EP4705484A1 - Zusammensetzungen und verfahren zur behandlung von tau-bedingten erkrankungen - Google Patents
Zusammensetzungen und verfahren zur behandlung von tau-bedingten erkrankungenInfo
- Publication number
- EP4705484A1 EP4705484A1 EP24727622.3A EP24727622A EP4705484A1 EP 4705484 A1 EP4705484 A1 EP 4705484A1 EP 24727622 A EP24727622 A EP 24727622A EP 4705484 A1 EP4705484 A1 EP 4705484A1
- Authority
- EP
- European Patent Office
- Prior art keywords
- seq
- amino acid
- acid sequence
- sequence
- nucleotide sequence
- 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.)
- Pending
Links
Classifications
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- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1037—Screening libraries presented on the surface of microorganisms, e.g. phage display, E. coli display
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- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1058—Directional evolution of libraries, e.g. evolution of libraries is achieved by mutagenesis and screening or selection of mixed population of organisms
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/525—Virus
- A61K2039/5256—Virus expressing foreign proteins
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
- C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
- C07K2317/33—Crossreactivity, e.g. for species or epitope, or lack of said crossreactivity
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
- C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
- C07K2317/565—Complementarity determining region [CDR]
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
- C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2317/00—Immunoglobulins specific features
- C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
- C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Virology (AREA)
- Neurosurgery (AREA)
- Medicinal Chemistry (AREA)
- Neurology (AREA)
- Hospice & Palliative Care (AREA)
- General Chemical & Material Sciences (AREA)
- Bioinformatics & Computational Biology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Ecology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202363463793P | 2023-05-03 | 2023-05-03 | |
| US202363593638P | 2023-10-27 | 2023-10-27 | |
| US202463564127P | 2024-03-12 | 2024-03-12 | |
| PCT/US2024/027742 WO2024229389A1 (en) | 2023-05-03 | 2024-05-03 | Compositions and methods for the treatment of tau-related disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| EP4705484A1 true EP4705484A1 (de) | 2026-03-11 |
Family
ID=91186590
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP24727622.3A Pending EP4705484A1 (de) | 2023-05-03 | 2024-05-03 | Zusammensetzungen und verfahren zur behandlung von tau-bedingten erkrankungen |
Country Status (2)
| Country | Link |
|---|---|
| EP (1) | EP4705484A1 (de) |
| WO (1) | WO2024229389A1 (de) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3861010A1 (de) | 2018-10-02 | 2021-08-11 | Voyager Therapeutics, Inc. | Umlenkung des tropismus von aav-kapsiden |
| AU2021273447B2 (en) | 2020-05-13 | 2026-03-05 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| IL311861A (en) | 2021-11-02 | 2024-06-01 | Voyager Therapeutics Inc | Aav capsid variants and uses thereof |
| CN120019071A (zh) | 2022-09-15 | 2025-05-16 | 沃雅戈治疗公司 | Tau结合化合物 |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0755454B1 (de) | 1994-04-13 | 2008-02-13 | The Rockefeller University | Aav-vermittelte überbringung von dna in zellen des nervensystems |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| CN107012171B (zh) * | 2011-04-22 | 2021-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| US10975391B2 (en) | 2014-04-25 | 2021-04-13 | University Of Massachusetts | Recombinant AAV vectors useful for reducing immunity against transgene products |
| EP3875593A1 (de) * | 2015-12-11 | 2021-09-08 | California Institute of Technology | Zielpeptide zum leiten von adeno-assoziierten viren (aavs) |
| US20190224339A1 (en) | 2016-04-29 | 2019-07-25 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2018119330A2 (en) | 2016-12-22 | 2018-06-28 | Oregon Health & Science University | Adeno associated viral vectors |
| EP3793686A1 (de) * | 2018-05-16 | 2021-03-24 | Voyager Therapeutics, Inc. | Aav-serotypen zur gehirnspezifischen payload-übertragung |
| EP3861010A1 (de) * | 2018-10-02 | 2021-08-11 | Voyager Therapeutics, Inc. | Umlenkung des tropismus von aav-kapsiden |
| CA3123600A1 (en) | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| TW202102526A (zh) * | 2019-04-04 | 2021-01-16 | 美商銳進科斯生物股份有限公司 | 重組腺相關病毒及其用途 |
| WO2020223276A1 (en) | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tauopathy |
| WO2021202651A1 (en) * | 2020-04-01 | 2021-10-07 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| BR112022020753A2 (pt) | 2020-04-15 | 2022-12-20 | Voyager Therapeutics Inc | Compostos de ligação a tau |
| AU2021273447B2 (en) | 2020-05-13 | 2026-03-05 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| MX2023012513A (es) * | 2021-04-23 | 2023-12-15 | Univ Pennsylvania | Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen. |
| IL311861A (en) * | 2021-11-02 | 2024-06-01 | Voyager Therapeutics Inc | Aav capsid variants and uses thereof |
| US20250034559A1 (en) * | 2021-11-17 | 2025-01-30 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tau-related disorders |
| EP4522757A2 (de) * | 2022-05-13 | 2025-03-19 | Voyager Therapeutics, Inc. | Zusammensetzungen und verfahren zur behandlung von her2-positivem krebs |
| EP4532515A1 (de) * | 2022-06-02 | 2025-04-09 | Voyager Therapeutics, Inc. | Aav-kapsidvarianten und verwendungen davon |
| WO2023250388A1 (en) * | 2022-06-22 | 2023-12-28 | Voyager Therapeutics, Inc. | Tau binding compounds |
| JP2025528068A (ja) * | 2022-08-03 | 2025-08-26 | ボイジャー セラピューティクス インコーポレイテッド | 血液脳関門を通過させるための組成物及び方法 |
-
2024
- 2024-05-03 EP EP24727622.3A patent/EP4705484A1/de active Pending
- 2024-05-03 WO PCT/US2024/027742 patent/WO2024229389A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| WO2024229389A1 (en) | 2024-11-07 |
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Legal Events
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| STAA | Information on the status of an ep patent application or granted ep patent |
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| STAA | Information on the status of an ep patent application or granted ep patent |
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| PUAI | Public reference made under article 153(3) epc to a published international application that has entered the european phase |
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| STAA | Information on the status of an ep patent application or granted ep patent |
Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE |
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| 17P | Request for examination filed |
Effective date: 20251008 |
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