ES2676550T5 - Vectores AAV para su uso en genoterapia de coroideremia - Google Patents
Vectores AAV para su uso en genoterapia de coroideremia Download PDFInfo
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- ES2676550T5 ES2676550T5 ES12706308T ES12706308T ES2676550T5 ES 2676550 T5 ES2676550 T5 ES 2676550T5 ES 12706308 T ES12706308 T ES 12706308T ES 12706308 T ES12706308 T ES 12706308T ES 2676550 T5 ES2676550 T5 ES 2676550T5
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Classifications
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- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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- C—CHEMISTRY; METALLURGY
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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- C12N9/10—Transferases (2.)
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- C—CHEMISTRY; METALLURGY
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- 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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- 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
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| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
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| MA39082B2 (fr) | 2013-12-06 | 2021-11-30 | Inst Nat Sante Rech Med | Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| LT3265568T (lt) * | 2015-03-06 | 2020-08-25 | Massachusetts Eye & Ear Infirmary | Genų augmentacijos terapijos, skirtos paveldimai tinklainės degeneracijai, sukeltai prpf31 geno mutacijos |
| MX2018007230A (es) * | 2015-12-14 | 2018-11-09 | Univ Pennsylvania | Terapia genica para trastornos oculares. |
| US20190017096A1 (en) * | 2016-03-10 | 2019-01-17 | Nightstarx Limited | Prenylation assay |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2018014152A (es) | 2016-05-18 | 2019-03-28 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la enfermedad de huntington. |
| 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 |
| US10633689B2 (en) | 2016-11-07 | 2020-04-28 | Spark Therapeutics, Inc. | Rab Escort Protein potency assay |
| AU2018207259B2 (en) * | 2017-01-10 | 2023-11-09 | Children's Medical Research Institute | Polynucleotides and vectors for the expression of transgenes |
| 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 |
| AU2018261003A1 (en) | 2017-05-05 | 2019-11-14 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's Disease |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| WO2018232149A1 (en) | 2017-06-14 | 2018-12-20 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖを送達するための組成物および方法 |
| CA3075656A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| KR20200078513A (ko) * | 2017-10-03 | 2020-07-01 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| WO2019079240A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US20200341015A1 (en) | 2017-10-17 | 2020-10-29 | Nightstarx Ltd. | Prenylation assay |
| AU2018378622A1 (en) * | 2017-12-06 | 2020-05-28 | Ovid Therapeutics Inc. | Use of miR101 or miR128 in the treatment of seizure disorders |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| BR112020015798A2 (pt) | 2018-02-01 | 2021-03-09 | Homology Medicines, Inc. | Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas |
| EP3755795A4 (en) | 2018-02-19 | 2022-07-20 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof |
| EP3775234A1 (en) * | 2018-04-05 | 2021-02-17 | NightstaRx Limited | Aav compositions, methods of making and methods of use |
| NZ759802A (en) * | 2018-05-07 | 2025-08-29 | Univ North Carolina Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| AU2019346655B2 (en) | 2018-09-28 | 2025-03-13 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| US20220193207A1 (en) * | 2019-04-30 | 2022-06-23 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of pompe disease |
| 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 |
| 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 |
| MX2023001998A (es) | 2020-08-17 | 2023-05-04 | Massachusetts Inst Technology | Enfoques de terapia con el gen shank3. |
| WO2022109247A1 (en) | 2020-11-20 | 2022-05-27 | Biogen Ma Inc. | Viral vector potency assay |
| EP4482970A1 (en) | 2022-02-23 | 2025-01-01 | Massachusetts Institute of Technology | Methods for upregulating shank3 expression |
| WO2024033834A1 (en) | 2022-08-11 | 2024-02-15 | Institute Of Molecular And Clinical Opthalmology Basel (Iob) | Promoters for specific expression of genes in cone photoreceptors |
| KR20250051697A (ko) | 2022-08-11 | 2025-04-17 | 인스티튜트 오브 몰레큘러 앤드 클리니컬 오프탈몰로지 바젤 (아이오비) | 인간 원추형 광수용체 광유전학 작제물 |
| WO2024233422A1 (en) | 2023-05-05 | 2024-11-14 | Massachusetts Institute Of Technology | Shank3 gene therapy approaches |
| WO2025090942A1 (en) * | 2023-10-26 | 2025-05-01 | University Of Notre Dame Du Lac | Gene therapy for treatment of neurometabolic disease |
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| EP0856585A1 (en) | 1997-01-29 | 1998-08-05 | Introgene B.V. | A conditional replication and expression system |
| JP2003526377A (ja) * | 2000-03-14 | 2003-09-09 | ニユーロロジクス・インコーポレーテツド | キメラキャプシドベクターの製造 |
| CA2442670A1 (en) | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| US20040208847A1 (en) * | 2003-03-28 | 2004-10-21 | Fabienne Rolling | Method and vectors for selectively transducing retinal pigment epithelium cells |
| KR20070114761A (ko) * | 2005-02-23 | 2007-12-04 | 디나벡크 가부시키가이샤 | Ｓｉｖｐｅｄｆ 벡터를 사용한 안조직 세포에 있어서의아포토시스 변성을 동반하는 질환의 치료제 |
| JP2008539698A (ja) * | 2005-04-29 | 2008-11-20 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 転写後レベルでの核酸発現調節のための方法および組成物 |
| US20090022731A1 (en) * | 2006-08-25 | 2009-01-22 | Wyeth | Arthritis-associated B cell gene expression |
| CN101854947A (zh) * | 2007-08-16 | 2010-10-06 | 圣文森特医院悉尼有限公司 | 用于调节巨噬细胞抑制因子(mic-1)活性的药剂和方法 |
| WO2009097129A1 (en) | 2008-01-29 | 2009-08-06 | Applied Genetic Technologies Corporation | Recombinant virus production using mammalian cells in suspension |
| AU2009212309B2 (en) * | 2008-02-07 | 2014-04-03 | Ceregene, Inc. | Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein |
| HUE072183T2 (hu) * | 2009-05-02 | 2025-10-28 | Genzyme Corp | Génterápia neurodegeneratív betegségekre |
| US9169494B2 (en) * | 2010-01-12 | 2015-10-27 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
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