AU2016366549B2 - Targeting peptides for directing adeno-associated viruses (AAVs) - Google Patents
Targeting peptides for directing adeno-associated viruses (AAVs) Download PDFInfo
- Publication number
- AU2016366549B2 AU2016366549B2 AU2016366549A AU2016366549A AU2016366549B2 AU 2016366549 B2 AU2016366549 B2 AU 2016366549B2 AU 2016366549 A AU2016366549 A AU 2016366549A AU 2016366549 A AU2016366549 A AU 2016366549A AU 2016366549 B2 AU2016366549 B2 AU 2016366549B2
- Authority
- AU
- Australia
- Prior art keywords
- aav
- amino acids
- seq
- sequence
- peptide
- Prior art date
- Legal status (The legal status 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 status listed.)
- Ceased
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
-
- 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
-
- 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
- A61K38/19—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
- 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
- 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
-
- 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)
-
- 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
- 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
- A61K38/16—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
- A61K38/43—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
-
- 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
- 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
- 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
- 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 nucleic acids [NA]
- 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
- 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)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Psychology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Pain & Pain Management (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266184P | 2015-12-11 | 2015-12-11 | |
| US62/266,184 | 2015-12-11 | ||
| US201662421891P | 2016-11-14 | 2016-11-14 | |
| US62/421,891 | 2016-11-14 | ||
| PCT/US2016/065969 WO2017100671A1 (en) | 2015-12-11 | 2016-12-09 | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2016366549A1 AU2016366549A1 (en) | 2018-07-12 |
| AU2016366549B2 true AU2016366549B2 (en) | 2022-11-10 |
Family
ID=59013356
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2016366549A Ceased AU2016366549B2 (en) | 2015-12-11 | 2016-12-09 | Targeting peptides for directing adeno-associated viruses (AAVs) |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US11499165B2 (enExample) |
| EP (2) | EP3387137B1 (enExample) |
| JP (1) | JP7066619B2 (enExample) |
| KR (1) | KR102423442B1 (enExample) |
| CN (1) | CN108699565B (enExample) |
| AU (1) | AU2016366549B2 (enExample) |
| BR (1) | BR112018011711A2 (enExample) |
| CA (1) | CA3007495C (enExample) |
| CL (1) | CL2018001522A1 (enExample) |
| CO (1) | CO2018007203A2 (enExample) |
| DK (1) | DK3387137T3 (enExample) |
| ES (1) | ES2869284T3 (enExample) |
| IL (1) | IL259842B (enExample) |
| MX (1) | MX2018006840A (enExample) |
| RU (1) | RU2727672C2 (enExample) |
| SG (1) | SG11201804713VA (enExample) |
| WO (1) | WO2017100671A1 (enExample) |
| ZA (1) | ZA201803808B (enExample) |
Families Citing this family (155)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| JP6754361B2 (ja) * | 2014-12-16 | 2020-09-09 | ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ | 若年型バッテン病のための遺伝子療法 |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| AU2016366549B2 (en) | 2015-12-11 | 2022-11-10 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | 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 |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| US12138298B2 (en) | 2017-01-30 | 2024-11-12 | Brainvectis | Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias |
| US10898585B2 (en) | 2017-04-14 | 2021-01-26 | Ptc Therapeutics .Inc. | Gene therapy for AADC deficiency |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| EP3662060A2 (en) * | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| AU2018320849A1 (en) * | 2017-08-25 | 2020-03-05 | Ovid Therapeutics Inc. | Recombinant adeno-associated vectors |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| CN111601884A (zh) * | 2017-10-16 | 2020-08-28 | 维格内罗有限责任公司 | Aav载体 |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | 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 |
| WO2019090000A1 (en) | 2017-11-02 | 2019-05-09 | 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 |
| JP7430652B2 (ja) | 2018-03-05 | 2024-02-13 | ザ チルドレンズ メディカル センター コーポレーション | 蝸牛および前庭細胞に核酸を送達するための組成物および方法 |
| 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 |
| WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| EP3781174A4 (en) * | 2018-04-09 | 2022-01-26 | Allen Institute | RESCUE OF VOLTAGE CONTROLLED SODIUM CHANNEL FUNCTION IN INHIBITORY NEURONS |
| US12054724B2 (en) | 2018-04-10 | 2024-08-06 | President And Fellows Of Harvard College | AAV vectors encoding clarin-1 or GJB2 and uses thereof |
| 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 |
| BR112020021903A2 (pt) | 2018-04-27 | 2021-03-02 | Rocket Pharmaceuticals, Ltd. | terapia genética para degeneração do snc |
| TW202015742A (zh) | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | 投遞腺相關病毒(aav)之組成物和方法 |
| MX2020012077A (es) | 2018-05-15 | 2021-03-09 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de parkinson. |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| EP3820885A4 (en) * | 2018-07-11 | 2022-04-20 | The Brigham & Women's Hospital, Inc. | METHODS AND COMPOSITIONS FOR ADMINISTRATION OF DRUGS ACROSS THE BLOOD-BRAIN BARRIER |
| WO2020023612A1 (en) | 2018-07-24 | 2020-01-30 | 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 |
| KR20210084459A (ko) | 2018-09-26 | 2021-07-07 | 캘리포니아 인스티튜트 오브 테크놀로지 | 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물 |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| AU2019354995B2 (en) | 2018-10-02 | 2024-12-12 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| EP3861113A1 (en) | 2018-10-04 | 2021-08-11 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| EP3861107A1 (en) | 2018-10-05 | 2021-08-11 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| EP3864150A4 (en) | 2018-10-08 | 2022-08-24 | Allen Institute | Artificial expression constructs for selectively modulating gene expression in interneurons |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| JP2022505106A (ja) | 2018-10-15 | 2022-01-14 | ボイジャー セラピューティクス インコーポレイテッド | バキュロウイルス/Sf9システムにおけるrAAVの大規模産生のための発現ベクター |
| EP3873511B1 (en) | 2018-10-29 | 2022-12-07 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Expression vector for cholesterol 24-hydrolase in therapy of amyotrophic lateral sclerosis |
| US20210393713A1 (en) * | 2018-11-20 | 2021-12-23 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for targeting the blood-brain barrier |
| CN113631225A (zh) | 2019-01-18 | 2021-11-09 | 沃雅戈治疗公司 | 用于生产aav颗粒的方法和系统 |
| CA3128205A1 (en) * | 2019-01-30 | 2020-08-06 | The Broad Institute, Inc. | Systems for evolved adeno-associated viruses (aavs) for targeted delivery |
| 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 |
| US20230193315A1 (en) * | 2019-01-31 | 2023-06-22 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of aav capsids |
| EP3923995A4 (en) * | 2019-02-15 | 2023-05-17 | Allen Institute | ARTIFICIAL EXPRESSION RECOMBINANTS FOR THE SELECTIVE MODULATION OF GENE EXPRESSION IN SELECTED NEURON CELL POPULATIONS |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| AU2020248116B2 (en) * | 2019-03-28 | 2025-12-04 | President And Fellows Of Harvard College | Engineered adeno-associated (AAV) vectors for transgene expression |
| KR20220007056A (ko) * | 2019-04-11 | 2022-01-18 | 캘리포니아 인스티튜트 오브 테크놀로지 | 뇌에서 증진된 특이성을 갖는 바이러스 조성물 |
| 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 |
| BR112021021720A2 (pt) * | 2019-04-30 | 2021-12-28 | Univ Pennsylvania | Composições úteis para tratamento de doença de pompe |
| EP3966227A1 (en) | 2019-05-07 | 2022-03-16 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| JP7624410B2 (ja) * | 2019-05-21 | 2025-01-30 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター |
| US20220281922A1 (en) * | 2019-08-02 | 2022-09-08 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| WO2021030125A1 (en) | 2019-08-09 | 2021-02-18 | 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 |
| CA3151087A1 (en) * | 2019-08-29 | 2021-03-04 | The Board Of Trustees Of The Leland Stanford Junior University | Adeno-associated viral vectors for crossing the human blood brain barrier |
| WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| GB201912863D0 (en) * | 2019-09-06 | 2019-10-23 | Babraham Inst | Novel method |
| GB201913974D0 (en) * | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| WO2021076983A1 (en) * | 2019-10-17 | 2021-04-22 | The Penn State Research Foundation | Regenerating functional neurons for treatment of spinal cord injury and als |
| TW202128736A (zh) * | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療c型尼曼—匹克病之腺相關病毒載體 |
| MX2022004353A (es) * | 2019-10-17 | 2022-07-19 | Casete de transferencia de aav. | |
| AU2020388634A1 (en) * | 2019-11-19 | 2022-06-23 | AskBio Inc. | Therapeutic adeno-associated virus comprising liver-specific promoters for treating Pompe disease and lysosomal disorders |
| MX2022006188A (es) * | 2019-11-22 | 2022-08-22 | Childrens Hospital Philadelphia | Variantes del vector viral adenoasociado. |
| AU2021206256A1 (en) | 2020-01-10 | 2022-07-28 | The Brigham And Women's Hospital, Inc. | Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer |
| WO2021163357A2 (en) | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| MY209860A (en) | 2020-02-13 | 2025-08-07 | Tenaya Therapeutics Inc | Gene therapy vectors for treating heart disease |
| US20230220014A1 (en) * | 2020-04-20 | 2023-07-13 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| 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 |
| EP4142800A1 (en) * | 2020-04-28 | 2023-03-08 | Genethon | Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders |
| CA3181623A1 (en) | 2020-04-29 | 2021-11-04 | The Broad Institute, Inc. | Machine learning accelerated protein engineering through fitness prediction |
| EP4143305A4 (en) * | 2020-05-01 | 2024-09-11 | The Broad Institute, Inc. | GENETICALLY MODIFIED CENTRAL NERVOUS SYSTEM COMPOSITIONS |
| US20230203102A1 (en) * | 2020-05-13 | 2023-06-29 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| 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 |
| CA3190309A1 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| US20230285596A1 (en) | 2020-07-27 | 2023-09-14 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| GB202011871D0 (en) * | 2020-07-30 | 2020-09-16 | Cambridge Entpr Ltd | Composition and method |
| BR112023001336A2 (pt) | 2020-08-05 | 2023-02-14 | Spacecraft Seven Llc | Terapia gênica de csrp3 (proteína 3 rica em cisteína e glicina) |
| US20230295656A1 (en) | 2020-08-06 | 2023-09-21 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| CN116194154A (zh) | 2020-08-07 | 2023-05-30 | 太空飞船七有限责任公司 | 使用aav载体的plakophilin-2(pkp2)基因疗法 |
| EP4196494A1 (en) | 2020-08-17 | 2023-06-21 | Massachusetts Institute of Technology | Shank3 gene therapy approaches |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| 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 |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| IL301525A (en) * | 2020-09-21 | 2023-05-01 | Asklepios Biopharmaceutical Inc | Methods for treating neurological disease |
| 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 |
| AU2021363098A1 (en) | 2020-10-15 | 2023-05-18 | F. Hoffmann-La Roche Ag | Nucleic acid constructs for simultaneous gene activation |
| TW202233584A (zh) | 2020-11-11 | 2022-09-01 | 歐洲分子生物學實驗室 | 用於基因治療之經修飾病毒顆粒 |
| EP4256065A2 (en) | 2020-12-01 | 2023-10-11 | The Trustees of The University of Pennsylvania | Novel compositions with tissue-specific targeting motifs and compositions containing same |
| WO2022187473A2 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| US20240141378A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| JP2024511808A (ja) * | 2021-03-29 | 2024-03-15 | 南京大学 | ウイルスベクターに基づくrna送達システム及びその使用 |
| WO2022221421A2 (en) * | 2021-04-13 | 2022-10-20 | Capsida, Inc. | Aav compositions with high brain expression for treating mucopolysaccharidosis ii |
| WO2022221529A1 (en) | 2021-04-16 | 2022-10-20 | Asklepios Biopharmaceutical, Inc. | Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response |
| 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 |
| AU2022307365A1 (en) | 2021-07-08 | 2024-02-01 | Tenaya Therapeutics, Inc. | Optimized expression cassettes for gene therapy |
| WO2023049710A1 (en) * | 2021-09-21 | 2023-03-30 | California Institute Of Technology | Viral compositions for targeting the nervous system and the lung |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| WO2023073526A1 (en) | 2021-10-25 | 2023-05-04 | Novartis Ag | Methods for improving adeno-associated virus (aav) delivery |
| KR20240113624A (ko) | 2021-11-02 | 2024-07-22 | 보이저 테라퓨틱스, 인크. | Aav 캡시드 변이체 및 이의 용도 |
| WO2023093905A1 (zh) | 2021-11-29 | 2023-06-01 | 上海瑞宏迪医药有限公司 | Aadc、gdnf多核苷酸及其用于治疗帕金森病 |
| 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 |
| WO2023155918A1 (zh) | 2022-02-21 | 2023-08-24 | 上海瑞宏迪医药有限公司 | Vegf结合分子及其医药用途 |
| EP4482970A1 (en) | 2022-02-23 | 2025-01-01 | Massachusetts Institute of Technology | Methods for upregulating shank3 expression |
| JP2025514794A (ja) * | 2022-04-22 | 2025-05-09 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | ヒト上衣特異的プロモーターおよびその使用方法 |
| 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 |
| TW202421787A (zh) | 2022-09-06 | 2024-06-01 | 美商特納亞治療股份有限公司 | 保護心臟之心臟病療法 |
| 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 |
| WO2024175604A1 (en) | 2023-02-21 | 2024-08-29 | Institut National de la Santé et de la Recherche Médicale | Methods and pharmaceutical composition for the treatment of sanfilippo syndrome type iiib |
| CN120936709A (zh) * | 2023-03-09 | 2025-11-11 | 新加坡科技研究局 | 经工程化以增强组织转导和特异性的腺相关病毒的新组合物 |
| TW202502803A (zh) | 2023-03-10 | 2025-01-16 | 美商戴諾治療公司 | 衣殼多肽及其使用方法 |
| WO2024194491A1 (en) | 2023-03-22 | 2024-09-26 | Ospedale San Raffaele S.R.L. | Gene therapy |
| AU2024242380A1 (en) * | 2023-03-31 | 2025-11-06 | Exegenesis Bio Singapore Pte. Ltd. | Variant aav9 capsid proteins and uses thereof |
| WO2024215653A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Guide rnas, vectors, and virions for targeting mutations in the pln gene |
| WO2024215655A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Cardioprotective bag3 therapies |
| WO2024229389A1 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tau-related disorders |
| WO2024229425A1 (en) * | 2023-05-04 | 2024-11-07 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2024233422A1 (en) | 2023-05-05 | 2024-11-14 | Massachusetts Institute Of Technology | Shank3 gene therapy approaches |
| WO2024238807A2 (en) | 2023-05-16 | 2024-11-21 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| WO2025021839A1 (en) | 2023-07-25 | 2025-01-30 | Institut National de la Santé et de la Recherche Médicale | Method to treat metabolic disorders |
| WO2025032158A1 (en) | 2023-08-08 | 2025-02-13 | Institut National de la Santé et de la Recherche Médicale | Method to treat tauopathies |
| TW202525832A (zh) | 2023-08-31 | 2025-07-01 | 美商戴諾治療公司 | 衣殼多肽及其使用方法(二) |
| 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 |
| WO2025073890A1 (en) | 2023-10-06 | 2025-04-10 | Institut National de la Santé et de la Recherche Médicale | Method to capture circulating tumor extracellular vesicles |
| GB202316264D0 (en) | 2023-10-24 | 2023-12-06 | Ospendale San Raffaele S R L | Gene therapy |
| WO2025090858A1 (en) | 2023-10-27 | 2025-05-01 | Biogen Ma Inc. | Methods for identifying aav capsid variants with desired characteristics |
| US20250152744A1 (en) * | 2023-11-15 | 2025-05-15 | Genzyme Corporation | Methods of treating neurodegenerative disorders |
| WO2025157848A1 (en) | 2024-01-23 | 2025-07-31 | Institut National de la Santé et de la Recherche Médicale | Methods for the intravenous treatment of sanfilippo syndrome type iiib |
| WO2025171227A1 (en) | 2024-02-08 | 2025-08-14 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2025186293A1 (en) | 2024-03-06 | 2025-09-12 | Institut National de la Santé et de la Recherche Médicale | Chmp2b mutants for use for treating infection |
| GB202406331D0 (en) | 2024-05-07 | 2024-06-19 | Sania Rx Ltd | Capsid |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009012176A2 (en) * | 2007-07-14 | 2009-01-22 | The University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| US20150079038A1 (en) * | 2013-09-13 | 2015-03-19 | California Institute Of Technology | Selective recovery |
| WO2015168666A2 (en) * | 2014-05-02 | 2015-11-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
Family Cites Families (29)
| 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 |
| AU722375B2 (en) | 1996-09-06 | 2000-08-03 | Trustees Of The University Of Pennsylvania, The | 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 |
| US6429001B1 (en) | 2000-01-26 | 2002-08-06 | Chiron Corporation | Recombinant AAV packaging systems |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| US6410271B1 (en) | 2000-06-23 | 2002-06-25 | Genetastix Corporation | Generation of highly diverse library of expression vectors via homologous recombination in yeast |
| US20070172460A1 (en) * | 2003-03-19 | 2007-07-26 | Jurgen Kleinschmidt | 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 |
| EP2298926A1 (en) * | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| WO2006099615A2 (en) | 2005-03-16 | 2006-09-21 | The Johns Hopkins University | Adenoviral fiber exchange shuttle system |
| US8512981B2 (en) * | 2006-06-21 | 2013-08-20 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV |
| EP2132309A4 (en) | 2007-02-23 | 2011-01-05 | Univ Florida | COMPOSITIONS AND METHODS OF TREATING GLYCOLOGICAL MEMORY DISEASES |
| US9018138B2 (en) | 2007-08-16 | 2015-04-28 | The Johns Hopkins University | Compositions and methods for generating and screening adenoviral libraries |
| US20110223635A1 (en) | 2008-08-11 | 2011-09-15 | Karl Deisseroth | Method and Composition for Controlling Gene Expression |
| EP3514232A1 (en) | 2010-04-23 | 2019-07-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| JP6091435B2 (ja) | 2011-02-22 | 2017-03-08 | カリフォルニア インスティチュート オブ テクノロジー | アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達 |
| RS66184B1 (sr) | 2011-04-22 | 2024-12-31 | Univ California | Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| MX369156B (es) | 2013-09-19 | 2019-10-30 | Janssen Vaccines & Prevention Bv | Formulaciones de adenovirus mejoradas. |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| AU2016366549B2 (en) | 2015-12-11 | 2022-11-10 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
| EP3619310A4 (en) * | 2017-05-05 | 2021-01-27 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| AU2018261790B2 (en) * | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| WO2018204803A1 (en) * | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| EP3662060A2 (en) * | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
-
2016
- 2016-12-09 AU AU2016366549A patent/AU2016366549B2/en not_active Ceased
- 2016-12-09 WO PCT/US2016/065969 patent/WO2017100671A1/en not_active Ceased
- 2016-12-09 JP JP2018530606A patent/JP7066619B2/ja active Active
- 2016-12-09 MX MX2018006840A patent/MX2018006840A/es unknown
- 2016-12-09 KR KR1020187019747A patent/KR102423442B1/ko active Active
- 2016-12-09 EP EP16873985.2A patent/EP3387137B1/en active Active
- 2016-12-09 IL IL259842A patent/IL259842B/en unknown
- 2016-12-09 CA CA3007495A patent/CA3007495C/en active Active
- 2016-12-09 RU RU2018120736A patent/RU2727672C2/ru active
- 2016-12-09 DK DK16873985.2T patent/DK3387137T3/da active
- 2016-12-09 SG SG11201804713VA patent/SG11201804713VA/en unknown
- 2016-12-09 BR BR112018011711A patent/BR112018011711A2/pt active Search and Examination
- 2016-12-09 EP EP21152352.7A patent/EP3875593A1/en not_active Withdrawn
- 2016-12-09 CN CN201680081708.1A patent/CN108699565B/zh not_active Expired - Fee Related
- 2016-12-09 CO CONC2018/0007203A patent/CO2018007203A2/es unknown
- 2016-12-09 US US15/374,596 patent/US11499165B2/en active Active
- 2016-12-09 ES ES16873985T patent/ES2869284T3/es active Active
-
2018
- 2018-06-07 CL CL2018001522A patent/CL2018001522A1/es unknown
- 2018-06-07 ZA ZA201803808A patent/ZA201803808B/en unknown
-
2022
- 2022-10-17 US US18/047,233 patent/US20230295659A1/en active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009012176A2 (en) * | 2007-07-14 | 2009-01-22 | The University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| US20150079038A1 (en) * | 2013-09-13 | 2015-03-19 | California Institute Of Technology | Selective recovery |
| WO2015168666A2 (en) * | 2014-05-02 | 2015-11-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
Also Published As
| Publication number | Publication date |
|---|---|
| BR112018011711A2 (pt) | 2018-12-04 |
| EP3387137A4 (en) | 2019-06-12 |
| US11499165B2 (en) | 2022-11-15 |
| EP3387137A1 (en) | 2018-10-17 |
| KR20180097629A (ko) | 2018-08-31 |
| MX2018006840A (es) | 2019-03-28 |
| KR102423442B1 (ko) | 2022-07-20 |
| US20230295659A1 (en) | 2023-09-21 |
| RU2727672C2 (ru) | 2020-07-22 |
| JP7066619B2 (ja) | 2022-05-13 |
| ES2869284T3 (es) | 2021-10-25 |
| US20170166926A1 (en) | 2017-06-15 |
| HK1257563A1 (en) | 2019-10-25 |
| AU2016366549A1 (en) | 2018-07-12 |
| CA3007495C (en) | 2023-04-11 |
| CA3007495A1 (en) | 2017-06-15 |
| CO2018007203A2 (es) | 2018-09-20 |
| EP3875593A1 (en) | 2021-09-08 |
| RU2018120736A (ru) | 2020-01-13 |
| CN108699565A (zh) | 2018-10-23 |
| SG11201804713VA (en) | 2018-07-30 |
| RU2018120736A3 (enExample) | 2020-01-13 |
| IL259842A (en) | 2018-07-31 |
| CL2018001522A1 (es) | 2018-12-21 |
| ZA201803808B (en) | 2020-11-25 |
| EP3387137B1 (en) | 2021-02-03 |
| IL259842B (en) | 2022-08-01 |
| CN108699565B (zh) | 2023-08-08 |
| WO2017100671A1 (en) | 2017-06-15 |
| JP2019506141A (ja) | 2019-03-07 |
| DK3387137T3 (da) | 2021-05-03 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20230295659A1 (en) | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) | |
| US11932668B2 (en) | Selective recovery | |
| JPWO2014103957A1 (ja) | Aav変異体 | |
| US12421526B2 (en) | AAV mutant having brain-targeting property | |
| US20210087560A1 (en) | Oligodendrocyte-specific promoter, mirna specific to plp1 gene,vector including said promoter and/or mirna, and pharmaceutical composition including said vector | |
| HK1257563B (en) | Targeting peptides for directing adeno-associated viruses (aavs) | |
| CN116761812A (zh) | Neurod1和dlx2载体 | |
| EP4658800A1 (en) | An aav9 capsid variant for targeted gene transfer | |
| CN116710566A (zh) | Neurod1载体 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FGA | Letters patent sealed or granted (standard patent) | ||
| MK14 | Patent ceased section 143(a) (annual fees not paid) or expired |