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

Info

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
Authority
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
Other languages
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 Penn
Original Assignee
University of Pennsylvania Penn
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 University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Publication of CA3008280A1 publication Critical patent/CA3008280A1/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Marine Sciences & Fisheries (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Psychology (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 (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102621134B1 (ko) * 2016-07-26 2024-01-04 코넬 유니버시티 알데히드 데히드로게나제 결핍의 치료를 위한 유전자 요법
MX2019010275A (es) 2017-02-28 2019-10-15 Univ Pennsylvania Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este.
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CA3073937A1 (en) * 2017-08-25 2019-02-28 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
WO2020037161A1 (en) * 2018-08-15 2020-02-20 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
US20220042045A1 (en) * 2018-12-21 2022-02-10 Genethon Expression cassettes for gene therapy vectors
WO2021030766A1 (en) * 2019-08-15 2021-02-18 Biogen Ma Inc. Combination therapy for spinal muscular 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
WO2021219762A1 (en) * 2020-04-28 2021-11-04 Genethon Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders
CA3177407A1 (en) 2020-05-12 2021-11-18 James M. Wilson 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
BR112023015177A2 (pt) * 2021-01-29 2023-11-14 Biocad Joint Stock Co Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
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
CA3237804A1 (en) * 2021-11-09 2023-05-19 Asimov Inc. Stable production systems for aav 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
HUE054805T2 (hu) * 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
JP6417322B2 (ja) 2012-06-21 2018-11-07 アソシアシオン・アンスティテュ・ドゥ・ミオロジーＡｓｓｏｃｉａｔｉｏｎ Ｉｎｓｔｉｔｕｔ Ｄｅ Ｍｙｏｌｏｇｉｅ 遺伝子治療ベクターの広範な遺伝子送達
BR112015027336A2 (pt) * 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
CA2928040A1 (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
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
EP3394270A1 (en) 2018-10-31
AU2016370630B2 (en) 2023-04-13
ZA201803956B (en) 2019-04-24
MX2018007234A (es) 2018-11-09
WO2017106354A1 (en) 2017-06-22
JP7082050B2 (ja) 2022-06-07
US20180353624A1 (en) 2018-12-13
MA44119A (fr) 2018-10-31
JP2018537984A (ja) 2018-12-27
BR112018011975A2 (pt) 2018-12-11
KR20180086266A (ko) 2018-07-30
IL259877A (en) 2018-07-31
AU2016370630A1 (en) 2018-06-28
US20220265861A1 (en) 2022-08-25
CN109072254A (zh) 2018-12-21

Similar Documents

Publication Publication Date Title
US20220265861A1 (en) Adeno-associated viral vectors useful in treatment of spinal muscular atropy
US11732246B2 (en) Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
JP2025120285A (ja) 繊毛病のための遺伝子治療
JP2024111074A (ja) 脊髄性筋萎縮症のための併用療法
JP2025138648A (ja) Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達
US20230167455A1 (en) Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
US20220370638A1 (en) Compositions and methods for treatment of maple syrup urine disease
US20240269328A1 (en) Recombinant adeno-associated viruses for lesch-nyhan disorders and uses thereof
JP7862321B2 (ja) Ｃｄｋｌ５欠損症（ｃｄｄ）の治療において有用な組成物
KR20260066056A (ko) 척수성 근위축증 치료를 위한 조성물 및 방법
CN119143858A (zh) 一种kcc2截短体或其编码序列及其应用
EP4711461A1 (en) Viral vector carrying smn gene expression cassette and use thereof
CN121335978A (zh) 一种人源MeCp2启动子及其用途
WO2025039622A1 (zh) 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
WO2025035143A1 (en) Compositions and methods for treatment of spinal muscular atrophy
CN119144657A (zh) 用于治疗脊髓损伤的腺相关病毒载体及其用途
CN116670159A (zh) 组合物及其用于治疗安格尔曼综合征的用途
EP4323520A1 (en) Compositions useful for treating spinal and bulbar muscular atrophy (sbma)

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220302