genentech, Inc. Isoquinolines as inhibitors of HPK1

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015084936A1 (en) * 2013-12-04 2015-06-11 The Scripps Research Institute Novel compounds as jnk kinase inhibitors
WO2016205615A1 (en) * 2015-06-17 2016-12-22 The Johns Hopkins University Tdp-43 in degenerative disease
AR108326A1 (en) 2016-04-27 2018-08-08 Samumed Llc ISOQUINOLIN-3-IL CARBOXAMIDS AND PREPARATION AND USE OF THE SAME
AR108325A1 (en) * 2016-04-27 2018-08-08 Samumed Llc ISOQUINOLIN-3-IL CARBOXAMIDS AND PREPARATION AND USE OF THE SAME
WO2018029336A1 (en) 2016-08-12 2018-02-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for determining whether a subject was administered with an activator of the ppar beta/delta pathway.
WO2019079626A1 (en) * 2017-10-19 2019-04-25 Samumed, Llc 6-(5-membered heteroaryl)isoquinolin-3-yl carboxamides and preparation and use thereof
US10413537B2 (en) 2017-10-27 2019-09-17 Samumed, Llc 6-(5-membered heteroaryl)isoquinolin-3-yl-(5-membered heteroaryl) carboxamides and preparation and use thereof
WO2019084497A1 (en) * 2017-10-27 2019-05-02 Samumed, Llc 6-(6-membered heteroaryl & aryl)isoquinolin-3-yl carboxamides and preparation and use thereof
US10703748B2 (en) * 2017-10-31 2020-07-07 Samumed, Llc Diazanaphthalen-3-yl carboxamides and preparation and use thereof
TW202019398A (en) * 2018-06-28 2020-06-01 張翔毓 Methods for treating or preventing conformation diseases and methods for drug screening
US11612606B2 (en) 2018-10-03 2023-03-28 Genentech, Inc. 8-aminoisoquinoline compounds and uses thereof
WO2020150290A2 (en) * 2019-01-14 2020-07-23 President And Fellows Of Harvard College Methods and compositions for restoring stmn2 levels

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5891855A (en) * 1996-02-12 1999-04-06 The Scripps Research Institute Inhibitors of leaderless protein export
US20060173020A1 (en) * 2004-10-08 2006-08-03 Shenlin Huang 1,2,4-Triazolylaminoaryl (heteroaryl) sulfonamide derivatives
US20070244080A1 (en) * 2004-06-25 2007-10-18 Pharmagenesis, Inc. Method for Treatment of Inflammatory Disorders Using Triptolide Compounds
WO2010111587A1 (en) * 2009-03-26 2010-09-30 Trustees Of The University Of Pennsylvania Modulators of tdp-43 mediated toxicity
US20100331328A1 (en) * 2008-02-19 2010-12-30 Cancer Research Technology Limited Bicyclylaryl-aryl-amine compounds and their use

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5891855A (en) * 1996-02-12 1999-04-06 The Scripps Research Institute Inhibitors of leaderless protein export
US20070244080A1 (en) * 2004-06-25 2007-10-18 Pharmagenesis, Inc. Method for Treatment of Inflammatory Disorders Using Triptolide Compounds
US20060173020A1 (en) * 2004-10-08 2006-08-03 Shenlin Huang 1,2,4-Triazolylaminoaryl (heteroaryl) sulfonamide derivatives
US20100331328A1 (en) * 2008-02-19 2010-12-30 Cancer Research Technology Limited Bicyclylaryl-aryl-amine compounds and their use
WO2010111587A1 (en) * 2009-03-26 2010-09-30 Trustees Of The University Of Pennsylvania Modulators of tdp-43 mediated toxicity

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MEYEROWITZ ET AL.: "C-Jun N-terminal kinase controls TDP-43 accumulation in stress granules induced by oxidative stress", MOLECULAR NEURODEGENERATION, vol. 6, no. 57, 2011 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11566003B2 (en) 2017-03-30 2023-01-31 Genentech, Inc. Isoquinolines as inhibitors of HPK1

Also Published As

Publication number Publication date
WO2013169793A2 (en) 2013-11-14

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