PL3119892T3 - Warianty białka beta-heksozaminidazy oraz związane z nimi sposoby do leczenia gangliozydoz gm2 - Google Patents

Warianty białka beta-heksozaminidazy oraz związane z nimi sposoby do leczenia gangliozydoz gm2

Info

Publication number
PL3119892T3
PL3119892T3 PL15772341T PL15772341T PL3119892T3 PL 3119892 T3 PL3119892 T3 PL 3119892T3 PL 15772341 T PL15772341 T PL 15772341T PL 15772341 T PL15772341 T PL 15772341T PL 3119892 T3 PL3119892 T3 PL 3119892T3
Authority
PL
Poland
Prior art keywords
gangliosdoses
beta
treating
associated methods
protein variants
Prior art date
Application number
PL15772341T
Other languages
English (en)
Inventor
Don Mahuran
Brian Mark
Original Assignee
The Hospital For Sick Children
The University 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 The Hospital For Sick Children, The University Of Manitoba filed Critical The Hospital For Sick Children
Publication of PL3119892T3 publication Critical patent/PL3119892T3/pl

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)
PL15772341T 2014-03-17 2015-03-17 Warianty białka beta-heksozaminidazy oraz związane z nimi sposoby do leczenia gangliozydoz gm2 PL3119892T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461954098P 2014-03-17 2014-03-17
EP15772341.2A EP3119892B1 (en) 2014-03-17 2015-03-17 Beta-hexosaminidase protein variants and associated methods for treating gm2 gangliosdoses
PCT/IB2015/001208 WO2015150922A2 (en) 2014-03-17 2015-03-17 β-HEXOSAMINIDASE PROTEIN VARIANTS AND ASSOCIATED METHODS FOR TREATING GM2 GANGLIOSIDOSES

Publications (1)

Publication Number Publication Date
PL3119892T3 true PL3119892T3 (pl) 2021-11-29

Family

ID=54067789

Family Applications (1)

Application Number Title Priority Date Filing Date
PL15772341T PL3119892T3 (pl) 2014-03-17 2015-03-17 Warianty białka beta-heksozaminidazy oraz związane z nimi sposoby do leczenia gangliozydoz gm2

Country Status (6)

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

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US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP2485761B1 (en) 2009-10-09 2019-02-27 Armagen, Inc. Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
EP3540055A1 (en) 2010-04-23 2019-09-18 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
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
HUE054748T2 (hu) 2014-03-17 2021-09-28 Hospital For Sick Children Béta-hexózaminidáz fehérje változatai és kapcsolódó eljárások GM2 gangliozidózis kezelésére
JP6756700B2 (ja) 2014-03-18 2020-09-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
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
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
US11020443B2 (en) 2015-04-23 2021-06-01 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
DK3364997T5 (da) 2015-10-22 2024-09-30 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP3411059A4 (en) 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017218852A1 (en) 2016-06-15 2017-12-21 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
US11859179B2 (en) 2017-05-09 2024-01-02 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
EP3744846A4 (en) * 2018-01-26 2021-11-17 Tokushima University NEW ADENO-ASSOCIATED VIRUS VIRION TO TREAT TAY-SACHS DISEASE AND SANDHOFF'S DISEASE
BR112020020836A2 (pt) 2018-04-13 2021-01-19 University Of Massachusetts Vetores de aav bicistrônicos codificando subunidades alfa e beta de hexosaminidase e usos dos mesmos
EP3833689A4 (en) * 2018-08-07 2022-05-25 Armagen, Inc. METHODS AND COMPOSITIONS FOR INCREASING THE ACTIVITY IN THE CENTRAL NERVOUS SYSTEM OF HEXOSAMINIDASE A, ACID SPHINGOMYELINASE AND PALMITOYL-PROTEIN THIOESTERASE 1
US12448629B2 (en) 2018-10-05 2025-10-21 University Of Massachusetts rAAV vectors for the treatment of GM1 and GM2 gangliosidosis
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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US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
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JP5678664B2 (ja) * 2009-01-16 2015-03-04 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
HUE054748T2 (hu) 2014-03-17 2021-09-28 Hospital For Sick Children Béta-hexózaminidáz fehérje változatai és kapcsolódó eljárások GM2 gangliozidózis kezelésére

Also Published As

Publication number Publication date
EP3119892A2 (en) 2017-01-25
WO2015150922A3 (en) 2016-01-28
WO2015150922A2 (en) 2015-10-08
HUE054748T2 (hu) 2021-09-28
US10400227B2 (en) 2019-09-03
EP3119892A4 (en) 2017-08-09
ES2881341T3 (es) 2021-11-29
US20150258180A1 (en) 2015-09-17
EP3119892B1 (en) 2021-03-10
CA2940728A1 (en) 2015-10-08

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