BR112020017348A2 - Vetores de vírus adenoassociados (aav), vetores de aav tendo desamidação de capsídeos reduzida e usos dos mesmos - Google Patents
Vetores de vírus adenoassociados (aav), vetores de aav tendo desamidação de capsídeos reduzida e usos dos mesmos Download PDFInfo
- Publication number
- BR112020017348A2 BR112020017348A2 BR112020017348-7A BR112020017348A BR112020017348A2 BR 112020017348 A2 BR112020017348 A2 BR 112020017348A2 BR 112020017348 A BR112020017348 A BR 112020017348A BR 112020017348 A2 BR112020017348 A2 BR 112020017348A2
- Authority
- BR
- Brazil
- Prior art keywords
- hsa
- mir
- capsid
- deamidation
- seq
- Prior art date
Links
Classifications
-
- 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
- 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
- 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
- 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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862635964P | 2018-02-27 | 2018-02-27 | |
| US62/635,964 | 2018-02-27 | ||
| US201862677471P | 2018-05-29 | 2018-05-29 | |
| US201862667585P | 2018-05-29 | 2018-05-29 | |
| US62/667,585 | 2018-05-29 | ||
| US62/677,471 | 2018-05-29 | ||
| US201862703670P | 2018-07-26 | 2018-07-26 | |
| US62/703,670 | 2018-07-26 | ||
| US201862722382P | 2018-08-24 | 2018-08-24 | |
| US62/722,382 | 2018-08-24 | ||
| PCT/US2019/019804 WO2019168961A1 (en) | 2018-02-27 | 2019-02-27 | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112020017348A2 true BR112020017348A2 (pt) | 2020-12-29 |
Family
ID=67805569
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112020017348-7A BR112020017348A2 (pt) | 2018-02-27 | 2019-02-27 | Vetores de vírus adenoassociados (aav), vetores de aav tendo desamidação de capsídeos reduzida e usos dos mesmos |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20200407750A1 (https=) |
| EP (1) | EP3758724A4 (https=) |
| JP (4) | JP2021516046A (https=) |
| KR (1) | KR20210010434A (https=) |
| CN (2) | CN119842635A (https=) |
| AU (1) | AU2019227726B2 (https=) |
| BR (1) | BR112020017348A2 (https=) |
| CA (1) | CA3091806A1 (https=) |
| CL (2) | CL2020002200A1 (https=) |
| IL (1) | IL276859B2 (https=) |
| MX (1) | MX2020008933A (https=) |
| SG (1) | SG11202008182TA (https=) |
| WO (1) | WO2019168961A1 (https=) |
Families Citing this family (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| BR112021006052A2 (pt) | 2018-10-01 | 2021-09-08 | Ultragenyx Pharmaceutical Inc. | Terapia gênica para tratar acidemia propiônica |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| SG11202111375RA (en) | 2019-04-29 | 2021-11-29 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
| WO2021067598A1 (en) | 2019-10-04 | 2021-04-08 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
| EP4048681A4 (en) * | 2019-10-21 | 2024-03-20 | The Trustees of The University of Pennsylvania | AAV3B VARIANTS WITH IMPROVED PRODUCTION YIELD AND LIVER TROPISM |
| CN114902051B (zh) * | 2020-01-03 | 2026-04-17 | 萨勒普塔医疗公司 | 用于分析aav衣壳蛋白的方法 |
| WO2023172491A1 (en) | 2022-03-07 | 2023-09-14 | Ultragenyx Pharmaceutical Inc. | Modified batch aav production systems and methods |
| JP2023514204A (ja) * | 2020-02-14 | 2023-04-05 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | Ｃｄｋｌ５欠損障害を処置するための遺伝子治療 |
| WO2021202532A1 (en) | 2020-03-31 | 2021-10-07 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| KR20230003569A (ko) * | 2020-04-27 | 2023-01-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Cdkl5 결핍 장애 (cdd)의 치료에 유용한 조성물 |
| AU2021292200A1 (en) | 2020-06-17 | 2023-02-02 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of gene therapy patients |
| IL299762A (en) | 2020-07-13 | 2023-03-01 | Univ Pennsylvania | Useful preparations for the treatment of Charcot-Marietot disease |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| WO2022082109A1 (en) * | 2020-10-18 | 2022-04-21 | The Trustees Of The University Of Pennsylvania | Improved adeno-associated virus (aav) vector and uses therefor |
| JP2023550581A (ja) * | 2020-10-29 | 2023-12-04 | 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 | Ａａｖカプシド及びそれを含有する組成物 |
| TW202237850A (zh) | 2020-12-01 | 2022-10-01 | 賓州大學委員會 | 具有組織特異性靶向基序的新穎構成物及含有其之組成物 |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| IL307633A (en) | 2021-04-12 | 2023-12-01 | Univ Pennsylvania | Useful preparations in the treatment of spinal muscular atrophy (SBMA) |
| CA3216004A1 (en) | 2021-04-23 | 2022-10-27 | The Trustees Of The University Of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| AU2022423978A1 (en) * | 2021-12-28 | 2024-07-18 | Chengdu Origen Biotechnology Co., Ltd. | Modified aav capsid protein and use thereof |
| WO2023133574A1 (en) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for treatment of c9orf72-mediated disorders |
| WO2023133584A1 (en) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| CN119317718A (zh) | 2022-01-25 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于改善心脏转导和肝脏去靶向的aav衣壳 |
| WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| CN115011601B (zh) * | 2022-06-27 | 2023-07-21 | 山东大学齐鲁医院 | 一种干扰JUND表达的shRNA、重组腺相关病毒载体及其应用 |
| US20260021205A1 (en) * | 2022-07-15 | 2026-01-22 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| CN116350801A (zh) * | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| EP4634394A2 (en) | 2022-12-17 | 2025-10-22 | The Trustees of The University of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| WO2024138129A2 (en) * | 2022-12-23 | 2024-06-27 | Spark Therapeutics, Inc. | Adeno-associated virus formulations |
| KR20260021096A (ko) * | 2023-05-03 | 2026-02-12 | 보이저 테라퓨틱스, 인크. | Cdkl5 결핍과 관련된 장애의 치료를 위한 조성물 및 방법 |
| WO2024229161A1 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency |
| 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 |
| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
| WO2026074494A1 (en) | 2024-10-03 | 2026-04-09 | Sarepta Therapeutics, Inc. | Modified viral capsids and uses thereof |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2345731B1 (en) * | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) * | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| NZ701693A (en) * | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| IL297919A (en) * | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| WO2017100674A1 (en) * | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| SG11201808812RA (en) * | 2016-04-15 | 2018-11-29 | Univ Pennsylvania | Novel aav8 mutant capsids and compositions containing same |
| CA3033856A1 (en) * | 2016-08-15 | 2018-02-22 | Genzyme Corporation | Methods for detecting aav |
| PT3589730T (pt) * | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
-
2019
- 2019-02-26 IL IL276859A patent/IL276859B2/en unknown
- 2019-02-27 KR KR1020207027457A patent/KR20210010434A/ko not_active Ceased
- 2019-02-27 EP EP19760264.2A patent/EP3758724A4/en active Pending
- 2019-02-27 US US16/975,541 patent/US20200407750A1/en active Pending
- 2019-02-27 MX MX2020008933A patent/MX2020008933A/es unknown
- 2019-02-27 WO PCT/US2019/019804 patent/WO2019168961A1/en not_active Ceased
- 2019-02-27 AU AU2019227726A patent/AU2019227726B2/en active Active
- 2019-02-27 CN CN202411591073.2A patent/CN119842635A/zh active Pending
- 2019-02-27 CA CA3091806A patent/CA3091806A1/en active Pending
- 2019-02-27 SG SG11202008182TA patent/SG11202008182TA/en unknown
- 2019-02-27 CN CN201980028806.2A patent/CN112352050A/zh active Pending
- 2019-02-27 BR BR112020017348-7A patent/BR112020017348A2/pt unknown
- 2019-02-27 JP JP2020545160A patent/JP2021516046A/ja active Pending
-
2020
- 2020-08-26 CL CL2020002200A patent/CL2020002200A1/es unknown
-
2022
- 2022-12-26 CL CL2022003757A patent/CL2022003757A1/es unknown
-
2023
- 2023-08-14 JP JP2023131761A patent/JP2023159235A/ja active Pending
-
2024
- 2024-01-18 JP JP2024005924A patent/JP2024041967A/ja active Pending
-
2025
- 2025-11-07 JP JP2025188265A patent/JP2026027394A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2019227726A1 (en) | 2020-09-10 |
| EP3758724A4 (en) | 2022-07-06 |
| JP2026027394A (ja) | 2026-02-18 |
| MX2020008933A (es) | 2021-01-15 |
| CN112352050A (zh) | 2021-02-09 |
| CA3091806A1 (en) | 2019-09-06 |
| JP2021516046A (ja) | 2021-07-01 |
| SG11202008182TA (en) | 2020-09-29 |
| CL2020002200A1 (es) | 2021-01-29 |
| EP3758724A1 (en) | 2021-01-06 |
| KR20210010434A (ko) | 2021-01-27 |
| IL276859B1 (en) | 2025-08-01 |
| CN119842635A (zh) | 2025-04-18 |
| IL276859A (en) | 2020-10-29 |
| CL2022003757A1 (es) | 2023-05-19 |
| AU2019227726B2 (en) | 2025-09-04 |
| WO2019168961A1 (en) | 2019-09-06 |
| JP2024041967A (ja) | 2024-03-27 |
| US20200407750A1 (en) | 2020-12-31 |
| IL276859B2 (en) | 2025-12-01 |
| JP2023159235A (ja) | 2023-10-31 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B350 | Update of information on the portal [chapter 15.35 patent gazette] | ||
| B06W | Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette] |