AU2018319565B2 - Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients - Google Patents
Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients Download PDFInfo
- Publication number
- AU2018319565B2 AU2018319565B2 AU2018319565A AU2018319565A AU2018319565B2 AU 2018319565 B2 AU2018319565 B2 AU 2018319565B2 AU 2018319565 A AU2018319565 A AU 2018319565A AU 2018319565 A AU2018319565 A AU 2018319565A AU 2018319565 B2 AU2018319565 B2 AU 2018319565B2
- Authority
- AU
- Australia
- Prior art keywords
- patient
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- ala
- gly
- 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.)
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Classifications
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- 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
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
- A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
-
- 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
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- 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)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Rheumatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Diabetes (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2025203384A AU2025203384A1 (en) | 2017-08-24 | 2025-05-12 | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762549732P | 2017-08-24 | 2017-08-24 | |
| US62/549,732 | 2017-08-24 | ||
| EP17306720 | 2017-12-07 | ||
| EP17306720.8 | 2017-12-07 | ||
| PCT/IB2018/056346 WO2019038685A2 (en) | 2017-08-24 | 2018-08-22 | TREATMENT OF ABNORMAL BONE CONDITIONS IN PATIENTS WITH ACIDIC SPHINGOMYELASE DISEASE |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2025203384A Division AU2025203384A1 (en) | 2017-08-24 | 2025-05-12 | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2018319565A1 AU2018319565A1 (en) | 2020-04-09 |
| AU2018319565B2 true AU2018319565B2 (en) | 2025-02-20 |
Family
ID=63556374
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2018319565A Active AU2018319565B2 (en) | 2017-08-24 | 2018-08-22 | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
| AU2025203384A Pending AU2025203384A1 (en) | 2017-08-24 | 2025-05-12 | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2025203384A Pending AU2025203384A1 (en) | 2017-08-24 | 2025-05-12 | Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US11898175B2 (enExample) |
| EP (1) | EP3672622B1 (enExample) |
| JP (3) | JP7216075B2 (enExample) |
| KR (2) | KR20250025051A (enExample) |
| CN (2) | CN118453843A (enExample) |
| AU (2) | AU2018319565B2 (enExample) |
| BR (1) | BR112020003541A2 (enExample) |
| CA (1) | CA3073648A1 (enExample) |
| CO (1) | CO2020001801A2 (enExample) |
| ES (1) | ES2970423T3 (enExample) |
| IL (1) | IL272757B2 (enExample) |
| MX (2) | MX2020002106A (enExample) |
| NZ (1) | NZ762860A (enExample) |
| PL (1) | PL3672622T3 (enExample) |
| SG (1) | SG11202001544VA (enExample) |
| TW (1) | TWI791040B (enExample) |
| WO (1) | WO2019038685A2 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102094253B1 (ko) * | 2009-08-28 | 2020-03-31 | 이칸 스쿨 오브 메디슨 엣 마운트 시나이 | 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 |
| UY38238A (es) * | 2018-05-25 | 2019-12-31 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
| JP7568648B2 (ja) * | 2019-05-31 | 2024-10-16 | ジェンザイム・コーポレーション | 二次元ｌｃ－ｍｓ／ｍｓシステム |
| CN112773779B (zh) * | 2021-02-04 | 2022-08-16 | 上海交通大学医学院附属新华医院 | 氨溴索、千金藤和汉防已在治疗酸性鞘磷脂酶缺乏症中的应用 |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011025996A2 (en) * | 2009-08-28 | 2011-03-03 | Mount Sinai School Of Medicine Of New York University | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| CN1302897A (zh) | 1999-10-28 | 2001-07-11 | 上海博容基因开发有限公司 | 一种新的多肽——人酸性鞘磷酯酶相似的磷酸二酯酶21和编码这种多肽的多核苷酸 |
| WO2006130769A2 (en) * | 2005-06-02 | 2006-12-07 | Tercica, Inc. | Methods for treatment of growth disorders |
| HK1207005A1 (en) | 2012-03-02 | 2016-01-22 | 夏尔人类遗传性治疗公司 | Compositions and methods for treating type iii gaucher disease |
| WO2014093354A1 (en) | 2012-12-12 | 2014-06-19 | Teva Pharmaceutical Industries Ltd. | Fusion of human growth hormone and albumin, formulation and uses thereof |
| PL3004896T3 (pl) | 2013-06-07 | 2020-04-30 | Genzyme Corporation | Marker zaburzeń kwaśnej spfingomielinazy i jego zastosowania |
-
2018
- 2018-08-22 SG SG11202001544VA patent/SG11202001544VA/en unknown
- 2018-08-22 MX MX2020002106A patent/MX2020002106A/es unknown
- 2018-08-22 KR KR1020257004704A patent/KR20250025051A/ko active Pending
- 2018-08-22 PL PL18768948.4T patent/PL3672622T3/pl unknown
- 2018-08-22 BR BR112020003541-6A patent/BR112020003541A2/pt unknown
- 2018-08-22 CA CA3073648A patent/CA3073648A1/en active Pending
- 2018-08-22 ES ES18768948T patent/ES2970423T3/es active Active
- 2018-08-22 CN CN202410508209.2A patent/CN118453843A/zh active Pending
- 2018-08-22 AU AU2018319565A patent/AU2018319565B2/en active Active
- 2018-08-22 EP EP18768948.4A patent/EP3672622B1/en active Active
- 2018-08-22 TW TW107129257A patent/TWI791040B/zh active
- 2018-08-22 NZ NZ762860A patent/NZ762860A/en unknown
- 2018-08-22 WO PCT/IB2018/056346 patent/WO2019038685A2/en not_active Ceased
- 2018-08-22 JP JP2020511325A patent/JP7216075B2/ja active Active
- 2018-08-22 US US16/641,116 patent/US11898175B2/en active Active
- 2018-08-22 KR KR1020207008213A patent/KR102769578B1/ko active Active
- 2018-08-22 CN CN201880067498.XA patent/CN111344003B/zh active Active
- 2018-08-22 IL IL272757A patent/IL272757B2/en unknown
-
2020
- 2020-02-18 CO CONC2020/0001801A patent/CO2020001801A2/es unknown
- 2020-02-24 MX MX2023010908A patent/MX2023010908A/es unknown
-
2023
- 2023-01-18 JP JP2023005602A patent/JP7431356B2/ja active Active
-
2024
- 2024-01-10 US US18/409,535 patent/US20240218338A1/en active Pending
- 2024-01-31 JP JP2024012534A patent/JP2024050732A/ja active Pending
-
2025
- 2025-05-12 AU AU2025203384A patent/AU2025203384A1/en active Pending
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011025996A2 (en) * | 2009-08-28 | 2011-03-03 | Mount Sinai School Of Medicine Of New York University | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
Non-Patent Citations (1)
| Title |
|---|
| WASSERSTEIN M. P., et al., 2015, "Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency", Molecular Genetics and Metabolism, 116(1), pages 88-97 * |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PC1 | Assignment before grant (sect. 113) |
Owner name: GENZYME CORPORATION Free format text: FORMER APPLICANT(S): SANOFI |
|
| FGA | Letters patent sealed or granted (standard patent) |