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AR125406A1 - Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen - Google Patents

Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen

Info

Publication number
AR125406A1
AR125406A1 ARP220101045A ARP220101045A AR125406A1 AR 125406 A1 AR125406 A1 AR 125406A1 AR P220101045 A ARP220101045 A AR P220101045A AR P220101045 A ARP220101045 A AR P220101045A AR 125406 A1 AR125406 A1 AR 125406A1
Authority
AR
Argentina
Prior art keywords
peptide
acid sequence
protein
gnpa
seq
Prior art date
Application number
ARP220101045A
Other languages
English (en)
Inventor
Joshua Joyner Sims
Yuan Yuan
James M Wilson
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 AR125406A1 publication Critical patent/AR125406A1/es

Links

Classifications

    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Se proporcionan en el presente documento composiciones que incluyen péptidos de unión a los capilares cerebrales y/o que atraviesan la barrera hematoencefálica (BBB), vinculados a estos o insertos en una proteína selectiva de un vector recombinante que tiene al menos un péptido exógeno que comprende una secuencia de aminoácidos de Y-G/A/R/K-Y/H-GNPA-T/R/HRYFD-V/K. Se proporcionan composiciones que proporcionan dichos conjugados, péptidos selectivos o vectores recombinantes que tienen una cápside mutante o proteína de envoltura, así como usos de estos. Reivindicación 1: Una partícula de virus adenoasociado recombinante (rAAV) caracterizada porque comprende: (a) una cápside de AAV que comprende proteína VP1, proteínas VP2 y proteínas VP3, donde la proteína VP3 tiene una secuencia de aminoácidos que comprende una región hipervariable que comprende un péptido de direccionamiento exógeno que tiene la secuencia: conector de N-terminal opcional- Y-X’-X’’-GNPA-X’’’-RYFD-X’’’’, donde X’ es G, A, R, K, X’’ es Y o H, X’’’ es T, R o H, y X’’’’ es V o K (SEQ ID Nº 14) - conector de C-terminal opcional; y (b) un genoma vectorial empaquetado en la cápside de AAV, donde el genoma vectorial comprende una secuencia de ácidos nucleicos que codifica un producto génico bajo el control de secuencias que direccionan la expresión de este. Reivindicación 10: Un péptido de direccionamiento a células cerebrales recombinante, el péptido caracterizado porque comprende un motivo dirigido al núcleo de una secuencia de aminoácidos de Y-X’-X’’-GNPA-X’’’-RYFD-X’’’’, donde X’ es G, A, R, K, X’’ es Y o H, X’’’ es T, R o H, y X’’’’ es V o K (SEQ ID Nº 14), opcionalmente flanqueada en el amino terminal y/o en el carboxi terminal de la SEQ ID Nº 14 por dos aminoácidos a siete aminoácidos, y opcionalmente el péptido o péptido con conectores está conjugado con una nanopartícula, una segunda molécula o una proteína de cápside viral recombinante. Reivindicación 21: Un método para aumentar la transducción de células de producción de AAV in vitro caracterizado porque comprende transducir células con una secuencia de ácidos nucleicos que codifica una cápside de AAV que comprende un péptido de direccionamiento exógeno de Y-X’-X’’-GNPA-X’’’-RYFD-X’’’’, donde X’ es G, A, R, K, X’’ es Y o H, X’’’ es T, R o H, y X’’’’ es V o K (SEQ ID Nº 14) motivo del núcleo.
ARP220101045A 2021-04-23 2022-04-22 Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen AR125406A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163178881P 2021-04-23 2021-04-23

Publications (1)

Publication Number Publication Date
AR125406A1 true AR125406A1 (es) 2023-07-12

Family

ID=81585505

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220101045A AR125406A1 (es) 2021-04-23 2022-04-22 Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen

Country Status (10)

Country Link
US (1) US20240207452A1 (es)
EP (1) EP4334334A1 (es)
AR (1) AR125406A1 (es)
AU (1) AU2022262771A1 (es)
BR (1) BR112023021971A2 (es)
CA (1) CA3216004A1 (es)
CO (1) CO2023014723A2 (es)
MX (1) MX2023012513A (es)
TW (1) TW202305124A (es)
WO (1) WO2022226263A1 (es)

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WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
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JP2022551986A (ja) * 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
CN115968302A (zh) 2020-06-17 2023-04-14 宾夕法尼亚州大学信托人 用于治疗基因疗法患者的组合物和方法
KR20230051208A (ko) 2020-08-14 2023-04-17 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 신규 aav 캡시드 및 이를 함유하는 조성물

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US20240207452A1 (en) 2024-06-27
TW202305124A (zh) 2023-02-01
CO2023014723A2 (es) 2023-11-20
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BR112023021971A2 (pt) 2024-02-20
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