BR112019023869A2 - variantes de sulfamidase (sgsh), vetores, composições e métodos e usos para tratar mucopolissacaridose tipo iiia (mps iiia) - Google Patents
variantes de sulfamidase (sgsh), vetores, composições e métodos e usos para tratar mucopolissacaridose tipo iiia (mps iiia) Download PDFInfo
- Publication number
- BR112019023869A2 BR112019023869A2 BR112019023869A BR112019023869A BR112019023869A2 BR 112019023869 A2 BR112019023869 A2 BR 112019023869A2 BR 112019023869 A BR112019023869 A BR 112019023869A BR 112019023869 A BR112019023869 A BR 112019023869A BR 112019023869 A2 BR112019023869 A2 BR 112019023869A2
- Authority
- BR
- Brazil
- Prior art keywords
- sgsh
- iiia
- variants
- sulfamidase
- vectors
- Prior art date
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Classifications
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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)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- A61K48/0083—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 administration regime
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y310/00—Hydrolases acting on sulfur-nitrogen bonds (3.10)
- C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
- C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762505423P | 2017-05-12 | 2017-05-12 | |
| PCT/US2018/032454 WO2018209317A1 (en) | 2017-05-12 | 2018-05-11 | Sulfamidase (sgsh) variants, vectors, compositions and methods and uses for treating mucopolysaccharidosis type iiia (mps iiia) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112019023869A2 true BR112019023869A2 (pt) | 2020-06-09 |
Family
ID=64104969
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112019023869A BR112019023869A2 (pt) | 2017-05-12 | 2018-05-11 | variantes de sulfamidase (sgsh), vetores, composições e métodos e usos para tratar mucopolissacaridose tipo iiia (mps iiia) |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12221635B2 (https=) |
| EP (1) | EP3622060A4 (https=) |
| JP (1) | JP7196099B2 (https=) |
| CN (1) | CN110809626A (https=) |
| AU (1) | AU2018265869C1 (https=) |
| BR (1) | BR112019023869A2 (https=) |
| CA (1) | CA3061925A1 (https=) |
| MX (1) | MX2019013528A (https=) |
| RU (1) | RU2019140862A (https=) |
| WO (1) | WO2018209317A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN108728495A (zh) * | 2018-05-31 | 2018-11-02 | 深圳市免疫基因治疗研究院 | 一种Sanfilippo A综合症慢病毒载体、慢病毒及其制备方法和应用 |
| ES3035673T3 (en) * | 2018-12-20 | 2025-09-08 | Esteve Pharmaceuticals Sa | Recombinant vectors for the long term treatment of mucchopolysacharidosis |
| EP3999647A1 (en) * | 2019-07-18 | 2022-05-25 | Lysogene | Compositions and methods for the treatment of sanfilippo disease and other disorders |
| KR20220116459A (ko) * | 2019-11-22 | 2022-08-23 | 더 칠드런스 호스피탈 오브 필라델피아 | 아데노(adeno)-관련 바이러스 벡터 변이체 |
| CA3174928A1 (en) * | 2020-03-11 | 2021-09-16 | Remotor Therapeutics, Inc. | Methods and materials for disseminating a protein throughout the central nervous system |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| KR20240133693A (ko) * | 2021-11-12 | 2024-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 점액다당류증 iiia의 치료를 위한 유전자 요법 |
| WO2023086939A1 (en) * | 2021-11-12 | 2023-05-19 | Amicus Therapeutics, Inc. | Compositions and methods for treating mucopolysaccharidosis iiia |
| US20250257338A1 (en) * | 2022-04-19 | 2025-08-14 | Jcr Pharmaceuticals Co., Ltd. | Glycoprotein having reduced number of mannose-6-phosphates |
| CN115029360B (zh) * | 2022-05-30 | 2024-08-02 | 上海勉亦生物科技有限公司 | 用于治疗粘多糖贮积症iiia型的转基因表达盒 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5863782A (en) * | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| KR20130010121A (ko) * | 2010-03-23 | 2013-01-25 | 인트렉손 코포레이션 | 치료적 단백질을 조건부로 발현하는 벡터，상기 벡터를 포함하는 숙주 세포 및 이의 용도 |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| WO2012135857A1 (en) * | 2011-03-31 | 2012-10-04 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP3166626B1 (en) | 2014-07-11 | 2021-04-28 | Biostrategies LC | Materials and methods for treating disorders associated with sulfatase enzymes |
-
2018
- 2018-05-11 RU RU2019140862A patent/RU2019140862A/ru unknown
- 2018-05-11 JP JP2019562293A patent/JP7196099B2/ja active Active
- 2018-05-11 CN CN201880043043.4A patent/CN110809626A/zh active Pending
- 2018-05-11 BR BR112019023869A patent/BR112019023869A2/pt unknown
- 2018-05-11 AU AU2018265869A patent/AU2018265869C1/en active Active
- 2018-05-11 MX MX2019013528A patent/MX2019013528A/es unknown
- 2018-05-11 CA CA3061925A patent/CA3061925A1/en active Pending
- 2018-05-11 US US16/611,458 patent/US12221635B2/en active Active
- 2018-05-11 EP EP18798587.4A patent/EP3622060A4/en active Pending
- 2018-05-11 WO PCT/US2018/032454 patent/WO2018209317A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| CA3061925A1 (en) | 2018-11-15 |
| EP3622060A4 (en) | 2021-02-17 |
| JP2020519629A (ja) | 2020-07-02 |
| CN110809626A (zh) | 2020-02-18 |
| AU2018265869C1 (en) | 2025-01-30 |
| US12221635B2 (en) | 2025-02-11 |
| EP3622060A1 (en) | 2020-03-18 |
| RU2019140862A3 (https=) | 2021-08-17 |
| RU2019140862A (ru) | 2021-06-15 |
| WO2018209317A1 (en) | 2018-11-15 |
| AU2018265869A1 (en) | 2019-11-21 |
| AU2018265869B2 (en) | 2024-10-24 |
| MX2019013528A (es) | 2020-07-14 |
| US20210324354A1 (en) | 2021-10-21 |
| JP7196099B2 (ja) | 2022-12-26 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B350 | Update of information on the portal [chapter 15.35 patent gazette] | ||
| B06W | Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette] |