HUE059891T2 - MRNS splicing javítására alkalmas vegyületek - Google Patents

MRNS splicing javítására alkalmas vegyületek

Info

Publication number
HUE059891T2
HUE059891T2 HUE16737944A HUE16737944A HUE059891T2 HU E059891 T2 HUE059891 T2 HU E059891T2 HU E16737944 A HUE16737944 A HU E16737944A HU E16737944 A HUE16737944 A HU E16737944A HU E059891 T2 HUE059891 T2 HU E059891T2
Authority
HU
Hungary
Prior art keywords
compounds
mrna splicing
improving mrna
improving
splicing
Prior art date
Application number
HUE16737944A
Other languages
English (en)
Inventor
Susan Slaugenhaupt
Graham Johnson
William Paquette
Wei Zhang
Juan MARUGAN
Original Assignee
Massachusetts Gen Hospital
Us Health
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 Massachusetts Gen Hospital, Us Health filed Critical Massachusetts Gen Hospital
Publication of HUE059891T2 publication Critical patent/HUE059891T2/hu

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D473/00Heterocyclic compounds containing purine ring systems
    • C07D473/26Heterocyclic compounds containing purine ring systems with an oxygen, sulphur, or nitrogen atom directly attached in position 2 or 6, but not in both
    • C07D473/32Nitrogen atom
    • C07D473/34Nitrogen atom attached in position 6, e.g. adenine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52Purines, e.g. adenine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D405/00Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/02Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
    • C07D405/12Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D417/00Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
    • C07D417/02Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings
    • C07D417/12Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings linked by a chain containing hetero atoms as chain links
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D473/00Heterocyclic compounds containing purine ring systems
    • C07D473/02Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6
    • C07D473/16Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6 two nitrogen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic 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
    • C07D487/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D495/00Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/02Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D495/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D513/00Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00
    • C07D513/02Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00 in which the condensed system contains two hetero rings
    • C07D513/04Ortho-condensed systems
HUE16737944A 2015-01-16 2016-01-15 MRNS splicing javítására alkalmas vegyületek HUE059891T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562104547P 2015-01-16 2015-01-16
US201562180380P 2015-06-16 2015-06-16

Publications (1)

Publication Number Publication Date
HUE059891T2 true HUE059891T2 (hu) 2023-01-28

Family

ID=56406434

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE16737944A HUE059891T2 (hu) 2015-01-16 2016-01-15 MRNS splicing javítására alkalmas vegyületek

Country Status (16)

Country Link
US (3) US10676475B2 (hu)
EP (2) EP3244891B9 (hu)
AU (2) AU2016206589B2 (hu)
BR (1) BR112017015019A2 (hu)
CA (2) CA2973949C (hu)
CL (2) CL2017001823A1 (hu)
DK (1) DK3244891T3 (hu)
EA (1) EA037663B1 (hu)
ES (1) ES2928714T3 (hu)
HU (1) HUE059891T2 (hu)
IL (2) IL253291B (hu)
MX (2) MX2017009295A (hu)
PL (1) PL3244891T3 (hu)
PT (1) PT3244891T (hu)
TW (2) TW202204345A (hu)
WO (1) WO2016115434A1 (hu)

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JP7129095B2 (ja) * 2017-02-20 2022-09-01 国立大学法人京都大学 スプライシング異常に起因する遺伝性疾病のための医薬組成物及び治療方法
CN111065626A (zh) 2017-06-05 2020-04-24 Ptc医疗公司 用于治疗亨廷顿氏舞蹈病的化合物
EP3645121A4 (en) 2017-06-28 2021-03-17 PTC Therapeutics, Inc. HUNTINGTON'S DISEASE TREATMENT METHODS
US11382918B2 (en) 2017-06-28 2022-07-12 Ptc Therapeutics, Inc. Methods for treating Huntington's Disease
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EA202092896A1 (ru) 2018-06-27 2021-04-13 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Гетероциклические и гетероарильные соединения для лечения болезни гентингтона
AU2019355870B2 (en) * 2018-10-01 2023-11-16 Genzyme Corporation Thieno[3,2-b]pyridine derivatives as UDP glycosyltransferase inhibitors and methods of use
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US20230096028A1 (en) * 2019-03-01 2023-03-30 Revolution Medicines, Inc. Bicyclic heterocyclyl compounds and uses thereof
KR20210146287A (ko) * 2019-03-01 2021-12-03 레볼루션 메디슨즈, 인크. 이환식 헤테로아릴 화합물 및 이의 용도
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JP2023506488A (ja) 2019-12-12 2023-02-16 ピーティーシー セラピューティクス, インコーポレイテッド 家族性自律神経失調症を処置するための化合物
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CN111777612B (zh) * 2020-06-05 2022-04-19 广东达元绿洲食品安全科技股份有限公司 一种6-苄基腺嘌呤半抗原、人工抗原及其在免疫检测中的应用
WO2022169864A1 (en) * 2021-02-05 2022-08-11 Ptc Therapeutics Inc. Methods for treating spinocerebellar ataxia type 3
CN113151296B (zh) * 2021-03-22 2022-09-13 云南中烟工业有限责任公司 一种烟草热激蛋白相关的基因及其应用

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Also Published As

Publication number Publication date
US20180118748A1 (en) 2018-05-03
US11702417B2 (en) 2023-07-18
EP3244891A1 (en) 2017-11-22
EA037663B1 (ru) 2021-04-28
AU2020277224B2 (en) 2022-12-22
EP4115882A1 (en) 2023-01-11
EP3244891B9 (en) 2022-11-16
AU2020277224A1 (en) 2020-12-24
IL253291A0 (en) 2017-09-28
CA2973949A1 (en) 2016-07-21
PL3244891T3 (pl) 2022-12-27
EP3244891B1 (en) 2022-07-20
TW202204345A (zh) 2022-02-01
MX2017009295A (es) 2018-03-07
ES2928714T9 (es) 2022-11-29
AU2016206589A1 (en) 2017-07-27
DK3244891T3 (da) 2022-10-24
EP3244891A4 (en) 2018-12-19
MX2022015737A (es) 2023-01-18
US20200283441A1 (en) 2020-09-10
TW201636341A (zh) 2016-10-16
ES2928714T3 (es) 2022-11-22
IL270945A (en) 2020-01-30
WO2016115434A1 (en) 2016-07-21
BR112017015019A2 (pt) 2018-01-30
CA3199430A1 (en) 2016-07-21
IL253291B (en) 2021-10-31
PT3244891T (pt) 2022-10-20
US10676475B2 (en) 2020-06-09
EA201791624A1 (ru) 2018-06-29
US20230279007A1 (en) 2023-09-07
CL2017001823A1 (es) 2018-04-13
TWI727939B (zh) 2021-05-21
CL2019002167A1 (es) 2019-12-13
CA2973949C (en) 2023-07-11
AU2016206589B2 (en) 2020-09-10

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