WO2017066796A3 - Modulators of telomere disease - Google Patents

Modulators of telomere disease Download PDF

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Publication number
WO2017066796A3
WO2017066796A3 PCT/US2016/057409 US2016057409W WO2017066796A3 WO 2017066796 A3 WO2017066796 A3 WO 2017066796A3 US 2016057409 W US2016057409 W US 2016057409W WO 2017066796 A3 WO2017066796 A3 WO 2017066796A3
Authority
WO
WIPO (PCT)
Prior art keywords
modulators
telomere disease
telomere
disease
diagnosing
Prior art date
Application number
PCT/US2016/057409
Other languages
French (fr)
Other versions
WO2017066796A2 (en
Inventor
Suneet Agarwal
Diane MOON
Baris BOYRAZ
Matthew SEGAL
Original Assignee
The Children's Medical Center Corporation
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
Priority claimed from PCT/US2016/057229 external-priority patent/WO2017066712A2/en
Application filed by The Children's Medical Center Corporation filed Critical The Children's Medical Center Corporation
Priority to US15/768,424 priority Critical patent/US11220689B2/en
Priority to EP16856437.5A priority patent/EP3364974B1/en
Publication of WO2017066796A2 publication Critical patent/WO2017066796A2/en
Publication of WO2017066796A3 publication Critical patent/WO2017066796A3/en
Priority to US17/533,950 priority patent/US20220220479A1/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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/7036—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin having at least one amino group directly attached to the carbocyclic ring, e.g. streptomycin, gentamycin, amikacin, validamycin, fortimicins
    • 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
    • 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
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The disclosure relates to treating and diagnosing telomere diseases, and methods of screening agents for treating and diagnosing telomere diseases.
PCT/US2016/057409 2015-10-16 2016-10-17 Modulators of telomere disease WO2017066796A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
US15/768,424 US11220689B2 (en) 2015-10-16 2016-10-17 Modulators of telomere disease
EP16856437.5A EP3364974B1 (en) 2015-10-16 2016-10-17 Modulators of telomere disease
US17/533,950 US20220220479A1 (en) 2015-10-16 2021-11-23 Modulators of telomere disease

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201562242970P 2015-10-16 2015-10-16
US62/242,970 2015-10-16
US201662308427P 2016-03-15 2016-03-15
US62/308,427 2016-03-15
USPCT/US2016/057229 2016-10-14
PCT/US2016/057229 WO2017066712A2 (en) 2015-10-16 2016-10-14 Modulators of telomere disease

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2016/057229 Continuation-In-Part WO2017066712A2 (en) 2015-10-16 2016-10-14 Modulators of telomere disease

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US15/768,424 A-371-Of-International US11220689B2 (en) 2015-10-16 2016-10-17 Modulators of telomere disease
US17/533,950 Continuation US20220220479A1 (en) 2015-10-16 2021-11-23 Modulators of telomere disease

Publications (2)

Publication Number Publication Date
WO2017066796A2 WO2017066796A2 (en) 2017-04-20
WO2017066796A3 true WO2017066796A3 (en) 2017-06-22

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2016/057409 WO2017066796A2 (en) 2015-10-16 2016-10-17 Modulators of telomere disease

Country Status (1)

Country Link
WO (1) WO2017066796A2 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102431353B1 (en) * 2017-10-16 2022-08-10 에프. 호프만-라 로슈 아게 NUCLEIC ACID MOLECULE FOR REDUCTION OF PAPD5 AND PAPD7 mRNA FOR TREATING HEPATITIS B INFECTION
EP3700527A4 (en) * 2017-10-25 2021-03-10 Children's Medical Center Corporation Papd5 inhibitors and methods of use thereof
CA3111792A1 (en) * 2018-09-05 2020-03-12 Children's Medical Center Corporation Papd5 inhibitors and methods of use thereof
CN114144231B (en) * 2019-05-15 2024-05-24 得克萨斯系统大学评议会 CRISPR method for treating cancer
EP4320117A2 (en) * 2021-04-04 2024-02-14 Baruch S. Blumberg Institute Novel hepatoselective polyadenylating polymerases inhibitors and their method of use
EP4116434A1 (en) * 2021-07-07 2023-01-11 Universidad Autónoma de Madrid In vitro method for quantifying the cellular content of specific rnas

Citations (9)

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WO2004081179A2 (en) * 2003-03-06 2004-09-23 The Government Of The United States As Represented By The Secretary Department Of Health And Human Services Regulation of rna stability
US20060052324A1 (en) * 2004-08-05 2006-03-09 Artandi Steven E Methods and compositions for cell activation
US20100008917A1 (en) * 2006-08-25 2010-01-14 Nancy Hosken Treatment of aplastic anemia
US20100204217A1 (en) * 2008-09-17 2010-08-12 Kallick Charles A Treatment for leukemia and idiopathic aplastic anemia
WO2011108699A1 (en) * 2010-03-04 2011-09-09 国立大学法人九州大学 Gene related to the action of isoflavones
US20120071349A1 (en) * 2009-04-10 2012-03-22 Haiyan Qi Novel anti-aging agents and methods to identify them
US20130323220A1 (en) * 2012-05-29 2013-12-05 The General Hospital Corporation TAL-Tet1 Fusion Proteins and Methods of Use Thereof
WO2014127290A2 (en) * 2013-02-14 2014-08-21 The Regents Of The University Of Colorado Methods for predicting risk of interstitial pneumonia
US20140304845A1 (en) * 2011-10-31 2014-10-09 Merck Sharp & Dohme Corp. Alzheimer's disease signature markers and methods of use

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DE3023787A1 (en) 1980-06-25 1982-01-21 Studiengesellschaft Kohle mbH, 4330 Mülheim METHOD FOR INCREASING THE INCORPORATION AND EXPRESSION OF GENETIC MATERIAL IN THE CORE OF INTACT CELLS BY LIPOSOME
US4980286A (en) 1985-07-05 1990-12-25 Whitehead Institute For Biomedical Research In vivo introduction and expression of foreign genetic material in epithelial cells
AU6131086A (en) 1985-07-05 1987-01-30 Whitehead Institute For Biomedical Research Epithelial cells expressing foreign genetic material
WO1989002468A1 (en) 1987-09-11 1989-03-23 Whitehead Institute For Biomedical Research Transduced fibroblasts and uses therefor
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US20060052324A1 (en) * 2004-08-05 2006-03-09 Artandi Steven E Methods and compositions for cell activation
US20100008917A1 (en) * 2006-08-25 2010-01-14 Nancy Hosken Treatment of aplastic anemia
US20100204217A1 (en) * 2008-09-17 2010-08-12 Kallick Charles A Treatment for leukemia and idiopathic aplastic anemia
US20120071349A1 (en) * 2009-04-10 2012-03-22 Haiyan Qi Novel anti-aging agents and methods to identify them
WO2011108699A1 (en) * 2010-03-04 2011-09-09 国立大学法人九州大学 Gene related to the action of isoflavones
US20140304845A1 (en) * 2011-10-31 2014-10-09 Merck Sharp & Dohme Corp. Alzheimer's disease signature markers and methods of use
US20130323220A1 (en) * 2012-05-29 2013-12-05 The General Hospital Corporation TAL-Tet1 Fusion Proteins and Methods of Use Thereof
WO2014127290A2 (en) * 2013-02-14 2014-08-21 The Regents Of The University Of Colorado Methods for predicting risk of interstitial pneumonia

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Title
MOON, DI ET AL.: "Poly (A)-Specific Ribonuclease (PARN) Mediates 3 [Prime]-End Maturation Of The Telomerase RNA Component", NATURE GENETICS, vol. 47, no. 12, December 2015 (2015-12-01), pages 1482 - 1488, XP055391255 *
See also references of EP3364974A4 *

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