MX2020005663A - Terapia genica para mucopolisacaridosis iii a. - Google Patents

Terapia genica para mucopolisacaridosis iii a.

Info

Publication number
MX2020005663A
MX2020005663A MX2020005663A MX2020005663A MX2020005663A MX 2020005663 A MX2020005663 A MX 2020005663A MX 2020005663 A MX2020005663 A MX 2020005663A MX 2020005663 A MX2020005663 A MX 2020005663A MX 2020005663 A MX2020005663 A MX 2020005663A
Authority
MX
Mexico
Prior art keywords
aav
raav
itr
gene therapy
hsgsh
Prior art date
Application number
MX2020005663A
Other languages
English (en)
Inventor
James M Wilson
Nathan Katz
Juliette Hordeaux
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of MX2020005663A publication Critical patent/MX2020005663A/es

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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K39/12—Viral antigens
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y310/00—Hydrolases acting on sulfur-nitrogen bonds (3.10)
    • C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
    • C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

En la presente se proporciona un AAV recombinante (rAAV) que comprende una cápside de AAV y un genoma de vector allí envuelto, donde el genoma de vector comprende una repetición terminal invertida (ITR, por su sigla en inglés) 5' de AAV, una secuencia de ácido nucleico modificada genéticamente que codifica un hSGSH funcional, una secuencia reguladora que dirige la expresión de hSGSH en una célula objetivo, y un 3' ITR de AAV; también se proporciona una composición farmacéutica que comprende un rAAV tal como se describe en la presente en un amortiguador de formulación, y un método para tratar un sujeto humano diagnosticado con MPS IIIA.
MX2020005663A 2017-11-30 2018-11-29 Terapia genica para mucopolisacaridosis iii a. MX2020005663A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762593081P 2017-11-30 2017-11-30
PCT/US2018/063168 WO2019108857A1 (en) 2017-11-30 2018-11-29 Gene therapy for mucopolysaccharidosis iiia

Publications (1)

Publication Number Publication Date
MX2020005663A true MX2020005663A (es) 2020-08-20

Family

ID=66665823

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020005663A MX2020005663A (es) 2017-11-30 2018-11-29 Terapia genica para mucopolisacaridosis iii a.

Country Status (10)

Country Link
US (2) US11555206B2 (es)
EP (1) EP3717653A4 (es)
JP (2) JP7389744B2 (es)
KR (1) KR20200104307A (es)
AU (1) AU2018375164A1 (es)
BR (1) BR112020010735A2 (es)
CA (1) CA3083416A1 (es)
IL (1) IL274939A (es)
MX (1) MX2020005663A (es)
WO (1) WO2019108857A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
MX2020005663A (es) * 2017-11-30 2020-08-20 Univ Pennsylvania Terapia genica para mucopolisacaridosis iii a.
WO2020127678A1 (en) * 2018-12-20 2020-06-25 Esteve Pharmaceuticals, S.A. Recombinant vectors for the long term treatment of mucchopolysacharidosis
CA3146192A1 (en) * 2019-07-18 2021-01-21 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
MX2022014258A (es) 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN115029360A (zh) * 2022-05-30 2022-09-09 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒

Family Cites Families (25)

* 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
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
SG10201912509RA (en) 2001-11-13 2020-02-27 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
CN102212558B (zh) 2003-09-30 2016-08-03 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
CN106906241B (zh) 2005-04-07 2020-09-11 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
JP6087504B2 (ja) 2008-11-07 2017-03-01 マサチューセッツ インスティテュート オブ テクノロジー アミノアルコールリピドイドおよびその使用
EP2553106A2 (en) 2010-03-29 2013-02-06 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (en) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
KR20200084048A (ko) 2011-06-08 2020-07-09 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
EP2492347A1 (en) 2012-05-22 2012-08-29 Laboratorios Del. Dr. Esteve, S.A. Methods for the production of vectors
KR20150020288A (ko) 2012-06-08 2015-02-25 에트리스 게엠베하 메신저 rna의 폐전달
WO2014125647A1 (ja) 2013-02-18 2014-08-21 富士通オプティカルコンポーネンツ株式会社 光受信装置
EP3044318B1 (en) 2013-09-13 2019-05-01 California Institute of Technology Selective recovery
WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
SG11201806270XA (en) 2016-02-03 2018-08-30 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
WO2017136533A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses
KR20190132639A (ko) 2017-02-28 2019-11-28 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아데노-연관 바이러스(aav) 클레이드 f 벡터 및 이의 용도
MX2020005663A (es) * 2017-11-30 2020-08-20 Univ Pennsylvania Terapia genica para mucopolisacaridosis iii a.

Also Published As

Publication number Publication date
WO2019108857A1 (en) 2019-06-06
BR112020010735A2 (pt) 2020-11-10
AU2018375164A1 (en) 2020-06-04
WO2019108857A8 (en) 2020-07-16
JP2023159274A (ja) 2023-10-31
US20200291429A1 (en) 2020-09-17
JP7389744B2 (ja) 2023-11-30
US20230279430A1 (en) 2023-09-07
US11555206B2 (en) 2023-01-17
IL274939A (en) 2020-07-30
KR20200104307A (ko) 2020-09-03
CA3083416A1 (en) 2019-06-06
JP2021508241A (ja) 2021-03-04
EP3717653A1 (en) 2020-10-07
EP3717653A4 (en) 2021-12-01

Similar Documents

Publication Publication Date Title
MX2020005663A (es) Terapia genica para mucopolisacaridosis iii a.
MX2020005673A (es) Terapia génica para mucopolisacaridosis iiib.
PH12021551341A1 (en) Compositions for drg-specific reduction of transgene expression
MX2019009720A (es) Metodos y composiciones para la transferencia de genes a traves de la vasculatura.
MX2017012097A (es) Variantes de virus adenoasociado y métodos de uso de estas.
PH12019550149A1 (en) Compositions useful in treatment of spinal muscular atrophy
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
PH12018501055A1 (en) Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
CL2016000502A1 (es) Vectores mejorados del factor viii del virus adeno-asociado de menos de 5,0 kb de longitud, que producen un polipéptido del factor viii funcionalmente activo para el tratamiento de la hemofilia a.
PE20160188A1 (es) Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos
MX2020010464A (es) Vectores de virus que evitan anticuerpos.
AR118850A1 (es) Composiciones útiles en el tratamiento de leucodistrofia metacromática
MX2021013364A (es) Composiciones utiles para el tratamiento de la enfermedad de pompe.
MX2022014258A (es) Composiciones para la reducción específica de un fármaco de la expresión de transgén.
MX2021009696A (es) Vectores de genoterapia para el tratamiento de la enfermedad de danon.
MX2022010388A (es) Polipéptidos de la cápside y vectores de virus adenoasociado.
SA518391787B1 (ar) تركيبة لعلاج متلازمة كريغلر نجار
PH12020550569A1 (en) A zika virus chimeric polyepitope comprising non-structural proteins and its use in an immunogenic composition
MX2020006476A (es) Vacuna contra el virus de lassa.
MX2022004345A (es) Construcciones de igf2 variantes.
MX2021010169A (es) Transactivadores de dominios de union a adn y usos de estos.
WO2023086928A3 (en) Gene therapy for treatment of mucopolysaccharidosis iiia
MX2022015048A (es) Viriones de virus adenoasociados con variantes de capsides y metodos de uso de estos.
MX2023006694A (es) Tratamiento de la enfermedad de danon.
MX2022013963A (es) Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas.