CN115968302A - 用于治疗基因疗法患者的组合物和方法 - Google Patents

用于治疗基因疗法患者的组合物和方法 Download PDF

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Publication number
CN115968302A
CN115968302A CN202180050281.XA CN202180050281A CN115968302A CN 115968302 A CN115968302 A CN 115968302A CN 202180050281 A CN202180050281 A CN 202180050281A CN 115968302 A CN115968302 A CN 115968302A
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seq
fcrn
ligand
aav
vector
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Chinese (zh)
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C·欣德尔
堀内真
J·M·威尔逊
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/21—Interferons [IFN]
    • A61K38/217—IFN-gamma
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/70535—Fc-receptors, e.g. CD16, CD32, CD64 (CD2314/705F)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55—Fab or Fab'
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
    • C07K2317/565—Complementarity determining region [CDR]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Dermatology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
CN202180050281.XA 2020-06-17 2021-06-16 用于治疗基因疗法患者的组合物和方法 Pending CN115968302A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063040381P 2020-06-17 2020-06-17
US63/040381 2020-06-17
US202163135998P 2021-01-11 2021-01-11
US63/135998 2021-01-11
US202163152085P 2021-02-22 2021-02-22
US63/152085 2021-02-22
PCT/US2021/037575 WO2021257668A1 (en) 2020-06-17 2021-06-16 Compositions and methods for treatment of gene therapy patients

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CN115968302A true CN115968302A (zh) 2023-04-14

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US (1) US20230220069A1 (https=)
EP (1) EP4171738A1 (https=)
JP (1) JP2023531451A (https=)
KR (1) KR20230025000A (https=)
CN (1) CN115968302A (https=)
AU (1) AU2021292200A1 (https=)
CA (1) CA3183153A1 (https=)
IL (1) IL299167A (https=)
MX (1) MX2022016528A (https=)
TW (1) TW202214695A (https=)
WO (1) WO2021257668A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117126270A (zh) * 2023-10-25 2023-11-28 首都儿科研究所 一种2型人博卡病毒型别特异性抗体及其应用
CN120272478A (zh) * 2025-03-28 2025-07-08 山西医科大学 一种抗寨卡病毒的靶点抑制剂、重组基因、表达载体及应用

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113518783A (zh) 2018-07-20 2021-10-19 动量制药公司 FcRn抗体组合物
EP4007605A4 (en) * 2019-08-01 2023-08-16 Momenta Pharmaceuticals, Inc. ANTI-FCRN ANTIBODIES AND METHODS OF USE THEREOF
IL325936A (en) * 2020-01-22 2026-03-01 Spark Therapeutics Inc Compositions and methods for enhancing or improving transduction of gene therapy vectors and removing or reducing immunoglobulins
CA3216004A1 (en) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
EP4409010A1 (en) 2021-10-02 2024-08-07 The Trustees of The University of Pennsylvania Novel aav capsids and compositions containing same
EP4504797A1 (en) 2022-04-06 2025-02-12 The Trustees of The University of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating spinal muscular atrophy
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
EP4699615A1 (en) * 2024-08-22 2026-02-25 Universität Zürich Fcrn inhibitor and therapeutic polypeptide

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000020041A2 (en) * 1998-10-07 2000-04-13 Calydon, Inc. Removal of preexisting antibodies to enhance the effectiveness of a therapeutic viral immunogenic agent
WO2007098420A2 (en) * 2006-02-17 2007-08-30 Syntonix Pharmaceuticals, Inc. Peptides that block the binding of igg to fcrn
WO2016142782A1 (en) * 2015-03-09 2016-09-15 Argen-X N.V. Methods of reducing serum levels of fc-containing agents using fcrn antagonsits
CN113769058A (zh) * 2020-06-10 2021-12-10 上海宝济药业有限公司 一种药物组合及其应用

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5786464C1 (en) 1994-09-19 2012-04-24 Gen Hospital Corp Overexpression of mammalian and viral proteins
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
CA2606378A1 (en) * 2005-04-29 2006-11-09 The Jackson Laboratory Fcrn antibodies and uses thereof
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
US20070027438A1 (en) 2005-07-26 2007-02-01 Frieder Loesel System and method for compensating a corneal dissection
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20070292922A1 (en) 2006-03-31 2007-12-20 Cell Genesys, Inc. Regulated expression of recombinant proteins from adeno-associated viral vectors
WO2015081096A2 (en) 2013-11-26 2015-06-04 The Brigham And Women's Hospital, Inc. Receptor-targeted nanoparticles for enhanced transcytosis mediated drug delivery
CA2729961C (en) 2008-07-09 2018-05-01 Biogen Idec Ma Inc. Li113, li62 variant co2, anti-lingo antibodies
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
AU2012262007B2 (en) 2011-06-02 2017-06-22 Takeda Pharmaceutical Company Limited Fc receptor binding proteins
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
US20150126589A1 (en) 2012-06-08 2015-05-07 Ethris Gmbh Pulmonary Delivery of Messenger RNA
AU2014235096B2 (en) 2013-03-15 2020-03-19 The Trustees Of The University Of Pennsylvania Compositions and methods for treating MPSI
US9527890B2 (en) 2013-06-18 2016-12-27 The Brigham And Womens's Hospital, Inc. FC receptor (FcRn) binding peptides and uses thereof
PL3253453T3 (pl) 2015-02-04 2020-06-01 Raichle, Marianne Sposób wytwarzania mleka o wysokiej zawartości natywnej witaminy d
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
US20190002915A1 (en) 2015-12-14 2019-01-03 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
KR102904658B1 (ko) * 2017-12-13 2025-12-29 모멘타 파머슈티컬스 인코포레이티드 FcRn 항체 및 이의 사용 방법
IL276859B2 (en) 2018-02-27 2025-12-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
EP3768695A4 (en) 2018-02-27 2022-04-06 The Trustees of the University of Pennsylvania NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
WO2020132455A1 (en) 2018-12-21 2020-06-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
SG11202111375RA (en) 2019-04-29 2021-11-29 Univ Pennsylvania Novel aav capsids and compositions containing same
JP7585233B2 (ja) 2019-04-30 2024-11-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ポンペ病の治療のために有用な組成物

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000020041A2 (en) * 1998-10-07 2000-04-13 Calydon, Inc. Removal of preexisting antibodies to enhance the effectiveness of a therapeutic viral immunogenic agent
WO2007098420A2 (en) * 2006-02-17 2007-08-30 Syntonix Pharmaceuticals, Inc. Peptides that block the binding of igg to fcrn
WO2016142782A1 (en) * 2015-03-09 2016-09-15 Argen-X N.V. Methods of reducing serum levels of fc-containing agents using fcrn antagonsits
CN113769058A (zh) * 2020-06-10 2021-12-10 上海宝济药业有限公司 一种药物组合及其应用

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
LEONA E LING等: "M281, an Anti-FcRn Antibody: Pharmacodynamics, Pharmacokinetics, and Safety Across the Full Range of IgG Reduction in a First-in-Human Study", CLIN PHARMACOL THER, vol. 105, no. 4, 30 April 2019 (2019-04-30) *
MOTLEY FOOL TRANSCRIBERS: "Momenta Pharmaceuticals Inc(MNTA) Q1 2019 Earnings Call Transcript", pages 3, Retrieved from the Internet <URL:https://www.fool.com/earnings/call-transcripts/2019/05/02/momenta-pharmaceuticals-inc-mnta-q1-2019-earnings.aspx> *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117126270A (zh) * 2023-10-25 2023-11-28 首都儿科研究所 一种2型人博卡病毒型别特异性抗体及其应用
CN117126270B (zh) * 2023-10-25 2024-02-13 首都儿科研究所 一种2型人博卡病毒型别特异性抗体及其应用
CN120272478A (zh) * 2025-03-28 2025-07-08 山西医科大学 一种抗寨卡病毒的靶点抑制剂、重组基因、表达载体及应用

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TW202214695A (zh) 2022-04-16
AU2021292200A1 (en) 2023-02-02
KR20230025000A (ko) 2023-02-21
WO2021257668A1 (en) 2021-12-23
JP2023531451A (ja) 2023-07-24
IL299167A (en) 2023-02-01
US20230220069A1 (en) 2023-07-13
MX2022016528A (es) 2023-06-02
EP4171738A1 (en) 2023-05-03

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