CA2773133A1 - Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency - Google Patents

Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency Download PDF

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Publication number
CA2773133A1
CA2773133A1 CA2773133A CA2773133A CA2773133A1 CA 2773133 A1 CA2773133 A1 CA 2773133A1 CA 2773133 A CA2773133 A CA 2773133A CA 2773133 A CA2773133 A CA 2773133A CA 2773133 A1 CA2773133 A1 CA 2773133A1
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CA
Canada
Prior art keywords
dose
hours
asm
rhasm
administered
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
CA2773133A
Other languages
English (en)
French (fr)
Inventor
Edward H. Schuchman
Robert J. Desnick
Gerald F. Cox
Laura P. Andrews
James M. Murray
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
Icahn School of Medicine at Mount Sinai
Original Assignee
Genzyme Corp
Mount Sinai School of Medicine
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=43625265&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CA2773133(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp, Mount Sinai School of Medicine filed Critical Genzyme Corp
Publication of CA2773133A1 publication Critical patent/CA2773133A1/en
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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pulmonology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Dermatology (AREA)
CA2773133A 2009-08-28 2010-08-28 Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency Pending CA2773133A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US23811309P 2009-08-28 2009-08-28
US61/238,113 2009-08-28
PCT/US2010/047057 WO2011025996A2 (en) 2009-08-28 2010-08-28 Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

Publications (1)

Publication Number Publication Date
CA2773133A1 true CA2773133A1 (en) 2011-03-03

Family

ID=43625265

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2773133A Pending CA2773133A1 (en) 2009-08-28 2010-08-28 Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

Country Status (30)

Country Link
US (9) US8349319B2 (enExample)
EP (4) EP2470199B1 (enExample)
JP (8) JP5955219B2 (enExample)
KR (6) KR102094253B1 (enExample)
CN (3) CN105999238A (enExample)
AU (6) AU2010286550B2 (enExample)
BR (1) BR112012004377A2 (enExample)
CA (1) CA2773133A1 (enExample)
CL (1) CL2012000495A1 (enExample)
CY (1) CY1122128T1 (enExample)
DK (3) DK3482767T3 (enExample)
ES (3) ES3052684T3 (enExample)
FI (2) FI3998078T3 (enExample)
FR (1) FR22C1062I2 (enExample)
HR (3) HRP20251310T1 (enExample)
HU (3) HUE057440T2 (enExample)
IL (4) IL314807A (enExample)
LT (4) LT3998078T (enExample)
LU (1) LUC00288I2 (enExample)
MX (2) MX356873B (enExample)
NL (1) NL301211I2 (enExample)
NO (1) NO2022054I1 (enExample)
PL (3) PL2470199T3 (enExample)
PT (3) PT3482767T (enExample)
RU (2) RU2731616C2 (enExample)
SI (3) SI2470199T1 (enExample)
SM (1) SMT202500432T1 (enExample)
TR (1) TR201911293T4 (enExample)
WO (1) WO2011025996A2 (enExample)
ZA (2) ZA201201435B (enExample)

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Publication number Publication date
CN106047835A (zh) 2016-10-26
NL301211I2 (nl) 2023-02-02
KR20210107153A (ko) 2021-08-31
US20180055916A1 (en) 2018-03-01
ES3052684T3 (en) 2026-01-13
JP7605949B2 (ja) 2024-12-24
PL2470199T3 (pl) 2019-11-29
HUS2200050I1 (hu) 2022-12-28
ZA201201435B (en) 2020-05-27
SI2470199T1 (sl) 2019-11-29
RU2020128994A (ru) 2022-03-02
TR201911293T4 (tr) 2019-08-21
SI3998078T1 (sl) 2026-02-27
EP4681775A2 (en) 2026-01-21
US20110052559A1 (en) 2011-03-03
IL266915B1 (en) 2023-03-01
RU2569744C2 (ru) 2015-11-27
AU2016216625B2 (en) 2018-07-05
LT2470199T (lt) 2019-10-10
JP7395693B2 (ja) 2023-12-11
US20130078231A1 (en) 2013-03-28
CN102596232A (zh) 2012-07-18
ES2902229T3 (es) 2022-03-25
LUC00288I2 (enExample) 2025-05-12
DK3998078T3 (da) 2025-11-17
IL300625B2 (en) 2025-01-01
IL300625B1 (en) 2024-09-01
JP7167281B2 (ja) 2022-11-08
AU2020256322A1 (en) 2020-11-12
US20190151419A1 (en) 2019-05-23
JP6952810B2 (ja) 2021-10-20
AU2025205539A1 (en) 2025-08-07
AU2020256322B2 (en) 2024-02-29
RU2731616C2 (ru) 2020-09-07
LTC3482767I2 (enExample) 2024-03-25
PL3998078T3 (pl) 2026-02-16
JP2024026214A (ja) 2024-02-28
DK3482767T3 (da) 2021-12-20
FI3998078T3 (fi) 2025-12-04
US8658162B2 (en) 2014-02-25
CL2012000495A1 (es) 2014-02-07
KR102893390B1 (ko) 2025-11-28
US8349319B2 (en) 2013-01-08
EP3482767B1 (en) 2021-10-06
IL218324A0 (en) 2012-04-30
BR112012004377A2 (pt) 2017-12-12
SMT202500432T1 (it) 2026-01-12
JP2020090513A (ja) 2020-06-11
HUE046598T2 (hu) 2020-03-30
FR22C1062I1 (fr) 2023-02-03
PT3482767T (pt) 2021-12-29
KR20220104271A (ko) 2022-07-26
JP5955219B2 (ja) 2016-07-20
RU2015145255A (ru) 2019-01-11
AU2010286550A1 (en) 2012-03-15
HRP20211992T1 (hr) 2022-04-01
WO2011025996A3 (en) 2012-03-15
AU2024201085B2 (en) 2025-04-24
NO2022054I1 (no) 2022-12-01
KR20250171458A (ko) 2025-12-08
JP2013502933A (ja) 2013-01-31
US10188705B2 (en) 2019-01-29
AU2018204156B2 (en) 2020-07-16
CN105999238A (zh) 2016-10-12
EP3482767A1 (en) 2019-05-15
FIC20220051I1 (fi) 2022-12-20
KR102608551B1 (ko) 2023-12-04
ZA201601362B (en) 2017-05-31
RU2012111823A (ru) 2013-10-10
PT2470199T (pt) 2019-08-07
AU2010286550B2 (en) 2016-05-19
MX356873B (es) 2018-06-18
NL301211I1 (nl) 2022-12-28
KR102094253B1 (ko) 2020-03-31
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