SG11201907298WA - Methods and compositions for gene transfer across the vasculature - Google Patents
Methods and compositions for gene transfer across the vasculatureInfo
- Publication number
- SG11201907298WA SG11201907298WA SG11201907298WA SG11201907298WA SG11201907298WA SG 11201907298W A SG11201907298W A SG 11201907298WA SG 11201907298W A SG11201907298W A SG 11201907298WA SG 11201907298W A SG11201907298W A SG 11201907298WA SG 11201907298W A SG11201907298W A SG 11201907298WA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- north
- pct
- publication
- date
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 2
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 210000005166 vasculature Anatomy 0.000 title abstract 2
- 241000700605 Viruses Species 0.000 abstract 4
- 108090000565 Capsid Proteins Proteins 0.000 abstract 2
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 239000013598 vector Substances 0.000 abstract 2
- 101100064317 Arabidopsis thaliana DTX41 gene Proteins 0.000 abstract 1
- 235000014036 Castanea Nutrition 0.000 abstract 1
- 241001070941 Castanea Species 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 210000003618 cortical neuron Anatomy 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 238000012986 modification Methods 0.000 abstract 1
- 230000004048 modification Effects 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 230000026683 transduction Effects 0.000 abstract 1
- 238000010361 transduction Methods 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- 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
-
- 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
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Epidemiology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Vascular Medicine (AREA)
- Urology & Nephrology (AREA)
- Immunology (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762459286P | 2017-02-15 | 2017-02-15 | |
| PCT/US2018/018381 WO2018152333A1 (en) | 2017-02-15 | 2018-02-15 | Methods and compositions for gene transfer across the vasculature |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201907298WA true SG11201907298WA (en) | 2019-09-27 |
Family
ID=63169716
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201907298WA SG11201907298WA (en) | 2017-02-15 | 2018-02-15 | Methods and compositions for gene transfer across the vasculature |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US11905312B2 (es) |
| EP (1) | EP3583220A4 (es) |
| JP (2) | JP7291397B2 (es) |
| KR (1) | KR20190117571A (es) |
| CN (1) | CN110520536B (es) |
| AU (1) | AU2018221738B2 (es) |
| BR (1) | BR112019016769A2 (es) |
| CA (1) | CA3052829A1 (es) |
| EA (1) | EA038695B1 (es) |
| IL (2) | IL268534B2 (es) |
| MX (1) | MX2019009720A (es) |
| SG (1) | SG11201907298WA (es) |
| WO (1) | WO2018152333A1 (es) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| WO2017058892A2 (en) | 2015-09-28 | 2017-04-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| JP2020533968A (ja) * | 2017-08-25 | 2020-11-26 | オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． | 組換えアデノ随伴ベクター |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| MX2020010466A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus que evitan anticuerpos. | |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| CA3100006A1 (en) | 2018-05-11 | 2019-11-14 | Massachusetts Eye And Ear Infirmary | Altering tissue tropism of adeno-associated viruses |
| CN112703198A (zh) | 2018-07-11 | 2021-04-23 | 布里格姆妇女医院 | 用于跨血脑屏障递送试剂的方法和组合物 |
| WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| KR20220011616A (ko) | 2019-03-21 | 2022-01-28 | 스트라이드바이오 인코포레이티드 | 재조합 아데노 관련 바이러스 벡터 |
| 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 |
| JP2022546236A (ja) * | 2019-08-14 | 2022-11-04 | ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． | 遺伝子治療のためのａａｖカプシドバリアント |
| WO2021050614A2 (en) * | 2019-09-09 | 2021-03-18 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav |
| AU2020367532A1 (en) | 2019-10-17 | 2022-05-12 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick disease type C |
| US20220411821A1 (en) * | 2019-10-28 | 2022-12-29 | University Of Florida Research Foundation, Incorporated | Gene therapy vectors |
| BR112022009864A2 (pt) * | 2019-11-22 | 2022-08-02 | Childrens Hospital Philadelphia | Variantes do vetor viral adenoassociado |
| US11981705B2 (en) | 2020-01-10 | 2024-05-14 | The Brigham And Women's Hospital, Inc. | Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer |
| WO2021163357A2 (en) | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| AU2021218411A1 (en) | 2020-02-13 | 2022-10-06 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| BR112022016965A2 (pt) * | 2020-02-25 | 2022-12-06 | Childrens Medical Res Institute | Polipeptídeos e vetores de capsídeo do vírus adenoassociado |
| KR20230068444A (ko) | 2020-08-19 | 2023-05-17 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터 |
| WO2022076750A2 (en) * | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
| WO2022150634A2 (en) * | 2021-01-08 | 2022-07-14 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav |
| US20240082351A1 (en) * | 2021-01-13 | 2024-03-14 | Duke University | Compositions for and methods of improving fluid flux in the brain |
| JP2024523707A (ja) | 2021-07-08 | 2024-06-28 | テナヤ セラピューティクス， インコーポレイテッド | 遺伝子治療のための最適化された発現カセット |
| WO2023288184A2 (en) * | 2021-07-14 | 2023-01-19 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods for treating chronic pain and for retrograde transduction of neurons |
| CN113563430B (zh) * | 2021-07-30 | 2022-05-31 | 上海信致医药科技有限公司 | 用于治疗眼部疾病的基因递送系统及其应用 |
| CN113480615B (zh) * | 2021-07-30 | 2022-05-31 | 上海信致医药科技有限公司 | 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| WO2023023781A1 (en) * | 2021-08-25 | 2023-03-02 | Children's Medical Research Institute | Modified aav capsid polypeptides and vectors |
| WO2023060272A2 (en) * | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns tropic delivery |
| CN114516901B (zh) * | 2022-03-11 | 2023-01-17 | 上海勉亦生物科技有限公司 | 一种神经系统高亲和性的aav载体及其应用 |
| WO2023201277A1 (en) * | 2022-04-14 | 2023-10-19 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns tropic delivery |
| TW202421787A (zh) | 2022-09-06 | 2024-06-01 | 美商特納亞治療股份有限公司 | 保護心臟之心臟病療法 |
| KR20240048599A (ko) * | 2022-10-06 | 2024-04-16 | 주식회사 글루진테라퓨틱스 | 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2340230T3 (es) * | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| DE19933288A1 (de) * | 1999-07-15 | 2001-01-18 | Medigene Ag | Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung |
| NZ561656A (en) * | 2002-05-01 | 2009-03-31 | Univ Florida | Improved rAAV expression systems for genetic modification of specific capsid proteins |
| EP2396343B1 (en) * | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| WO2013078400A1 (en) * | 2011-11-22 | 2013-05-30 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
| PL2900686T3 (pl) * | 2012-09-28 | 2021-01-25 | The University Of North Carolina At Chapel Hill | Wektory aav ukierunkowane na oligodendrocyty |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2017015102A1 (en) * | 2015-07-17 | 2017-01-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for achieving high levels of transduction in human liver cells |
-
2018
- 2018-02-15 EA EA201991911A patent/EA038695B1/ru unknown
- 2018-02-15 MX MX2019009720A patent/MX2019009720A/es unknown
- 2018-02-15 EP EP18754551.2A patent/EP3583220A4/en active Pending
- 2018-02-15 SG SG11201907298WA patent/SG11201907298WA/en unknown
- 2018-02-15 JP JP2019542561A patent/JP7291397B2/ja active Active
- 2018-02-15 US US16/485,094 patent/US11905312B2/en active Active
- 2018-02-15 CA CA3052829A patent/CA3052829A1/en active Pending
- 2018-02-15 WO PCT/US2018/018381 patent/WO2018152333A1/en unknown
- 2018-02-15 AU AU2018221738A patent/AU2018221738B2/en active Active
- 2018-02-15 IL IL268534A patent/IL268534B2/en unknown
- 2018-02-15 IL IL312788A patent/IL312788A/en unknown
- 2018-02-15 KR KR1020197025393A patent/KR20190117571A/ko not_active Application Discontinuation
- 2018-02-15 CN CN201880025093.XA patent/CN110520536B/zh active Active
- 2018-02-15 BR BR112019016769-2A patent/BR112019016769A2/pt unknown
-
2023
- 2023-01-20 JP JP2023007075A patent/JP2023052541A/ja active Pending
-
2024
- 2024-01-10 US US18/409,304 patent/US20240209031A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2020505936A (ja) | 2020-02-27 |
| WO2018152333A9 (en) | 2019-01-24 |
| AU2018221738A1 (en) | 2019-08-22 |
| IL312788A (en) | 2024-07-01 |
| US11905312B2 (en) | 2024-02-20 |
| AU2018221738B2 (en) | 2024-06-27 |
| IL268534B2 (en) | 2024-10-01 |
| US20190367562A1 (en) | 2019-12-05 |
| WO2018152333A1 (en) | 2018-08-23 |
| CA3052829A1 (en) | 2018-08-23 |
| CN110520536A (zh) | 2019-11-29 |
| EA201991911A1 (ru) | 2020-02-20 |
| KR20190117571A (ko) | 2019-10-16 |
| EP3583220A4 (en) | 2021-07-07 |
| EP3583220A1 (en) | 2019-12-25 |
| JP7291397B2 (ja) | 2023-06-15 |
| US20240209031A1 (en) | 2024-06-27 |
| CN110520536B (zh) | 2024-07-09 |
| MX2019009720A (es) | 2019-10-07 |
| BR112019016769A2 (pt) | 2020-05-26 |
| IL268534A (en) | 2019-09-26 |
| JP2023052541A (ja) | 2023-04-11 |
| IL268534B1 (en) | 2024-06-01 |
| EA038695B1 (ru) | 2021-10-06 |
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