PE20220930A1 - Composiciones utiles en el tratamiento de leucodistrofia metacromatica - Google Patents

Composiciones utiles en el tratamiento de leucodistrofia metacromatica

Info

Publication number
PE20220930A1
PE20220930A1 PE2021001824A PE2021001824A PE20220930A1 PE 20220930 A1 PE20220930 A1 PE 20220930A1 PE 2021001824 A PE2021001824 A PE 2021001824A PE 2021001824 A PE2021001824 A PE 2021001824A PE 20220930 A1 PE20220930 A1 PE 20220930A1
Authority
PE
Peru
Prior art keywords
harsa
seq
amino acid
acid sequence
treatment
Prior art date
Application number
PE2021001824A
Other languages
English (en)
Inventor
Juliette Hordeaux
James 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 PE20220930A1 publication Critical patent/PE20220930A1/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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K47/02—Inorganic compounds
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06001—Arylsulfatase (3.1.6.1)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/106—Primate
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Abstract

La presente invencion se refiere a un virus adenoasociado recombinante (rAAV) que comprende: (a) una capside de AAVhub68; y (b) un genoma de vector empaquetado en la capside de AAV de (a), en donde un genoma de vector comprende repeticiones terminales invertidas (ITR) y una secuencia de acidos nucleicos que codifica una arisulfatasa A funcional humana (hARSA) controlada por secuencias reguladoras que dirigen la expresion de hARSA, en donde la secuencia codificante de hARSA comprende una secuencia de nucleotidos (nt 55 al nt 1521 de SEQ ID NO: 1); adicionalmente, la proteina funcional hARSA comprende un peptido de senal y una secuencia de aminoacidos del aminoacido (aa) 19 al aa 507 de SEQ ID NO: 2, y el peptido de senal tiene una secuencia de aminoacidos del aa 1 al aa 18 de SEQ ID NO: 2 o una secuencia de aminoacidos del aa 1 al aa 20 de SEQ ID NO: 4. Tambien se refiere a una composicion farmaceutica, a un vector y a su metodo de produccion. Dicho virus adenoasociado recombinante (rAAV) es util en el tratamiento de pacientes con leucodistrofia metacromatica (MLD).
PE2021001824A 2019-05-03 2020-05-02 Composiciones utiles en el tratamiento de leucodistrofia metacromatica PE20220930A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962843091P 2019-05-03 2019-05-03
PCT/US2020/031207 WO2020227166A1 (en) 2019-05-03 2020-05-02 Compositions useful in treatment of metachromatic leukodystrophy

Publications (1)

Publication Number Publication Date
PE20220930A1 true PE20220930A1 (es) 2022-05-31

Family

ID=73050896

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2021001824A PE20220930A1 (es) 2019-05-03 2020-05-02 Composiciones utiles en el tratamiento de leucodistrofia metacromatica

Country Status (17)

Country Link
US (1) US20220228170A1 (es)
EP (1) EP3962454A4 (es)
JP (1) JP2022531861A (es)
KR (1) KR20220020261A (es)
CN (1) CN114072129A (es)
AR (1) AR118850A1 (es)
AU (1) AU2020270421A1 (es)
BR (1) BR112021021908A2 (es)
CA (1) CA3135539A1 (es)
CL (1) CL2021002881A1 (es)
CO (1) CO2021016323A2 (es)
IL (1) IL287715A (es)
MX (1) MX2021013420A (es)
PE (1) PE20220930A1 (es)
SG (1) SG11202111867XA (es)
TW (1) TW202108760A (es)
WO (1) WO2020227166A1 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020251954A1 (en) * 2019-06-10 2020-12-17 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
KR20230010670A (ko) * 2020-05-12 2023-01-19 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이식유전자 발현의 drg-특이적 감소를 위한 조성물
RU2769577C1 (ru) * 2021-06-01 2022-04-04 федеральное государственное автономное образовательное учреждение высшего образования "Казанский (Приволжский) федеральный университет" (ФГАОУ ВО КФУ) Препарат для лечения метахроматической лейкодистрофии и способ ее лечения
TW202338086A (zh) * 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
WO2023196863A1 (en) * 2022-04-05 2023-10-12 Astellas Gene Therapies, Inc. Adeno-associated virus potency assay and uses thereof
US20230398192A1 (en) * 2022-05-16 2023-12-14 Genzyme Corporation Methods of treating metachromatic leukodystrophy
WO2024079361A1 (en) * 2022-10-14 2024-04-18 Neuway Pharma Gmbh A PROTEIN- OR PEPTIDE-BASED CAPSULE (PPC), PREFERABLY A VLP, LOADED WITH A MESSENGER RNA (mRNA) AND A METHOD OF PRODUCTION AND PURIFICATION THEREOF

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000026393A1 (en) * 1998-10-31 2000-05-11 University Of Medicine And Dentistry Of New Jersey A myeloid precursor cell useful for gene therapy and for modulation of immune responses
AU2002317700A1 (en) * 2001-06-07 2002-12-16 Hemebiotech A/S Production of recombinant human arylsulfatase a
JP4586027B2 (ja) * 2004-01-30 2010-11-24 シャイア ファーマシューティカルズ アイルランド リミテッド 組換えアリールスルファターゼａの製造及び精製
WO2013096899A2 (en) * 2011-12-23 2013-06-27 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
DK3294323T3 (da) * 2015-05-15 2022-01-10 Univ Minnesota Adeno-associeret virus til terapeutisk indgivelse til centralnervesystemet
SG10201912969XA (en) * 2017-02-28 2020-02-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
WO2019051424A2 (en) * 2017-09-08 2019-03-14 Poseida Therapeutics, Inc. COMPOSITIONS AND METHODS FOR CONDITIONAL GENE EXPRESSION MEDIATED BY A CHIMERIC LIGAND RECEPTOR (CLR)

Also Published As

Publication number Publication date
EP3962454A1 (en) 2022-03-09
BR112021021908A2 (pt) 2022-02-01
CL2021002881A1 (es) 2022-07-29
AU2020270421A1 (en) 2021-11-18
IL287715A (en) 2021-12-01
JP2022531861A (ja) 2022-07-12
CO2021016323A2 (es) 2021-12-10
TW202108760A (zh) 2021-03-01
KR20220020261A (ko) 2022-02-18
US20220228170A1 (en) 2022-07-21
CA3135539A1 (en) 2020-11-12
MX2021013420A (es) 2022-02-03
WO2020227166A1 (en) 2020-11-12
CN114072129A (zh) 2022-02-18
AR118850A1 (es) 2021-11-03
EP3962454A4 (en) 2023-05-31
SG11202111867XA (en) 2021-11-29

Similar Documents

Publication Publication Date Title
PE20220930A1 (es) Composiciones utiles en el tratamiento de leucodistrofia metacromatica
PH12021551341A1 (en) Compositions for drg-specific reduction of transgene expression
MX2020008932A (es) Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
PE20191261A1 (es) Composiciones utiles para el tratamiento de atrofia muscular espinal
MX2020008933A (es) Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
MX2019010275A (es) Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este.
CO2020004738A2 (es) Variantes de cápsides de virus adenoasociados y métodos de uso de estas
MX2021005517A (es) Terapia genica para lipofuscinosis neuronal ceroidea.
PE20160188A1 (es) Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos
JP2020510430A5 (es)
PE20231949A1 (es) VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
RU2017142006A (ru) Векторы на основе аденоассоциированных вирусов для лечения мукополисахаридозов
BR112019023303A2 (pt) Terapia de gene para lipofuscinoses ceroides neuronais
JP2020510428A5 (es)
CO2021016198A2 (es) Composiciones útiles para el tratamiento de la enfermedad de pompe
BR112022004027A2 (pt) Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte
PE20220429A1 (es) Vectores viricos que codifican variantes del fviii recombinantes con mayor expresion para la genoterapia de la hemofilia a
PE20212072A1 (es) Composiciones utiles en el tratamiento de la enfermedad de krabbe
CO2022017224A2 (es) Composiciones útiles para el tratamiento de la enfermedad de pompe
AR123369A1 (es) VACUNA BASADA EN AAV5 PARA LA INDUCCIÓN DE INMUNIDAD ESPECÍFICA CONTRA EL SARS-CoV-2 Y/O LA PREVENCIÓN DE LA INFECCIÓN POR CORONAVIRUS RELACIONADA CON EL SARS-CoV-2
MX2019014697A (es) Proteínas del receptor para productos finales de la glicación avanzada (rage) para el tratamiento de fibrosis y enfermedades mediadas por daño al adn.
BR112022003206A2 (pt) Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte
AR122251A1 (es) Composiciones útiles para el tratamiento de la enfermedad de krabbe
AR118004A1 (es) Administración del virus adenoasociado de polinucleótido de cln6
EA202191744A1 (ru) Композиции для drg-специфического снижения экспрессии трансгена