CA3008280A1 - Adeno-associated viral vectors useful in treatment of spinal muscular atropy - Google Patents

Adeno-associated viral vectors useful in treatment of spinal muscular atropy Download PDF

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Publication number
CA3008280A1
CA3008280A1 CA3008280A CA3008280A CA3008280A1 CA 3008280 A1 CA3008280 A1 CA 3008280A1 CA 3008280 A CA3008280 A CA 3008280A CA 3008280 A CA3008280 A CA 3008280A CA 3008280 A1 CA3008280 A1 CA 3008280A1
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CA
Canada
Prior art keywords
vector
aav
sequence
composition
promoter
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3008280A
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English (en)
French (fr)
Inventor
James M. Wilson
Christain HINDERER
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Pennsylvania
Original Assignee
University of Pennsylvania
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Filing date
Publication date
Application filed by University of Pennsylvania filed Critical University of Pennsylvania
Publication of CA3008280A1 publication Critical patent/CA3008280A1/en
Pending legal-status Critical Current

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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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3008280A 2015-12-14 2016-12-14 Adeno-associated viral vectors useful in treatment of spinal muscular atropy Pending CA3008280A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267012P 2015-12-14 2015-12-14
US62/267,012 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
CA3008280A1 true CA3008280A1 (en) 2017-06-22

Family

ID=57737990

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3008280A Pending CA3008280A1 (en) 2015-12-14 2016-12-14 Adeno-associated viral vectors useful in treatment of spinal muscular atropy

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)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190160187A1 (en) * 2016-07-26 2019-05-30 Cornell University Gene therapy for the treatment of aldehyde dehydrogenase deficiency
RS65241B1 (sr) 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
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
JP2021534154A (ja) * 2018-08-15 2021-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. 脊髄性筋萎縮症のための併用療法
US20220042045A1 (en) * 2018-12-21 2022-02-10 Genethon Expression cassettes for gene therapy vectors
EP4013387A4 (en) * 2019-08-15 2023-09-27 Biogen MA Inc. COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY
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
US20230173102A1 (en) * 2020-04-28 2023-06-08 Genethon Use of a Synthetic AAV Capsid for Gene Therapy of Muscle and Central Nervous System Disorders
IL298138A (en) 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
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
WO2022119826A1 (en) * 2020-12-01 2022-06-09 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of peripheral myelin protein-22
CN117545842A (zh) * 2021-01-29 2024-02-09 拜奥卡德联合股份公司 SMN1和miR-23a在治疗脊髓性肌萎缩中的协同效应
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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
IL312487A (en) * 2021-11-09 2024-07-01 Asimov Inc Stable production systems for adeno-associated virus vector production
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

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1668143B1 (en) * 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
CN104704123A (zh) * 2012-06-21 2015-06-10 肌肉学研究协会 基因治疗载体的广泛的基因递送
BR112015027336A2 (pt) * 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
AU2014340149B2 (en) * 2013-10-22 2020-12-24 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
EP4134375A1 (en) * 2015-07-30 2023-02-15 Massachusetts Eye & Ear Infirmary Ancestral adeno associated virus sequences and uses thereof

Also Published As

Publication number Publication date
AU2016370630A1 (en) 2018-06-28
JP7082050B2 (ja) 2022-06-07
US20220265861A1 (en) 2022-08-25
MA44119A (fr) 2018-10-31
MX2018007234A (es) 2018-11-09
IL259877A (en) 2018-07-31
ZA201803956B (en) 2019-04-24
JP2018537984A (ja) 2018-12-27
AU2016370630B2 (en) 2023-04-13
EP3394270A1 (en) 2018-10-31
US20180353624A1 (en) 2018-12-13
KR20180086266A (ko) 2018-07-30
CN109072254A (zh) 2018-12-21
WO2017106354A1 (en) 2017-06-22
BR112018011975A2 (pt) 2018-12-11

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