AR129733A1 - Variantes de cápsides de aav y sus usos - Google Patents
Variantes de cápsides de aav y sus usosInfo
- Publication number
- AR129733A1 AR129733A1 ARP230101649A ARP230101649A AR129733A1 AR 129733 A1 AR129733 A1 AR 129733A1 AR P230101649 A ARP230101649 A AR P230101649A AR P230101649 A ARP230101649 A AR P230101649A AR 129733 A1 AR129733 A1 AR 129733A1
- Authority
- AR
- Argentina
- Prior art keywords
- acid sequence
- amino acid
- seq
- cell
- capsid variant
- Prior art date
Links
Classifications
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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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- 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
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
- G01N33/569—Immunoassay; Biospecific binding assay; Materials therefor for microorganisms, e.g. protozoa, bacteria, viruses
- G01N33/56983—Viruses
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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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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/005—Assays involving biological materials from specific organisms or of a specific nature from viruses
- G01N2333/01—DNA viruses
- G01N2333/075—Adenoviridae
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Virology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- Plant Pathology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
- Cell Biology (AREA)
- Tropical Medicine & Parasitology (AREA)
- Food Science & Technology (AREA)
- Analytical Chemistry (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Abstract
Reivindicación 1: Una variante de la cápside de AAV5 caracterizada porque comprende la secuencia de aminoácidos de las posiciones 193 - 724 de la SEQ ID Nº 982, o una secuencia de aminoácidos al menos 95% idéntica a esta, en donde la variante de la cápside de AAV comprende una N en la posición 578 y una A en la posición 580, numeradas de acuerdo con la SEQ ID Nº 982. Reivindicación 2: Una variante de la cápside de AAV5 caracterizada porque comprende la secuencia de aminoácidos de las posiciones 137 - 724 de la SEQ ID Nº 982, o una secuencia de aminoácidos al menos 95% idéntica a esta, en donde la variante de la cápside de AAV comprende una N en la posición 578 y una A en la posición 580, numeradas de acuerdo con la SEQ ID Nº 982. Reivindicación 3: Una variante de la cápside de AAV5 caracterizada porque comprende la secuencia de aminoácidos de la SEQ ID Nº 982, o una secuencia de aminoácidos al menos 95% idéntica a esta, en donde la variante de la cápside de AAV comprende una N en la posición 578 y una A en la posición 580, numeradas de acuerdo con la SEQ ID Nº 982. Reivindicación 6: Una variante de la cápside de AAV5 caracterizada porque comprende la secuencia de aminoácidos de las posiciones 193 - 724 de la SEQ ID Nº 982, o una secuencia de aminoácidos al menos 95% idéntica a esta, en donde la variante de la cápside de AAV comprende una N en la posición 578, A en la posición 580, Q en la posición 581, A en la posición 582, e Y en la posición 583, numeradas de acuerdo con la SEQ ID Nº 982. Reivindicación 7: Una variante de la cápside de AAV5 caracterizada porque comprende la secuencia de aminoácidos de las posiciones 137 - 724 de la SEQ ID Nº 982, o una secuencia de aminoácidos al menos 95% idéntica a esta, en donde la variante de la cápside de AAV comprende una N en la posición 578, A en la posición 580, Q en la posición 581, A en la posición 582, e Y en la posición 583, numeradas de acuerdo con a SEQ ID Nº 982. Reivindicación 8: Una variante de la cápside de AAV5 caracterizada porque comprende la secuencia de aminoácidos de la SEQ ID Nº 982, o una secuencia de aminoácidos al menos 95% idéntica a esta, en donde la variante de la cápside de AAV comprende una N en la posición 578, A en la posición 580, Q en la posición 581, A en la posición 582, e Y en la posición 583, numeradas de acuerdo con la SEQ ID Nº 982. Reivindicación 17: Un péptido, caracterizado porque comprende: (a) la secuencia de aminoácidos de la SEQ ID Nº 943; (b) una secuencia de aminoácidos que comprende al menos 4 o 5 aminoácidos consecutivos de la SEQ ID Nº 943; (c) una secuencia de aminoácidos que comprende uno, dos o tres, pero no más de cuatro, aminoácidos diferentes, con respecto a la secuencia de aminoácidos de la SEQ ID Nº 943; o (d) una secuencia de aminoácidos que comprende una, dos o tres, pero no más de cuatro, modificaciones, por ejemplo, sustituciones (por ejemplo, sustituciones conservadoras), inserciones o eliminaciones, con respecto a la secuencia de aminoácidos de la SEQ ID Nº 943. Reivindicación 29: Una célula, por ejemplo, una célula hospedadora, caracterizada porque comprende la variante de la cápside de AAV5 de cualquiera de las reivindicaciones 1 - 14, 18, o 27, el polinucleótido de cualquiera de las reivindicaciones 15, 16 o 27, un polinucleótido que codifica el péptido de la reivindicación 17 o 27, la partícula de AAV de cualquiera de las reivindicaciones 19 - 27, o el vector de la reivindicación 28, opcionalmente en donde: (i) la célula es una célula de mamífero o una célula de insecto; (ii) la célula es una célula de una región del cerebro o una región de la médula espinal, opcionalmente una célula de la ínsula de Reil, corteza motora, putamen, tálamo, o cerebelo (por ejemplo, la capa molecular y granular del cerebelo); y/o (iii) la célula es una neurona, una neurona sensorial, una neurona motora, un astrocito, una célula glial, un oligodendrocito o una célula muscular (por ejemplo, una célula del corazón, diafragma, o cuádriceps). Reivindicación 46: Uso de la composición farmacéutica de la reivindicación 31, la partícula de AAV de cualquiera de las reivindicaciones 19 - 27, una partícula de AAV que comprende la variante de la cápside de AAV de cualquiera de las reivindicaciones 1 - 14, 18, o 27, o una partícula de AAV que comprende el péptido de la reivindicación 17 o 27, en la fabricación de un medicamento para el tratamiento de un trastorno genético, un trastorno neurológico, un trastorno neurodegenerativo, un trastorno muscular, una distrofia muscular, un trastorno neuromuscular, o un trastorno neurooncológico.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263356337P | 2022-06-28 | 2022-06-28 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR129733A1 true AR129733A1 (es) | 2024-09-25 |
Family
ID=87556475
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP230101649A AR129733A1 (es) | 2022-06-28 | 2023-06-27 | Variantes de cápsides de aav y sus usos |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20250215453A1 (es) |
| EP (1) | EP4547690A1 (es) |
| AR (1) | AR129733A1 (es) |
| CA (1) | CA3259902A1 (es) |
| TW (1) | TW202417467A (es) |
| WO (1) | WO2024006741A1 (es) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| IL311861A (en) | 2021-11-02 | 2024-06-01 | Voyager Therapeutics Inc | Aav capsid variants and uses thereof |
| WO2025137219A1 (en) * | 2023-12-21 | 2025-06-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025147436A1 (en) * | 2024-01-03 | 2025-07-10 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
Family Cites Families (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2640638B1 (fr) | 1988-12-20 | 1991-02-15 | Commissariat Energie Atomique | Bioreacteur et dispositif pour la culture de cellules animales |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6281010B1 (en) | 1995-06-05 | 2001-08-28 | The Trustees Of The University Of Pennsylvania | Adenovirus gene therapy vehicle and cell line |
| US5756283A (en) | 1995-06-05 | 1998-05-26 | The Trustees Of The University Of Pennsylvania | Method for improved production of recombinant adeno-associated viruses for gene therapy |
| US6506379B1 (en) | 1995-06-07 | 2003-01-14 | Ariad Gene Therapeutics, Inc. | Intramuscular delivery of recombinant AAV |
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| WO1998022588A2 (en) | 1996-11-20 | 1998-05-28 | Introgen Therapeutics, Inc. | An improved method for the production and purification of adenoviral vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| AU9319198A (en) | 1997-09-19 | 1999-04-05 | Trustees Of The University Of Pennsylvania, The | Methods and vector constructs useful for production of recombinant aav |
| EP1015619A1 (en) | 1997-09-19 | 2000-07-05 | The Trustees Of The University Of Pennsylvania | Methods and cell line useful for production of recombinant adeno-associated viruses |
| US6953690B1 (en) | 1998-03-20 | 2005-10-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| ES2235470T3 (es) | 1998-03-20 | 2005-07-01 | The Trustees Of The University Of Pennsylvania | Composiciones y metodos para la produccion libre de cooperadores de virus adnoasociados recombinantes. |
| DE69941905D1 (de) | 1998-11-10 | 2010-02-25 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| JP4693244B2 (ja) | 1999-03-18 | 2011-06-01 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法 |
| JP2003501067A (ja) | 1999-06-02 | 2003-01-14 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ヘルパーウイルスを要求する組み換えウイルス産生のために有用な組成物及び方法 |
| US6365394B1 (en) | 1999-09-29 | 2002-04-02 | The Trustees Of The University Of Pennsylvania | Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus |
| US6399385B1 (en) | 1999-09-29 | 2002-06-04 | The Trustees Of The University Of Pennsylvania | Methods for rapid PEG-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor |
| JP4677187B2 (ja) | 2001-11-13 | 2011-04-27 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 新規なアデノ随伴ウイルス（ａａｖ）７配列、それを含むベクターおよびそれらの使用 |
| US7605249B2 (en) | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| PL2277996T3 (pl) | 2003-05-21 | 2015-03-31 | Genzyme Corp | Sposoby wytwarzania preparatów rekombinowanych wirionów AAV zasadniczo niezawierających pustych kapsydów |
| HUE034597T2 (en) | 2003-06-19 | 2018-02-28 | Genzyme Corp | Reduced immunoreactivity of AAV virions and their applications |
| US7291498B2 (en) | 2003-06-20 | 2007-11-06 | The Trustees Of The University Of Pennsylvania | Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses |
| ES2478625T3 (es) | 2003-06-20 | 2014-07-22 | The Trustees Of The University Of Pennsylvania | Método de generación de adenovirus quiméricos y usos de tales adenovirus quiméricos |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| NZ545628A (en) | 2003-09-30 | 2009-04-30 | Univ Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor |
| WO2005072364A2 (en) | 2004-01-27 | 2005-08-11 | University Of Florida | A modified baculovirus expression system for production of pseudotyped raav vector |
| CA2566286A1 (en) | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same |
| US7427396B2 (en) | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
| CN101287837B (zh) | 2005-10-20 | 2015-03-18 | 尤尼克尔生物制药股份有限公司 | 昆虫细胞中生产的改进的aav载体 |
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| EP3134522B1 (en) | 2014-04-25 | 2021-10-06 | University of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| BR112021011143A2 (pt) | 2018-12-21 | 2022-01-25 | Univ Pennsylvania | Composições para redução específica de drg da expressão de transgene |
| EP3947700A4 (en) * | 2019-04-01 | 2023-01-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| WO2021202651A1 (en) | 2020-04-01 | 2021-10-07 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| CA3182970A1 (en) | 2020-05-13 | 2021-11-18 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| US20230048732A1 (en) | 2020-05-26 | 2023-02-16 | Shape Therapeutics Inc. | High throughput engineering of functional aav capsids |
-
2023
- 2023-06-27 CA CA3259902A patent/CA3259902A1/en active Pending
- 2023-06-27 TW TW112123932A patent/TW202417467A/zh unknown
- 2023-06-27 US US18/876,521 patent/US20250215453A1/en active Pending
- 2023-06-27 AR ARP230101649A patent/AR129733A1/es unknown
- 2023-06-27 EP EP23751189.4A patent/EP4547690A1/en not_active Withdrawn
- 2023-06-27 WO PCT/US2023/069146 patent/WO2024006741A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| EP4547690A1 (en) | 2025-05-07 |
| US20250215453A1 (en) | 2025-07-03 |
| TW202417467A (zh) | 2024-05-01 |
| CA3259902A1 (en) | 2024-01-04 |
| WO2024006741A1 (en) | 2024-01-04 |
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