SG11201804965SA - Augmented acid alpha-glucosidase for the treatment of pompe disease - Google Patents
Augmented acid alpha-glucosidase for the treatment of pompe diseaseInfo
- Publication number
- SG11201804965SA SG11201804965SA SG11201804965SA SG11201804965SA SG11201804965SA SG 11201804965S A SG11201804965S A SG 11201804965SA SG 11201804965S A SG11201804965S A SG 11201804965SA SG 11201804965S A SG11201804965S A SG 11201804965SA SG 11201804965S A SG11201804965S A SG 11201804965SA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- glucosidase
- december
- new jersey
- cranbury
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
-
- 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
- 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
- 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)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- 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)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Molecular Biology (AREA)
- Physiology (AREA)
- Dermatology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Nutrition Science (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization 111111110111101110101011111010111110111011101011101110111011111111101111011111 International Bureau 0.. .... .. (10) International Publication Number (43) International Publication Date ..... ...r .....1 WO 2017/117407 Al 6 July 2017 (06.07.2017) WI P0 I P CT (51) International Patent Classification: (81) Designated States (unless otherwise indicated, for every A61K 38/47 (2006.01) A61K 31/445 (2006.01) kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (21) International Application Number: BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, PCT/US2016/069243 DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (22) International Filing Date: HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, 29 December 2016 (29.12.2016) KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (25) Filing Language: English NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, (26) Publication Language: English RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, (30) Priority Data: ZA, ZM, ZW. 62/272,890 30 December 2015 (30.12.2015) US 62/300,479 26 February 2016 (26.02.2016) US (84) Designated States (unless otherwise indicated, for every 62/315,412 30 March 2016 (30.03.2016) US kind of regional protection available): ARIPO (BW, GH, 62/402,454 30 September 2016 (30.09.2016) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 62/428,867 1 December 2016 (01.12.2016) US TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 62/431,791 8 December 2016 (08.12.2016) US TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, 15/394,135 29 December 2016 (29.12.2016) US DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, (71) Applicant: AMICUS THERAPEUTICS, INC. [US/US]; SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, 1 Cedar Brook Drive, Cranbury, New Jersey 08512 (US). GW, KM, ML, MR, NE, SN, TD, TG). (72) Inventors: DO, Hung V.; 1 Cedarbrook Drive, Cranbury, Published: New Jersey 08512 (US). KHANNA, Richie; 1 Cedar — with international search report (Art. 21(3)) Brook Drive, Cranbury, New Jersey 08512 (US). GOTSCHALL, Russell; 1 Cedar Brook Drive, Cranbury, — with sequence listing part of description (Rule 5.2(a)) New Jersey 08512 (US). Agent: ALEGRIA, Rory P.; Servilla Whitney LLC, 33 (74) Wood Avenue South, Suite 830, Iselin, New Jersey 08830 (US). 1-1 IN © 7r IN 11 11 IN 1-1 (54) Title: AUGMENTED ACID ALPHA-GLUCOSIDASE FOR THE TREATMENT OF POMPE DISEASE 0 N (57) : A method for treating Pompe disease including administration of recombinant human acid a-glucosidase having op - en. ) timal glycosylation with mannose-6-phosphate residues in combination with an amount of miglustat effective to maximize tissue up- take of recombinant human acid a-glucosidase while minimizing inhibition of the enzymatic activity of the recombinant human acid a-glucosidase is provided.
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562272890P | 2015-12-30 | 2015-12-30 | |
| US201662300479P | 2016-02-26 | 2016-02-26 | |
| US201662315412P | 2016-03-30 | 2016-03-30 | |
| US201662402454P | 2016-09-30 | 2016-09-30 | |
| US201662428867P | 2016-12-01 | 2016-12-01 | |
| US201662431791P | 2016-12-08 | 2016-12-08 | |
| US15/394,135 US20170189497A1 (en) | 2015-12-30 | 2016-12-29 | Augmented Acid Alpha-Glucosidase For The Treatment Of Pompe Disease |
| PCT/US2016/069243 WO2017117407A1 (en) | 2015-12-30 | 2016-12-29 | Augmented acid alpha-glucosidase for the treatment of pompe disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201804965SA true SG11201804965SA (en) | 2018-07-30 |
Family
ID=57822129
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201804965SA SG11201804965SA (en) | 2015-12-30 | 2016-12-29 | Augmented acid alpha-glucosidase for the treatment of pompe disease |
Country Status (15)
| Country | Link |
|---|---|
| US (4) | US20170189497A1 (en) |
| JP (3) | JP2019501178A (en) |
| KR (2) | KR102510941B1 (en) |
| AU (2) | AU2016381832B2 (en) |
| BR (1) | BR112018013151A2 (en) |
| CA (1) | CA3010205A1 (en) |
| CL (1) | CL2018001773A1 (en) |
| CO (1) | CO2018007680A2 (en) |
| EA (1) | EA201891507A1 (en) |
| IL (3) | IL299470A (en) |
| MX (2) | MX2018008185A (en) |
| MY (1) | MY198085A (en) |
| PH (1) | PH12018501313A1 (en) |
| SG (1) | SG11201804965SA (en) |
| WO (1) | WO2017117407A1 (en) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10172924B2 (en) | 2015-03-19 | 2019-01-08 | Translate Bio, Inc. | MRNA therapy for pompe disease |
| KR102618519B1 (en) * | 2016-03-30 | 2023-12-28 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Formulations comprising recombinant acid alpha-glucosidase |
| US11944628B2 (en) | 2017-05-12 | 2024-04-02 | Duke University | Methods for the use of low-dose immune modulators transiently for treating patients undergoing protein replacement therapy |
| JP7193476B2 (en) * | 2017-05-15 | 2022-12-20 | アミカス セラピューティックス インコーポレイテッド | recombinant human acid alpha-glycosidase |
| IL271193B1 (en) | 2017-06-07 | 2024-09-01 | Regeneron Pharma | Compositions and methods for internalizing enzymes |
| WO2020163480A1 (en) * | 2019-02-05 | 2020-08-13 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase and uses thereof |
| BR112022004000A2 (en) | 2019-09-06 | 2022-05-31 | Amicus Therapeutics Inc | Method for capturing and purifying biologicals |
| WO2022174037A1 (en) * | 2021-02-11 | 2022-08-18 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase and uses thereof |
| WO2023215865A1 (en) * | 2022-05-05 | 2023-11-09 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
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2016
- 2016-12-29 WO PCT/US2016/069243 patent/WO2017117407A1/en active Application Filing
- 2016-12-29 SG SG11201804965SA patent/SG11201804965SA/en unknown
- 2016-12-29 AU AU2016381832A patent/AU2016381832B2/en active Active
- 2016-12-29 MX MX2018008185A patent/MX2018008185A/en unknown
- 2016-12-29 BR BR112018013151A patent/BR112018013151A2/en active Search and Examination
- 2016-12-29 KR KR1020187021154A patent/KR102510941B1/en active IP Right Grant
- 2016-12-29 US US15/394,135 patent/US20170189497A1/en not_active Abandoned
- 2016-12-29 EA EA201891507A patent/EA201891507A1/en unknown
- 2016-12-29 KR KR1020237008744A patent/KR20230041833A/en active IP Right Grant
- 2016-12-29 JP JP2018533817A patent/JP2019501178A/en not_active Withdrawn
- 2016-12-29 MY MYPI2018001191A patent/MY198085A/en unknown
- 2016-12-29 CA CA3010205A patent/CA3010205A1/en active Pending
- 2016-12-29 IL IL299470A patent/IL299470A/en unknown
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2018
- 2018-04-11 US US15/950,347 patent/US10857212B2/en active Active
- 2018-06-07 IL IL259876A patent/IL259876B/en active IP Right Grant
- 2018-06-20 PH PH12018501313A patent/PH12018501313A1/en unknown
- 2018-06-28 CL CL2018001773A patent/CL2018001773A1/en unknown
- 2018-06-29 MX MX2022010311A patent/MX2022010311A/en unknown
- 2018-07-25 CO CONC2018/0007680A patent/CO2018007680A2/en unknown
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2020
- 2020-10-02 US US17/061,691 patent/US11278601B2/en active Active
-
2021
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| IL283837B2 (en) | 2023-06-01 |
| CL2018001773A1 (en) | 2018-08-24 |
| IL259876A (en) | 2018-07-31 |
| US20220370571A1 (en) | 2022-11-24 |
| AU2024200071A1 (en) | 2024-02-01 |
| JP2019501178A (en) | 2019-01-17 |
| CO2018007680A2 (en) | 2018-08-10 |
| WO2017117407A1 (en) | 2017-07-06 |
| KR20180099753A (en) | 2018-09-05 |
| PH12018501313A1 (en) | 2019-02-27 |
| KR20230041833A (en) | 2023-03-24 |
| IL299470A (en) | 2023-02-01 |
| CA3010205A1 (en) | 2017-07-06 |
| JP2022058423A (en) | 2022-04-12 |
| MX2018008185A (en) | 2018-08-28 |
| AU2016381832A1 (en) | 2018-06-28 |
| AU2016381832B2 (en) | 2024-01-25 |
| IL283837A (en) | 2021-07-29 |
| KR102510941B1 (en) | 2023-03-20 |
| IL259876B (en) | 2021-06-30 |
| BR112018013151A2 (en) | 2018-12-18 |
| US20210268074A1 (en) | 2021-09-02 |
| MY198085A (en) | 2023-07-31 |
| US10857212B2 (en) | 2020-12-08 |
| US20180228877A1 (en) | 2018-08-16 |
| US20170189497A1 (en) | 2017-07-06 |
| EA201891507A1 (en) | 2018-12-28 |
| JP7358446B2 (en) | 2023-10-10 |
| JP2024009829A (en) | 2024-01-23 |
| US11278601B2 (en) | 2022-03-22 |
| MX2022010311A (en) | 2022-09-19 |
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