CN119136803A - 用于治疗硫酸乙酰肝素蓄积疾病的治疗组合物 - Google Patents

用于治疗硫酸乙酰肝素蓄积疾病的治疗组合物 Download PDF

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Publication number
CN119136803A
CN119136803A CN202380034938.2A CN202380034938A CN119136803A CN 119136803 A CN119136803 A CN 119136803A CN 202380034938 A CN202380034938 A CN 202380034938A CN 119136803 A CN119136803 A CN 119136803A
Authority
CN
China
Prior art keywords
disease
iminosugars
mps
accumulation
treatment
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.)
Pending
Application number
CN202380034938.2A
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English (en)
Chinese (zh)
Inventor
路易吉·米歇尔·帕沃纳
安娜丽莎·瓜拉尼亚
瓦莱丽亚·德帕斯夸尔
安娜·埃斯波西托
马西莫·达戈斯提诺
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.)
Sverre Therapy LLC
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CN119136803A publication Critical patent/CN119136803A/zh
Pending legal-status Critical Current

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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
    • A61K31/33—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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D211/40—Oxygen atoms
    • C07D211/44—Oxygen atoms attached in position 4
    • C07D211/46—Oxygen atoms attached in position 4 having a hydrogen atom as the second substituent in position 4

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Oncology (AREA)
  • Hospice & Palliative Care (AREA)
  • Communicable Diseases (AREA)
  • Psychiatry (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202380034938.2A 2022-04-20 2023-04-18 用于治疗硫酸乙酰肝素蓄积疾病的治疗组合物 Pending CN119136803A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
IT102022000007808A IT202200007808A1 (it) 2022-04-20 2022-04-20 Composizioni terapeutiche per malattie causate da accumulo di eparan solfato
IT102022000007808 2022-04-20
PCT/EP2023/059966 WO2023203004A1 (en) 2022-04-20 2023-04-18 Therapeutic compositions with imino sugars for the treatment of diseases with accumulation of heparan sulfate

Publications (1)

Publication Number Publication Date
CN119136803A true CN119136803A (zh) 2024-12-13

Family

ID=82483266

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380034938.2A Pending CN119136803A (zh) 2022-04-20 2023-04-18 用于治疗硫酸乙酰肝素蓄积疾病的治疗组合物

Country Status (9)

Country Link
US (1) US20250255856A1 (enExample)
EP (1) EP4511032A1 (enExample)
JP (1) JP2025513384A (enExample)
CN (1) CN119136803A (enExample)
AU (1) AU2023257557A1 (enExample)
CA (1) CA3249619A1 (enExample)
IT (1) IT202200007808A1 (enExample)
MX (1) MX2024012934A (enExample)
WO (1) WO2023203004A1 (enExample)

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2083205C1 (ru) 1994-01-19 1997-07-10 Товарищество с ограниченной ответственностью предприятие "БИОМ" Способ лечения мукополисахаридоза
RU2196988C2 (ru) 2000-04-19 2003-01-20 Государственный новосибирский областной клинический диагностический центр Способ диагностики мукополисахаридозов
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
AU2002241853A1 (en) 2001-01-12 2002-07-24 Oxford Glycosciences (Uk) Ltd. Mucopolysaccharidosis therapies
JP2003265196A (ja) 2002-03-15 2003-09-24 Seikagaku Kogyo Co Ltd ムコ多糖症のスクリーニング方法
AUPS293002A0 (en) 2002-06-14 2002-07-04 Women's And Children's Hospital Identification of oligosaccharides and their use in the diagnosis and evaluation of mucopolysaccharidoses and other related disorders
KR100762945B1 (ko) 2004-09-08 2007-10-04 진동규 동물 세포주를 이용한 이듀로네이트-설파타제의 발현 방법및 그 발현을 위한 세포주
PL377180A1 (pl) 2005-09-21 2007-04-02 Instytut Farmaceutyczny Zastosowanie izoflawonów i ich pochodnych w leczeniu mukopolisacharydoz
US8105788B2 (en) 2005-12-08 2012-01-31 The University Of British Columbia Mucopolysaccharidosis (MPS) diagnostic methods, systems, kits and assays associated therewith
US20070161074A1 (en) 2005-12-27 2007-07-12 Daiichi Pharmaceutical Co., Ltd. Diagnostic method of mucopolysaccharidoses
KR101497194B1 (ko) * 2009-09-04 2015-02-27 유나이티드 세러퓨틱스 코오포레이션 오르토믹소바이러스 감염의 치료 방법
AU2010366066B2 (en) 2010-12-22 2016-01-14 Fondazione Telethon Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses
WO2012177778A1 (en) 2011-06-20 2012-12-27 Mount Sinai School Of Medicine Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders
HK1221871A1 (zh) * 2013-05-02 2017-06-16 The Chancellor, Masters And Scholars Of The University Of Oxford 使用亚氨基糖进行的糖脂抑制
WO2017201030A1 (en) * 2016-05-16 2017-11-23 Emergent Virology Llc Methods of treating viral infection

Also Published As

Publication number Publication date
AU2023257557A1 (en) 2024-12-05
IT202200007808A1 (it) 2023-10-20
MX2024012934A (es) 2024-12-06
EP4511032A1 (en) 2025-02-26
CA3249619A1 (en) 2023-10-26
US20250255856A1 (en) 2025-08-14
JP2025513384A (ja) 2025-04-24
WO2023203004A1 (en) 2023-10-26

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Applicant before: Luigi Michele. Pavona

Country or region before: Italy

Applicant before: Anna Lisa Guarania

Applicant before: Valeria Depascal

Applicant before: Anna Esposito

Applicant before: Massimo Dagustino

TA01 Transfer of patent application right