CA3041548A1 - Gene transfer compositions, methods and uses for treating neurodegenerative diseases - Google Patents
Gene transfer compositions, methods and uses for treating neurodegenerative diseases Download PDFInfo
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- A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
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- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
- C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
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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
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- 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
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- C12N2750/14011—Parvoviridae
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- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- 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)
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| US201662418033P | 2016-11-04 | 2016-11-04 | |
| US62/418,033 | 2016-11-04 | ||
| PCT/US2017/059986 WO2018085688A1 (en) | 2016-11-04 | 2017-11-03 | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
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| US9818239B2 (en) | 2015-08-20 | 2017-11-14 | Zendrive, Inc. | Method for smartphone-based accident detection |
| MY198804A (en) | 2017-06-07 | 2023-09-28 | Regeneron Pharma | Compositions and methods for internalizing enzymes |
| WO2019079807A1 (en) | 2017-10-20 | 2019-04-25 | Zendrive, Inc. | METHOD AND SYSTEM FOR VEHICULAR COMMUNICATIONS |
| BR112020016005A2 (pt) | 2018-02-07 | 2020-12-15 | Regeneron Pharmaceuticals, Inc. | Método de administração de uma proteína terapêutica ao sistema nervoso central, proteína terapêutica multidomínio, polinucleotídeo, vetor de terapia gênica, e, uso de um nucleotídeo que codifica a proteína terapêutica multidomínio |
| CN112135624B (zh) | 2018-05-17 | 2024-07-19 | 瑞泽恩制药公司 | 抗cd63抗体、缀合物和其用途 |
| WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| IL282808B2 (en) * | 2018-11-14 | 2025-02-01 | Regenxbio Inc | Gene therapy for neural ceroid lipofuscinosis |
| JP2022518931A (ja) * | 2019-02-01 | 2022-03-17 | スパーク セラピューティクス インコーポレイテッド | 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法 |
| JP2023504269A (ja) | 2019-12-03 | 2023-02-02 | ゼンドライヴ，インコーポレイテッド | ルートのリスク判定方法およびシステム |
| EP4118219A4 (en) * | 2020-03-11 | 2024-04-17 | Shanghai Belief-Delivery Biomed Co., Ltd. | Novel use of aspirin compound in increasing nucleic acid expression |
| WO2025231406A1 (en) * | 2024-05-02 | 2025-11-06 | The Children's Hospital Of Philadelphia | Methods to increase transduction of ependyma cells in brain |
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| US20070003518A1 (en) * | 2002-04-19 | 2007-01-04 | Atkinson Mark A | Raav vector-based compositions and methods for the prevention and treatment of mammalian diseases |
| US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
| KR20070004637A (ko) * | 2004-01-22 | 2007-01-09 | 가부시키가이샤 디나벡크 겐큐쇼 | 바이러스 벡터의 제조방법 |
| EP3456331B1 (en) * | 2006-02-08 | 2021-05-26 | Genzyme Corporation | Gene therapy for niemann-pick disease type a |
| ES2635726T3 (es) * | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
| PT2691529T (pt) * | 2011-03-31 | 2019-09-27 | Univ Iowa Res Found | Partícula de aav2 que compreende uma proteína do capsídeo de aav2 e um vetor que compreende um ácido nucleico que codifica uma tripeptidil peptidase 1 (tpp1) para utilização no tratamento da lipofuscinose ceroide infantil tardia (lincl) num mamífero não roedor por injeção intraventricular ou entrega icv |
| KR102380265B1 (ko) * | 2013-07-22 | 2022-03-29 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| EP3024497B1 (en) * | 2013-07-26 | 2021-01-13 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP3060669B1 (en) * | 2013-10-24 | 2024-12-11 | uniQure IP B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| KR20160079891A (ko) * | 2013-11-20 | 2016-07-06 | 유니버시티 오브 아이오와 리써치 파운데이션 | 아밀로이드 침착의 치료를 위한 방법 및 조성물 |
| SG11201803218PA (en) * | 2015-10-23 | 2018-05-30 | Univ Iowa Res Found | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| IL270422B2 (en) * | 2017-05-11 | 2025-04-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinosis |
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| Publication number | Publication date |
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| WO2018085688A1 (en) | 2018-05-11 |
| EP3534892A4 (en) | 2020-05-27 |
| EP3534892A1 (en) | 2019-09-11 |
| US20190269797A1 (en) | 2019-09-05 |
| RU2019117062A (ru) | 2020-12-04 |
| JP2019537576A (ja) | 2019-12-26 |
| RU2019117062A3 (cg-RX-API-DMAC7.html) | 2021-03-11 |
| MX2019005266A (es) | 2019-09-27 |
| BR112019009074A2 (pt) | 2019-07-16 |
| AU2017355502B2 (en) | 2023-08-31 |
| JP2023002721A (ja) | 2023-01-10 |
| CN110198712A (zh) | 2019-09-03 |
| AU2017355502A1 (en) | 2019-05-16 |
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