SG11201901494UA - Acid-alpha glucosidase variants and uses thereof - Google Patents

Acid-alpha glucosidase variants and uses thereof

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Publication number
SG11201901494UA
SG11201901494UA SG11201901494UA SG11201901494UA SG11201901494UA SG 11201901494U A SG11201901494U A SG 11201901494UA SG 11201901494U A SG11201901494U A SG 11201901494UA SG 11201901494U A SG11201901494U A SG 11201901494UA SG 11201901494U A SG11201901494U A SG 11201901494UA
Authority
SG
Singapore
Prior art keywords
international
rue
paris
evry
september
Prior art date
Application number
SG11201901494UA
Inventor
Federico Mingozzi
Giuseppe Ronzitti
Original Assignee
Genethon
Inst Nat Sante Rech Med
Univ Devry Val Dessonne
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
Priority claimed from EP16306150.0A external-priority patent/EP3293260A1/en
Application filed by Genethon, Inst Nat Sante Rech Med, Univ Devry Val Dessonne filed Critical Genethon
Publication of SG11201901494UA publication Critical patent/SG11201901494UA/en

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    • 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
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    • 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)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
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    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/34—Muscles; Smooth muscle cells; Heart; Cardiac stem cells; Myoblasts; Myocytes; Cardiomyocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/37—Digestive system
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0658—Skeletal muscle cells, e.g. myocytes, myotubes, myoblasts
    • C—CHEMISTRY; METALLURGY
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/067—Hepatocytes
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
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    • C12N2510/00—Genetically modified cells
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    • 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
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

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Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 15 March 2018 (15.03.2018) WIP0 I PCT mu °million °nolo ImiloomomommovoimIE (10) International Publication Number WO 2018/046774 Al (51) International Patent Classification: C12N 9/26 (2006.01) (21) International Application Number: PCT/EP2017/072944 (22) International Filing Date: 12 September 2017 (12.09.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 16306150.0 12 September 2016 (12.09.2016) EP 16306187.2 16 September 2016 (16.09.2016) EP (71) Applicants: GENETHON [FR/FR]; 1 bis, rue de l'Internationale, 91000 EVRY (FR). INSERM (INSTI- TUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) [FR/FR]; 101 rue de Tol- biac, 75013 PARIS (FR). UNIVERSITE D'EVRY VAL D'ESSONNE [FR/FR]; Boulevard Francois Mitterrand, 91000 EVRY (FR). = (72) Inventors: MINGOZZI, Federico; 104, rue Oberkampf, 75011 PARIS (FR). RONZITTI, Giuseppe; 134, rue du chemin vert, 75011 PARIS (FR). (74) Agent: CABINET BECKER ET ASSOCIES; 25, rue Louis le Grand, 75002 PARIS (FR). = (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. = (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, 1-1 TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, • KM, ML, MR, NE, SN, TD, TG). N 71' Published: ▪ — with international search report (Art. 21(3)) 71- — with sequence listing part of description (Rule 5.2(a)) O 00 1-1 (54) Title: ACID-ALPHA GLUCOSIDASE VARIANTS AND USES THEREOF • (57) : The present invention relates to variants of acid-alpha glucosidase and uses thereof. C
SG11201901494UA 2016-09-12 2017-09-12 Acid-alpha glucosidase variants and uses thereof SG11201901494UA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16306150.0A EP3293260A1 (en) 2016-09-12 2016-09-12 Acid-alpha glucosidase variants and uses thereof
EP16306187 2016-09-16
PCT/EP2017/072944 WO2018046774A1 (en) 2016-09-12 2017-09-12 Acid-alpha glucosidase variants and uses thereof

Publications (1)

Publication Number Publication Date
SG11201901494UA true SG11201901494UA (en) 2019-03-28

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ID=59887252

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SG11201901494UA SG11201901494UA (en) 2016-09-12 2017-09-12 Acid-alpha glucosidase variants and uses thereof

Country Status (17)

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US (2) US11339406B2 (en)
EP (1) EP3510149A1 (en)
JP (2) JP7245155B2 (en)
KR (2) KR102617502B1 (en)
CN (2) CN117384890A (en)
AU (2) AU2017322376B2 (en)
BR (1) BR112019004785A2 (en)
CA (1) CA3035868A1 (en)
CL (1) CL2019000609A1 (en)
CO (1) CO2019002252A2 (en)
IL (2) IL299325A (en)
MX (1) MX2019002842A (en)
PE (1) PE20191203A1 (en)
PH (1) PH12019500408A1 (en)
SG (1) SG11201901494UA (en)
WO (1) WO2018046774A1 (en)
ZA (1) ZA201902141B (en)

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