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)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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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CA2694762A1 (en) 2007-07-27 2009-02-05 Armagen Technologies, Inc. Methods and compositions for increasing alpha-l-iduronidase activity in the cns
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
ES2725200T3 (es) 2009-10-09 2019-09-20 Armagen Inc Métodos y composiciones para aumentar la actividad de iduronato 2-sulfatasa en el SNC
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
ES2661680T3 (es) 2011-04-21 2018-04-03 University Of Massachusetts Composiciones basadas en VAAr y métodos para tratar deficiencias de alfa-1 anti-tripsina
EP3119892B1 (en) 2014-03-17 2021-03-10 The Hospital For Sick Children Beta-hexosaminidase protein variants and associated methods for treating gm2 gangliosdoses
EP4410805A2 (en) 2014-03-18 2024-08-07 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3209311B1 (en) 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
WO2016172155A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
CA3021949C (en) 2015-04-24 2023-10-17 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
US11253576B2 (en) 2015-10-22 2022-02-22 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
US11060088B2 (en) 2016-02-12 2021-07-13 University Of Massachusetts Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts COMPOSITIONS AND METHODS FOR SELECTIVE INHIBITION OF EXPRESSION OF GRAINHEAD PROTEIN
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
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
AU2018338188A1 (en) 2017-09-22 2020-04-02 University Of Massachusetts SOD1 dual expression vectors and uses thereof
EP3744846A4 (en) * 2018-01-26 2021-11-17 Tokushima University NEW ADENO-ASSOCIATED VIRUS VIRION TO TREAT TAY-SACHS DISEASE AND SANDHOFF'S DISEASE
CA3107749A1 (en) * 2018-08-07 2020-02-13 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
AR126237A1 (es) * 2021-06-25 2023-10-04 Biomarin Pharm Inc COMPOSICIONES DE VARIANTES DE b-HEXOSAMINIDASA Y USOS DE ESTAS

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US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4598049A (en) 1983-08-31 1986-07-01 Systec Inc. General purpose gene synthesizer
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US20080140008A1 (en) 2006-12-06 2008-06-12 Medtronic, Inc. Intrathecal catheter
JP2008206428A (ja) * 2007-02-24 2008-09-11 Univ Of Tokushima β−ヘキソサミニダーゼＡ（ＨｅｘＡ）を構成するα−サブユニット、前記α−サブユニットを有するＨｅｘＡ、前記α−サブユニットをコードする遺伝子、ベクターおよび形質転換細胞
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
WO2014061735A1 (ja) * 2012-10-19 2014-04-24 国立大学法人徳島大学 ヒトβ－ヘキソサミニダーゼＢの基質特異性を変換し、且つ、プロテアーゼ抵抗性を付与した新規高機能酵素
EP3119892B1 (en) 2014-03-17 2021-03-10 The Hospital For Sick Children Beta-hexosaminidase protein variants and associated methods for treating gm2 gangliosdoses

Also Published As

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

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