IL278964A - Pharmaceutical compositions for the treatment of sphingomyelinic acid deficiency - Google Patents
Pharmaceutical compositions for the treatment of sphingomyelinic acid deficiencyInfo
- Publication number
- IL278964A IL278964A IL278964A IL27896420A IL278964A IL 278964 A IL278964 A IL 278964A IL 278964 A IL278964 A IL 278964A IL 27896420 A IL27896420 A IL 27896420A IL 278964 A IL278964 A IL 278964A
- Authority
- IL
- Israel
- Prior art keywords
- pharmaceutical compositions
- acid sphingomyelinase
- treating acid
- sphingomyelinase deficiency
- deficiency
- Prior art date
Links
- 208000034012 Acid sphingomyelinase deficiency Diseases 0.000 title 1
- 239000008194 pharmaceutical composition Substances 0.000 title 1
Classifications
-
- 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
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/20—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/08—Solutions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
- A61K9/1605—Excipients; Inactive ingredients
- A61K9/1611—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
- A61K9/1605—Excipients; Inactive ingredients
- A61K9/1617—Organic compounds, e.g. phospholipids, fats
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
- A61K9/1605—Excipients; Inactive ingredients
- A61K9/1617—Organic compounds, e.g. phospholipids, fats
- A61K9/1623—Sugars or sugar alcohols, e.g. lactose; Derivatives thereof; Homeopathic globules
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
-
- 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
- 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
- C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Inorganic Chemistry (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862676525P | 2018-05-25 | 2018-05-25 | |
| PCT/US2019/033983 WO2019227029A1 (en) | 2018-05-25 | 2019-05-24 | Pharmaceutical compositions for treating acid sphingomyelinase deficiency |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL278964A true IL278964A (en) | 2021-01-31 |
Family
ID=66912954
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL278964A IL278964A (en) | 2018-05-25 | 2020-11-25 | Pharmaceutical compositions for the treatment of sphingomyelinic acid deficiency |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20210145941A1 (https=) |
| EP (1) | EP3802805A1 (https=) |
| JP (2) | JP7497300B2 (https=) |
| KR (1) | KR102852946B1 (https=) |
| CN (1) | CN112424347A (https=) |
| AU (2) | AU2019275109C1 (https=) |
| BR (1) | BR112020023829A2 (https=) |
| CA (1) | CA3101688A1 (https=) |
| CO (1) | CO2020014399A2 (https=) |
| IL (1) | IL278964A (https=) |
| MX (2) | MX2020012700A (https=) |
| PY (1) | PY1939780A (https=) |
| SG (1) | SG11202011488WA (https=) |
| UY (1) | UY38238A (https=) |
| WO (1) | WO2019227029A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024506860A (ja) * | 2021-02-01 | 2024-02-15 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ニーマンピック病ａ型を治療するための組成物及び方法 |
| TW202403043A (zh) | 2022-03-18 | 2024-01-16 | 美商健臻公司 | 重組人類酸性鞘磷脂酶醫藥組合物及方法 |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| ES2351786T3 (es) * | 2001-11-13 | 2011-02-10 | Genentech, Inc. | Formulaciones con ligando apo2/trail y usos de las mismas. |
| WO2004112826A1 (en) | 2003-06-20 | 2004-12-29 | Ares Trading Sa | Freeze-dried fsh / lh formulations |
| JP2007514661A (ja) * | 2003-11-25 | 2007-06-07 | マウント シナイ スクール オブ メディスン オブ ニューヨーク ユニバーシティー | ニーマンピック病のシャペロンに基づく治療方法 |
| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| HRP20120994T1 (hr) | 2006-02-07 | 2012-12-31 | Shire Human Genetic Therapies, Inc. | Stabilizirana smjesa glukocerebrozidaze |
| MX2008011842A (es) * | 2006-03-13 | 2008-10-02 | Encysive Pharmaceuticals Inc | Procedimientos y composiciones para el tratamiento de insuficiencia cardiaca diastolica. |
| CN102943106A (zh) * | 2006-03-13 | 2013-02-27 | 珀金埃尔默健康科学股份有限公司 | 用于质谱法检测的底物和内标 |
| AU2010263058A1 (en) | 2009-06-18 | 2012-01-12 | Wyeth Llc | Lyophilized formulations for small modular immunopharmaceuticals |
| SI3998078T1 (sl) | 2009-08-28 | 2026-02-27 | Genzyme Corporation | Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze |
| NZ605874A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of heparan n-sulfatase |
| WO2012134240A2 (ko) * | 2011-03-31 | 2012-10-04 | (주)메디톡스 | 보툴리눔 독소의 동결건조제제 |
| JP6480154B2 (ja) | 2014-11-06 | 2019-03-06 | 持田製薬株式会社 | エタネルセプトの凍結乾燥製剤 |
| FI126979B (en) * | 2016-02-29 | 2017-09-15 | Faron Pharmaceuticals Oy | Lyophilized pharmaceutical formulation and its use |
| KR20250025051A (ko) * | 2017-08-24 | 2025-02-20 | 사노피 | 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료 |
-
2019
- 2019-05-23 PY PY201901939780A patent/PY1939780A/es unknown
- 2019-05-23 UY UY0001038238A patent/UY38238A/es not_active Application Discontinuation
- 2019-05-24 CN CN201980048133.7A patent/CN112424347A/zh active Pending
- 2019-05-24 MX MX2020012700A patent/MX2020012700A/es unknown
- 2019-05-24 WO PCT/US2019/033983 patent/WO2019227029A1/en not_active Ceased
- 2019-05-24 SG SG11202011488WA patent/SG11202011488WA/en unknown
- 2019-05-24 EP EP19731419.8A patent/EP3802805A1/en active Pending
- 2019-05-24 AU AU2019275109A patent/AU2019275109C1/en active Active
- 2019-05-24 JP JP2020565415A patent/JP7497300B2/ja active Active
- 2019-05-24 KR KR1020207037328A patent/KR102852946B1/ko active Active
- 2019-05-24 CA CA3101688A patent/CA3101688A1/en active Pending
- 2019-05-24 BR BR112020023829-5A patent/BR112020023829A2/pt unknown
-
2020
- 2020-11-20 CO CONC2020/0014399A patent/CO2020014399A2/es unknown
- 2020-11-25 IL IL278964A patent/IL278964A/en unknown
- 2020-11-25 US US17/104,593 patent/US20210145941A1/en active Pending
- 2020-11-25 MX MX2025002733A patent/MX2025002733A/es unknown
-
2024
- 2024-03-14 JP JP2024039768A patent/JP7832245B2/ja active Active
-
2025
- 2025-07-17 AU AU2025205577A patent/AU2025205577A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3802805A1 (en) | 2021-04-14 |
| UY38238A (es) | 2019-12-31 |
| WO2019227029A1 (en) | 2019-11-28 |
| PY1939780A (es) | 2019-12-04 |
| JP2021525083A (ja) | 2021-09-24 |
| AU2025205577A1 (en) | 2025-08-07 |
| US20210145941A1 (en) | 2021-05-20 |
| MX2025002733A (es) | 2025-04-02 |
| JP7497300B2 (ja) | 2024-06-10 |
| JP7832245B2 (ja) | 2026-03-17 |
| CO2020014399A2 (es) | 2020-12-10 |
| CA3101688A1 (en) | 2019-11-28 |
| AU2019275109C1 (en) | 2025-11-13 |
| CN112424347A (zh) | 2021-02-26 |
| MX2020012700A (es) | 2021-04-28 |
| BR112020023829A2 (pt) | 2021-05-11 |
| JP2024069459A (ja) | 2024-05-21 |
| AU2019275109B2 (en) | 2025-04-17 |
| AU2019275109A1 (en) | 2021-01-21 |
| SG11202011488WA (en) | 2020-12-30 |
| KR20210015903A (ko) | 2021-02-10 |
| KR102852946B1 (ko) | 2025-09-02 |
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