SG11201901494UA - Acid-alpha glucosidase variants and uses thereof - Google Patents
Acid-alpha glucosidase variants and uses thereofInfo
- 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
Links
- 102000004366 Glucosidases Human genes 0.000 title abstract 3
- 108010056771 Glucosidases Proteins 0.000 title abstract 3
- 230000008520 organization Effects 0.000 abstract 1
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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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)
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- 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
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- 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
- A61K35/407—Liver; Hepatocytes
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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
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- 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
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- 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
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- A—HUMAN NECESSITIES
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- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P3/00—Drugs for disorders of the metabolism
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- 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
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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
- C12N5/067—Hepatocytes
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- 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
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- 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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- C12N2750/00—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
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- C12N2830/00—Vector systems having a special element relevant for transcription
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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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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
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- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
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- Veterinary Medicine (AREA)
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- Pharmacology & Pharmacy (AREA)
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- Developmental Biology & Embryology (AREA)
- Virology (AREA)
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- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Rheumatology (AREA)
- Nutrition Science (AREA)
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- Plant Pathology (AREA)
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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
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 |
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SG11201901494UA true SG11201901494UA (en) | 2019-03-28 |
Family
ID=59887252
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201901494UA SG11201901494UA (en) | 2016-09-12 | 2017-09-12 | Acid-alpha glucosidase variants and uses thereof |
Country Status (17)
Country | Link |
---|---|
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) |
Families Citing this family (15)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
WO2018226861A1 (en) | 2017-06-07 | 2018-12-13 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
WO2019222411A1 (en) * | 2018-05-16 | 2019-11-21 | Spark Therapeutics, Inc. | Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same |
EP3952920A1 (en) | 2019-04-08 | 2022-02-16 | Genethon | Hybrid promoters for muscle expression |
KR20220008280A (en) * | 2019-04-30 | 2022-01-20 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Compositions useful for the treatment of Pompe disease |
US20220331408A1 (en) | 2019-07-09 | 2022-10-20 | Genethon | Treatment of glycogen storage disease (gsd) |
CA3145112A1 (en) * | 2019-07-26 | 2021-02-04 | Chunping Qiao | Engineered nucleic acid regulatory element and methods of uses thereof |
WO2021078834A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric acid-alpha glucosidase polypeptides and uses thereof |
CA3151442A1 (en) * | 2019-10-22 | 2021-04-29 | Pasqualina COLELLA | Sortilin receptor ligand based chimeric polypeptides and uses thereof |
JP2023545731A (en) | 2020-10-07 | 2023-10-31 | アスクレピオス バイオファーマシューティカル, インコーポレイテッド | Therapeutic adeno-associated virus delivery of fukutin-related protein (FKRP) to treat dystroglycanopathy disorders including limb girdle type 2I (LGMD2I) |
CA3193833A1 (en) | 2020-10-09 | 2022-04-14 | Juliette HORDEAUX | Compositions and methods for treatment of fabry disease |
CA3226119A1 (en) | 2021-08-04 | 2023-02-09 | Giuseppe RONZITTI | Hybrid promoters for gene expression in muscles and in the cns |
WO2023102517A1 (en) | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
TW202332767A (en) * | 2022-02-02 | 2023-08-16 | 美商雷傑納榮製藥公司 | Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease |
Family Cites Families (29)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6858425B1 (en) | 1998-12-04 | 2005-02-22 | Genzyme Corporation | Human acid alpha glucosidase gene and bovine alpha-S1 casein gene sequences |
EP1137762B1 (en) | 1998-12-07 | 2008-10-08 | Genzyme Corporation | Treatment of pompe's disease |
US20070037165A1 (en) | 2000-09-08 | 2007-02-15 | Applera Corporation | Polymorphisms in known genes associated with human disease, methods of detection and uses thereof |
US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
EP1587923B1 (en) | 2003-01-22 | 2011-08-24 | Duke University | Improved constructs for expressing lysosomal polypeptides |
US20050244400A1 (en) * | 2004-02-10 | 2005-11-03 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
US7704721B2 (en) | 2004-06-01 | 2010-04-27 | Genzyme Corporation | Compositions and methods to prevent AAV vector aggregation |
WO2010005565A2 (en) | 2008-07-08 | 2010-01-14 | Duke University | Method of treating glycogen storage disease |
KR101618767B1 (en) * | 2008-09-02 | 2016-05-09 | 한국생명공학연구원 | Effective leader sequences needed for high expression of heterologous proteins |
US9018001B2 (en) * | 2008-10-02 | 2015-04-28 | Unitargeting Research As | Kit for the optimisation of protein synthesis/secretion |
KR102000383B1 (en) | 2009-09-29 | 2019-07-15 | 유니버시테이트 젠트 | Hydrolysis of mannose-1-phospho-6-mannose linkage to phospho-6-mannose |
US9347050B2 (en) | 2010-09-29 | 2016-05-24 | Oxyrane Uk Limited | Mannosidases capable of uncapping mannose-1-phospho-6-mannose linkages and demannosylating phosphorylated N-glycans and methods of facilitating mammalian cellular uptake of glycoproteins |
JP6023073B2 (en) * | 2010-12-22 | 2016-11-09 | フォンダッツィオーネ・テレソン | Therapeutic strategy for treating CNS lesions in mucopolysaccharidosis |
EP2471929A1 (en) | 2010-12-29 | 2012-07-04 | Algenics | Production of high mannose glycosylated proteins stored in the plastid of microalgae |
BR112013026976A2 (en) | 2011-04-22 | 2019-09-24 | Genzyme Corp | alpha accelerated modified modified acid glucosity |
WO2013013017A2 (en) | 2011-07-21 | 2013-01-24 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for modifying the glycosylation of lysosomal storage disorder therapeutics |
EP3501550A1 (en) | 2012-04-02 | 2019-06-26 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
US9540657B2 (en) | 2012-05-25 | 2017-01-10 | California Institute Of Technology | Expression of secreted and cell-surface polypeptides |
JP2015523998A (en) | 2012-06-19 | 2015-08-20 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | Compositions and methods for treating diseases |
DK2880151T3 (en) | 2012-08-06 | 2020-08-17 | Brainstorm Cell Therapeutics Ltd | METHODS FOR THE PRODUCTION OF MESENKYMAL STEM CELLS THAT SEPARATE NEUROTROPHIC FACTORS |
JP2016514096A (en) * | 2013-02-20 | 2016-05-19 | バレリオン セラピューティクス, エルエルシー | Methods and compositions for the treatment of Pompe disease |
ES2774966T3 (en) | 2013-07-22 | 2020-07-23 | Childrens Hospital Philadelphia | Variants of aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
WO2015126729A1 (en) | 2014-02-19 | 2015-08-27 | Bioasis Technologies, Inc. | P97-ids fusion proteins |
FI3851537T3 (en) | 2014-04-25 | 2024-03-21 | Genethon | Treatment of hyperbilirubinemia |
AU2015274348A1 (en) | 2014-06-13 | 2017-01-12 | Valerion Therapeutics, Llc | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
US10781459B2 (en) | 2014-06-20 | 2020-09-22 | University Of Florida Research Foundation, Incorporated | Methods of packaging multiple adeno-associated virus vectors |
EP3220958A1 (en) * | 2014-10-24 | 2017-09-27 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting of enzymes, and uses thereof |
EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
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2017
- 2017-09-12 BR BR112019004785A patent/BR112019004785A2/en unknown
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JP2023072007A (en) | 2023-05-23 |
CA3035868A1 (en) | 2018-03-15 |
IL265084B2 (en) | 2023-05-01 |
CO2019002252A2 (en) | 2019-05-21 |
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BR112019004785A2 (en) | 2019-06-04 |
RU2019110773A (en) | 2020-10-12 |
CN110050063A (en) | 2019-07-23 |
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KR20240001721A (en) | 2024-01-03 |
JP7245155B2 (en) | 2023-03-23 |
CN110050063B (en) | 2023-11-03 |
AU2017322376B2 (en) | 2023-08-17 |
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AU2023258449A1 (en) | 2023-11-23 |
IL265084B1 (en) | 2023-01-01 |
AU2017322376A1 (en) | 2019-03-28 |
PE20191203A1 (en) | 2019-09-10 |
MX2019002842A (en) | 2019-08-29 |
US20190390225A1 (en) | 2019-12-26 |
JP2019533990A (en) | 2019-11-28 |
IL299325A (en) | 2023-02-01 |
IL265084A (en) | 2019-04-30 |
EP3510149A1 (en) | 2019-07-17 |
CN117384890A (en) | 2024-01-12 |
US20220275396A1 (en) | 2022-09-01 |
ZA201902141B (en) | 2024-03-27 |
KR102617502B1 (en) | 2023-12-26 |
RU2019110773A3 (en) | 2020-11-25 |
KR20190057327A (en) | 2019-05-28 |
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