MX2022004770A - Vectores de virus adeno-asociados (aav) para el tratamiento de la degeneracion macular relacionada con la edad y otras enfermedades y trastornos oculares. - Google Patents

Vectores de virus adeno-asociados (aav) para el tratamiento de la degeneracion macular relacionada con la edad y otras enfermedades y trastornos oculares.

Info

Publication number
MX2022004770A
MX2022004770A MX2022004770A MX2022004770A MX2022004770A MX 2022004770 A MX2022004770 A MX 2022004770A MX 2022004770 A MX2022004770 A MX 2022004770A MX 2022004770 A MX2022004770 A MX 2022004770A MX 2022004770 A MX2022004770 A MX 2022004770A
Authority
MX
Mexico
Prior art keywords
vectors
age
treatment
related macular
aav
Prior art date
Application number
MX2022004770A
Other languages
English (en)
Spanish (es)
Inventor
Mark Shearman
Adrian Timmers
Judith Newmark
Steven Pennock
Rakshaa Mureli
Chunjuan Song
Lisa Keyes
Original Assignee
Applied Genetic Tech Corporation
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 Applied Genetic Tech Corporation filed Critical Applied Genetic Tech Corporation
Publication of MX2022004770A publication Critical patent/MX2022004770A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/472Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/40Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (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)
MX2022004770A 2019-10-22 2020-10-22 Vectores de virus adeno-asociados (aav) para el tratamiento de la degeneracion macular relacionada con la edad y otras enfermedades y trastornos oculares. MX2022004770A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962924338P 2019-10-22 2019-10-22
PCT/US2020/056862 WO2021081203A1 (en) 2019-10-22 2020-10-22 Adeno-associated virus (aav)vectors for the treatment of age-related macular degeneration and other ocular diseases and disorders

Publications (1)

Publication Number Publication Date
MX2022004770A true MX2022004770A (es) 2022-10-07

Family

ID=75586875

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022004770A MX2022004770A (es) 2019-10-22 2020-10-22 Vectores de virus adeno-asociados (aav) para el tratamiento de la degeneracion macular relacionada con la edad y otras enfermedades y trastornos oculares.

Country Status (10)

Country Link
US (1) US20210123076A1 (https=)
EP (1) EP4048321A4 (https=)
JP (1) JP2022553309A (https=)
KR (1) KR20230019062A (https=)
CN (1) CN115175706A (https=)
AU (1) AU2020371663A1 (https=)
CA (1) CA3158517A1 (https=)
IL (1) IL292379A (https=)
MX (1) MX2022004770A (https=)
WO (1) WO2021081203A1 (https=)

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7745389B2 (en) * 2005-02-14 2010-06-29 University Of Iowa Research Foundation Methods for treatment of age-related macular degeneration
CN101160412A (zh) * 2005-02-14 2008-04-09 爱荷华大学研究基金会 治疗和诊断年龄相关性黄斑变性的方法和试剂
WO2007127428A2 (en) * 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
EP2479284B1 (en) * 2006-07-13 2017-09-20 University of Iowa Research Foundation Methods and reagents for treatment and diagnosis of vascular disorders and age-related macular degeneration
CN108291216B (zh) * 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
WO2017114401A1 (zh) * 2015-12-31 2017-07-06 江苏匡亚生物医药科技有限公司 具有补体调节活性的重组补体因子h-免疫球蛋白融合蛋白及其制备方法与应用
GB201706808D0 (en) * 2017-04-28 2017-06-14 Univ Newcastle Modified complement proteins and uses thereof
EP3697920A4 (en) * 2017-10-20 2022-03-02 Gemini Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION

Also Published As

Publication number Publication date
EP4048321A4 (en) 2023-11-22
WO2021081203A1 (en) 2021-04-29
US20210123076A1 (en) 2021-04-29
CA3158517A1 (en) 2021-04-29
AU2020371663A1 (en) 2022-05-19
EP4048321A1 (en) 2022-08-31
IL292379A (en) 2022-06-01
KR20230019062A (ko) 2023-02-07
JP2022553309A (ja) 2022-12-22
CN115175706A (zh) 2022-10-11

Similar Documents

Publication Publication Date Title
DOP2016000280A (es) Vectores de aav para la terapia génica de la retina y el snc
MX2022004771A (es) Vectores de virus adeno-asociados (aav) de triple funcion para el tratamiento de enfermedades asociadas a c9orf72.
WO2012094560A3 (en) Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
PH12021552038A1 (en) Compositions and methods to treat bietti crystalline dystrophy
ZA201705628B (en) Methods and compositions for treating genetic eye diseases
AU2018261769A1 (en) Compositions and methods for expressing Otoferlin
WO2020093018A8 (en) A codon optimized otoferlin aav dual vector gene therapy
PH12021551155A1 (en) Recombinant viral vectors and nucleic acids for producing the same
EP4219695A3 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
PH12021550794A1 (en) Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
IL286427B2 (en) Inhibitors bcl-2 anti-apoptotic protein family members for treatment of non cancer pulmonary disease or an opthalmic disease
PH12018502387A1 (en) Adeno-associated virus variant capsids and methods of use thereof
MX2020010968A (es) Formulaciones de bevacizumab tamponadas para usarse en el tratamiento de enfermedades.
WO2019087130A3 (en) Method of inhibiting angiogenesis
PH12020551642A1 (en) Aav compositions, methods of making and methods of use
EP4055174A4 (en) TRANSGENIC CASSETTES, AAV VECTORS AND AAV VECTORS FOR EXPRESSING HUMAN CODON-OPTIMIZED SLCA1
WO2020264438A3 (en) Enhancing aav-mediated transduction of ocular tissues with hyaluronic acid
MX2020010477A (es) Composiciones y metodos para el tratamiento de la distrofia macular.
WO2019169371A8 (en) Drug stabilized therapeutic transgenes delivered by adeno-associated virus expression
MX2025009248A (es) Composiciones y metodos de terapia genica para tratar enfermedades de la retina
MX2022004770A (es) Vectores de virus adeno-asociados (aav) para el tratamiento de la degeneracion macular relacionada con la edad y otras enfermedades y trastornos oculares.
EP4223320A3 (en) Codon-optimized transgene for the treatment of progressive familiar intrahepatic cholestasis type 3 (pfic3)
WO2019046341A3 (en) Methods and compositions for treating cone-rod retinal dystrophy
ZA202206245B (en) On-bipolar cell-specific promoters for ocular gene delivery
EP4356727A3 (en) Compositions and methods for the treatment of dominantly-inherited catecholaminergic polymorphic ventricular tachycardia