CN109072254A - 用于治疗脊髓性肌萎缩的腺相关病毒载体 - Google Patents

用于治疗脊髓性肌萎缩的腺相关病毒载体 Download PDF

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Publication number
CN109072254A
CN109072254A CN201680081819.2A CN201680081819A CN109072254A CN 109072254 A CN109072254 A CN 109072254A CN 201680081819 A CN201680081819 A CN 201680081819A CN 109072254 A CN109072254 A CN 109072254A
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aav
sequence
composition
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J.M.威尔逊
C.欣德勒
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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    • 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
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
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  • Marine Sciences & Fisheries (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN201680081819.2A 2015-12-14 2016-12-14 用于治疗脊髓性肌萎缩的腺相关病毒载体 Pending CN109072254A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267012P 2015-12-14 2015-12-14
US62/267012 2015-12-14
PCT/US2016/066669 WO2017106354A1 (en) 2015-12-14 2016-12-14 Adeno-associated viral vectors useful in treatment of spinal muscular atropy

Publications (1)

Publication Number Publication Date
CN109072254A true CN109072254A (zh) 2018-12-21

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CN201680081819.2A Pending CN109072254A (zh) 2015-12-14 2016-12-14 用于治疗脊髓性肌萎缩的腺相关病毒载体

Country Status (13)

Country Link
US (2) US20180353624A1 (enExample)
EP (1) EP3394270A1 (enExample)
JP (1) JP7082050B2 (enExample)
KR (1) KR20180086266A (enExample)
CN (1) CN109072254A (enExample)
AU (1) AU2016370630B2 (enExample)
BR (1) BR112018011975A2 (enExample)
CA (1) CA3008280A1 (enExample)
IL (1) IL259877A (enExample)
MA (1) MA44119A (enExample)
MX (1) MX2018007234A (enExample)
WO (1) WO2017106354A1 (enExample)
ZA (1) ZA201803956B (enExample)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112011571A (zh) * 2020-04-26 2020-12-01 辉大(上海)生物科技有限公司 一种用于治疗脊髓性肌萎缩的基因治疗药物
CN112852882A (zh) * 2021-02-04 2021-05-28 中吉智药(南京)生物技术有限公司 一种杆状病毒感染昆虫细胞生产aav基因药物的系统及方法
CN113755524A (zh) * 2020-06-02 2021-12-07 舒泰神（北京）生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途
CN115996759A (zh) * 2020-04-28 2023-04-21 吉尼松公司 合成aav衣壳用于肌肉和中枢神经系统障碍的基因治疗的用途

Families Citing this family (14)

* Cited by examiner, † Cited by third party
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US20190160187A1 (en) * 2016-07-26 2019-05-30 Cornell University Gene therapy for the treatment of aldehyde dehydrogenase deficiency
WO2018160582A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP2020533968A (ja) * 2017-08-25 2020-11-26 オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． 組換えアデノ随伴ベクター
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
WO2020127813A1 (en) * 2018-12-21 2020-06-25 Genethon Expression cassettes for gene therapy vectors
JP7689110B2 (ja) * 2019-08-15 2025-06-05 バイオジェン・エムエイ・インコーポレイテッド 脊髄性筋萎縮症のための併用療法
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
MX2024005636A (es) * 2021-11-09 2024-07-19 Asimov Inc Sistemas de producción estables para la producción de vectores del virus adenoasociado (aav).
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
KR102717399B1 (ko) * 2022-05-10 2024-10-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
WO2024165839A1 (en) * 2023-02-06 2024-08-15 Royal Holloway And Bedford New College Transgenes

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WO2013190059A1 (en) * 2012-06-21 2013-12-27 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
WO2014178863A1 (en) * 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy

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ES2874298T3 (es) * 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
WO2015061461A1 (en) * 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
PT3872085T (pt) * 2015-07-30 2023-04-13 Massachusetts Eye & Ear Infirmary Sequências de aav ancestrais e utilizações destas

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WO2013190059A1 (en) * 2012-06-21 2013-12-27 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
WO2014178863A1 (en) * 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy

Non-Patent Citations (5)

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CHUHONG HU等: ""RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy"", 《THE JOURNAL OF GENE MEDICINE》 *
ELISA DOMINGUEZ等: ""Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice"", 《HUMAN MOLECULAR GENETICS》 *
SANDRA I. DUQUE等: ""A large animal model of spinal muscular atrophy and correction of phenotype"", 《ANNALS OF NEUROLOG》 *
SOFIA BENKHELIFA-ZIYYAT等: ""Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice"", 《MOLECULAR THERAPY》 *
YANNICK TANGUY等: ""Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice"", 《FRONT.MOL.NEUROSCI》 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112011571A (zh) * 2020-04-26 2020-12-01 辉大(上海)生物科技有限公司 一种用于治疗脊髓性肌萎缩的基因治疗药物
CN115996759A (zh) * 2020-04-28 2023-04-21 吉尼松公司 合成aav衣壳用于肌肉和中枢神经系统障碍的基因治疗的用途
CN113755524A (zh) * 2020-06-02 2021-12-07 舒泰神（北京）生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途
CN113755524B (zh) * 2020-06-02 2023-11-03 舒泰神（北京）生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途
CN112852882A (zh) * 2021-02-04 2021-05-28 中吉智药(南京)生物技术有限公司 一种杆状病毒感染昆虫细胞生产aav基因药物的系统及方法

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BR112018011975A2 (pt) 2018-12-11
AU2016370630B2 (en) 2023-04-13
JP7082050B2 (ja) 2022-06-07
EP3394270A1 (en) 2018-10-31
MX2018007234A (es) 2018-11-09
AU2016370630A1 (en) 2018-06-28
MA44119A (fr) 2018-10-31
WO2017106354A1 (en) 2017-06-22
ZA201803956B (en) 2019-04-24
CA3008280A1 (en) 2017-06-22
US20220265861A1 (en) 2022-08-25
US20180353624A1 (en) 2018-12-13
IL259877A (en) 2018-07-31
KR20180086266A (ko) 2018-07-30
JP2018537984A (ja) 2018-12-27

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