MD3589730T2 - Vector de virus adeno-asociat (AAV) de cladă F și utilizările acestuia - Google Patents
Vector de virus adeno-asociat (AAV) de cladă F și utilizările acestuiaInfo
- Publication number
- MD3589730T2 MD3589730T2 MDE20200060T MDE20200060T MD3589730T2 MD 3589730 T2 MD3589730 T2 MD 3589730T2 MD E20200060 T MDE20200060 T MD E20200060T MD E20200060 T MDE20200060 T MD E20200060T MD 3589730 T2 MD3589730 T2 MD 3589730T2
- Authority
- MD
- Moldova
- Prior art keywords
- vector
- raav
- associated virus
- seq
- adeno
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 abstract 2
- 150000001413 amino acids Chemical group 0.000 abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 2
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 abstract 1
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Natural products NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 abstract 1
- 239000004471 Glycine Substances 0.000 abstract 1
- DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical compound OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-REOHCLBHSA-N 0.000 abstract 1
- CKLJMWTZIZZHCS-REOHCLBHSA-N L-aspartic acid Chemical group OC(=O)[C@@H](N)CC(O)=O CKLJMWTZIZZHCS-REOHCLBHSA-N 0.000 abstract 1
- 235000001014 amino acid Nutrition 0.000 abstract 1
- 229960001230 asparagine Drugs 0.000 abstract 1
- 235000009582 asparagine Nutrition 0.000 abstract 1
- 239000002775 capsule Substances 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 235000018102 proteins Nutrition 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
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
- 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
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- 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
- 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
- C07K16/32—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
-
- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- 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
-
- 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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Microbiology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Mycology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Oncology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
Abstract
Vector de virus recombinant adeno-asociat (rAAV) care cuprinde o capsidă AAVhu68produsă într-un sistem de producţie care cuprinde o secvenţă de nucleotide din ID NO SECV: 1, sau o secvenţă cel puţin 75% identică cu aceasta care codifică ID NO SECV: 2. Capsida AAVhu68 cuprinde subpopulaţii de reziduuri de asparagină puternic deamidate în perechile asparagină - glicină în secvenţa de aminoacizi din ID NO SECV: 2. Se furnizează de asemeni compoziţii care conţin rAAV-ul şi utilizările acestora. Suplimentar, se furnizează rAAV care are o capsulă AVV modificată genetic care cuprinde cel puţin o subpopulaţie de proteine vp1 sau vp2 având o Va1 în poziţia de aminoacid 157 faţă de numărătoarea AAVhu68 vp1.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762464748P | 2017-02-28 | 2017-02-28 | |
US201762591002P | 2017-11-27 | 2017-11-27 | |
US201862614002P | 2018-01-05 | 2018-01-05 | |
PCT/US2018/019992 WO2018160582A1 (en) | 2017-02-28 | 2018-02-27 | Adeno-associated virus (aav) clade f vector and uses therefor |
Publications (1)
Publication Number | Publication Date |
---|---|
MD3589730T2 true MD3589730T2 (ro) | 2024-04-30 |
Family
ID=61628475
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MDE20200060T MD3589730T2 (ro) | 2017-02-28 | 2018-02-27 | Vector de virus adeno-asociat (AAV) de cladă F și utilizările acestuia |
Country Status (25)
Country | Link |
---|---|
US (2) | US11827906B2 (ro) |
EP (2) | EP3589730B1 (ro) |
JP (2) | JP7455579B2 (ro) |
KR (1) | KR20190132639A (ro) |
CN (1) | CN110741082A (ro) |
AU (1) | AU2018227440B2 (ro) |
BR (1) | BR112019017839A2 (ro) |
CA (1) | CA3053399A1 (ro) |
CL (1) | CL2019002474A1 (ro) |
CO (1) | CO2019009525A2 (ro) |
DK (1) | DK3589730T3 (ro) |
ES (1) | ES2971872T3 (ro) |
FI (1) | FI3589730T3 (ro) |
HR (1) | HRP20240257T1 (ro) |
IL (1) | IL268644A (ro) |
LT (1) | LT3589730T (ro) |
MD (1) | MD3589730T2 (ro) |
MX (1) | MX2019010275A (ro) |
PE (1) | PE20191362A1 (ro) |
PT (1) | PT3589730T (ro) |
RS (1) | RS65241B1 (ro) |
SG (4) | SG10201912969XA (ro) |
SI (1) | SI3589730T1 (ro) |
WO (1) | WO2018160582A1 (ro) |
ZA (1) | ZA201905407B (ro) |
Families Citing this family (55)
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HU230406B1 (hu) | 2001-11-13 | 2016-04-28 | The Trustees Of The University Of Pennsylvania | Eljárás adeno-asszociált vírus(AAV)szekvenciák detektálására és/vagy azonosítására és az azzal azonosított új szekvenciák izolálására |
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 |
ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
JP7260898B2 (ja) * | 2016-01-15 | 2023-04-19 | アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド | ガンマデルタt細胞の活性化のための方法および組成物 |
SI3589730T1 (sl) | 2017-02-28 | 2024-04-30 | The Trustees Of The University Of Pennsylvania | Adeno-povezan virus (AAV) clade F vektor in njihova uporaba |
JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
AU2018265531A1 (en) | 2017-05-11 | 2019-11-14 | The Trustees Of The University Of Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
BR112020010735A2 (pt) | 2017-11-30 | 2020-11-10 | The Trustees Of The University Of Pennsylvania | terapia genética para mucopolissacaridose iii a |
US11723989B2 (en) | 2017-11-30 | 2023-08-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis IIIB |
JP7498665B2 (ja) * | 2018-02-27 | 2024-06-12 | 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 | 新規アデノ随伴ウイルス(aav)ベクター、低減されたカプシド脱アミド化を有するaavベクター、およびその使用 |
MX2020008933A (es) * | 2018-02-27 | 2021-01-15 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
KR20210006357A (ko) | 2018-04-03 | 2021-01-18 | 스트라이드바이오 인코포레이티드 | 항체-회피 바이러스 벡터 |
PE20211596A1 (es) * | 2018-10-01 | 2021-08-18 | Univ Pennsylvania | Composiciones utiles para tratar la gangliosidosis gm1 |
UY38407A (es) | 2018-10-15 | 2020-05-29 | Novartis Ag | Anticuerpos estabilizadores de trem2 |
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PE20211819A1 (es) | 2019-02-22 | 2021-09-14 | Univ Pennsylvania | Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn |
US20220118108A1 (en) | 2019-02-26 | 2022-04-21 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
KR20220011616A (ko) | 2019-03-21 | 2022-01-28 | 스트라이드바이오 인코포레이티드 | 재조합 아데노 관련 바이러스 벡터 |
JP2022530095A (ja) * | 2019-04-24 | 2022-06-27 | 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 | レット症候群の治療に有用な組成物 |
EP3962454A4 (en) * | 2019-05-03 | 2023-05-31 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR THE TREATMENT OF METACHROMATIC LEUKODYSTROPHY |
EP4045525A1 (en) | 2019-10-17 | 2022-08-24 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
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US20230365955A1 (en) | 2020-10-09 | 2023-11-16 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
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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 |
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AU2022290521A1 (en) * | 2021-06-08 | 2023-12-21 | The Trustees Of The University Of Pennsylvania | Recombinant adeno-associated viruses for lesch-nyhan disorders and uses thereof |
WO2023056399A1 (en) | 2021-10-02 | 2023-04-06 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
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