CO2018007203A2 - Focalización de péptidos para dirigir virus adenoasociados (aavs) - Google Patents
Focalización de péptidos para dirigir virus adenoasociados (aavs)Info
- Publication number
- CO2018007203A2 CO2018007203A2 CONC2018/0007203A CO2018007203A CO2018007203A2 CO 2018007203 A2 CO2018007203 A2 CO 2018007203A2 CO 2018007203 A CO2018007203 A CO 2018007203A CO 2018007203 A2 CO2018007203 A2 CO 2018007203A2
- Authority
- CO
- Colombia
- Prior art keywords
- aavs
- adeno
- associated viruses
- peptides
- focalization
- Prior art date
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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/02—Peptides of undefined number of amino acids; Derivatives thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/08—Peptides having 5 to 11 amino acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
- A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/19—Cytokines; Lymphokines; Interferons
- A61K38/20—Interleukins [IL]
- A61K38/2093—Leukaemia inhibitory factor [LIF]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
- A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
-
- 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
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- A61P25/04—Centrally acting analgesics, e.g. opioids
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/06—Antiarrhythmics
-
- 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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
- C07K14/01—DNA viruses
- C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
- C07K7/04—Linear peptides containing only normal peptide links
- C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
-
- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
- C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
- C07K2317/622—Single chain antibody (scFv)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/122—Hairpin
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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
- C12N2750/14141—Use of virus, viral particle or viral elements as a 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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- 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
- C12N2750/14171—Demonstrated in vivo effect
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
- C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/01—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
- C12Y305/01015—Aspartoacylase (3.5.1.15)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Psychology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pain & Pain Management (AREA)
Abstract
Aquí se divulgan secuencias de péptidos capaces de dirigir virus adenoasociados (AAV) para focalizar ambientes específicos, por ejemplo el sistema nervioso y el corazón, en un sujeto. También se divulgan AAVs que tienen proteínas de la cápside que no ocurren naturalmente comprendiendo las secuencias de péptidos divulgadas, y métodos para utilizar los AAVs para tratar enfermedades.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562266184P | 2015-12-11 | 2015-12-11 | |
US201662421891P | 2016-11-14 | 2016-11-14 | |
PCT/US2016/065969 WO2017100671A1 (en) | 2015-12-11 | 2016-12-09 | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2018007203A2 true CO2018007203A2 (es) | 2018-09-20 |
Family
ID=59013356
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2018/0007203A CO2018007203A2 (es) | 2015-12-11 | 2016-12-09 | Focalización de péptidos para dirigir virus adenoasociados (aavs) |
Country Status (19)
Country | Link |
---|---|
US (2) | US11499165B2 (es) |
EP (2) | EP3875593A1 (es) |
JP (1) | JP7066619B2 (es) |
KR (1) | KR102423442B1 (es) |
CN (1) | CN108699565B (es) |
AU (1) | AU2016366549B2 (es) |
BR (1) | BR112018011711A2 (es) |
CA (1) | CA3007495C (es) |
CL (1) | CL2018001522A1 (es) |
CO (1) | CO2018007203A2 (es) |
DK (1) | DK3387137T3 (es) |
ES (1) | ES2869284T3 (es) |
HK (1) | HK1257563A1 (es) |
IL (1) | IL259842B (es) |
MX (1) | MX2018006840A (es) |
RU (1) | RU2727672C2 (es) |
SG (1) | SG11201804713VA (es) |
WO (1) | WO2017100671A1 (es) |
ZA (1) | ZA201803808B (es) |
Families Citing this family (109)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3044318B1 (en) | 2013-09-13 | 2019-05-01 | California Institute of Technology | Selective recovery |
WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
CA2967468A1 (en) * | 2014-12-16 | 2016-06-23 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
US11499165B2 (en) | 2015-12-11 | 2022-11-15 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
BR112018073384A2 (pt) | 2016-05-18 | 2019-03-06 | Voyager Therapeutics, Inc. | polinucleotídeos moduladores |
US11951121B2 (en) | 2016-05-18 | 2024-04-09 | Voyager Therapeutics, Inc. | Compositions and methods for treating Huntington's disease |
WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
MX2019012113A (es) | 2017-04-14 | 2020-09-10 | Univ Nat Taiwan | Terapia génica para la deficiencia de la aadc. |
MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
CN110913866A (zh) | 2017-05-05 | 2020-03-24 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
CN111163811A (zh) * | 2017-08-25 | 2020-05-15 | 奥维德医疗公司 | 重组腺相关载体 |
US20200263199A1 (en) * | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
US11458129B2 (en) | 2017-11-02 | 2022-10-04 | California Institute Of Technology | Neurokinin antagonists and uses thereof |
EP3703716A4 (en) | 2017-11-02 | 2021-12-01 | California Institute of Technology | EXPRESSION OF NEUROPEPTIDES |
US20190202866A1 (en) * | 2017-12-22 | 2019-07-04 | William Marsh Rice University | Viral nanoparticles and methods of use thereof |
JP7378417B2 (ja) * | 2018-03-30 | 2023-11-13 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 増強されたヒト膵臓トロピズムを有する新規な組換えアデノ随伴ウイルスキャプシド |
KR20210006357A (ko) | 2018-04-03 | 2021-01-18 | 스트라이드바이오 인코포레이티드 | 항체-회피 바이러스 벡터 |
CA3096407A1 (en) * | 2018-04-09 | 2019-10-17 | Allen Institute | Rescuing voltage-gated sodium channel function in inhibitory neurons |
WO2019198084A1 (en) * | 2018-04-12 | 2019-10-17 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Anti-aging compositions and methods of use |
WO2019222329A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
WO2019222328A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
EP3807404A1 (en) | 2018-06-13 | 2021-04-21 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
US20210277066A1 (en) | 2018-07-11 | 2021-09-09 | The Brigham And Women`S Hospital, Inc. | Methods and compositions for delivery of agents across the blood-brain barrier |
US20210355454A1 (en) | 2018-07-24 | 2021-11-18 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
WO2020028751A2 (en) | 2018-08-03 | 2020-02-06 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
CN113966399A (zh) | 2018-09-26 | 2022-01-21 | 加州理工学院 | 用于靶向基因疗法的腺相关病毒组合物 |
CN113383010A (zh) | 2018-09-28 | 2021-09-10 | 沃雅戈治疗公司 | 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法 |
US20210380969A1 (en) | 2018-10-02 | 2021-12-09 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
US20210348242A1 (en) | 2018-10-04 | 2021-11-11 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
CA3115248A1 (en) | 2018-10-05 | 2020-04-09 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
US20210371470A1 (en) | 2018-10-12 | 2021-12-02 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
CA3116701A1 (en) | 2018-10-15 | 2020-04-23 | Voyager Therapeutics, Inc. | Expression vectors for large-scale production of raav in the baculovirus/sf9 system |
EP4205757A1 (en) | 2018-10-29 | 2023-07-05 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Expression vector for cholesterol 24-hydrolase for the prevention of amyotrophic lateral sclerosis |
MX2021008542A (es) | 2019-01-18 | 2021-11-12 | Voyager Therapeutics Inc | Métodos y sistemas para la producción de partículas de aav. |
CN110437317B (zh) * | 2019-01-30 | 2023-05-02 | 上海科技大学 | 具有变异衣壳蛋白的腺相关病毒及其用途 |
WO2020160337A1 (en) * | 2019-01-30 | 2020-08-06 | The Broad Institute, Inc. | Systems for evolved adeno-associated viruses (aavs) for targeted delivery |
CA3142948A1 (en) * | 2019-02-15 | 2020-08-20 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations |
KR20220011616A (ko) | 2019-03-21 | 2022-01-28 | 스트라이드바이오 인코포레이티드 | 재조합 아데노 관련 바이러스 벡터 |
WO2020210655A1 (en) * | 2019-04-11 | 2020-10-15 | California Institute Of Technology | Virus compositions with enhanced specificity in the brain |
GB201905301D0 (en) | 2019-04-15 | 2019-05-29 | Ospedale San Raffaele Srl | Gene therapy |
WO2020223280A1 (en) | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
US20220243225A1 (en) | 2019-04-29 | 2022-08-04 | Voyager Therapeutics, Inc. | SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS |
WO2020223362A1 (en) * | 2019-04-30 | 2020-11-05 | The Trustees Of The University Ofpennsylvania | Compositions useful for treatment of pompe disease |
US20240124889A1 (en) | 2019-05-07 | 2024-04-18 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
US20220233654A1 (en) | 2019-05-21 | 2022-07-28 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome |
WO2021025995A1 (en) | 2019-08-02 | 2021-02-11 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
US20220290182A1 (en) | 2019-08-09 | 2022-09-15 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
EP4022070A1 (en) | 2019-08-26 | 2022-07-06 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
US20220380413A1 (en) * | 2019-08-29 | 2022-12-01 | The Board Of Trustees Of The Leland Stanford Junior University | Adeno-Associated Viral Vectors for Crossing the Human Blood Brain Barrier |
US20220333133A1 (en) | 2019-09-03 | 2022-10-20 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
GB201913974D0 (en) * | 2019-09-27 | 2019-11-13 | King S College London | Vector |
EP4045525A1 (en) * | 2019-10-17 | 2022-08-24 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
US20210162002A1 (en) * | 2019-10-17 | 2021-06-03 | The Penn State Research Foundation | Regenerating functional neurons for treatment of spinal cord injury and als |
US20240131185A1 (en) * | 2019-10-17 | 2024-04-25 | Stridebio, Inc. | Aav transfer cassette |
KR20220098384A (ko) * | 2019-11-19 | 2022-07-12 | 아스클레피오스 바이오파마슈티컬, 인크. | 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스 |
EP4087602A4 (en) | 2020-01-10 | 2023-11-29 | The Brigham & Women's Hospital, Inc. | METHODS AND COMPOSITIONS FOR DELIVERING IMMUNOTHERAPEUTIC ACTIVE INGREDIENTS THROUGH THE BLOOD-BRAIN BARRIER TO TREAT BRAIN TUMORS |
WO2021163357A2 (en) | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
AU2021218411A1 (en) | 2020-02-13 | 2022-10-06 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
CN112011571A (zh) * | 2020-04-26 | 2020-12-01 | 辉大(上海)生物科技有限公司 | 一种用于治疗脊髓性肌萎缩的基因治疗药物 |
EP4150626A1 (en) | 2020-04-29 | 2023-03-22 | The Broad Institute Inc. | Machine learning accelerated protein engineering through fitness prediction |
MX2022014256A (es) * | 2020-05-13 | 2023-01-11 | Voyager Therapeutics Inc | Redirección del tropismo de las cápsides de aav. |
WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
GB202010981D0 (en) | 2020-07-16 | 2020-09-02 | Ucl Business Ltd | Gene therapy for neuromuscular and neuromotor disorders |
WO2022017630A1 (en) | 2020-07-23 | 2022-01-27 | Ucl Business Ltd | GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF |
US20230285596A1 (en) * | 2020-07-27 | 2023-09-14 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
IL300187A (en) | 2020-08-05 | 2023-03-01 | Spacecraft Seven Llc | Gene therapy CSRP3 (protein 3 rich in cysteine and glycine) |
EP4192514A1 (en) | 2020-08-06 | 2023-06-14 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
MX2023001615A (es) | 2020-08-07 | 2023-03-08 | Spacecraft Seven Llc | Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav. |
CN116406305A (zh) | 2020-08-17 | 2023-07-07 | 麻省理工学院 | Shank3基因治疗方法 |
JP2023538129A (ja) * | 2020-08-21 | 2023-09-06 | カプシダ, インコーポレイテッド | 好ましい発現レベルを有するアデノ随伴ウイルス組成物 |
JP2023542211A (ja) * | 2020-09-21 | 2023-10-05 | アスクレピオス バイオファーマシューティカル, インコーポレイテッド | 神経疾患を処置するための方法 |
CN112194706B (zh) * | 2020-09-30 | 2022-03-08 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
CA3197726A1 (en) | 2020-10-15 | 2022-04-21 | Simon Auslaender | Nucleic acid constructs for simultaneous gene activation |
CA3201533A1 (en) | 2020-11-11 | 2022-05-19 | European Molecular Biology Laboratory | Modified viral particles for gene therapy |
IL303236A (en) | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | New compounds with specific tissue-targeting motifs and preparations containing them |
US20240141378A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
TW202246516A (zh) | 2021-03-03 | 2022-12-01 | 美商航海家醫療公司 | 病毒蛋白之控制表現 |
WO2022221421A2 (en) * | 2021-04-13 | 2022-10-20 | Capsida, Inc. | Aav compositions with high brain expression for treating mucopolysaccharidosis ii |
JP2024515626A (ja) | 2021-04-16 | 2024-04-10 | アスクレピオス バイオファーマシューティカル, インコーポレイテッド | 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドaavビリオン |
EP4334334A1 (en) | 2021-04-23 | 2024-03-13 | The Trustees of The University of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
TW202317768A (zh) | 2021-07-08 | 2023-05-01 | 美商特納亞治療股份有限公司 | 用於基因療法的優化表現卡匣 |
WO2023049710A1 (en) * | 2021-09-21 | 2023-03-30 | California Institute Of Technology | Viral compositions for targeting the nervous system and the lung |
CN118043039A (zh) | 2021-10-25 | 2024-05-14 | 诺华股份有限公司 | 用于改善腺相关病毒(aav)递送的方法 |
IL311861A (en) * | 2021-11-02 | 2024-06-01 | Voyager Therapeutics Inc | Adeno-associated virus capsid variants and their uses |
CN114107231B (zh) * | 2021-12-13 | 2023-08-18 | 重庆大学 | 实现全脑突触后神经元胞体标记的重组腺相关病毒及其应用 |
WO2023147374A2 (en) | 2022-01-25 | 2023-08-03 | Voyager Therapeutics, Inc. | Baculovirus expression system |
CN116554278A (zh) * | 2022-01-30 | 2023-08-08 | 上海玮美基因科技有限责任公司 | 变异型腺相关病毒及其在疾病治疗中的应用 |
WO2023164545A1 (en) | 2022-02-23 | 2023-08-31 | Massachusetts Institute Of Technology | Methods for upregulating shank3 expression |
WO2023205397A1 (en) * | 2022-04-22 | 2023-10-26 | The Children's Hospital Of Philadelphia | Human ependyma-specific promoter and uses thereof |
WO2023240158A2 (en) * | 2022-06-07 | 2023-12-14 | Oregon Health & Science University | Mutant adeno-associated virus (aav) capsids |
WO2023240236A1 (en) * | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
WO2024054864A1 (en) | 2022-09-06 | 2024-03-14 | Tenaya Therapeutics, Inc. | Cardioprotective heart disease therapies |
WO2024054983A1 (en) | 2022-09-08 | 2024-03-14 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
GB2622629A (en) | 2022-09-23 | 2024-03-27 | Sania Rx Ltd | Method |
WO2024100145A1 (en) | 2022-11-08 | 2024-05-16 | Ospedale San Raffaele S.R.L. | Polynucleotide and vector |
CN117264912A (zh) * | 2023-09-25 | 2023-12-22 | 中国科学院深圳先进技术研究院 | 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用 |
Family Cites Families (32)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5919676A (en) | 1993-06-24 | 1999-07-06 | Advec, Inc. | Adenoviral vector system comprising Cre-loxP recombination |
US6379943B1 (en) | 1999-03-05 | 2002-04-30 | Merck & Co., Inc. | High-efficiency Cre/loxp based system for construction of adenovirus vectors |
US7279313B2 (en) * | 1995-09-15 | 2007-10-09 | Centelion | Circular DNA molecule having a conditional origin of replication, process for their preparation and their use in gene therapy |
AU2319497A (en) | 1996-03-07 | 1997-09-22 | Regents Of The University Of California, The | Helper-free, totally defective adenovirus for gene therapy |
CA2264499A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | Methods using cre-lox for production of recombinant adeno-associated viruses |
DE19933719A1 (de) * | 1999-07-19 | 2001-01-25 | Medigene Ag | Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung |
CA2397532A1 (en) | 2000-01-26 | 2001-08-02 | Chiron Corporation | Recombinant aav packaging systems |
NZ522840A (en) | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
US6410271B1 (en) | 2000-06-23 | 2002-06-25 | Genetastix Corporation | Generation of highly diverse library of expression vectors via homologous recombination in yeast |
WO2004083441A2 (en) | 2003-03-19 | 2004-09-30 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Random peptide library displayed on aav vectors |
WO2005017101A2 (en) | 2003-05-19 | 2005-02-24 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH | Avian adenoassociated virus (aaav) and uses thereof |
US9233131B2 (en) * | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
WO2005033321A2 (en) * | 2003-09-30 | 2005-04-14 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
WO2006099615A2 (en) | 2005-03-16 | 2006-09-21 | The Johns Hopkins University | Adenoviral fiber exchange shuttle system |
CA2655957C (en) * | 2006-06-21 | 2016-05-03 | Amsterdam Molecular Therapeutics (Amt) B.V. | Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells |
AU2008218109A1 (en) | 2007-02-23 | 2008-08-28 | University Of Florida Research Foundation, Inc. | Compositions and methods for treating glycogen storage diseases |
WO2009012176A2 (en) | 2007-07-14 | 2009-01-22 | The University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
US9018138B2 (en) | 2007-08-16 | 2015-04-28 | The Johns Hopkins University | Compositions and methods for generating and screening adenoviral libraries |
WO2010019619A1 (en) | 2008-08-11 | 2010-02-18 | The Board Of Trustees Of The Leland Stanford Junior Univeristy | Method and composition for controlling gene expression |
EP3540055A1 (en) | 2010-04-23 | 2019-09-18 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
EP2678433B1 (en) | 2011-02-22 | 2017-05-03 | California Institute of Technology | Delivery of proteins using adeno-associated virus (aav) vectors |
EP2699270B1 (en) | 2011-04-22 | 2017-06-21 | The Regents of The University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
WO2013029030A1 (en) | 2011-08-24 | 2013-02-28 | The Board Of Trustees Of The Leland Stanford Junior University | New aav capsid proteins for nucleic acid transfer |
EP3044318B1 (en) | 2013-09-13 | 2019-05-01 | California Institute of Technology | Selective recovery |
JP6013654B2 (ja) | 2013-09-19 | 2016-10-25 | クルセル ホランド ベー ヴェー | 改良されたアデノウイルス製剤 |
GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
DK3137497T3 (da) | 2014-05-02 | 2021-07-12 | Genzyme Corp | Aav-vektorer til retinal- og cns-genterapi |
US11499165B2 (en) | 2015-12-11 | 2022-11-15 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
CA3061365A1 (en) * | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
MX2019013172A (es) * | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
CN110913866A (zh) * | 2017-05-05 | 2020-03-24 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
EP3662060A2 (en) * | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
-
2016
- 2016-12-09 US US15/374,596 patent/US11499165B2/en active Active
- 2016-12-09 SG SG11201804713VA patent/SG11201804713VA/en unknown
- 2016-12-09 MX MX2018006840A patent/MX2018006840A/es unknown
- 2016-12-09 EP EP21152352.7A patent/EP3875593A1/en active Pending
- 2016-12-09 EP EP16873985.2A patent/EP3387137B1/en active Active
- 2016-12-09 RU RU2018120736A patent/RU2727672C2/ru active
- 2016-12-09 CO CONC2018/0007203A patent/CO2018007203A2/es unknown
- 2016-12-09 DK DK16873985.2T patent/DK3387137T3/da active
- 2016-12-09 KR KR1020187019747A patent/KR102423442B1/ko active IP Right Grant
- 2016-12-09 BR BR112018011711A patent/BR112018011711A2/pt active Search and Examination
- 2016-12-09 ES ES16873985T patent/ES2869284T3/es active Active
- 2016-12-09 WO PCT/US2016/065969 patent/WO2017100671A1/en active Application Filing
- 2016-12-09 IL IL259842A patent/IL259842B/en unknown
- 2016-12-09 CA CA3007495A patent/CA3007495C/en active Active
- 2016-12-09 JP JP2018530606A patent/JP7066619B2/ja active Active
- 2016-12-09 CN CN201680081708.1A patent/CN108699565B/zh active Active
- 2016-12-09 AU AU2016366549A patent/AU2016366549B2/en active Active
-
2018
- 2018-06-07 CL CL2018001522A patent/CL2018001522A1/es unknown
- 2018-06-07 ZA ZA201803808A patent/ZA201803808B/en unknown
- 2018-12-27 HK HK18116620.4A patent/HK1257563A1/zh unknown
-
2022
- 2022-10-17 US US18/047,233 patent/US20230295659A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
CA3007495C (en) | 2023-04-11 |
CN108699565B (zh) | 2023-08-08 |
HK1257563A1 (zh) | 2019-10-25 |
KR20180097629A (ko) | 2018-08-31 |
US20230295659A1 (en) | 2023-09-21 |
BR112018011711A2 (pt) | 2018-12-04 |
AU2016366549B2 (en) | 2022-11-10 |
JP2019506141A (ja) | 2019-03-07 |
AU2016366549A1 (en) | 2018-07-12 |
IL259842B (en) | 2022-08-01 |
CA3007495A1 (en) | 2017-06-15 |
RU2018120736A (ru) | 2020-01-13 |
CL2018001522A1 (es) | 2018-12-21 |
EP3875593A1 (en) | 2021-09-08 |
ZA201803808B (en) | 2020-11-25 |
US20170166926A1 (en) | 2017-06-15 |
MX2018006840A (es) | 2019-03-28 |
SG11201804713VA (en) | 2018-07-30 |
RU2018120736A3 (es) | 2020-01-13 |
EP3387137B1 (en) | 2021-02-03 |
CN108699565A (zh) | 2018-10-23 |
EP3387137A4 (en) | 2019-06-12 |
RU2727672C2 (ru) | 2020-07-22 |
DK3387137T3 (da) | 2021-05-03 |
ES2869284T3 (es) | 2021-10-25 |
KR102423442B1 (ko) | 2022-07-20 |
WO2017100671A1 (en) | 2017-06-15 |
IL259842A (en) | 2018-07-31 |
JP7066619B2 (ja) | 2022-05-13 |
US11499165B2 (en) | 2022-11-15 |
EP3387137A1 (en) | 2018-10-17 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
CO2018007203A2 (es) | Focalización de péptidos para dirigir virus adenoasociados (aavs) | |
CL2019000167A1 (es) | Novedosas proteínas de la cápside del virus adenoasociado. | |
CL2018000740A1 (es) | Vectores del factor viii del virus adeno-asociado, partículas víricas asociadas y formulaciones terapéuticas que los comprenden | |
UY36458A (es) | Anticuerpos humanos para hemaglutinina influenza. | |
CO2017012562A2 (es) | Vectores virales adenoasociados que codifican para la enzima iduronato-2-sulfatasa | |
BR112017010447A2 (pt) | vetores de aav direcionados ao sistema nervoso central | |
CL2019000261A1 (es) | Polipéptidos modificados y usos de los mismos. | |
BR112019001737A2 (pt) | expressão de pten-long com vírus oncolíticos | |
BR112018011881A2 (pt) | proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus | |
CL2017002731A1 (es) | Adenovirus oncolítico que codifica una proteína b7 | |
BR112017012859A2 (pt) | fosforamidatos para o tratamento do vírus da hepatite b | |
CL2023000264A1 (es) | Nuevos vectores virales adeno-asociados dirigidos al hígado | |
BR112015022790A8 (pt) | molécula biespecífica, composição farmacêutica, método de tratamento de uma infecção por vírus latente em um indivíduo em necessidade de tal tratamento, método de tratamento de uma infecção por vírus persistente em um indivíduo em necessidade de tal tratamento, método de tratamento de uma infecção por vírus inativo em um indivíduo em necessidade de tal tratamento, método para exterminar uma célula que contém um genoma viral, e, método para exterminar uma célula que expressa uma proteína viral | |
EA201792573A1 (ru) | Триспецифические связанные белки и способы их применения | |
DK3294323T3 (da) | Adeno-associeret virus til terapeutisk indgivelse til centralnervesystemet | |
CR20170139A (es) | Proteínas de fusión, bacterias recombinantes y métodos de uso de las bacterias recombinantes | |
CO2019009215A2 (es) | Sal hemi-sulfato de nucleótido para el tratamiento del virus de la hepatitis c | |
BR112018073238A2 (pt) | vírus oncolíticos compreendendo esrage e métodos de tratamento de câncer | |
CU24580B1 (es) | Vectores atenuados de influenza para la prevención y/o tratamiento de enfermedades infecciosas y para el tratamiento de enfermedades oncológicas | |
BR112017005235A2 (pt) | vetores virais adeno-associados para o tratamento de glaucoma de miocilina (myoc) | |
DK3146042T3 (da) | Rekombinant klassisk svinepestvirus (CSFV) med substitution i E2-proteinets TAV-epitop | |
BR112018070497A2 (pt) | métodos e formulações farmacêuticas para tratamento de condições oculares | |
DOP2016000202A (es) | Proteínas de fusión uti | |
EA201792571A1 (ru) | Терапевтическое применение l-4-хлорокинуренина | |
DK3274447T3 (da) | Rekombinant fåresygevirus jeryl lynn 2 baseret vaccine |