[go: up one dir, main page]

MX2024004420A - Variantes de alfa-glucosidasa acida y usos de las mismas. - Google Patents

Variantes de alfa-glucosidasa acida y usos de las mismas.

Info

Publication number
MX2024004420A
MX2024004420A MX2024004420A MX2024004420A MX2024004420A MX 2024004420 A MX2024004420 A MX 2024004420A MX 2024004420 A MX2024004420 A MX 2024004420A MX 2024004420 A MX2024004420 A MX 2024004420A MX 2024004420 A MX2024004420 A MX 2024004420A
Authority
MX
Mexico
Prior art keywords
acid
alpha glucosidase
glucosidase variants
variants
alpha
Prior art date
Application number
MX2024004420A
Other languages
English (en)
Inventor
Federico Mingozzi
Giuseppe Ronzitti
Original Assignee
Genethon
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 filed Critical Genethon
Publication of MX2024004420A publication Critical patent/MX2024004420A/es

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
    • 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
    • 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
    • A61K35/407—Liver; Hepatocytes
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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)
    • 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)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • 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
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • 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
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Rheumatology (AREA)
  • Plant Pathology (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)

Abstract

La presente invención se refiere a variantes de alfa-glucosidasa ácida y a usos de las mismas.
MX2024004420A 2016-09-12 2019-03-12 Variantes de alfa-glucosidasa acida y usos de las mismas. MX2024004420A (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
MX2024004420A true MX2024004420A (es) 2024-05-06

Family

ID=59887252

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2019002842A MX2019002842A (es) 2016-09-12 2017-09-12 Variantes de alfa-glucosidasa acida y usos de las mismas.
MX2024004420A MX2024004420A (es) 2016-09-12 2019-03-12 Variantes de alfa-glucosidasa acida y usos de las mismas.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2019002842A MX2019002842A (es) 2016-09-12 2017-09-12 Variantes de alfa-glucosidasa acida y usos de las mismas.

Country Status (17)

Country Link
US (3) US11339406B2 (es)
EP (1) EP3510149A1 (es)
JP (3) JP7245155B2 (es)
KR (2) KR102617502B1 (es)
CN (2) CN110050063B (es)
AU (2) AU2017322376B2 (es)
BR (1) BR112019004785A2 (es)
CA (1) CA3035868A1 (es)
CL (1) CL2019000609A1 (es)
CO (1) CO2019002252A2 (es)
IL (3) IL299325B2 (es)
MX (2) MX2019002842A (es)
PE (2) PE20250258A1 (es)
PH (1) PH12019500408A1 (es)
SG (1) SG11201901494UA (es)
WO (1) WO2018046774A1 (es)
ZA (1) ZA201902141B (es)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
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
CA3066569A1 (en) 2017-06-07 2018-12-13 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
JP7779653B2 (ja) 2018-02-07 2025-12-03 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 治療用タンパク質送達のための方法および組成物
SG11202011191WA (en) * 2018-05-16 2020-12-30 Spark Therapeutics Inc Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same
KR20250140631A (ko) 2018-05-17 2025-09-25 리제너론 파마슈티칼스 인코포레이티드 항-cd63 항체, 콘쥬게이트, 및 이의 용도
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
BR112021003399A2 (pt) 2018-08-24 2021-05-18 Spark Therapeutics, Inc. sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso
KR20210148273A (ko) 2019-04-08 2021-12-07 제네똥 근육 발현을 위한 하이브리드 프로모터
AU2020266829A1 (en) * 2019-04-30 2021-11-11 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of Pompe disease
EP3980549A4 (en) * 2019-06-05 2023-07-26 Orchard Therapeutics (Europe) Limited COMPOSITIONS AND METHODS FOR MODIFICATION OF EUKARYOTIC CELLS
EP3996696A1 (en) 2019-07-09 2022-05-18 Genethon Treatment of glycogen storage disease (gsd)
JP2022544004A (ja) * 2019-07-26 2022-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
WO2021078834A1 (en) 2019-10-22 2021-04-29 Genethon Chimeric acid-alpha glucosidase polypeptides and uses thereof
EP4048786A1 (en) * 2019-10-22 2022-08-31 Genethon Chimeric polypeptides and uses thereof
EP3913060A1 (en) * 2020-05-22 2021-11-24 Genethon Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy
WO2022076556A2 (en) 2020-10-07 2022-04-14 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)
CA3193833A1 (en) 2020-10-09 2022-04-14 Juliette HORDEAUX Compositions and methods for treatment of fabry disease
US20240350670A1 (en) 2021-08-04 2024-10-24 Genethon Hybrid promoters for gene expression in muscles and in the cns
US20240408157A1 (en) 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CA3242731A1 (en) * 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
CN118667797A (zh) * 2023-03-14 2024-09-20 北京据德医药科技有限公司 一种重组酸性α-葡萄糖苷酶及其用途
WO2025064350A1 (en) * 2023-09-18 2025-03-27 AskBio Inc. Therapeutic adeno-associated virus using nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with heterologous signal peptide

Family Cites Families (29)

* Cited by examiner, † Cited by third party
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
JP4990434B2 (ja) 1998-12-07 2012-08-01 ジェンザイム・コーポレーション ポンペ病の処置
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
EP1716232B9 (en) * 2004-02-10 2010-10-13 ZyStor Therapeutics , Inc. Acid alpha-glucosidase and fragments thereof
EP3290513B1 (en) 2004-06-01 2022-09-07 Genzyme Corporation Compositions and methods to prevent aav vector aggregation
PL2318037T3 (pl) 2008-07-08 2015-07-31 Univ Duke Sposób leczenia Choroby Spichrzeniowej Glikogenu
KR101618767B1 (ko) * 2008-09-02 2016-05-09 한국생명공학연구원 외래단백질을 고효율로 생산하는 리더서열
US9018001B2 (en) * 2008-10-02 2015-04-28 Unitargeting Research As Kit for the optimisation of protein synthesis/secretion
CN104651428A (zh) * 2009-09-29 2015-05-27 根特大学 水解甘露糖-1-磷酸-6-甘露糖连接为磷酸-6-甘露糖
JP6151183B2 (ja) * 2010-09-29 2017-06-21 オキシレイン ユーケー リミテッド マンノース−１−ホスホ−６−マンノース結合からキャップを外すことおよびリン酸化ｎ−グリカンを脱マンノシル化することができるマンノシダーゼならびに哺乳動物細胞による糖タンパク質の取り込みを促進する方法
AU2010366066B2 (en) * 2010-12-22 2016-01-14 Fondazione Telethon Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses
EP2471929A1 (en) * 2010-12-29 2012-07-04 Algenics Production of high mannose glycosylated proteins stored in the plastid of microalgae
SG194486A1 (en) 2011-04-22 2013-12-30 Genzyme Corp Modified acid alpha glucosidase with accelerated processing
WO2013013017A2 (en) 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for modifying the glycosylation of lysosomal storage disorder therapeutics
HK1206779A1 (en) 2012-04-02 2016-01-15 Modernatx, Inc. Modified polynucleotides for the production of nuclear proteins
WO2013177533A1 (en) 2012-05-25 2013-11-28 California Institute Of Technology Expression of secreted and cell-surface polypeptides
SMT202000445T1 (it) 2012-06-19 2020-09-10 Univ Florida Composizioni e metodi per trattare patologie
US10046010B2 (en) * 2012-08-06 2018-08-14 Brainstorm Cell Therapeutics Ltd. Methods of generating mesenchymal stem cells which secrete neurotrophic factors
JP2016514096A (ja) * 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 ポンぺ病の処置のための方法および組成物
NZ716102A (en) 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
AU2015219339B2 (en) * 2014-02-19 2020-03-05 Bioasis Technologies Inc. P97-IDS fusion proteins
EP3851537B1 (en) 2014-04-25 2023-12-27 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
WO2015196179A1 (en) 2014-06-20 2015-12-23 University Of Florida Research Foundation, Inc. Methods of packaging multiple adeno-associated virus vectors
MA40791A (fr) * 2014-10-24 2017-09-27 Shire Human Genetic Therapies Ciblage lysosomal d'enzymes et utilisations associées
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof

Also Published As

Publication number Publication date
JP7689149B2 (ja) 2025-06-05
PE20250258A1 (es) 2025-01-29
IL299325B1 (en) 2025-04-01
US20220275396A1 (en) 2022-09-01
KR102617502B1 (ko) 2023-12-26
US11339406B2 (en) 2022-05-24
JP2019533990A (ja) 2019-11-28
WO2018046774A1 (en) 2018-03-15
AU2017322376B2 (en) 2023-08-17
IL299325A (en) 2023-02-01
IL319481A (en) 2025-05-01
KR20240001721A (ko) 2024-01-03
CN110050063B (zh) 2023-11-03
CN117384890A (zh) 2024-01-12
JP7245155B2 (ja) 2023-03-23
KR20190057327A (ko) 2019-05-28
RU2019110773A (ru) 2020-10-12
CN110050063A (zh) 2019-07-23
BR112019004785A2 (pt) 2019-06-04
US20250270590A1 (en) 2025-08-28
JP2023072007A (ja) 2023-05-23
AU2017322376A1 (en) 2019-03-28
MX2019002842A (es) 2019-08-29
IL299325B2 (en) 2025-08-01
IL265084B2 (en) 2023-05-01
US12305187B2 (en) 2025-05-20
IL265084B1 (en) 2023-01-01
EP3510149A1 (en) 2019-07-17
RU2019110773A3 (es) 2020-11-25
PE20191203A1 (es) 2019-09-10
JP2025060967A (ja) 2025-04-10
CO2019002252A2 (es) 2019-05-21
IL265084A (es) 2019-04-30
US20190390225A1 (en) 2019-12-26
ZA201902141B (en) 2024-03-27
CA3035868A1 (en) 2018-03-15
AU2023258449A1 (en) 2023-11-23
SG11201901494UA (en) 2019-03-28
PH12019500408A1 (en) 2020-01-20
CL2019000609A1 (es) 2019-07-05

Similar Documents

Publication Publication Date Title
ZA201902141B (en) Acid-alpha glucosidase variants and uses thereof
ZA201902140B (en) Acid-alpha glucosidase variants and uses thereof
MY202415A (en) Subcutaneous formulations of anti-cd38 antibodies and their uses
PH12018500711A1 (en) Anti-pd1 antibodies and methods of use
MX2019002843A (es) Variantes de alfa-glucosidasa acida y usos de las mismas.
EP4335874A3 (en) Anti-hla-g antibodies and use thereof
MX2019003623A (es) Composiciones y metodos para el tratamiento de afecciones oftalmicas.
MX2016003843A (es) Inhibidores de cinasas asociadas al receptor de interleucina 1 (irak) y usos de los mismos.
PH12016501763A1 (en) Multispecific antibodies
AU2015287674B2 (en) Topical antiviral compositions and methods of using the same
PH12018501279A1 (en) Compositions comprising 15-hepe and methods of using the same
AU2017317563A8 (en) Detergent compositions comprising xanthan lyase variants I
MX2018004674A (es) Moduladores de interacción de sestrina-gator2 y sus usos.
MX2017011223A (es) Variantes de protoxina ii y metodos de uso.
EA201600026A1 (ru) Бактериальная гиалуронидаза и способ ее производства
IN2013MU00848A (es)
MX2017016623A (es) Variantes de quimosina con propiedades mejoradas.
MX2017012654A (es) Variantes de protoxina-ii y metodos de uso.
PH12016501201A1 (en) Synthesis of ent-progesterone and intermediates thereof
AU358315S (en) Novelty sconce
AU357826S (en) Novelty chandelier sconce
PH12017500499A1 (en) Synthesis of ent-progesterone and intermediates thereof
PH12018501166B1 (en) Concentrated gibberellin solution formulations
MX2015012902A (es) Sintesis de ent-progesterona e intermediarios de la misma.
AU365961S (en) Clasp