MX2019013528A - Variantes de sulfamidasa (sgsh) y vectores, composiciones, metodos y usos para tratar la mucopolisacaridosis tipo iiia (mps iiia). - Google Patents
Variantes de sulfamidasa (sgsh) y vectores, composiciones, metodos y usos para tratar la mucopolisacaridosis tipo iiia (mps iiia).Info
- Publication number
- MX2019013528A MX2019013528A MX2019013528A MX2019013528A MX2019013528A MX 2019013528 A MX2019013528 A MX 2019013528A MX 2019013528 A MX2019013528 A MX 2019013528A MX 2019013528 A MX2019013528 A MX 2019013528A MX 2019013528 A MX2019013528 A MX 2019013528A
- Authority
- MX
- Mexico
- Prior art keywords
- sgsh
- variants
- iiia
- sulfamidase
- vectors
- 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
- C12N9/14—Hydrolases (3)
-
- 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
-
- 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
-
- 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)
-
- 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
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)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Neurosurgery (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)
Abstract
La invención se refiere a sulfamidasa (SGSH) y a variantes de SGSH. La SGSH y variantes de SGSH pueden suministrarse por medio de una partícula de virus adenoasociado recombinante (rAAV) al sistema nervioso central (CNS) de un mamífero, para transducir células del CNS que contactan el líquido cefalorraquídeo (CSF). Los mamíferos objetivo, para la administración de SGSH y variantes de SGSH, incluyen mamíferos con una deficiencia o defecto en la expresión o función de SGSH.
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 |
---|---|
MX2019013528A true MX2019013528A (es) | 2020-07-14 |
Family
ID=64104969
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2019013528A MX2019013528A (es) | 2017-05-12 | 2018-05-11 | Variantes de sulfamidasa (sgsh) y vectores, composiciones, metodos y usos para tratar la mucopolisacaridosis tipo iiia (mps iiia). |
Country Status (10)
Country | Link |
---|---|
US (1) | US20210324354A1 (es) |
EP (1) | EP3622060A4 (es) |
JP (1) | JP7196099B2 (es) |
CN (1) | CN110809626A (es) |
AU (1) | AU2018265869A1 (es) |
BR (1) | BR112019023869A2 (es) |
CA (1) | CA3061925A1 (es) |
MX (1) | MX2019013528A (es) |
RU (1) | RU2019140862A (es) |
WO (1) | WO2018209317A1 (es) |
Families Citing this family (7)
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 |
WO2020127678A1 (en) * | 2018-12-20 | 2020-06-25 | Esteve Pharmaceuticals, S.A. | Recombinant vectors for the long term treatment of mucchopolysacharidosis |
AU2020388404A1 (en) * | 2019-11-22 | 2022-06-09 | The Children's Hospital Of Philadelphia | Adeno-associated viral vector variants |
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 |
WO2023086939A1 (en) * | 2021-11-12 | 2023-05-19 | Amicus Therapeutics, Inc. | Compositions and methods for treating mucopolysaccharidosis iiia |
JP2023159049A (ja) * | 2022-04-19 | 2023-10-31 | Jcrファーマ株式会社 | マンノース-6-リン酸の数を減少させた糖蛋白質 |
CN115029360A (zh) * | 2022-05-30 | 2022-09-09 | 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 |
TWI688395B (zh) * | 2010-03-23 | 2020-03-21 | 英翠克頌公司 | 條件性表現治療性蛋白質之載體、包含該載體之宿主細胞及彼等之用途 |
EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
CA2832151C (en) * | 2011-03-31 | 2021-06-15 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
US20170191041A1 (en) * | 2014-07-11 | 2017-07-06 | Biostrategies LC | Materials and methods for treating disorders associated with sulfatase enzymes |
-
2018
- 2018-05-11 AU AU2018265869A patent/AU2018265869A1/en active Pending
- 2018-05-11 JP JP2019562293A patent/JP7196099B2/ja active Active
- 2018-05-11 RU RU2019140862A patent/RU2019140862A/ru unknown
- 2018-05-11 BR BR112019023869A patent/BR112019023869A2/pt unknown
- 2018-05-11 EP EP18798587.4A patent/EP3622060A4/en active Pending
- 2018-05-11 MX MX2019013528A patent/MX2019013528A/es unknown
- 2018-05-11 WO PCT/US2018/032454 patent/WO2018209317A1/en active Application Filing
- 2018-05-11 CA CA3061925A patent/CA3061925A1/en active Pending
- 2018-05-11 US US16/611,458 patent/US20210324354A1/en active Pending
- 2018-05-11 CN CN201880043043.4A patent/CN110809626A/zh active Pending
Also Published As
Publication number | Publication date |
---|---|
EP3622060A1 (en) | 2020-03-18 |
RU2019140862A3 (es) | 2021-08-17 |
JP2020519629A (ja) | 2020-07-02 |
AU2018265869A1 (en) | 2019-11-21 |
WO2018209317A1 (en) | 2018-11-15 |
JP7196099B2 (ja) | 2022-12-26 |
BR112019023869A2 (pt) | 2020-06-09 |
RU2019140862A (ru) | 2021-06-15 |
US20210324354A1 (en) | 2021-10-21 |
EP3622060A4 (en) | 2021-02-17 |
CN110809626A (zh) | 2020-02-18 |
CA3061925A1 (en) | 2018-11-15 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
MX2019013528A (es) | Variantes de sulfamidasa (sgsh) y vectores, composiciones, metodos y usos para tratar la mucopolisacaridosis tipo iiia (mps iiia). | |
PH12017501436A1 (en) | Enhanced delivery of viral particles to the striatum and cortex | |
PH12018500594A1 (en) | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same | |
MX2022012490A (es) | Vectores de aav para la terapia genica de la retina y el snc. | |
AU2018253615A1 (en) | Adeno-associated virus mediated gene transfer to the central nervous system | |
MX2020012816A (es) | Anticuerpos antagonistas dirigidos contra el peptido relacionado con el gen de calcitonina y metodos que usan los mismos. | |
WO2016106404A3 (en) | Methods and compositions for treating malignant tumors associated with kras mutation | |
EP4299079A3 (en) | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid | |
MY190067A (en) | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a | |
IN2014KN02672A (es) | ||
BR112017019625A2 (pt) | udp-glicosiltransferases | |
MX2017011615A (es) | Construcciones de vectores virales adeno-asociados de glut1 recombinantes y métodos relacionados para restablecer la expresión de glut1. | |
PH12018502104A1 (en) | Column-based fully scalable raav manufacturing process | |
PH12017500098A1 (en) | Suspension compositions of cyclosporin a for subconjunctival and periocular injection | |
MX2019005266A (es) | Composiciones, metodos y usos de transferencia genetica para tratar enfermedades neurodegenerativas. | |
ZA201701881B (en) | Adeno-associated viral vectors for treating myocilin (myoc) glaucoma | |
EA201691011A1 (ru) | Последовательность модифицированного эндолизина el188 | |
EA201891138A1 (ru) | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a | |
MX2016006277A (es) | Secuencia de endolisina kz144 modificada. | |
WO2019222314A8 (en) | Raav vectors encoding of lysosomal beta-galactosidase (glb1) and cathepsin a | |
WO2014100809A3 (en) | Ghrh agonists for the treatment of ischemic disorders | |
WO2015120527A3 (en) | Enhancement of recombinant protein expression using a membrane-based cell retention system | |
MX2018007330A (es) | Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. | |
MX2021011598A (es) | Uso de mir101 o mir128 en el tratamiento de trastornos convulsivos. | |
MX2019001804A (es) | Peptidos modificados para uso en el tratamiento de trastornos neurodegenerativos. |