KR20220106982A - 레트 증후군의 치료 방법 및 조성물 - Google Patents

레트 증후군의 치료 방법 및 조성물 Download PDF

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KR20220106982A
KR20220106982A KR1020227018117A KR20227018117A KR20220106982A KR 20220106982 A KR20220106982 A KR 20220106982A KR 1020227018117 A KR1020227018117 A KR 1020227018117A KR 20227018117 A KR20227018117 A KR 20227018117A KR 20220106982 A KR20220106982 A KR 20220106982A
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tropinetide
administered
syndrome
rett
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모나 다위시
제임스 엠. 유아킴
로렌스 어윈 글래스
낸시 엘리자베스 존스
션 폴 오스터홀트
오스카 델라 파스쿠아
Original Assignee
아카디아 파마슈티칼스 인코포레이티드
뉴렌 파마슈티컬즈 리미티드
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/04Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/06Tripeptides
    • 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
    • A61K31/40Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • 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
    • A61K31/40Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/401Proline; Derivatives thereof, e.g. captopril
    • 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
    • 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
    • C07KPEPTIDES
    • C07K5/00Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/08Tripeptides

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Psychiatry (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Hospice & Palliative Care (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Detergent Compositions (AREA)
  • Chemical Or Physical Treatment Of Fibers (AREA)
  • Peptides Or Proteins (AREA)
KR1020227018117A 2019-10-28 2020-10-28 레트 증후군의 치료 방법 및 조성물 Pending KR20220106982A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962927008P 2019-10-28 2019-10-28
US62/927,008 2019-10-28
US202063031201P 2020-05-28 2020-05-28
US63/031,201 2020-05-28
PCT/US2020/057627 WO2021086892A1 (en) 2019-10-28 2020-10-28 Methods and compositions for treatment of rett syndrome

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KR20220106982A true KR20220106982A (ko) 2022-08-01

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KR1020227018117A Pending KR20220106982A (ko) 2019-10-28 2020-10-28 레트 증후군의 치료 방법 및 조성물

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US (1) US20220339138A1 (https=)
EP (1) EP4051308A4 (https=)
JP (2) JP2022553888A (https=)
KR (1) KR20220106982A (https=)
CN (1) CN115335071A (https=)
AU (1) AU2020376801A1 (https=)
BR (1) BR112022008095A2 (https=)
CA (1) CA3156680A1 (https=)
CL (1) CL2022001079A1 (https=)
CO (1) CO2022007501A2 (https=)
IL (1) IL292617B2 (https=)
MX (1) MX2022004785A (https=)
TW (1) TW202116300A (https=)
WO (1) WO2021086892A1 (https=)

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IL310045A (en) 2021-07-12 2024-03-01 Acadia Pharm Inc Crystalline forms of tropintide
WO2023242844A1 (en) * 2022-06-15 2023-12-21 Ramot At Tel-Aviv University Ltd. Method and therapeutic agent for treatment of disease or disorder associated with impaired firing rate and/or mitochondrial calcium homeostasis
WO2025262646A1 (en) * 2024-06-22 2025-12-26 Biophore India Pharmaceuticals Pvt. Ltd Dosage forms of trofinetide and its pharmaceutically acceptable salt thereof

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US6709817B1 (en) * 1999-09-07 2004-03-23 Baylor College Of Medicine Method of screening Rett syndrome by detecting a mutation in MECP2
US9708366B2 (en) * 2011-01-27 2017-07-18 Neuren Pharmaceuticals Ltd. Treatment of fragile X syndrome using glycyl-L-2-methylprolyl-L-glutamate
WO2014130691A2 (en) * 2013-02-20 2014-08-28 Theravasc Inc. Pharmaceutical formulations of nitrite and uses thereof
WO2015013397A2 (en) * 2013-07-25 2015-01-29 Neuren Pharmaceuticals Limited Neuroprotective bicyclic compounds and methods for their use in treating autism spectrum disorders and neurodevelopmental disorders
US20150224164A1 (en) * 2013-11-26 2015-08-13 Neuren Pharmaceuticals Limited Treatment of autism spectrum disorders using glycyl-l-2-methylprolyl-l-glumatic acid
GB2554064B (en) * 2016-09-01 2019-11-20 Jit Singh Shailinder An enteral feeding tube

Non-Patent Citations (66)

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CN115335071A (zh) 2022-11-11
TW202116300A (zh) 2021-05-01
CA3156680A1 (en) 2021-05-06
CL2022001079A1 (es) 2023-04-21
EP4051308A4 (en) 2023-08-23
MX2022004785A (es) 2022-05-16
CO2022007501A2 (es) 2022-08-30
US20220339138A1 (en) 2022-10-27
AU2020376801A1 (en) 2022-06-09
IL292617B1 (en) 2025-11-01
IL292617B2 (en) 2026-03-01
EP4051308A1 (en) 2022-09-07
WO2021086892A1 (en) 2021-05-06
BR112022008095A2 (pt) 2022-07-12
IL292617A (en) 2022-07-01
JP2025148461A (ja) 2025-10-07
JP2022553888A (ja) 2022-12-26

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