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 PDF

Info

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
Application number
BR112019023869A
Other languages
English (en)
Portuguese (pt)
Inventor
L Davidson Beverly
Hong Chen Yong
Original Assignee
Childrens Hospital Philadelphia
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Childrens Hospital Philadelphia filed Critical Childrens Hospital Philadelphia
Publication of BR112019023869A2 publication Critical patent/BR112019023869A2/pt

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
    • 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (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)
BR112019023869A 2017-05-12 2018-05-11 variantes de sulfamidase (sgsh), vetores, composições e métodos e usos para tratar mucopolissacaridose tipo iiia (mps iiia) BR112019023869A2 (pt)

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 (enExample)
EP (1) EP3622060A4 (enExample)
JP (1) JP7196099B2 (enExample)
CN (1) CN110809626A (enExample)
AU (1) AU2018265869C1 (enExample)
BR (1) BR112019023869A2 (enExample)
CA (1) CA3061925A1 (enExample)
MX (1) MX2019013528A (enExample)
RU (1) RU2019140862A (enExample)
WO (1) WO2018209317A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
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
US20220042043A1 (en) * 2018-12-20 2022-02-10 Esteve Pharmaceuticals, S.A. Recombinant vectors for the long term treatment of mucchopolysacharidosis
CN114746556A (zh) * 2019-07-18 2022-07-12 利索基因公司 用于治疗山菲立普病和其他障碍的组合物和方法
IL293140A (en) * 2019-11-22 2022-07-01 Childrens Hospital Philadelphia Vector variants of adenoviruses
EP4093859A4 (en) * 2020-03-11 2023-06-14 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
WO2023086939A1 (en) * 2021-11-12 2023-05-19 Amicus Therapeutics, Inc. Compositions and methods for treating mucopolysaccharidosis iiia
WO2023204202A1 (ja) * 2022-04-19 2023-10-26 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
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)

* Cited by examiner, † Cited by third party
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
EP2691529B1 (en) * 2011-03-31 2019-06-12 University of Iowa Research Foundation AAV2 particle comprising an AAV2 capsid protein and a vector comprising a nucleic acid encoding a tripeptidyl peptidase 1 (TPP1) for use in treating late infantile ceroid lipofuscinosis (LINCL) in a non-rodent mammal by intraventricular injection or ICV delivery.
EP3912635A1 (en) * 2014-07-11 2021-11-24 Biostrategies LC Materials and methods for treating disorders associated with sulfatase enzymes

Also Published As

Publication number Publication date
US20210324354A1 (en) 2021-10-21
EP3622060A1 (en) 2020-03-18
JP2020519629A (ja) 2020-07-02
WO2018209317A1 (en) 2018-11-15
EP3622060A4 (en) 2021-02-17
AU2018265869A1 (en) 2019-11-21
AU2018265869C1 (en) 2025-01-30
RU2019140862A3 (enExample) 2021-08-17
JP7196099B2 (ja) 2022-12-26
CA3061925A1 (en) 2018-11-15
MX2019013528A (es) 2020-07-14
US12221635B2 (en) 2025-02-11
CN110809626A (zh) 2020-02-18
AU2018265869B2 (en) 2024-10-24
RU2019140862A (ru) 2021-06-15

Similar Documents

Publication Publication Date Title
BR112019023869A2 (pt) variantes de sulfamidase (sgsh), vetores, composições e métodos e usos para tratar mucopolissacaridose tipo iiia (mps iiia)
BR112018072946A2 (pt) proteínas de fusão a gdf15 e usos das mesmas
CL2022000036A1 (es) Polipéptidos modificados y usos de los mismos (divisional de solicitud no. 201900261)
BRPI0911385B8 (pt) polipeptídeo isolado, polipeptídeo de fusão, multímero e composição farmacêutica
BR112019009113A2 (pt) formulações de vírus adenoassociado
BR112016024379A2 (pt) variantes ldlr e seu uso em composições para reduzir os níveis de colesterol
CL2015002529A1 (es) Compuestos de tetrahidropirrolotiazina
CL2017002028A1 (es) Mejora del suministro de partículas virales al cuerpo estriado y al córtex.
PE20170771A1 (es) Composiciones y metodos de uso para tratar trastornos metabolicos
BR112019009074A2 (pt) composições, métodos e uso de transferência genética para o tratamento de doenças neurodegenerativas
BR112017008157A2 (pt) polipeptídeos de fgf-21 modificados e usos dos mesmos
UY32607A (es) Mutantes de fgf21 y usos del mismo
MX2015013948A (es) Composicion farmaceutica, metodos para tratamiento y sus usos.
BR112019000098A2 (pt) proteína de fornecimento ao cérebro, método de tratamento e/ou profilaxia de distúrbios cerebrais em mamíferos que possuem ou encontram-se em risco de desenvolver o mencionado distúrbio e método de diagnóstico e/ou detecção de distúrbios cerebrais em mamíferos suspeitos de possuir ou que se encontram em risco de desenvolver o mencionado distúrbio
BR112014028666A2 (pt) métodos e composições para o tratamento de depósitos amiloides
BR112019004353A2 (pt) métodos e vetores para tratar distúrbios do cns
BR112018009534A2 (pt) composição farmacêutica contendo 2-{4-[n-(5,6-difenilpirazin-2-il)-n-isopropilamino]butiloxi }-n-(metilsulfonil)acetamida
CR20200454A (es) Compuestos agonistas del factor de diferenciación del crecimiento 15 y sus métodos de uso
MX2018014718A (es) Compuestos y su uso para reducir los niveles de ácido úrico.
BR112018011046A2 (pt) agonistas receptores de piperidinona formil peptí-deo 2 e de formil peptídeo 1
MX2016009630A (es) Compuestos inmunomoduladores.
BR112017013453A2 (pt) novo peptídeo inibidor de pi3ky para o tratamento de doenças do sistema respiratório
BR112013021661A2 (pt) objetivação de fator de coagulação a tlt-1 sobre plaquetas ativadas
BR112017013999A2 (pt) ?composição derivada de uma bifidobactéria?
BR112018077225A2 (pt) proteínas de fusão tatk-cdkl5, composições, formulações e uso das mesmas

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]