NZ701693A - Composition and methods for highly efficient gene transfer using aav capsid variants - Google Patents
Composition and methods for highly efficient gene transfer using aav capsid variantsInfo
- Publication number
- NZ701693A NZ701693A NZ701693A NZ70169313A NZ701693A NZ 701693 A NZ701693 A NZ 701693A NZ 701693 A NZ701693 A NZ 701693A NZ 70169313 A NZ70169313 A NZ 70169313A NZ 701693 A NZ701693 A NZ 701693A
- Authority
- NZ
- New Zealand
- Prior art keywords
- methods
- aav
- composition
- gene transfer
- highly efficient
- 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
-
- 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
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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- 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
- C12N15/63—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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
-
- 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
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Hematology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261635273P | 2012-04-18 | 2012-04-18 | |
| US201361794995P | 2013-03-15 | 2013-03-15 | |
| PCT/US2013/037170 WO2013158879A1 (en) | 2012-04-18 | 2013-04-18 | Composition and methods for highly efficient gene transfer using aav capsid variants |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NZ701693A true NZ701693A (en) | 2017-02-24 |
Family
ID=49384070
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ701693A NZ701693A (en) | 2012-04-18 | 2013-04-18 | Composition and methods for highly efficient gene transfer using aav capsid variants |
Country Status (26)
Families Citing this family (132)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP5702519B2 (ja) * | 2005-04-07 | 2015-04-15 | 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ベクターの機能を上げる方法 |
| CA2720097C (en) | 2007-04-09 | 2016-07-19 | University Of Florida Research Foundation, Inc. | Raav vector compositions having tyrosine-modified capsid proteins and methods for use |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| CN117205331A (zh) | 2011-04-29 | 2023-12-12 | 西莱克塔生物科技公司 | 用于降低抗体应答的致耐受性合成纳米载体 |
| BR112014025985A2 (pt) | 2012-04-18 | 2017-07-11 | Childrens Hospital Philadelphia | composição e métodos para transferência de gene altamente eficaz com o uso de variantes de capsídeo de aav |
| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| CN110639024A (zh) | 2013-05-03 | 2020-01-03 | 西莱克塔生物科技公司 | 用于增强cd4+调节性t细胞的方法和组合物 |
| ES2897508T3 (es) * | 2013-05-31 | 2022-03-01 | Univ California | Variantes de virus adenoasociados y métodos de uso de las mismas |
| MX2016001044A (es) * | 2013-07-22 | 2016-09-09 | The Children's Hospital Of Philadelphia | Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos. |
| AU2014324717B2 (en) | 2013-09-26 | 2020-10-08 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial AAV capsid library for targeted gene therapy |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| US10308957B2 (en) | 2014-03-04 | 2019-06-04 | University Of Florida Research Foundation, Inc. | rAAV vectors and methods for transduction of photoreceptors and RPE cells |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| BR112017001601A2 (pt) | 2014-09-07 | 2017-11-21 | Selecta Biosciences Inc | métodos e composições para atenuar as respostas imunes do vetor de transferência antiviral de modulação da expressão genética |
| JP7023108B2 (ja) | 2014-10-21 | 2022-02-21 | ユニバーシティ オブ マサチューセッツ | 組み換えａａｖバリアントおよびその使用 |
| MX2017005834A (es) | 2014-11-05 | 2017-11-17 | Voyager Therapeutics Inc | Polinucleotidos aad para el tratamiento de la enfermedad de parkinson. |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| SG11201703281RA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| SG10202001102XA (en) | 2014-11-14 | 2020-03-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| WO2016126857A1 (en) | 2015-02-03 | 2016-08-11 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
| US10648000B2 (en) | 2015-02-16 | 2020-05-12 | University Of Florida Research Foundation, Incorporated | rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes |
| WO2016134337A1 (en) * | 2015-02-19 | 2016-08-25 | University Of Florida Research Foundation, Inc. | Site-specific integrating recombinant aav vectors for gene therapy and improved production methods |
| JP6519080B2 (ja) * | 2015-06-22 | 2019-05-29 | ライオン株式会社 | 繊維製品用抗ウイルス組成物 |
| KR102178322B1 (ko) | 2015-06-23 | 2020-11-13 | 더 칠드런스 호스피탈 오브 필라델피아 | 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도 |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| EP4316512A3 (en) | 2015-10-28 | 2024-04-24 | The Trustees of The University of Pennsylvania | Intrathecal administration of adeno-associated-viral vectors for gene therapy |
| PE20231949A1 (es) | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| AU2016362477A1 (en) | 2015-12-02 | 2018-06-14 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
| AU2017248840B2 (en) | 2016-04-16 | 2022-07-28 | University Of Florida Research Foundation, Incorporated | Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| CN109476707B (zh) | 2016-05-13 | 2022-12-02 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和其使用方法 |
| JP7066635B2 (ja) | 2016-05-18 | 2022-05-13 | ボイジャー セラピューティクス インコーポレイテッド | 調節性ポリヌクレオチド |
| WO2017201258A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| ES2863674T3 (es) * | 2016-08-15 | 2021-10-11 | Genzyme Corp | Métodos para detectar AAV |
| US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| WO2018071831A1 (en) | 2016-10-13 | 2018-04-19 | University Of Massachusetts | Aav capsid designs |
| CN110325203A (zh) | 2017-01-07 | 2019-10-11 | 西莱克塔生物科技公司 | 与合成纳米载体偶联的免疫抑制剂的模式化给药 |
| US11028131B2 (en) * | 2017-01-30 | 2021-06-08 | Nippon Medical School Foundation | Mutant of adeno-associated virus (AAV) capsid protein |
| EP3585883A4 (en) | 2017-02-21 | 2021-04-14 | University of Florida Research Foundation, Incorporated | MODIFIED AAV CAPSID PROTEINS AND USES THEREOF |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| MX2019010757A (es) | 2017-03-11 | 2020-01-20 | Selecta Biosciences Inc | Métodos y composiciones relacionados con el tratamiento combinado con antiinflamatorios y nanoportadores sintéticos que comprenden un inmunosupresor. |
| IL316259A (en) | 2017-03-15 | 2024-12-01 | Univ North Carolina Chapel Hill | Polyploid gland associated with virus vectors and methods for preparing and using the same. |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| IL269534B2 (en) | 2017-03-30 | 2025-01-01 | Univ Queensland | Chimeric molecules and their uses |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| AU2018261003A1 (en) | 2017-05-05 | 2019-11-14 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's Disease |
| CA3066358A1 (en) | 2017-06-07 | 2018-12-13 | Spark Therapeutics, Inc. | Enhancing agents for improved cell transfection and/or raav vector production |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN110461368A (zh) | 2017-06-30 | 2019-11-15 | 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 |
| US11497576B2 (en) | 2017-07-17 | 2022-11-15 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| US20210228738A1 (en) | 2017-07-17 | 2021-07-29 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins |
| EP3687561A4 (en) | 2017-09-01 | 2021-06-09 | The Australian National University | "immunoregulatory molecules and uses therefor" |
| ES2948902T3 (es) | 2017-09-20 | 2023-09-21 | 4D Molecular Therapeutics Inc | Variantes de cápsides de virus adenoasociado y procedimientos de utilización de las mismas |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| BR112020007157A2 (pt) | 2017-10-13 | 2020-09-24 | Selecta Biosciences, Inc. | métodos e composições para a atenuação de respostas de igm antivetor de transferência viral |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| CA3083472A1 (en) | 2017-11-27 | 2019-05-31 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and use for inhibiting angiogenesis |
| CN111727243A (zh) | 2017-12-19 | 2020-09-29 | 北卡罗来纳-查佩尔山大学 | 用于跨越血-脑屏障递送病毒载体的方法和组合物 |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| JP2021512871A (ja) | 2018-02-01 | 2021-05-20 | ホモロジー・メディシンズ・インコーポレイテッド | Ｐａｈ遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びそれらの使用方法 |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| KR20200139679A (ko) | 2018-02-26 | 2020-12-14 | 안톨알엑스, 인크. | 면역관용성 리포솜 및 그의 사용 방법 |
| 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. |
| BR112020018354A2 (pt) * | 2018-03-16 | 2020-12-29 | Research Institute At Nationwide Children's Hospital | Aumento de fornecimento gênico específico a tecido através de modificação de capsídeo |
| SG11202009450SA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Virus vectors for targeting ophthalmic tissues |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| US12091435B2 (en) | 2018-04-03 | 2024-09-17 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| BR112020020223A2 (pt) | 2018-04-05 | 2021-01-19 | Genethon | Sorotipo de vírus adeno-associado híbrido recombinante entre aav9 e aavrh74 com tropismo pelo fígado reduzido |
| US12233136B2 (en) | 2018-04-27 | 2025-02-25 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| WO2019210267A2 (en) * | 2018-04-27 | 2019-10-31 | Spark Therapeutics, Inc. | Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same |
| US12319929B2 (en) | 2018-05-15 | 2025-06-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| WO2019222411A1 (en) * | 2018-05-16 | 2019-11-21 | Spark Therapeutics, Inc. | Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same |
| KR20210034015A (ko) | 2018-07-16 | 2021-03-29 | 셀렉타 바이오사이언시즈, 인크. | Mma 구축물 및 벡터의 방법 및 조성물 |
| WO2020018583A1 (en) | 2018-07-16 | 2020-01-23 | Selecta Biosciences, Inc. | Methods and compositions of otc constructs and vectors |
| CN121160803A (zh) | 2018-09-28 | 2025-12-19 | 沃雅戈治疗公司 | 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法 |
| BR112021006052A2 (pt) | 2018-10-01 | 2021-09-08 | Ultragenyx Pharmaceutical Inc. | Terapia gênica para tratar acidemia propiônica |
| US11702673B2 (en) | 2018-10-18 | 2023-07-18 | University Of Florida Research Foundation, Incorporated | Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus |
| JP7578590B2 (ja) | 2018-10-18 | 2024-11-06 | インテリア セラピューティクス，インコーポレーテッド | 第ｉｘ因子を発現するための組成物及び方法 |
| JP7562544B2 (ja) | 2019-02-08 | 2024-10-07 | デシベル セラピューティクス インコーポレイテッド | ミオシン１５プロモーター及びその使用 |
| WO2020163743A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CN113474461A (zh) | 2019-02-25 | 2021-10-01 | 诺华股份有限公司 | 治疗bietti晶体营养不良的组合物和方法 |
| CR20210444A (es) | 2019-02-25 | 2021-11-02 | Novartis Ag | Composiciones y métodos para tratar distrofia cristalina de bietti |
| TW202102525A (zh) | 2019-03-21 | 2021-01-16 | 美商史崔德生物公司 | 重組腺相關病毒載體 |
| CA3138525A1 (en) | 2019-04-28 | 2020-11-05 | Selecta Biosciences, Inc. | Methods for treatment of subjects with preexisting immunity to viral transfer vectors |
| KR20220015425A (ko) | 2019-05-28 | 2022-02-08 | 셀렉타 바이오사이언시즈, 인크. | 약화된 항바이러스 전달 벡터 면역 반응을 위한 방법 및 조성물 |
| RU2751592C2 (ru) | 2019-08-22 | 2021-07-15 | Общество С Ограниченной Ответственностью "Анабион" | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе |
| WO2021067598A1 (en) | 2019-10-04 | 2021-04-08 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| AU2020367532A1 (en) | 2019-10-17 | 2022-05-12 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick disease type C |
| TWI896570B (zh) * | 2019-10-30 | 2025-09-11 | 美商迪賽博治療公司 | 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法 |
| EP4085255A1 (en) | 2020-01-03 | 2022-11-09 | Sarepta Therapeutics, Inc. | Methods for analyzing aav capsid proteins |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| CN115151648A (zh) | 2020-02-14 | 2022-10-04 | 阿尔特拉吉尼克斯制药公司 | 用于治疗cdkl5缺陷障碍的基因疗法 |
| EP4121544A1 (en) | 2020-03-19 | 2023-01-25 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| EP4127189A1 (en) | 2020-03-31 | 2023-02-08 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| BR112023003145A2 (pt) | 2020-08-19 | 2023-05-09 | Sarepta Therapeutics Inc | Vetores de vírus adenoassociado para tratamento da síndrome de rett |
| AR123776A1 (es) | 2020-10-15 | 2023-01-11 | Hoffmann La Roche | Construcciones de ácido nucleico para la activación simultánea de genes |
| CA3197730A1 (en) | 2020-10-15 | 2022-04-21 | F. Hoffman-La Roche Ag | Nucleic acid constructs for va rna transcription |
| EP4274571A1 (en) | 2021-01-05 | 2023-11-15 | Selecta Biosciences, Inc. | Viral vector dosing protocols |
| BR112023021495A2 (pt) * | 2021-04-23 | 2023-12-19 | Univ Florida | Vetores aavrh74 para terapia gênica de distrofias musculares |
| WO2023019168A1 (en) | 2021-08-11 | 2023-02-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
| AR126840A1 (es) | 2021-08-20 | 2023-11-22 | Llc «Anabion» | Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta |
| PE20241995A1 (es) | 2021-08-20 | 2024-09-27 | Biocad Joint Stock Co | Metodo para obtener una capside de virus adenoasociado modificada |
| AR126839A1 (es) | 2021-08-20 | 2023-11-22 | Llc «Anabion» | Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta |
| US20230086710A1 (en) * | 2021-09-14 | 2023-03-23 | California Institute Of Technology | Aav capsid variants |
| WO2023064350A1 (en) | 2021-10-12 | 2023-04-20 | Selecta Biosciences, Inc. | Viral vector dosing protocols |
| US20230141563A1 (en) | 2021-10-12 | 2023-05-11 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector igm responses |
| WO2023086615A1 (en) | 2021-11-14 | 2023-05-19 | Selecta Biosciences, Inc. | Multiple dosing with viral vectors |
| 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 |
| KR20240153391A (ko) | 2022-03-07 | 2024-10-22 | 울트라제닉스 파마수티컬 인코포레이티드 | 변형된 뱃치 aav 생산 시스템 및 방법 |
| WO2023172624A1 (en) | 2022-03-09 | 2023-09-14 | Selecta Biosciences, Inc. | Immunosuppressants in combination with anti-igm agents and related dosing |
| WO2023198685A1 (en) | 2022-04-13 | 2023-10-19 | F. Hoffmann-La Roche Ag | Method for determining aav genomes |
| IL316908A (en) * | 2022-05-11 | 2025-01-01 | Childrens Hospital Philadelphia | Adeno-associated viral vectors for targeting deep brain structures |
| JP2025517987A (ja) | 2022-05-23 | 2025-06-12 | エフ． ホフマン－ラ ロシュ アーゲー | Ａａｖ粒子血清型およびａａｖ粒子負荷状態の識別のためのラマンベースの方法 |
| CA3258165A1 (en) | 2022-06-03 | 2023-12-07 | F. Hoffmann-La Roche Ag | PROCESS FOR THE PRODUCTION OF RECOMBINED ADENOVIRUS-ASSOCIATED VIRUS (AAV) PARTICLES |
| AR129895A1 (es) | 2022-07-14 | 2024-10-09 | Hoffmann La Roche | Método para producir partículas de aav recombinantes |
| CN120813689A (zh) | 2022-08-11 | 2025-10-17 | 笛卡尔疗法股份有限公司 | 与免疫球蛋白蛋白酶及其融合体相关的组合物和方法 |
| JP2025526697A (ja) | 2022-08-15 | 2025-08-15 | エフ． ホフマン－ラ ロシュ アーゲー | 組換えウイルスベクターに関連する有害作用の予防または緩和 |
| IL318766A (en) | 2022-09-12 | 2025-04-01 | Hoffmann La Roche | Method for separating full and empty AAV particles |
| IL322001A (en) | 2023-02-07 | 2025-09-01 | Hoffmann La Roche | Method for detecting antibodies to anti-AAV particles |
| EP4683676A1 (en) | 2023-03-21 | 2026-01-28 | F. Hoffmann-La Roche AG | Method for the production of recombinant aav particle preparations |
| WO2024213596A1 (en) | 2023-04-13 | 2024-10-17 | F. Hoffmann-La Roche Ag | Improved recombinant polyadenylation signal sequences and use thereof |
| WO2025057182A1 (en) * | 2023-09-15 | 2025-03-20 | Indian Institute Of Technology Kanpur | Modified aav vectors for enhanced gene therapy in hemophilia b |
| WO2025082618A1 (en) | 2023-10-20 | 2025-04-24 | Fuse Vectors Aps | In vitro parvovirus capsid manufacturing |
| WO2025168663A1 (en) | 2024-02-09 | 2025-08-14 | F. Hoffmann-La Roche Ag | Method for producing recombinant adeno-associated viral particles |
| WO2025252480A1 (en) | 2024-06-07 | 2025-12-11 | F. Hoffmann-La Roche Ag | Method for purifying plasmid dna |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA1202962A (en) | 1982-10-19 | 1986-04-08 | David P. Clifford | Substituted n-phenyl-n'-benzoyl ureas and their use as insecticides and acaricides |
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| US4965188A (en) | 1986-08-22 | 1990-10-23 | Cetus Corporation | Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme |
| US6127525A (en) | 1995-02-21 | 2000-10-03 | Cornell Research Foundation, Inc. | Chimeric adenoviral coat protein and methods of using same |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| JP4888876B2 (ja) * | 2003-06-13 | 2012-02-29 | 田平 武 | アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター |
| JP5702519B2 (ja) | 2005-04-07 | 2015-04-15 | 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ベクターの機能を上げる方法 |
| US9198984B2 (en) | 2006-04-28 | 2015-12-01 | The Trustees Of The University Of Pennsylvania | Scalable production method for AAV |
| MX342858B (es) * | 2010-03-29 | 2016-10-13 | The Trustees Of The Univ Of Pennsylvania * | Sistema de ablacion transgenica inducida farmacologicamente. |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| BR112014025985A2 (pt) | 2012-04-18 | 2017-07-11 | Childrens Hospital Philadelphia | composição e métodos para transferência de gene altamente eficaz com o uso de variantes de capsídeo de aav |
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