JP2025509722A - 医薬組換えヒト酸性スフィンゴミエリナーゼ組成物及び方法 - Google Patents

医薬組換えヒト酸性スフィンゴミエリナーゼ組成物及び方法 Download PDF

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JP2025509722A
JP2025509722A JP2024555092A JP2024555092A JP2025509722A JP 2025509722 A JP2025509722 A JP 2025509722A JP 2024555092 A JP2024555092 A JP 2024555092A JP 2024555092 A JP2024555092 A JP 2024555092A JP 2025509722 A JP2025509722 A JP 2025509722A
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JP2025509722A5 (https=
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トーマス・エム・ワシィレンコ
ケヴィン・ブラウァー
シャオイン・ジン
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Genzyme Corp
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Genzyme Corp
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    • 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
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/10—Selective adsorption, e.g. chromatography characterised by constructional or operational features
    • B01D15/18—Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to flow patterns
    • B01D15/1864—Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to flow patterns using two or more columns
    • B01D15/1871—Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to flow patterns using two or more columns placed in series
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/10—Selective adsorption, e.g. chromatography characterised by constructional or operational features
    • B01D15/20—Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to the conditioning of the sorbent material
    • B01D15/203—Equilibration or regeneration
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/36—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
    • B01D15/361—Ion-exchange
    • B01D15/362—Cation-exchange
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/38—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups B01D15/265 and B01D15/30 - B01D15/36, e.g. affinity, ligand exchange or chiral chromatography
    • B01D15/3804—Affinity chromatography
    • B01D15/3828—Ligand exchange chromatography, e.g. complexation, chelation or metal interaction chromatography
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Analytical Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Obesity (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2024555092A 2022-03-18 2023-03-17 医薬組換えヒト酸性スフィンゴミエリナーゼ組成物及び方法 Pending JP2025509722A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263321636P 2022-03-18 2022-03-18
US63/321,636 2022-03-18
PCT/US2023/064657 WO2023178325A1 (en) 2022-03-18 2023-03-17 Pharmaceutical recombinant human acid sphingomyelinase compositions and methods

Publications (2)

Publication Number Publication Date
JP2025509722A true JP2025509722A (ja) 2025-04-11
JP2025509722A5 JP2025509722A5 (https=) 2026-03-24

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ID=86051983

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JP2024555092A Pending JP2025509722A (ja) 2022-03-18 2023-03-17 医薬組換えヒト酸性スフィンゴミエリナーゼ組成物及び方法

Country Status (12)

Country Link
US (1) US20230365946A1 (https=)
EP (1) EP4493684A1 (https=)
JP (1) JP2025509722A (https=)
KR (1) KR20240156645A (https=)
CN (1) CN119365593A (https=)
AR (1) AR128825A1 (https=)
AU (1) AU2023235321A1 (https=)
CA (1) CA3254814A1 (https=)
IL (1) IL315655A (https=)
MX (1) MX2024011382A (https=)
TW (1) TW202403043A (https=)
WO (1) WO2023178325A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4594462A1 (en) 2022-09-28 2025-08-06 Genzyme Corporation Systems and devices for producing biological products and methods of using the same

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4215051A (en) 1979-08-29 1980-07-29 Standard Oil Company (Indiana) Formation, purification and recovery of phthalic anhydride
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
US20020137173A1 (en) * 2001-02-05 2002-09-26 Dae-Kyong Kim Membrane-associated sphingomyelinase derived from bovine brain, isolation method therefor and anti-sphingomyelinase monoclonal anitibody
US7527956B2 (en) * 2003-06-12 2009-05-05 Genzyme Corporation Modified human acid sphingomyelinase having increased activity, and methods for making the same
WO2008085988A1 (en) 2007-01-05 2008-07-17 Amgen Inc. Methods of purifying proteins
KR20100103595A (ko) 2008-01-18 2010-09-27 에프. 호프만-라 로슈 아게 비-글라이코실화된 단백질의 정제
RU2012151132A (ru) 2010-05-19 2014-06-27 Ф. Хоффманн-Ля Рош Аг Хроматография с гиброфобным взаимодействием
AR082319A1 (es) * 2010-07-22 2012-11-28 Biomarin Pharm Inc Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos
EP2997036B1 (en) 2013-05-15 2021-03-03 Medimmune Limited Purification of recombinantly produced polypeptides
DK3728287T3 (da) 2017-12-19 2024-06-10 CSL Behring Lengnau AG Proteinoprensning og virusinaktivering med alkylglycosider
PY1939780A (es) 2018-05-25 2019-12-04 Genzyme Corp Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida

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Publication number Publication date
TW202403043A (zh) 2024-01-16
WO2023178325A1 (en) 2023-09-21
IL315655A (en) 2024-11-01
KR20240156645A (ko) 2024-10-30
US20230365946A1 (en) 2023-11-16
AR128825A1 (es) 2024-06-19
CA3254814A1 (en) 2023-09-21
AU2023235321A1 (en) 2024-10-31
MX2024011382A (es) 2024-09-23
CN119365593A (zh) 2025-01-24
EP4493684A1 (en) 2025-01-22

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