MA50546A - Compositions et méthodes pour l'internalisation d'enzymes - Google Patents

Compositions et méthodes pour l'internalisation d'enzymes

Info

Publication number
MA50546A
MA50546A MA050546A MA50546A MA50546A MA 50546 A MA50546 A MA 50546A MA 050546 A MA050546 A MA 050546A MA 50546 A MA50546 A MA 50546A MA 50546 A MA50546 A MA 50546A
Authority
MA
Morocco
Prior art keywords
internalization
enzymes
compositions
methods
Prior art date
Application number
MA050546A
Other languages
English (en)
Inventor
Andrew Baik
Katherine Cygnar
Christos Kyratsous
Christopher Schoenherr
Cheng Wang
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Publication of MA50546A publication Critical patent/MA50546A/fr

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596Molecules with a "CD"-designation not provided for elsewhere
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2896Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against molecules with a "CD"-designation, not provided for elsewhere
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405Glucanases
    • C12N9/2408Glucanases acting on alpha -1,4-glucosidic bonds
    • C12N9/2411Amylases
    • C12N9/2428Glucan 1,4-alpha-glucosidase (3.2.1.3), i.e. glucoamylase
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/20Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/30Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/31Immunoglobulins specific features characterized by aspects of specificity or valency multispecific
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/60Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77Internalization into the cell
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/90Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/92Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/33Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10041Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
MA050546A 2017-06-07 2018-06-06 Compositions et méthodes pour l'internalisation d'enzymes MA50546A (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762516656P 2017-06-07 2017-06-07
US201762574719P 2017-10-19 2017-10-19
US201862673098P 2018-05-17 2018-05-17

Publications (1)

Publication Number Publication Date
MA50546A true MA50546A (fr) 2020-09-16

Family

ID=62815133

Family Applications (1)

Application Number Title Priority Date Filing Date
MA050546A MA50546A (fr) 2017-06-07 2018-06-06 Compositions et méthodes pour l'internalisation d'enzymes

Country Status (17)

Country Link
US (3) US11208458B2 (fr)
EP (1) EP3635009B1 (fr)
JP (2) JP7348844B2 (fr)
KR (1) KR102760654B1 (fr)
CN (1) CN110809583A (fr)
AU (2) AU2018281280B2 (fr)
BR (1) BR112019025888A2 (fr)
CA (1) CA3066569A1 (fr)
CL (1) CL2019003586A1 (fr)
CO (1) CO2019014682A2 (fr)
IL (1) IL271193B2 (fr)
MA (1) MA50546A (fr)
MY (1) MY198804A (fr)
NZ (1) NZ760232A (fr)
PH (1) PH12019550261A1 (fr)
TW (2) TWI880364B (fr)
WO (1) WO2018226861A1 (fr)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MY171761A (en) 2012-03-14 2019-10-28 Regeneron Pharma Multispecific antigen-binding molecules and uses thereof
US11191844B2 (en) 2015-07-06 2021-12-07 Regeneran Pharmaceuticals, Inc. Multispecific antigen-binding molecules and uses thereof
IL287019B2 (en) * 2015-12-08 2025-10-01 Regeneron Pharma Preparations and methods for internalizing enzymes
EP3635009B1 (fr) 2017-06-07 2026-02-25 Regeneron Pharmaceuticals, Inc. Compositions et méthodes pour l'internalisation d'enzymes
IL276464B2 (en) 2018-02-07 2026-01-01 Regeneron Pharma Methods and compositions for administering therapeutic protein
MA52626A (fr) * 2018-05-17 2021-03-24 Regeneron Pharma Anticorps anti-cd63, conjugués et leurs utilisations
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
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2020028832A1 (fr) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
AU2019316103B2 (en) 2018-08-02 2026-02-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
AU2019384790A1 (en) 2018-11-21 2021-03-18 Regeneron Pharmaceuticals, Inc. Anti-staphylococcus antibodies and uses thereof
US20220023434A1 (en) 2018-12-19 2022-01-27 The Board Of Trustees Of The Leland Stanford Junior University Bifunctional Molecules for Lysosomal Targeting and Related Compositions and Methods
TW202134277A (zh) * 2019-11-05 2021-09-16 美商再生元醫藥公司 N—端scFv多特異性結合分子
BR112023001272A2 (pt) * 2020-07-24 2023-04-04 Massachusetts Gen Hospital Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células
US20240025971A1 (en) * 2020-10-23 2024-01-25 Atreca, Inc. Antibodies to coronavirus sars-cov-2
KR102494508B1 (ko) * 2020-12-23 2023-02-06 아주대학교산학협력단 CRISPR/Cas 시스템을 이용한 유도만능 줄기세포 제조방법
CA3211270A1 (fr) * 2021-02-19 2022-08-25 Vib Vzw Liants de recepteur de mannose-6-phosphate independants des cations
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス,インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
JP2024524614A (ja) 2021-07-14 2024-07-05 ライシア セラピューティクス, インコーポレイテッド Asgpr細胞表面受容体結合化合物及びコンジュゲート
CA3228014A1 (fr) * 2021-07-30 2023-02-16 Vib Vzm Liants du recepteur mannose-6-phosphate independants des cations pour la degradation ciblee de proteines
JP2025508677A (ja) 2022-02-02 2025-04-10 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 ポンペ病の処置のための抗TfR:GAA及び抗CD63:GAAの挿入
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
CN114731987B (zh) * 2022-05-19 2023-06-23 华芯微鱼(苏州)生物科技有限公司 颅缝早闭症小鼠模型、其构建方法及应用
CN120112164A (zh) 2022-07-29 2025-06-06 瑞泽恩制药公司 包含修饰的转铁蛋白受体基因座的非人动物
EP4569000A2 (fr) * 2022-08-12 2025-06-18 Lycia Therapeutics, Inc. Anticorps bispécifiques et constructions pour dégradation de ciblage lysosomal et leurs procédés d'utilisation
EP4612184A1 (fr) 2022-11-04 2025-09-10 Regeneron Pharmaceuticals, Inc. Protéines de liaison de sous-unité auxiliaire gamma 1 du canal calcique dépendant de la tension (cacng1) et administration médiée par cacng1 au muscle squelettique
WO2024107765A2 (fr) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions et procédés d'administration médiée par le récepteur 3 du facteur de croissance des fibroblastes à des astrocytes
AU2024317483A1 (en) 2023-07-28 2026-01-29 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
AU2024315073A1 (en) 2023-07-28 2026-01-22 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
TW202540428A (zh) 2024-01-26 2025-10-16 美商雷傑納榮製藥公司 使用漿細胞耗乏劑及/或b細胞耗乏劑遏制宿主抗aav抗體反應且實現aav轉導及重複給藥的方法及組成物
US20250276092A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response

Family Cites Families (195)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4444878A (en) 1981-12-21 1984-04-24 Boston Biomedical Research Institute, Inc. Bispecific antibody determinants
US5156965A (en) 1983-11-29 1992-10-20 Igen, Inc. Catalytic antibodies
US5229272A (en) 1989-04-25 1993-07-20 Igen, Inc. Catalytic antibody components
US5126258A (en) 1984-09-07 1992-06-30 Scripps Clinic And Research Foundation Molecules with antibody combining sites that exhibit catalytic properties
US5030717A (en) 1986-09-17 1991-07-09 Scripps Clinic And Research Foundation Antibodies which catalyze hydrolysis of ester bonds
IL85035A0 (en) 1987-01-08 1988-06-30 Int Genetic Eng Polynucleotide molecule,a chimeric antibody with specificity for human b cell surface antigen,a process for the preparation and methods utilizing the same
US4975278A (en) 1988-02-26 1990-12-04 Bristol-Myers Company Antibody-enzyme conjugates in combination with prodrugs for the delivery of cytotoxic agents to tumor cells
US5851527A (en) 1988-04-18 1998-12-22 Immunomedics, Inc. Method for antibody targeting of therapeutic agents
US5601819A (en) 1988-08-11 1997-02-11 The General Hospital Corporation Bispecific antibodies for selective immune regulation and for selective immune cell binding
US5208020A (en) 1989-10-25 1993-05-04 Immunogen Inc. Cytotoxic agents comprising maytansinoids and their therapeutic use
EP0940468A1 (fr) 1991-06-14 1999-09-08 Genentech, Inc. Domaine variable d'un anticorps humanisé
CA2161114A1 (fr) 1993-04-23 1994-11-10 Andrew D. Napper Anticorps catalytiques hydrolysant des amides primaires; methodes d'elicitation de ces anticorps
US5602021A (en) 1994-12-29 1997-02-11 Catalytic Antibodies, Inc. Method for generating proteolytic enzymes specific against a selected peptide sequence
US5585108A (en) 1994-12-30 1996-12-17 Nanosystems L.L.C. Formulations of oral gastrointestinal therapeutic agents in combination with pharmaceutically acceptable clays
US5731168A (en) 1995-03-01 1998-03-24 Genentech, Inc. Method for making heteromultimeric polypeptides
US5714586A (en) 1995-06-07 1998-02-03 American Cyanamid Company Methods for the preparation of monomeric calicheamicin derivative/carrier conjugates
CA2224907A1 (fr) 1995-07-25 1997-02-13 Introgene B.V. Procedes et moyens d'apport cible de genes
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
EP0932417B1 (fr) 1996-10-17 2003-03-05 Immunomedics, Inc. Conjugue d'une toxine non antigenique et proteine de fusion d'un systeme recepteur de penetration intracellulaire
US6372205B1 (en) 1997-06-11 2002-04-16 The School Of Pharmacy, University Of London Pharmaceutical compositions containing antibody-enzyme conjugates in combination with prodrugs
IT1293511B1 (it) 1997-07-30 1999-03-01 Gentili Ist Spa Anticorpi monoclonali catalitici ad attivita' proteasica per la lisi selettiva della componente proteica di placche e aggregati correlati
JP2002509158A (ja) 1998-01-15 2002-03-26 センター・フォー・モレキュラー・メディシン・アンド・イムノロジー アーム付きリガンドの送達向上のための抗体と受容体ターゲティング部分
US6703488B1 (en) 1998-01-15 2004-03-09 Center For Molecular Medicine And Immunology Antibody/receptor targeting moiety for enhanced delivery of armed ligand
US6235714B1 (en) 1998-03-23 2001-05-22 Sudhir Paul Methods for identifying inducers and inhibitors of proteolytic antibodies, compositions and their uses
US6855804B2 (en) 1998-03-23 2005-02-15 Board Of Regents, The University Of Texas System Covalently reactive transition state analogs and methods of use thereof
AU761430B2 (en) 1998-04-02 2003-06-05 Xintela Ab An integrin heterodimer and a subunit thereof
EP2080802B1 (fr) 1998-06-01 2017-03-29 Agensys, Inc. Nouveaux antigènes transmembranaires à serpentin exprimés dans les cancers humains et utilisations associées
US7138103B2 (en) 1998-06-22 2006-11-21 Immunomedics, Inc. Use of bi-specific antibodies for pre-targeting diagnosis and therapy
US7223556B1 (en) 1998-10-09 2007-05-29 President And Fellows Of Harvard College Targeted proteolysis by recruitment to ubiquitin protein ligases
EP1231944A2 (fr) 1999-11-15 2002-08-21 University Of Southern California Liberation ciblee de fractions therapeutiques et de diagnostic
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
WO2001097829A2 (fr) 2000-06-19 2001-12-27 Genzyme Corporation Traitement de substitution enzymatique, therapie genique et therapie par petites molecules pour maladies liees au stockage lysosomal
DE10034607A1 (de) 2000-07-20 2002-02-07 Gundram Jung Multispezifisches Reagenz zur selektiven Stimulierung von Zelloberflächenrezeptoren
AU2001295041A1 (en) 2000-09-08 2002-03-22 California Institute Of Technology Proteolysis targeting chimeric pharmaceutical
US6649138B2 (en) 2000-10-13 2003-11-18 Quantum Dot Corporation Surface-modified semiconductive and metallic nanoparticles having enhanced dispersibility in aqueous media
US7105348B2 (en) 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US20070258987A1 (en) 2000-11-28 2007-11-08 Seattle Genetics, Inc. Recombinant Anti-Cd30 Antibodies and Uses Thereof
US7431923B2 (en) 2005-01-03 2008-10-07 Arius Research Inc. Cytotoxicity mediation of cells evidencing surface expression of CD63
US7442777B2 (en) 2000-11-29 2008-10-28 Arius Research Inc. Cytotoxicity mediation of cells evidencing surface expression of CD63
US20060210474A1 (en) 2000-11-29 2006-09-21 Young David S Cytotoxicity mediation of cells evidencing surface expression of CD63
US7534429B2 (en) 2000-11-29 2009-05-19 Hoffmann-La Roche Inc. Cytotoxicity mediation of cells evidencing surface expression of CD63
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
JP4381140B2 (ja) 2001-10-12 2009-12-09 シェーリング コーポレイション 免疫応答を調節するための二重特異性抗体の使用
EP1474109B1 (fr) 2001-12-21 2010-08-25 Alcon, Inc. Utilisation de nanoparticules synthetiques inorganiques comme vecteurs de medicaments ophtalmiques
JP2006506317A (ja) 2002-01-11 2006-02-23 バイオマリン ファーマシューティカル インコーポレイテッド 治療用リソソーム酵素の送達のための酵素送達系としてのp97の使用
US7332585B2 (en) 2002-04-05 2008-02-19 The Regents Of The California University Bispecific single chain Fv antibody molecules and methods of use thereof
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
WO2004023973A2 (fr) 2002-09-12 2004-03-25 Incyte Corporation Molecules utilisees a des fins diagnostiques et therapeutiques
US20050142141A1 (en) 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
EP1587923B1 (fr) 2003-01-22 2011-08-24 Duke University Constructions ameliorees permettant d'exprimer des polypeptides lysosomiaux
SE0301087D0 (sv) 2003-04-14 2003-04-14 Cartela Ab New monoclonal antibody
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
PL1620133T3 (pl) 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
EP1633865B1 (fr) 2003-06-18 2011-09-28 Bayer Pharma Aktiengesellschaft Nouvelles entités biologiques et leur utilisation
WO2005007695A2 (fr) 2003-07-14 2005-01-27 Beth Israel Deaconess Medical Center, Inc. Anticorps anti-cd63 antibodies et leurs methodes d'utilisation
WO2005035753A1 (fr) 2003-10-10 2005-04-21 Chugai Seiyaku Kabushiki Kaisha Anticorps a double specificite remplaçant une proteine fonctionnelle
US7371539B2 (en) 2003-12-03 2008-05-13 President And Fellows Of Harvard College Targeted polypeptide degradation
US7235641B2 (en) 2003-12-22 2007-06-26 Micromet Ag Bispecific antibodies
EP1720405A4 (fr) 2004-02-06 2008-08-27 Biomarin Pharm Inc Fabrication d'enzymes lysosomales hautement phosphorylees et utilisations associees
JP4914224B2 (ja) 2004-02-10 2012-04-11 バイオマリン ファーマシューティカル インコーポレイテッド 酸性αグルコシダーゼおよびそのフラグメント
WO2005077333A2 (fr) * 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Administration sous forme de gel de vecteurs de virus associes aux adenovirus recombines
EP1718667B1 (fr) 2004-02-23 2013-01-09 Genentech, Inc. Liants et conjugues heterocycliques auto-immolateurs
TW200539855A (en) 2004-03-15 2005-12-16 Wyeth Corp Calicheamicin conjugates
US20060171926A1 (en) 2004-04-30 2006-08-03 Passini Marco A Gene therapy for neurometabolic disorders
US7892809B2 (en) 2004-12-15 2011-02-22 The University Of North Carolina At Chapel Hill Chimeric vectors
US7341720B2 (en) 2005-04-06 2008-03-11 Genzyme Corporation Targeting of glycoprotein therapeutics
PT2420256T (pt) 2005-05-02 2016-09-13 Genzyme Corp Terapia genética para distúrbios neurometabólicos
CN102016814B (zh) 2005-06-17 2013-10-23 北卡罗来纳大学查珀尔希尔分校 纳米粒子制备方法、系统及材料
RS63964B1 (sr) 2005-08-23 2023-03-31 Univ Pennsylvania Rnk koja sadrži modifikovane nukleozide i postupci za njenu upotrebu
JP5090366B2 (ja) 2005-12-16 2012-12-05 アイビーシー ファーマスーティカルズ,インク. 多価イムノグロブリン系生物活性アセンブリー
EP1981546B1 (fr) 2006-02-06 2014-06-04 The Medical Research and Infrastructure Fund of the Tel-Aviv Sourasky Medical Center Enzymotherapie de remplacement pour le traitement des maladies lysosomiales
US7750116B1 (en) 2006-02-18 2010-07-06 Seattle Genetics, Inc. Antibody drug conjugate metabolites
RS52176B (sr) 2006-06-02 2012-08-31 Regeneron Pharmaceuticals Inc. Antitela visokog afiniteta prema humanom il-6 receptoru
US20080089891A1 (en) 2006-07-26 2008-04-17 Arius Research, Inc. Cancerous disease modifying antibodies
US9127293B2 (en) 2006-07-26 2015-09-08 The University Of Chicago Receptor-mediated delivery: compositions and methods
HRP20140081T1 (hr) 2006-08-18 2014-03-28 Novartis Ag Prlr-specifiäśna antitijela i njihova uporaba
EP2099523A2 (fr) 2006-11-13 2009-09-16 ZyStor Therapeutics , Inc. Méthodes de traitement de la maladie de pompe
CA2680063A1 (fr) 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions et procedes pour le traitement de maladies liees au stockage du glycogene
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
WO2008122039A2 (fr) 2007-04-02 2008-10-09 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Molécules d'anticorps hybrides médiées par la sélénocystéine
JP5507242B2 (ja) 2007-05-18 2014-05-28 公益財団法人東京都医学総合研究所 酵素補充療法用医薬組成物
JP2010528285A (ja) 2007-05-23 2010-08-19 ベンタナ・メデイカル・システムズ・インコーポレーテツド 免疫組織化学およびinsituハイブリダーゼーションのためのポリマー担体
ES2522615T3 (es) 2007-06-04 2014-11-17 Regeneron Pharmaceuticals, Inc. Regiones de expresión y estabilidad potenciadas
WO2009018386A1 (fr) 2007-07-31 2009-02-05 Medimmune, Llc Protéines de liaison à épitope multispécifiques et leurs utilisations
WO2009075815A1 (fr) 2007-12-07 2009-06-18 Duke University Thérapie génique d'immunomodulation
EP2238162A1 (fr) 2008-01-24 2010-10-13 The Government of the United States of America as represented by The Secretary of the Department of Health and Human Services Internalisation induite de récepteurs de surface
DK2282764T3 (da) * 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
CN102083461B (zh) 2008-04-30 2014-09-17 伊缪诺金公司 有效的偶联物和亲水性连接体
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
ES2536882T3 (es) 2008-07-21 2015-05-29 Polytherics Limited Nuevos reactivos y procedimiento de conjugación de moléculas biológicas
BRPI0919382A2 (pt) 2008-09-26 2016-01-05 Roche Glycart Ag anticorpos bi-específicos anti-egfr/anti-igf-1r
MX2011010166A (es) 2009-04-07 2011-10-11 Roche Glycart Ag Anticuerpos biespecificos anti-erbb-3/anti-c-met.
EP2241576A1 (fr) 2009-04-17 2010-10-20 Trion Pharma Gmbh Utilisation des anticorps bispécifiques trifonctionnels pour le traitement des tumeurs associées aux cellules souches de cancer CD133+/EpCAM+
ES2758827T3 (es) 2009-06-17 2020-05-06 Biomarin Pharm Inc Formulaciones para enzimas lisosómicas
KR101747103B1 (ko) 2009-06-26 2017-06-14 리제너론 파마슈티칼스 인코포레이티드 천연 면역글로불린 포맷을 가지는 용이하게 분리된 이중특이성 항체
BR112012002291A2 (pt) 2009-07-31 2016-11-29 Ethris Gmbh "polirribonucleotídeo com uma sequência que codifica uma proteína ou fragmento de proteína, implante, e, processo para a seleção de sequências de nucleotídeos"
EP2464654B1 (fr) 2009-08-10 2014-10-08 UCL Business PLC Groupement protecteur thiol
WO2011029823A1 (fr) 2009-09-09 2011-03-17 Novartis Ag Anticorps monoclonal réactif avec cd63 lors de son expression à la surface de mastocytes dégranulés
SG181952A1 (en) * 2009-12-29 2012-07-30 Emergent Product Dev Seattle Heterodimer binding proteins and uses thereof
US10143186B2 (en) 2010-02-08 2018-12-04 Regeneron Pharmaceuticals, Inc. Common light chain mouse
WO2011130598A1 (fr) 2010-04-15 2011-10-20 Spirogen Limited Pyrrolobenzodiazépines et conjugués de celles-ci
WO2011140279A1 (fr) * 2010-05-04 2011-11-10 Wayne State University Ciblage sous-cellulaire faisant intervenir aav de rhodopsines hétérologues dans des cellules ganglionnaires rétiniennes
US8809282B2 (en) 2010-05-06 2014-08-19 Duke University Method of reducing titers of antibodies specific for a therapeutic agent
CA3051311A1 (fr) 2010-05-27 2011-12-01 Genmab A/S Anticorps monoclonaux contre her2
WO2012005982A2 (fr) 2010-07-06 2012-01-12 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Rapporteur pour une terminaison par la arn polymérase ii
BR112013007616B1 (pt) 2010-09-29 2023-01-24 Oxyrane Uk Limited Métodos para retirar o cap ("uncapping") de porções de manose-1- fosfo-6-manose e desmanosilar n-glicanos fosforilados em uma glicoproteína, para direcionar uma glicoproteína para o interior de uma célula de mamífero, e para converter uma glicoproteína de uma primeira forma que não se liga substancialmente a um receptor de manose-6-fosfato em uma segunda forma que se liga substancialmente a um receptor de manose-6-fosfato em uma célula de mamífero
US8679478B2 (en) 2010-10-04 2014-03-25 Duke University Methods of lysosomal storage disease therapy
WO2012051220A1 (fr) 2010-10-11 2012-04-19 Wichita State University Système de délivrance de médicaments de nanoparticules magnétiques composites
EP3461905B1 (fr) 2010-11-22 2020-08-05 Amicus Therapeutics, Inc. Nouvelles séquences de signaux pour améliorer l'expression des protéines et la sécrétion d'enzymes recombinantes et d'autres protéines
ME03732B (fr) 2011-02-25 2021-01-20 Regeneron Pharma Souris à ADAM6
US9493753B2 (en) 2011-03-16 2016-11-15 Amano Enzyme Inc. Modified α-glucosidase and applications of same
WO2012125987A2 (fr) 2011-03-17 2012-09-20 Massachusetts Institute Of Technology Système d'administration
CA2832151C (fr) 2011-03-31 2021-06-15 University Of Iowa Research Foundation Procedes et compositions pour traiter des maladies cerebrales
KR20140031217A (ko) 2011-04-20 2014-03-12 로슈 글리카트 아게 혈액-뇌 차단벽의 pH 의존적 통과를 위한 방법 및 구축물
EP2699676A1 (fr) 2011-04-22 2014-02-26 Genzyme Corporation Alpha-glucosidase acide modifiée à traitement accéléré
US20120283503A1 (en) 2011-04-29 2012-11-08 The Johns Hopkins University Nanoparticle loaded stem cells and their use in mri guided hyperthermia
RS56913B1 (sr) 2011-05-27 2018-05-31 Amicus Therapeutics Inc Postupci za kuplovanje peptida za ciljanje na rekombinantne lizosomalne encime za poboljšanje tretmana lizosomalnih bolesti taloženja
EP2714685B1 (fr) 2011-05-27 2016-10-19 Ambrx, Inc. Compositions contenant des dérivés de dolastatine liés à des acides aminés non naturel
US8815226B2 (en) 2011-06-10 2014-08-26 Mersana Therapeutics, Inc. Protein-polymer-drug conjugates
BR112014008888A2 (pt) 2011-10-14 2017-04-18 Seattle Genetics Inc pirrolobenzodiazepinas
MX358757B (es) 2011-10-14 2018-09-03 Seattle Genetics Inc Pirrolobenzodiazepinas y conjugados dirigidos.
EP2751076B1 (fr) 2011-10-14 2018-04-25 MedImmune Limited Procédé de synthèse et intermédiaires pouvant servir à préparer des pyrrolobenzodiazépines
US9526798B2 (en) 2011-10-14 2016-12-27 Seattle Genetics, Inc. Pyrrolobenzodiazepines and targeted conjugates
WO2013068874A1 (fr) 2011-11-11 2013-05-16 Pfizer Inc. Conjugués anticorps-médicament
MX2014006739A (es) 2011-12-05 2015-06-05 Igenica Biotherapeutics Inc Conjugados de anticuerpo-farmaco y compuestos relacionados, composiciones , y metodos.
ME03477B (fr) 2011-12-20 2020-01-20 Regeneron Pharma Souris à chaînes légères humanisées
MY171761A (en) 2012-03-14 2019-10-28 Regeneron Pharma Multispecific antigen-binding molecules and uses thereof
HUE051632T2 (hu) 2012-06-19 2021-03-29 Univ Florida Betegségek kezelésére szolgáló készítmények és eljárások
HUE051612T2 (hu) 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
KR20190121874A (ko) 2012-08-29 2019-10-28 에프. 호프만-라 로슈 아게 혈액 뇌 장벽 셔틀
SI2911699T2 (sl) 2012-10-23 2026-01-30 Synaffix B.V. Modificirano protitelo, protitelo-konjugat in proces za njihovo pripravo
DK3115372T3 (da) 2012-11-27 2019-06-11 Biomarin Pharm Inc Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf
TWI635098B (zh) 2013-02-01 2018-09-11 再生元醫藥公司 含嵌合恆定區之抗體
US10017581B2 (en) * 2013-02-20 2018-07-10 Valerion Therapeutics, Llc Methods and compositions for treatment of Pompe disease
EP2958936A4 (fr) 2013-02-20 2016-11-09 Valerion Therapeutics Llc Méthodes et compositions pour le traitement de la maladie de forbes-cori
AU2014233385C1 (en) 2013-03-15 2019-09-19 Regeneron Pharmaceuticals, Inc. Biologically active molecules, conjugates thereof, and therapeutic uses
JP2016520586A (ja) 2013-05-08 2016-07-14 ザイムワークス,インコーポレイテッド 二重特異性her2およびher3抗原結合性構築物
US9738717B2 (en) 2013-05-15 2017-08-22 The Translational Genomics Research Institute Hybridoma clones, monoclonal antibodies, and methods of use
CA2912678C (fr) 2013-05-15 2023-10-10 Regents Of The University Of Minnesota Transfert genique au systeme nerveux central a mediation par un virus adeno-associe
SG11201509566RA (en) 2013-05-20 2015-12-30 Genentech Inc Anti-transferrin receptor antibodies and methods of use
CN104211814A (zh) 2013-05-29 2014-12-17 三星电子株式会社 用于消耗靶膜蛋白的组合物
BR112016001592A2 (pt) 2013-07-26 2017-11-28 Univ Iowa Res Found métodos e composições para o tratamento de doenças cerebrais
TWI641620B (zh) 2013-08-21 2018-11-21 再生元醫藥公司 抗-prlr抗體及其用途
KR102252925B1 (ko) 2013-08-26 2021-05-18 리제너론 파마슈티칼스 인코포레이티드 마크롤라이드 디아스테레오머를 포함하는 약제학적 조성물, 이의 합성 방법 및 치료학적 용도
WO2015110449A1 (fr) 2014-01-21 2015-07-30 Vrije Universiteit Brussel Éléments régulateurs d'acide nucléique exprimé dans un muscle, méthodes et utilisation associées
US10815469B2 (en) * 2014-06-11 2020-10-27 Molecular Templates, Inc. Cell-targeting molecules comprising protease-cleavage resistant, Shiga toxin A subunit effector polypeptides and carboxy-terminal moieties
WO2016044947A1 (fr) 2014-09-26 2016-03-31 Exerkine Corporation Exosomes utiles pour traiter une maladie de stockage lysosomal
EP3220958A1 (fr) 2014-10-24 2017-09-27 Shire Human Genetic Therapies, Inc. Ciblage lysosomal d'enzymes et utilisations associées
EP3218411B1 (fr) 2014-11-14 2022-01-12 Ossianix, Inc. Récepteurs d'antigène nouveaux variables (vnars) dirigé contre récepteur de transferrine et leur utilisation
MA41022A (fr) 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
KR20240142591A (ko) 2015-03-27 2024-09-30 리제너론 파아마슈티컬스, 인크. 메이탄시노이드 유도체, 이의 컨주게이트, 및 사용 방법
TWI726879B (zh) 2015-05-04 2021-05-11 美商Cytomx生物製藥公司 抗cd71抗體類、可活化之抗cd71抗體類及使用彼等之方法
TWI769982B (zh) 2015-06-24 2022-07-11 日商Jcr製藥股份有限公司 通過血腦障壁之抗人類運鐵蛋白受體抗體
US11191844B2 (en) 2015-07-06 2021-12-07 Regeneran Pharmaceuticals, Inc. Multispecific antigen-binding molecules and uses thereof
US10292961B2 (en) * 2015-07-15 2019-05-21 Hangzhou Dac Biotech Co., Ltd. Disulfur bridge linkers for conjugation of a cell-binding molecule
CN108601771B (zh) 2015-10-23 2023-02-21 衣阿华大学研究基金会 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法
IL287019B2 (en) 2015-12-08 2025-10-01 Regeneron Pharma Preparations and methods for internalizing enzymes
CA3010205A1 (fr) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Alpha-glucosidase acide amelioree pour le traitement de la maladie de pompe
KR102885186B1 (ko) 2016-01-25 2025-11-17 리제너론 파아마슈티컬스, 인크. 메이탄시노이드 유도체, 그의 접합체, 및 사용 방법
AR107483A1 (es) 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
MA46419A (fr) 2016-02-05 2018-12-12 Genmab As Molécule de liaison aux antigènes multispécifique présentant de meilleures caractéristiques d'internalisation
EP4275751A3 (fr) 2016-02-24 2023-12-27 BioMarin Pharmaceutical Inc. Formulation de la protéine de fusion naglu améliorée
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
EP3448891A1 (fr) 2016-04-28 2019-03-06 Regeneron Pharmaceuticals, Inc. Procédés de production de molécules multispécifiques se liant à l'antigène
US11078247B2 (en) 2016-05-04 2021-08-03 Curevac Ag RNA encoding a therapeutic protein
WO2018031424A1 (fr) 2016-08-06 2018-02-15 Ossianix, Inc. Méthodes in vivo pour sélectionner des peptides qui traversent la barrière hémato-encéphalique, compositions apparentées et procédés d'utilisation
EP3293259A1 (fr) 2016-09-12 2018-03-14 Genethon Variants d'alpha-glucosidase acide et leurs utilisations
IL299325B2 (en) 2016-09-12 2025-08-01 Inst Nat Sante Rech Med Acid-alpha glucosidase variants and uses thereof
EP3293203A1 (fr) 2016-09-12 2018-03-14 Genethon Variants d'alpha-glucosidase acide et leurs utilisations
CA3041548A1 (fr) 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Compositions de transfert de gene, methodes et utilisations pour le traitement de maladies neurodegeneratives
CN109983031B (zh) 2016-12-26 2022-11-25 Jcr制药股份有限公司 通过血脑屏障的新的抗人转铁蛋白受体抗体
CN110234329A (zh) 2017-01-27 2019-09-13 辛特拉股份公司 骨骼和软骨损害或疾病的预防和治疗
PT3624831T (pt) 2017-05-15 2023-07-04 Amicus Therapeutics Inc Alfa-glucosidase ácida humana recombinante
EP3635009B1 (fr) * 2017-06-07 2026-02-25 Regeneron Pharmaceuticals, Inc. Compositions et méthodes pour l'internalisation d'enzymes
BR112020005436B1 (pt) 2017-09-20 2022-08-02 4D Molecular Therapeutics Inc Proteína do capsídeo de variante do vírus adenoassociado, virion do aav recombinante (raav) infeccioso, composições, composições farmacêuticas e usos de virion de raav ou de composições farmacêuticas
HUE054037T2 (hu) 2017-10-14 2021-08-30 Abbvie Inc CD71 elleni, aktiválható antitest hatóanyag konjugátumok és eljárások alkalmazásukra
EP3749756A4 (fr) 2018-02-05 2021-12-08 Audentes Therapeutics, Inc. Éléments de régulation de la transcription et utilisations associées
US20210038739A1 (en) 2018-02-05 2021-02-11 Jcr Pharmaceuticals Co., Ltd. Method for Delivering Drug to Muscle
IL276464B2 (en) 2018-02-07 2026-01-01 Regeneron Pharma Methods and compositions for administering therapeutic protein
US20210040464A1 (en) 2018-03-15 2021-02-11 Valerion Therapeutics, Llc Methods and compositions for treatment of polyglucosan disorders
EP3552631A1 (fr) 2018-04-10 2019-10-16 Inatherys Conjugués d'anticorps-médicament et leurs utilisations pour le traitement du cancer
CA3100213A1 (fr) 2018-05-16 2019-11-21 Spark Therapeutics, Inc. Cassettes d'expression d'alpha-glucosidase acide optimisees par des codons et leurs methodes d'utilisation
MA52626A (fr) 2018-05-17 2021-03-24 Regeneron Pharma Anticorps anti-cd63, conjugués et leurs utilisations
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
CN118662653A (zh) 2018-08-02 2024-09-20 达因疗法公司 肌肉靶向复合物及其用于治疗蓬佩病的用途
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
WO2020102645A1 (fr) 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Virus adéno-associé thérapeutique pour le traitement de la maladie de pompe
MX2021006646A (es) 2018-12-05 2021-12-10 Abeona Therapeutics Inc Vector viral adenoasociado recombinante para el suministro de genes.
WO2020163480A1 (fr) 2019-02-05 2020-08-13 Amicus Therapeutics, Inc. Alpha-glucosidase acide humaine recombinée et utilisations associées
KR20220004696A (ko) 2019-04-30 2022-01-11 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 폼페병의 치료에 유용한 조성물
WO2021005176A1 (fr) 2019-07-09 2021-01-14 Genethon Traitement de la glycogénose (gsd)
WO2023133579A1 (fr) 2022-01-10 2023-07-13 Regeneron Pharmaceuticals, Inc. Gaa ciblant la bhe administré en tant que thérapie génique pour traiter le snc et les muscles de souris modèles atteintes de la maladie de pompe
JP2025508677A (ja) 2022-02-02 2025-04-10 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 ポンペ病の処置のための抗TfR:GAA及び抗CD63:GAAの挿入

Also Published As

Publication number Publication date
JP2022121630A (ja) 2022-08-19
AU2018281280A1 (en) 2020-01-16
WO2018226861A4 (fr) 2019-01-24
JP2020523321A (ja) 2020-08-06
IL271193B1 (en) 2024-09-01
US11208458B2 (en) 2021-12-28
CA3066569A1 (fr) 2018-12-13
AU2018281280B2 (en) 2024-12-19
TW202405021A (zh) 2024-02-01
TWI880364B (zh) 2025-04-11
IL271193B2 (en) 2025-01-01
WO2018226861A1 (fr) 2018-12-13
KR20200015932A (ko) 2020-02-13
IL271193A (en) 2020-01-30
MY198804A (en) 2023-09-28
AU2025201922B2 (en) 2026-03-05
US20180355017A1 (en) 2018-12-13
US20220195011A1 (en) 2022-06-23
JP7348844B2 (ja) 2023-09-21
KR102760654B1 (ko) 2025-02-04
BR112019025888A2 (pt) 2020-06-30
TWI820027B (zh) 2023-11-01
CL2019003586A1 (es) 2020-06-05
PH12019550261A1 (en) 2020-07-13
EP3635009B1 (fr) 2026-02-25
NZ760232A (en) 2023-05-26
JP7538183B2 (ja) 2024-08-21
CN110809583A (zh) 2020-02-18
CO2019014682A2 (es) 2020-01-17
RU2019143567A3 (fr) 2022-02-15
AU2025201922A1 (en) 2025-04-10
RU2019143567A (ru) 2021-07-09
US12215138B2 (en) 2025-02-04
TW201920678A (zh) 2019-06-01
EP3635009A1 (fr) 2020-04-15
US20250171520A1 (en) 2025-05-29

Similar Documents

Publication Publication Date Title
MA50546A (fr) Compositions et méthodes pour l'internalisation d'enzymes
EP3679141C0 (fr) Méthodes et compositions pour inhiber l'expression de la ldha
EP3510152A4 (fr) Méthodes et compositions pour moduler l'expression génique
EP3897747A4 (fr) Molécules bi-fonctionnelles pour le ciblage des lysosomes, compositions et méthodes associées
MA46643A (fr) Méthodes et compositions pour le mappage d'arn
EP3707257A4 (fr) Méthodes et compositions pour des molécules d'arn circulaire
EP3781705A4 (fr) Compositions et méthodes pour l'édition génique
MA71468A (fr) Compositions et méthodes pour diminuer l'expression de tau
EP3402549A4 (fr) Compositions pour micro-aiguilles et méthodes d'utilisation de celles-ci
EP3445407A4 (fr) Compositions et méthodes d'immunothérapie cellulaire
EP4034253A4 (fr) Compositions et méthodes pour accroître l'efficacité d'immunothérapies et de vaccins
MA50833A (fr) Compositions et méthodes pour l'édition génique pour l'hémophilie a
EP3328363A4 (fr) Compositions et méthodes d'immunomodulation
EP3362104A4 (fr) Méthodes et compositions utilisant cpf1 pour l'édition génétique guidée par l'arn
EP3635100A4 (fr) Compositions et procédés pour exprimer l'otoferline
EP3271482A4 (fr) Compositions et méthodes pour inhiber l'expression génique du facteur xii
MA49693A (fr) Méthodes et compositions pour assurer l'immunisation contre l'arnrep hétérologue
MA43378A (fr) Compositions et méthodes pour la reduction de la réponse immunitaire contre récepteurs d'antigène chimériques
EP3302588A4 (fr) Compositions et méthodes pour l'adhérence à des surfaces
EP3713569A4 (fr) Compositions et méthodes d'administration d'une composition inhibitrice d'yap1/wwrt1 et d'une composition inhibitrice de gls1
EP3405191A4 (fr) Méthodes et compositions pour le traitement de l'hyperhidrose
EP3458079A4 (fr) Compositions et méthodes pour le traitement de l'acné
MA49576A (fr) Compositions et méthodes pour le traitement d'infections fongiques
EP3302503A4 (fr) Compositions et méthodes permettant d'inhiber l'expression du gène de hif2alpha
EP3630846A4 (fr) Compositions et méthodes d'immunothérapie cellulaire