WO2013169793A3 - Methods and compositions for tdp-43 proteinopathies - Google Patents
Methods and compositions for tdp-43 proteinopathies Download PDFInfo
- 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
Links
Classifications
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- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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.
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)
| 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)
| 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 |
| US10962551B2 (en) | 2015-06-17 | 2021-03-30 | The Johns Hopkins University | TDP-43 in degenerative disease |
| AR108325A1 (en) * | 2016-04-27 | 2018-08-08 | Samumed Llc | ISOQUINOLIN-3-IL CARBOXAMIDS AND PREPARATION AND USE OF THE SAME |
| AR108326A1 (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 |
| WO2019084496A1 (en) | 2017-10-27 | 2019-05-02 | 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 |
| WO2019089835A1 (en) * | 2017-10-31 | 2019-05-09 | 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 |
| CA3126918A1 (en) * | 2019-01-14 | 2020-07-23 | President And Fellows Of Harvard College | Methods and compositions for restoring stmn2 levels |
Citations (5)
| 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 |
-
2013
- 2013-05-07 WO PCT/US2013/039968 patent/WO2013169793A2/en active Application Filing
Patent Citations (5)
| 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)
| 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)
| 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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