HUE054748T2 - Béta-hexózaminidáz fehérje változatai és kapcsolódó eljárások GM2 gangliozidózis kezelésére - Google Patents

Béta-hexózaminidáz fehérje változatai és kapcsolódó eljárások GM2 gangliozidózis kezelésére

Info

Publication number
HUE054748T2
HUE054748T2 HUE15772341A HUE15772341A HUE054748T2 HU E054748 T2 HUE054748 T2 HU E054748T2 HU E15772341 A HUE15772341 A HU E15772341A HU E15772341 A HUE15772341 A HU E15772341A HU E054748 T2 HUE054748 T2 HU E054748T2
Authority
HU
Hungary
Prior art keywords
gangliosdoses
beta
treating
associated methods
protein variants
Prior art date
Application number
HUE15772341A
Other languages
English (en)
Inventor
Don Mahuran
Brian Mark
Original Assignee
Hospital For Sick Children
The Univ Of Manitoba
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 Hospital For Sick Children, The Univ Of Manitoba filed Critical Hospital For Sick Children
Publication of HUE054748T2 publication Critical patent/HUE054748T2/hu

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)
    • 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)
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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)
    • C12Y302/01052—Beta-N-acetylhexosaminidase (3.2.1.52)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
HUE15772341A 2014-03-17 2015-03-17 Béta-hexózaminidáz fehérje változatai és kapcsolódó eljárások GM2 gangliozidózis kezelésére HUE054748T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201461954098P 2014-03-17 2014-03-17

Publications (1)

Publication Number Publication Date
HUE054748T2 true HUE054748T2 (hu) 2021-09-28

Family

ID=54067789

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE15772341A HUE054748T2 (hu) 2014-03-17 2015-03-17 Béta-hexózaminidáz fehérje változatai és kapcsolódó eljárások GM2 gangliozidózis kezelésére

Country Status (7)

Country Link
US (1) US10400227B2 (hu)
EP (1) EP3119892B1 (hu)
CA (1) CA2940728A1 (hu)
ES (1) ES2881341T3 (hu)
HU (1) HUE054748T2 (hu)
PL (1) PL3119892T3 (hu)
WO (1) WO2015150922A2 (hu)

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US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2011044542A1 (en) 2009-10-09 2011-04-14 Armagen Technologies, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
WO2011133901A2 (en) 2010-04-23 2011-10-27 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
EP3318635A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
WO2015150922A2 (en) 2014-03-17 2015-10-08 The Hospital For Sick Children β-HEXOSAMINIDASE PROTEIN VARIANTS AND ASSOCIATED METHODS FOR TREATING GM2 GANGLIOSIDOSES
EP3750907A3 (en) 2014-03-18 2021-04-28 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
CA3019315A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
US10016514B2 (en) 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
DK3364997T3 (da) 2015-10-22 2024-04-22 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017176929A1 (en) 2016-04-05 2017-10-12 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
WO2018208972A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
WO2019060686A1 (en) 2017-09-22 2019-03-28 University Of Massachusetts NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF
WO2019146745A1 (ja) * 2018-01-26 2019-08-01 国立大学法人徳島大学 テイ－サックス病及びザンドホッフ病治療用の新規アデノ随伴ウイルスビリオン
AU2019317460A1 (en) * 2018-08-07 2021-03-18 Armagen, Inc. Methods and compositions for increasing the activity in the CNS of hexosaminidase a, acid sphingomyelinase, and palmitoyl-protein thioesterase 1
US20230374483A1 (en) * 2020-07-08 2023-11-23 Regents Of The University Of Minnesota Modified hexosaminidase and uses thereof
EP4359517A2 (en) * 2021-06-25 2024-05-01 BioMarin Pharmaceutical Inc. Compositions of beta-hexosaminidase variants and uses thereof

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US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
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WO2010082622A1 (ja) 2009-01-16 2010-07-22 学校法人明治薬科大学 ヒトβ-ヘキソサミニダーゼＢの基質特異性を変換した新規高機能酵素
RU2015118580A (ru) 2012-10-19 2016-12-10 Токусима Юниверсити НОВЫЙ ВЫСОКО ФУНКЦИОНАЛЬНЫЙ ФЕРМЕНТ, ИМЕЮЩИЙ МОДИФИЦИРОВАННУЮ СУБСТРАТНУЮ СПЕЦИФИЧНОСТЬ β-ГЕКСОЗАМИНИДАЗЫ ЧЕЛОВЕКА И ПРОЯВЛЯЮЩИЙ УСТОЙЧИВОСТЬ К ПРОТЕАЗЕ
WO2015150922A2 (en) 2014-03-17 2015-10-08 The Hospital For Sick Children β-HEXOSAMINIDASE PROTEIN VARIANTS AND ASSOCIATED METHODS FOR TREATING GM2 GANGLIOSIDOSES

Also Published As

Publication number Publication date
US10400227B2 (en) 2019-09-03
ES2881341T3 (es) 2021-11-29
EP3119892B1 (en) 2021-03-10
EP3119892A4 (en) 2017-08-09
PL3119892T3 (pl) 2021-11-29
US20150258180A1 (en) 2015-09-17
WO2015150922A3 (en) 2016-01-28
WO2015150922A2 (en) 2015-10-08
CA2940728A1 (en) 2015-10-08
EP3119892A2 (en) 2017-01-25

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