JP6990110B2 - タンパク質症を処置するための方法 - Google Patents

タンパク質症を処置するための方法 Download PDF

Info

Publication number
JP6990110B2
JP6990110B2 JP2017547448A JP2017547448A JP6990110B2 JP 6990110 B2 JP6990110 B2 JP 6990110B2 JP 2017547448 A JP2017547448 A JP 2017547448A JP 2017547448 A JP2017547448 A JP 2017547448A JP 6990110 B2 JP6990110 B2 JP 6990110B2
Authority
JP
Japan
Prior art keywords
compound
quinuclidine
mice
disease
proteinosis
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.)
Active
Application number
JP2017547448A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018507886A (ja
JP2018507886A5 (https=
Inventor
エイチ・セン・チェン
ラムヤ・シハブディン
パブロ・セルジオ・サルディ
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.)
Genzyme Corp
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of JP2018507886A publication Critical patent/JP2018507886A/ja
Publication of JP2018507886A5 publication Critical patent/JP2018507886A5/ja
Priority to JP2021078459A priority Critical patent/JP7374149B2/ja
Application granted granted Critical
Publication of JP6990110B2 publication Critical patent/JP6990110B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • A61K31/435Heterocyclic 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
    • A61K31/439Heterocyclic 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 the ring forming part of a bridged ring system, e.g. quinuclidine
    • 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
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D453/00Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids
    • C07D453/02Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids containing not further condensed quinuclidine ring systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
JP2017547448A 2015-03-10 2016-03-09 タンパク質症を処置するための方法 Active JP6990110B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2021078459A JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562131071P 2015-03-10 2015-03-10
US62/131,071 2015-03-10
PCT/US2016/021512 WO2016145046A1 (en) 2015-03-10 2016-03-09 Methods for treating proteinopathies

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021078459A Division JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

Publications (3)

Publication Number Publication Date
JP2018507886A JP2018507886A (ja) 2018-03-22
JP2018507886A5 JP2018507886A5 (https=) 2019-04-25
JP6990110B2 true JP6990110B2 (ja) 2022-02-10

Family

ID=55587378

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2017547448A Active JP6990110B2 (ja) 2015-03-10 2016-03-09 タンパク質症を処置するための方法
JP2021078459A Active JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2021078459A Active JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

Country Status (17)

Country Link
US (2) US20180036295A1 (https=)
EP (2) EP3267983B1 (https=)
JP (2) JP6990110B2 (https=)
KR (1) KR20170123329A (https=)
CN (1) CN107872976A (https=)
AU (1) AU2016229826A1 (https=)
CA (1) CA2978883A1 (https=)
EA (1) EA201791993A1 (https=)
ES (1) ES2973101T3 (https=)
HK (1) HK1244217A1 (https=)
HU (1) HUE065628T2 (https=)
IL (1) IL254393A0 (https=)
MX (1) MX2017011598A (https=)
PL (1) PL3267983T3 (https=)
PT (1) PT3267983T (https=)
TW (1) TW201642855A (https=)
WO (1) WO2016145046A1 (https=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA37975B2 (fr) 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide
WO2018234864A2 (en) 2017-06-19 2018-12-27 Kainos Medicine Inc. ALPHA-SYNUCLEINE MODULATORS
TWI644673B (zh) * 2017-07-27 2018-12-21 百朗克股份有限公司 頭孢曲松的用途
CN114040762A (zh) * 2019-02-04 2022-02-11 建新公司 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病
AU2020218496B2 (en) * 2019-02-04 2024-12-19 Genzyme Corporation Methods for treating symptoms and disorders associated with lysosomal storage diseases
PL4041733T3 (pl) * 2019-11-15 2024-09-23 Yuhan Corporation Pochodne mające ugrupowanie 2,3-dihydro-1h-indenu lub 2,3-dihydrobenzofuranu lub ich farmaceutycznie dopuszczalne sole oraz kompozycje farmaceutyczne je zawierające
TW202142236A (zh) 2020-02-03 2021-11-16 美商健臻公司 用於治療與溶體儲積症相關的神經症狀之方法
JP7699836B2 (ja) 2020-02-28 2025-06-30 プラクシス プレシジョン メディシンズ, インコーポレイテッド Kcnt1阻害剤、および使用方法
DK4122923T3 (da) * 2020-03-17 2025-12-15 Sumitomo Pharma Co Ltd Oxadiazolderivat
KR20230005168A (ko) * 2020-03-23 2023-01-09 프락시스 프리시젼 메디신즈, 인크. Kcnt1 억제제 및 사용 방법
KR20230043024A (ko) * 2020-07-24 2023-03-30 젠자임 코포레이션 벤글루스타트를 포함하는 제약 조성물
EP4188381A1 (en) * 2020-07-30 2023-06-07 Genzyme Corporation Methods for reducing glycosphingolipid concentration in brain tissue and methods of treatment of neurodegenerative diseases involving the same
US11773088B2 (en) 2020-11-02 2023-10-03 Praxis Precision Medicines, Inc. KCNT1 inhibitors and methods of use
CN120417900A (zh) 2022-12-01 2025-08-01 建新公司 文鲁司他与cyp3a4的强或中度抑制剂的组合
WO2025262570A1 (en) 2024-06-17 2025-12-26 Genzyme Corporation Venglustat as an inhibitor of glucosylceramide synthase in subjects having hepatic impairment

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014043068A1 (en) 2012-09-11 2014-03-20 Genzyme Corporation Glucosylceramide synthase inhibitors

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683A (en) 1846-08-08 waring and richard e
US195A (en) 1837-05-15 Machine fob cutting and dressing stone
BE640616A (https=) 1962-12-19
US3492397A (en) 1967-04-07 1970-01-27 Warner Lambert Pharmaceutical Sustained release dosage in the pellet form and process thereof
US4060598A (en) 1967-06-28 1977-11-29 Boehringer Mannheim G.M.B.H. Tablets coated with aqueous resin dispersions
US3538214A (en) 1969-04-22 1970-11-03 Merck & Co Inc Controlled release medicinal tablets
US4173626A (en) 1978-12-11 1979-11-06 Merck & Co., Inc. Sustained release indomethacin
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
AU2012231275A1 (en) * 2011-03-18 2013-10-17 Genzyme Corporation Glucosylceramide synthase inhibitors
WO2012177997A1 (en) * 2011-06-22 2012-12-27 The General Hospital Corporation Treatment of proteinopathies

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014043068A1 (en) 2012-09-11 2014-03-20 Genzyme Corporation Glucosylceramide synthase inhibitors

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
J Neurochem,2014年,129(5),884-894

Also Published As

Publication number Publication date
PT3267983T (pt) 2024-03-12
EP3267983A1 (en) 2018-01-17
JP2018507886A (ja) 2018-03-22
JP2021119185A (ja) 2021-08-12
JP7374149B2 (ja) 2023-11-06
MX2017011598A (es) 2017-12-20
KR20170123329A (ko) 2017-11-07
PL3267983T3 (pl) 2024-05-13
HK1244217A1 (zh) 2018-08-03
AU2016229826A1 (en) 2017-10-26
CN107872976A (zh) 2018-04-03
TW201642855A (zh) 2016-12-16
EP4349408A2 (en) 2024-04-10
EA201791993A1 (ru) 2017-12-29
WO2016145046A1 (en) 2016-09-15
EP3267983B1 (en) 2023-12-20
HUE065628T2 (hu) 2024-06-28
ES2973101T3 (es) 2024-06-18
CA2978883A1 (en) 2016-09-15
US20180036295A1 (en) 2018-02-08
EP4349408A3 (en) 2024-06-19
IL254393A0 (en) 2017-11-30
US20200197374A1 (en) 2020-06-25

Similar Documents

Publication Publication Date Title
JP6990110B2 (ja) タンパク質症を処置するための方法
US7829597B2 (en) Benzylether and benzylamino beta-secretase inhibitors for the treatment of alzheimer's disease
US8497260B2 (en) 2-arylbenzothiophene derivatives or pharmaceutically acceptable salts thereof, preparation method thereof, and pharmaceutical composition for the diagnosis or treatment of degenerative brain disease containing the same as active ingredient
EP0807111B1 (fr) Composes heterocycliques substitues, procede pour leur preparation et compositions pharmaceutiques les contenant
JPH09509935A (ja) モルホリンタキキニン受容体拮抗薬のプロドラッグ
TW200911254A (en) Oxadiazole derivatives and their use as metabotropic glutamate receptor potentiators-842
CN102369202A (zh) 氮杂喹啉酮衍生物及其应用
US12286420B2 (en) EAAT2 activators and methods of using thereof
JP2022519131A (ja) グルコシルセラミドシンターゼ(gcs)阻害剤を使用した繊毛病の処置
JP2020023512A (ja) S1p3アンタゴニスト
JP2025072556A (ja) リソソーム蓄積性疾患と関連する症状および障害を処置するための方法
CN113195084A (zh) 可用作分子伴侣介导的自噬调节剂的苯并噁唑及相关化合物
JP2015504861A (ja) ニコチン性受容体標的化合物および組成物
KR20230047146A (ko) 뇌 조직 내 글리코스핑고지질 농도를 감소시키는 방법 및 이를 수반하는 신경퇴행성 질환의 치료 방법
EP1395582A1 (fr) Nouveaux derives de piperidinecarboxamide, un procede pour leur preparation et les compositions pharmaceutiques les contenant
HK40108783A (en) Methods for treating proteinopathies
WO2019154196A1 (zh) 用于防治神经退行性疾病的新化合物及其应用
CN103354742A (zh) 作为鞘氨醇1-磷酸(s1p)受体调节剂的新型肟氮杂环丁烷衍生物
JP2025529136A (ja) キラルガンマラクタム誘導体またはその薬学的に許容可能な塩およびその製造方法
JP2026509274A (ja) ソルチリン活性の修飾剤

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190308

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190308

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190315

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200212

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200507

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200710

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200722

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20210105

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210506

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20210506

C11 Written invitation by the commissioner to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C11

Effective date: 20210518

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20210622

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20210629

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210803

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211020

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20211109

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20211203

R150 Certificate of patent or registration of utility model

Ref document number: 6990110

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250