ZA202300544B - Compositions and methods for treating late-onset pompe disease - Google Patents
Compositions and methods for treating late-onset pompe diseaseInfo
- Publication number
- ZA202300544B ZA202300544B ZA2023/00544A ZA202300544A ZA202300544B ZA 202300544 B ZA202300544 B ZA 202300544B ZA 2023/00544 A ZA2023/00544 A ZA 2023/00544A ZA 202300544 A ZA202300544 A ZA 202300544A ZA 202300544 B ZA202300544 B ZA 202300544B
- Authority
- ZA
- South Africa
- Prior art keywords
- compositions
- methods
- pompe disease
- onset pompe
- treating late
- Prior art date
Links
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
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
- A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
- A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
-
- 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
- A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
-
- 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
- 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
- A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Organic Chemistry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063038857P | 2020-06-14 | 2020-06-14 | |
| US202063076037P | 2020-09-09 | 2020-09-09 | |
| US202163147629P | 2021-02-09 | 2021-02-09 | |
| US202163157490P | 2021-03-05 | 2021-03-05 | |
| PCT/US2021/037111 WO2021257409A1 (en) | 2020-06-14 | 2021-06-11 | Compositions and methods for treating late-onset pompe disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ZA202300544B true ZA202300544B (en) | 2026-02-25 |
Family
ID=76797137
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ZA2023/00544A ZA202300544B (en) | 2020-06-14 | 2023-01-12 | Compositions and methods for treating late-onset pompe disease |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US12533396B2 (enExample) |
| EP (1) | EP4164678A1 (enExample) |
| JP (2) | JP2023530112A (enExample) |
| KR (1) | KR20230025874A (enExample) |
| CN (1) | CN115916245A (enExample) |
| AU (1) | AU2021293021A1 (enExample) |
| BR (1) | BR112022025314A2 (enExample) |
| CA (1) | CA3182409A1 (enExample) |
| IL (1) | IL299015A (enExample) |
| MX (1) | MX2022016019A (enExample) |
| TW (1) | TW202214302A (enExample) |
| WO (1) | WO2021257409A1 (enExample) |
| ZA (1) | ZA202300544B (enExample) |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7723296B2 (en) | 2001-01-18 | 2010-05-25 | Genzyme Corporation | Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof |
| JP5137814B2 (ja) | 2005-04-06 | 2013-02-06 | ジェンザイム・コーポレーション | 治療ターゲティングのための酸不安定リンカーを介するpegおよびポリシアルリソソーム酵素のコンジュゲート |
| CN102978185B (zh) | 2007-01-18 | 2015-02-11 | 建新公司 | 包含氨基氧基基团的寡糖及其轭合物 |
| EP3608330B1 (en) * | 2008-12-16 | 2022-11-09 | Genzyme Corporation | Synthetic intermediates for preparing oligosaccharide-protein conjugates |
| US8883979B2 (en) | 2012-08-31 | 2014-11-11 | Bayer Healthcare Llc | Anti-prolactin receptor antibody formulations |
| IL299470B2 (en) | 2015-12-30 | 2026-05-01 | Amicus Therapeutics Inc | Improved acid alpha-glucosidase for the treatment of Pompe disease |
| CA3019354A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
| KR102618519B1 (ko) | 2016-03-30 | 2023-12-28 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
| EP4100120A1 (en) | 2020-02-08 | 2022-12-14 | Genzyme Corporation | Compositions and methods for treating pompe disease |
-
2021
- 2021-06-11 BR BR112022025314A patent/BR112022025314A2/pt unknown
- 2021-06-11 CA CA3182409A patent/CA3182409A1/en active Pending
- 2021-06-11 KR KR1020237001571A patent/KR20230025874A/ko active Pending
- 2021-06-11 WO PCT/US2021/037111 patent/WO2021257409A1/en not_active Ceased
- 2021-06-11 IL IL299015A patent/IL299015A/en unknown
- 2021-06-11 EP EP21737902.3A patent/EP4164678A1/en active Pending
- 2021-06-11 CN CN202180041026.9A patent/CN115916245A/zh active Pending
- 2021-06-11 AU AU2021293021A patent/AU2021293021A1/en active Pending
- 2021-06-11 US US18/009,234 patent/US12533396B2/en active Active
- 2021-06-11 JP JP2022576792A patent/JP2023530112A/ja active Pending
- 2021-06-11 MX MX2022016019A patent/MX2022016019A/es unknown
- 2021-06-15 TW TW110121740A patent/TW202214302A/zh unknown
-
2023
- 2023-01-12 ZA ZA2023/00544A patent/ZA202300544B/en unknown
-
2025
- 2025-12-22 US US19/429,911 patent/US20260216302A1/en active Pending
-
2026
- 2026-04-02 JP JP2026060810A patent/JP2026117025A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2021257409A1 (en) | 2021-12-23 |
| US12533396B2 (en) | 2026-01-27 |
| US20230256062A1 (en) | 2023-08-17 |
| EP4164678A1 (en) | 2023-04-19 |
| JP2026117025A (ja) | 2026-07-14 |
| IL299015A (en) | 2023-02-01 |
| KR20230025874A (ko) | 2023-02-23 |
| BR112022025314A2 (pt) | 2023-01-03 |
| AU2021293021A1 (en) | 2023-02-23 |
| MX2022016019A (es) | 2023-04-10 |
| TW202214302A (zh) | 2022-04-16 |
| JP2023530112A (ja) | 2023-07-13 |
| CA3182409A1 (en) | 2021-12-23 |
| CN115916245A (zh) | 2023-04-04 |
| US20260216302A1 (en) | 2026-07-30 |
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