CL2024001307A1 - Partículas virales redirigidas a músculo esquelético - Google Patents

Partículas virales redirigidas a músculo esquelético

Info

Publication number
CL2024001307A1
CL2024001307A1 CL2024001307A CL2024001307A CL2024001307A1 CL 2024001307 A1 CL2024001307 A1 CL 2024001307A1 CL 2024001307 A CL2024001307 A CL 2024001307A CL 2024001307 A CL2024001307 A CL 2024001307A CL 2024001307 A1 CL2024001307 A1 CL 2024001307A1
Authority
CL
Chile
Prior art keywords
muscular dystrophy
viral particles
skeletal muscle
muscle
aav
Prior art date
Application number
CL2024001307A
Other languages
English (en)
Spanish (es)
Inventor
Kyratsous Christos
J Murphy Andrew
Sabin Leah
MOLLER-TANK Sven
Stec Michael
Samai Poulami
Original Assignee
Regeneron Pharma
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 Regeneron Pharma filed Critical Regeneron Pharma
Publication of CL2024001307A1 publication Critical patent/CL2024001307A1/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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0658—Skeletal muscle cells, e.g. myocytes, myotubes, myoblasts
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • 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
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • 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
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Cell Biology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Environmental Sciences (AREA)
  • Mycology (AREA)
  • Biodiversity & Conservation Biology (AREA)
CL2024001307A 2021-11-04 2024-04-29 Partículas virales redirigidas a músculo esquelético CL2024001307A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163275731P 2021-11-04 2021-11-04
US202263369761P 2022-07-28 2022-07-28

Publications (1)

Publication Number Publication Date
CL2024001307A1 true CL2024001307A1 (es) 2024-10-11

Family

ID=84537880

Family Applications (2)

Application Number Title Priority Date Filing Date
CL2024001307A CL2024001307A1 (es) 2021-11-04 2024-04-29 Partículas virales redirigidas a músculo esquelético
CL2025000839A CL2025000839A1 (es) 2021-11-04 2025-03-21 Partículas virales redirigidas a músculo esquelético

Family Applications After (1)

Application Number Title Priority Date Filing Date
CL2025000839A CL2025000839A1 (es) 2021-11-04 2025-03-21 Partículas virales redirigidas a músculo esquelético

Country Status (13)

Country Link
US (1) US20250304918A1 (https=)
EP (1) EP4426359A1 (https=)
JP (1) JP2024540181A (https=)
KR (1) KR20240095211A (https=)
CN (1) CN118201643A (https=)
AU (1) AU2022380841A1 (https=)
CA (1) CA3233698A1 (https=)
CL (2) CL2024001307A1 (https=)
CO (1) CO2024005350A2 (https=)
IL (1) IL312598A (https=)
MX (1) MX2024005314A (https=)
PE (1) PE20241212A1 (https=)
WO (1) WO2023081850A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4612184A1 (en) * 2022-11-04 2025-09-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
EP4665764A1 (en) * 2023-02-13 2025-12-24 Regeneron Pharmaceuticals, Inc. Treatment of muscle related disorders with anti-human cacng1 antibodies
CN121568720A (zh) * 2023-07-10 2026-02-24 瑞泽恩制药公司 抗人类cacng1抗体-药物缀合物及其用途
TW202528546A (zh) * 2023-09-07 2025-07-16 美商再生元醫藥公司 經共價表面修飾之腺相關病毒的生產及純化
WO2025087345A1 (en) * 2023-10-25 2025-05-01 Suzhou Cure Genetics Biosciences Co., Limited Targeted viral particles
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2224907A1 (en) 1995-07-25 1997-02-13 Introgene B.V. Methods and means for targeted gene delivery
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
ES2724975T3 (es) 2009-12-10 2019-09-18 Regeneron Pharma Ratones que producen anticuerpos de cadena pesada
GB201002362D0 (en) 2010-02-11 2010-03-31 Isis Innovation Peptide tag systems that spontaneously form an irreversible link to protein partners via isopeptide bonds
CA3124228C (en) 2014-03-21 2024-05-14 Regeneron Pharmaceuticals, Inc. Non-human animals that make single domain binding proteins
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
JP6832280B2 (ja) 2015-01-16 2021-02-24 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ 新規のマイクロジストロフィンおよび使用の関連する方法
CN117887804A (zh) 2017-02-02 2024-04-16 纽约基因组研究中心公司 用于识别或量化在生物样品中的靶标的方法和组合物
PE20200488A1 (es) 2017-06-27 2020-03-03 Regeneron Pharma Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas
ES2996886T3 (en) 2017-06-27 2025-02-13 Regeneron Pharma Rodents comprising a humanized asgr1 locus
LT3645553T (lt) 2017-06-27 2023-04-25 Regeneron Pharmaceuticals, Inc. Modifikuoto tropizmo rekombinantinės viruso dalelės ir jų panaudojimas genetinės medžiagos tiksliniam įvedimui į žmogaus ląsteles
WO2019084015A1 (en) 2017-10-23 2019-05-02 Daniel Schmidt PROGRAMMABLE SET OF VIRUS COMPOSITES FOR ADMINISTRATION OF GENES TARGETING A RECEPTOR
US12258597B2 (en) * 2018-02-07 2025-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
US20220028234A1 (en) 2020-02-27 2022-01-27 Mirza Faizan Active security system and a method to detect and neutralize armed intruders

Also Published As

Publication number Publication date
CN118201643A (zh) 2024-06-14
IL312598A (en) 2024-07-01
EP4426359A1 (en) 2024-09-11
PE20241212A1 (es) 2024-06-06
AU2022380841A1 (en) 2024-05-02
JP2024540181A (ja) 2024-10-31
CL2025000839A1 (es) 2025-05-30
WO2023081850A1 (en) 2023-05-11
MX2024005314A (es) 2024-05-17
CO2024005350A2 (es) 2024-05-10
KR20240095211A (ko) 2024-06-25
CA3233698A1 (en) 2023-05-11
US20250304918A1 (en) 2025-10-02

Similar Documents

Publication Publication Date Title
CL2024001307A1 (es) Partículas virales redirigidas a músculo esquelético
Kizima et al. A potent combination microbicide that targets SHIV-RT, HSV-2 and HPV
Zhao et al. Adenovirus-mediated gene transfer in mesenchymal stem cells can be significantly enhanced by the cationic polymer polybrene
BR112018071167A2 (pt) entrega de beta-sarcoglicano e microrna-29 por vírus adenoassociado e tratamento de distrofia muscular
CL2020002445A1 (es) Vectores virales que codifican variantes de fix recombinantes con mayor expresión para la terapia génica de hemofilia b (divisional de la solicitud no. 201903395)
BR112021019821A2 (pt) Sorotipo de vírus adenoassociado híbrido recombinante com modificação peptídica entre aav9 e aavrh74 com tropismo hepático reduzido e transdução muscular aumentada
BR112022022644A2 (pt) Composições de vírus adeno-associados compatíveis com espécies cruzadas e método de uso destas
AR118734A1 (es) Formulaciones y métodos de vectores de virus adenoasociados
BR0009580A (pt) Composição, processos para preparar vìrus da influenza, para preparar um veìculo de liberação de genes, para preparar vìrus da influenza defeituosos de replicação infecciosa, para imunizar um paciente contra um patógeno, para preparar vìrus infeccioso, e para preparar vìrus, vìrus, célula, vetor, célula hospedeira, e, uso do vìrus
CO6180445A2 (es) Anticuerpos agonistas anti notch3 y su uso en el tratamiento de las enfermedades relacionadas con el gen notch3
MX2024012617A (es) Uso de un modulador irak4 para terapia genica
AR113134A1 (es) Arni variante
BR112022009290A2 (pt) Terapia para malignidades de células hematopoiéticas com o uso de células t geneticamente modificadas visando cd70
PE20230433A1 (es) Composiciones de virus adenoasociados para restaurar la funcion del gen pah y los metodos de uso de estas
CO2020010783A2 (es) Composiciones de virus adeno-asociadas para la transferencia de genes pah y métodos de uso de los mismos
CO2020007601A2 (es) Cepa vacunal de brucella modificada para el tratamiento de brucelosis
CO2023015911A2 (es) Vectores aavrh74 para la terapia génica de distrofias musculares.
DOP2024000171A (es) Compuestos antivirales y métodos de elaboración y uso de los mismos
CO2024013743A2 (es) Terapia génica dirigida para la distrofia miotónica dm-1
CL2024002622A1 (es) Compuestos antivirales y métodos de elaboración y uso de los mismos
PE20231175A1 (es) Inmunoterapia oncolitica por remodelacion del microambiente tumoral
Lu et al. Knockdown of the HDAC1 promotes the directed differentiation of bone mesenchymal stem cells into cardiomyocytes
MX2024012618A (es) Ensayo de células dendríticas para determinar la inmunogenicidad innata frente a agentes de terapia génica
MX2022002060A (es) Un vector viral híbrido procariota-eucariota para el suministro de grandes cargas de genes y proteínas a células humanas.
CO2022013833A2 (es) Composiciones de virus adenoasociados para la transferencia del gen ids y sus métodos de uso