MX2019014412A - Productos terapeuticos para la enfermedad de almacenamiento de glucogeno de tipo iii. - Google Patents
Productos terapeuticos para la enfermedad de almacenamiento de glucogeno de tipo iii.Info
- Publication number
- MX2019014412A MX2019014412A MX2019014412A MX2019014412A MX2019014412A MX 2019014412 A MX2019014412 A MX 2019014412A MX 2019014412 A MX2019014412 A MX 2019014412A MX 2019014412 A MX2019014412 A MX 2019014412A MX 2019014412 A MX2019014412 A MX 2019014412A
- Authority
- MX
- Mexico
- Prior art keywords
- agl
- alpha
- glucogen
- type iii
- storage disease
- Prior art date
Links
Classifications
-
- 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
- C12N9/10—Transferases (2.)
- C12N9/1048—Glycosyltransferases (2.4)
- C12N9/1051—Hexosyltransferases (2.4.1)
-
- 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
-
- 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
- A61K38/45—Transferases (2)
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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)
- C12N9/2405—Glucanases
- C12N9/2451—Glucanases acting on alpha-1,6-glucosidic bonds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y204/00—Glycosyltransferases (2.4)
- C12Y204/01—Hexosyltransferases (2.4.1)
- C12Y204/01025—4-Alpha-glucanotransferase (2.4.1.25)
-
- 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)
- C12Y302/01033—Amylo-alpha-1,6-glucosidase (3.2.1.33)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Obesity (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762513350P | 2017-05-31 | 2017-05-31 | |
| PCT/US2018/035477 WO2018222926A1 (en) | 2017-05-31 | 2018-05-31 | Therapeutics for glycogen storage disease type iii |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2019014412A true MX2019014412A (es) | 2020-02-10 |
Family
ID=64456114
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2019014412A MX2019014412A (es) | 2017-05-31 | 2018-05-31 | Productos terapeuticos para la enfermedad de almacenamiento de glucogeno de tipo iii. |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US11377643B2 (enExample) |
| EP (1) | EP3630964B1 (enExample) |
| JP (2) | JP7284101B2 (enExample) |
| KR (1) | KR102636537B1 (enExample) |
| CN (1) | CN110719954B (enExample) |
| AR (1) | AR112706A1 (enExample) |
| AU (1) | AU2018278315B2 (enExample) |
| BR (1) | BR112019025224A2 (enExample) |
| CA (1) | CA3063907A1 (enExample) |
| CO (1) | CO2019013332A2 (enExample) |
| ES (1) | ES3048960T3 (enExample) |
| MX (1) | MX2019014412A (enExample) |
| TW (1) | TWI794237B (enExample) |
| WO (1) | WO2018222926A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2791160T (pt) | 2011-12-16 | 2022-07-04 | Modernatx Inc | Composições arnm modificado |
| EP3630964B1 (en) * | 2017-05-31 | 2025-08-13 | Ultragenyx Pharmaceutical Inc. | Therapeutics for glycogen storage disease type iii |
| ES2960692T3 (es) | 2018-12-06 | 2024-03-06 | Arcturus Therapeutics Inc | Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa |
| CN110314407A (zh) * | 2019-08-01 | 2019-10-11 | 山东新希望六和集团有限公司 | 一种脂肪快速提取装置 |
| US20220347298A1 (en) | 2019-10-04 | 2022-11-03 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| IT202000003371A1 (it) * | 2020-02-19 | 2021-08-19 | Enea Agenzia Naz Per Le Nuove Tecnologie Lenergia E Lo Sviluppo Economico Sostenibile | Composto per il trattamento di una glicogenosi |
| US12351834B2 (en) | 2020-03-03 | 2025-07-08 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| US11759515B2 (en) | 2020-03-09 | 2023-09-19 | Arcturus Therapeutics, Inc. | Compositions and methods for inducing immune responses |
| WO2022023284A1 (en) | 2020-07-27 | 2022-02-03 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| AU2021325945A1 (en) | 2020-08-14 | 2023-04-06 | Arcturus Therapeutics, Inc. | Method of lyophilizing lipid nanoparticles |
| CA3214538A1 (en) * | 2021-04-20 | 2022-10-27 | Joel DE BEER | Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof |
| WO2023131254A1 (zh) * | 2022-01-06 | 2023-07-13 | 上海吉量医药工程有限公司 | N1位修饰假尿嘧啶核苷及其在mRNA合成中的应用 |
| CN115960926A (zh) * | 2022-12-30 | 2023-04-14 | 南方科技大学 | 一种mRNA及含有mRNA的药物 |
| WO2025080939A1 (en) | 2023-10-13 | 2025-04-17 | Ultragenyx Pharmaceutical, Inc. | Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations |
| WO2025128853A2 (en) | 2023-12-13 | 2025-06-19 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating conditions associated with ube3a overexpression |
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| CA2457959C (en) | 2001-06-05 | 2014-10-28 | Curevac Gmbh | Pharmaceutical composition containing a stabilised mrna optimised for translation in its coding regions |
| WO2004039412A2 (en) | 2002-10-29 | 2004-05-13 | Engene, Inc. | Compositions for cancer treatment |
| TR201909609T4 (tr) | 2005-08-23 | 2019-07-22 | Univ Pennsylvania | Modifiye edilmiş nükleosidleri içeren rna ve kullanım yöntemleri. |
| JP2009544754A (ja) | 2006-07-28 | 2009-12-17 | アプライド バイオシステムズ， エルエルシー | ジヌクレオチドｍｒｎａキャップアナログ |
| WO2009058911A2 (en) | 2007-10-31 | 2009-05-07 | Applied Biosystems Inc. | Preparation and isolation of 5' capped mrna |
| ES2535419T3 (es) | 2007-12-27 | 2015-05-11 | Protiva Biotherapeutics Inc. | Silenciamiento de expresión de quinasa tipo polo usando ARN interferente |
| US20110117125A1 (en) | 2008-01-02 | 2011-05-19 | Tekmira Pharmaceuticals Corporation | Compositions and methods for the delivery of nucleic acids |
| ES2638448T3 (es) | 2008-04-15 | 2017-10-20 | Protiva Biotherapeutics Inc. | Novedosas formulaciones de lípidos para la administración de ácidos nucleicos |
| SI2318037T1 (sl) * | 2008-07-08 | 2015-07-31 | Duke University | Metoda za zdravljenje bolezni glikogenoze |
| WO2010037408A1 (en) | 2008-09-30 | 2010-04-08 | Curevac Gmbh | Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof |
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| PL4241767T3 (pl) | 2008-11-10 | 2025-03-03 | Arbutus Biopharma Corporation | Nowe lipidy i kompozycje do dostarczania środków terapeutycznych |
| EP2391343B1 (en) | 2009-01-29 | 2017-03-01 | Arbutus Biopharma Corporation | Improved lipid formulation for the delivery of nucleic acids |
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| PL3431076T3 (pl) | 2009-06-10 | 2022-01-31 | Arbutus Biopharma Corporation | Ulepszona formulacja lipidowa |
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| KR20150127582A (ko) | 2013-03-14 | 2015-11-17 | 샤이어 휴먼 지네틱 테라피즈 인크. | 4''-티오 개질된 뉴클레오티드를 갖는 리보핵산 및 관련 방법 |
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| AU2015274348A1 (en) | 2014-06-13 | 2017-01-12 | Valerion Therapeutics, Llc | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
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| WO2017054086A1 (en) | 2015-10-01 | 2017-04-06 | Exerkine Corporation | Treatment of genetic myopathies using bioengineered exosomes |
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| IL308824A (en) | 2016-08-17 | 2024-01-01 | Factor Bioscience Inc | Nucleic acid products and methods of administration thereof |
| JP7289265B2 (ja) | 2016-10-26 | 2023-06-09 | キュアバック エスイー | 脂質ナノ粒子ｍＲＮＡワクチン |
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| EP3630964B1 (en) * | 2017-05-31 | 2025-08-13 | Ultragenyx Pharmaceutical Inc. | Therapeutics for glycogen storage disease type iii |
-
2018
- 2018-05-31 EP EP18810235.4A patent/EP3630964B1/en active Active
- 2018-05-31 US US16/617,431 patent/US11377643B2/en active Active
- 2018-05-31 AR ARP180101448A patent/AR112706A1/es unknown
- 2018-05-31 BR BR112019025224-0A patent/BR112019025224A2/pt unknown
- 2018-05-31 ES ES18810235T patent/ES3048960T3/es active Active
- 2018-05-31 CA CA3063907A patent/CA3063907A1/en active Pending
- 2018-05-31 KR KR1020197036416A patent/KR102636537B1/ko active Active
- 2018-05-31 MX MX2019014412A patent/MX2019014412A/es unknown
- 2018-05-31 AU AU2018278315A patent/AU2018278315B2/en active Active
- 2018-05-31 WO PCT/US2018/035477 patent/WO2018222926A1/en not_active Ceased
- 2018-05-31 CN CN201880036551.XA patent/CN110719954B/zh active Active
- 2018-05-31 JP JP2019565348A patent/JP7284101B2/ja active Active
- 2018-05-31 TW TW107118693A patent/TWI794237B/zh active
-
2019
- 2019-11-27 CO CONC2019/0013332A patent/CO2019013332A2/es unknown
-
2022
- 2022-05-04 US US17/736,798 patent/US20220340886A1/en active Pending
-
2023
- 2023-03-13 JP JP2023038257A patent/JP2023075248A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20200014319A (ko) | 2020-02-10 |
| CN110719954A (zh) | 2020-01-21 |
| EP3630964A1 (en) | 2020-04-08 |
| KR102636537B1 (ko) | 2024-02-15 |
| US11377643B2 (en) | 2022-07-05 |
| US20220340886A1 (en) | 2022-10-27 |
| EP3630964B1 (en) | 2025-08-13 |
| AU2018278315A1 (en) | 2019-12-12 |
| AU2018278315B2 (en) | 2024-01-18 |
| CO2019013332A2 (es) | 2020-02-18 |
| ES3048960T3 (en) | 2025-12-12 |
| WO2018222926A1 (en) | 2018-12-06 |
| EP3630964C0 (en) | 2025-08-13 |
| BR112019025224A2 (pt) | 2020-12-08 |
| CN110719954B (zh) | 2023-12-26 |
| CA3063907A1 (en) | 2018-12-06 |
| TW201903151A (zh) | 2019-01-16 |
| US20200149017A1 (en) | 2020-05-14 |
| AR112706A1 (es) | 2019-12-04 |
| JP2020522244A (ja) | 2020-07-30 |
| JP2023075248A (ja) | 2023-05-30 |
| EP3630964A4 (en) | 2021-03-03 |
| JP7284101B2 (ja) | 2023-05-30 |
| TWI794237B (zh) | 2023-03-01 |
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