JP2018506304A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018506304A5 JP2018506304A5 JP2017560478A JP2017560478A JP2018506304A5 JP 2018506304 A5 JP2018506304 A5 JP 2018506304A5 JP 2017560478 A JP2017560478 A JP 2017560478A JP 2017560478 A JP2017560478 A JP 2017560478A JP 2018506304 A5 JP2018506304 A5 JP 2018506304A5
- Authority
- JP
- Japan
- Prior art keywords
- rnai
- vector
- guide region
- sequence
- strand
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 230000009368 gene silencing by RNA Effects 0.000 claims 46
- 108091030071 RNAI Proteins 0.000 claims 45
- 239000013598 vector Substances 0.000 claims 35
- 239000002245 particle Substances 0.000 claims 33
- 150000007523 nucleic acids Chemical group 0.000 claims 25
- 241000702421 Dependoparvovirus Species 0.000 claims 18
- 241000124008 Mammalia Species 0.000 claims 13
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 13
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 12
- 108020004707 nucleic acids Proteins 0.000 claims 12
- 102000039446 nucleic acids Human genes 0.000 claims 12
- 239000002773 nucleotide Substances 0.000 claims 11
- 125000003729 nucleotide group Chemical group 0.000 claims 11
- 241000700605 Viruses Species 0.000 claims 9
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 8
- 230000008488 polyadenylation Effects 0.000 claims 8
- 210000000234 capsid Anatomy 0.000 claims 7
- 230000002401 inhibitory effect Effects 0.000 claims 7
- 208000023105 Huntington disease Diseases 0.000 claims 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 6
- 229920001184 polypeptide Polymers 0.000 claims 6
- 108090000765 processed proteins & peptides Proteins 0.000 claims 6
- 102000004196 processed proteins & peptides Human genes 0.000 claims 6
- 230000003612 virological effect Effects 0.000 claims 6
- 239000013608 rAAV vector Substances 0.000 claims 5
- 241000711975 Vesicular stomatitis virus Species 0.000 claims 4
- 208000035475 disorder Diseases 0.000 claims 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 3
- 241000283690 Bos taurus Species 0.000 claims 3
- 241000701022 Cytomegalovirus Species 0.000 claims 3
- 241000712899 Lymphocytic choriomeningitis mammarenavirus Species 0.000 claims 3
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims 3
- 108700011259 MicroRNAs Proteins 0.000 claims 3
- 241000714474 Rous sarcoma virus Species 0.000 claims 3
- 108010006025 bovine growth hormone Proteins 0.000 claims 3
- 210000004027 cell Anatomy 0.000 claims 3
- 239000002679 microRNA Substances 0.000 claims 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 2
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 2
- 241001115402 Ebolavirus Species 0.000 claims 2
- 206010066919 Epidemic polyarthritis Diseases 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
- 108010043121 Green Fluorescent Proteins Proteins 0.000 claims 2
- 102000004144 Green Fluorescent Proteins Human genes 0.000 claims 2
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 2
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 2
- 241001115401 Marburgvirus Species 0.000 claims 2
- 208000001089 Multiple system atrophy Diseases 0.000 claims 2
- 241000711798 Rabies lyssavirus Species 0.000 claims 2
- 241000710942 Ross River virus Species 0.000 claims 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 238000009825 accumulation Methods 0.000 claims 2
- 210000004227 basal ganglia Anatomy 0.000 claims 2
- 210000003169 central nervous system Anatomy 0.000 claims 2
- 210000003710 cerebral cortex Anatomy 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 230000007850 degeneration Effects 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 239000005090 green fluorescent protein Substances 0.000 claims 2
- 230000001697 htt accumulation Effects 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 201000002212 progressive supranuclear palsy Diseases 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 101710081722 Antitrypsin Proteins 0.000 claims 1
- 208000003174 Brain Neoplasms Diseases 0.000 claims 1
- 241000282465 Canis Species 0.000 claims 1
- 206010012289 Dementia Diseases 0.000 claims 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 1
- 101000933465 Homo sapiens Beta-glucuronidase Proteins 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 201000009906 Meningitis Diseases 0.000 claims 1
- 108091033773 MiR-155 Proteins 0.000 claims 1
- 241000713869 Moloney murine leukemia virus Species 0.000 claims 1
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 241001494479 Pecora Species 0.000 claims 1
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 claims 1
- 108010077524 Peptide Elongation Factor 1 Proteins 0.000 claims 1
- 108010071690 Prealbumin Proteins 0.000 claims 1
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 claims 1
- 208000006011 Stroke Diseases 0.000 claims 1
- 108010017842 Telomerase Proteins 0.000 claims 1
- 239000004098 Tetracycline Substances 0.000 claims 1
- 108010022394 Threonine synthase Proteins 0.000 claims 1
- 102000009190 Transthyretin Human genes 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 230000001475 anti-trypsic effect Effects 0.000 claims 1
- 208000015114 central nervous system disease Diseases 0.000 claims 1
- 210000003161 choroid Anatomy 0.000 claims 1
- 210000000349 chromosome Anatomy 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 102000004419 dihydrofolate reductase Human genes 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 206010015037 epilepsy Diseases 0.000 claims 1
- 230000030279 gene silencing Effects 0.000 claims 1
- 238000012226 gene silencing method Methods 0.000 claims 1
- AWUCVROLDVIAJX-UHFFFAOYSA-N glycerol 1-phosphate Chemical compound OCC(O)COP(O)(O)=O AWUCVROLDVIAJX-UHFFFAOYSA-N 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000004698 lymphocyte Anatomy 0.000 claims 1
- 210000005171 mammalian brain Anatomy 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000013646 rAAV2 vector Substances 0.000 claims 1
- 230000001177 retroviral effect Effects 0.000 claims 1
- 230000001568 sexual effect Effects 0.000 claims 1
- 230000002123 temporal effect Effects 0.000 claims 1
- 229960002180 tetracycline Drugs 0.000 claims 1
- 229930101283 tetracycline Natural products 0.000 claims 1
- 235000019364 tetracycline Nutrition 0.000 claims 1
- 150000003522 tetracyclines Chemical class 0.000 claims 1
- 230000001988 toxicity Effects 0.000 claims 1
- 231100000419 toxicity Toxicity 0.000 claims 1
- 239000002753 trypsin inhibitor Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021078464A JP7526710B2 (ja) | 2015-02-10 | 2021-05-06 | バリアントＲＮＡｉ |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562114578P | 2015-02-10 | 2015-02-10 | |
| US62/114,578 | 2015-02-10 | ||
| PCT/US2016/017207 WO2016130589A2 (en) | 2015-02-10 | 2016-02-09 | VARIANT RNAi |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021078464A Division JP7526710B2 (ja) | 2015-02-10 | 2021-05-06 | バリアントＲＮＡｉ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018506304A JP2018506304A (ja) | 2018-03-08 |
| JP2018506304A5 true JP2018506304A5 (enExample) | 2019-03-22 |
Family
ID=56614905
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017560478A Pending JP2018506304A (ja) | 2015-02-10 | 2016-02-09 | バリアントＲＮＡｉ |
| JP2021078464A Active JP7526710B2 (ja) | 2015-02-10 | 2021-05-06 | バリアントＲＮＡｉ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021078464A Active JP7526710B2 (ja) | 2015-02-10 | 2021-05-06 | バリアントＲＮＡｉ |
Country Status (25)
| Country | Link |
|---|---|
| US (4) | US10450563B2 (enExample) |
| EP (1) | EP3256588A2 (enExample) |
| JP (2) | JP2018506304A (enExample) |
| KR (1) | KR102670852B1 (enExample) |
| CN (1) | CN107438671B (enExample) |
| AR (1) | AR103646A1 (enExample) |
| AU (3) | AU2016219396B2 (enExample) |
| CA (2) | CA2976075C (enExample) |
| CL (1) | CL2017002027A1 (enExample) |
| CO (1) | CO2017009083A2 (enExample) |
| CR (1) | CR20170406A (enExample) |
| EA (1) | EA201791805A1 (enExample) |
| EC (1) | ECSP17059343A (enExample) |
| HK (1) | HK1247641A1 (enExample) |
| IL (1) | IL253893B (enExample) |
| MA (1) | MA40819B1 (enExample) |
| MX (2) | MX2017010369A (enExample) |
| MY (2) | MY181458A (enExample) |
| PE (1) | PE20171382A1 (enExample) |
| PH (1) | PH12017501432A1 (enExample) |
| SG (3) | SG11201706444TA (enExample) |
| TN (1) | TN2017000354A1 (enExample) |
| TW (1) | TWI781079B (enExample) |
| UY (1) | UY36554A (enExample) |
| WO (1) | WO2016130589A2 (enExample) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| JP6246121B2 (ja) | 2012-07-13 | 2017-12-13 | 株式会社新日本科学 | キラル核酸アジュバント |
| CN112007045B (zh) | 2012-07-13 | 2026-01-06 | 波涛生命科学有限公司 | 手性控制 |
| DK2872485T3 (da) | 2012-07-13 | 2021-03-08 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| KR102423317B1 (ko) | 2014-01-16 | 2022-07-22 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN112410338A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| US10450563B2 (en) | 2015-02-10 | 2019-10-22 | Genzyme Corporation | Variant RNAi |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| WO2017015575A1 (en) | 2015-07-22 | 2017-01-26 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| US20180228830A1 (en) * | 2015-10-23 | 2018-08-16 | Rena Therapeutics Inc. | Nucleic acid complex having at least one bulge structure |
| JP2019516680A (ja) | 2016-05-04 | 2019-06-20 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド組成物およびその方法 |
| KR20220108216A (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| SG11201809699XA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| CA3064590A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | Two-tailed self-delivering sirna and related methods |
| IL273394B2 (en) | 2017-09-22 | 2025-04-01 | Genzyme Corp | RNAI variant |
| EP3684388A4 (en) * | 2017-09-22 | 2021-09-22 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF HUNTINGTON'S DISEASE |
| KR20200119782A (ko) * | 2017-12-06 | 2020-10-20 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 mir101 또는 mir128의 용도 |
| EP3768386A4 (en) | 2018-03-23 | 2022-04-13 | University of Massachusetts | GENE THERAPY FOR THE TREATMENT OF BONE DISORDERS |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| JP2021530548A (ja) | 2018-07-24 | 2021-11-11 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. | 遺伝子治療製剤を生産するための系および方法 |
| CA3108526A1 (en) * | 2018-08-03 | 2020-02-06 | Genzyme Corporation | Variant rnai against alpha-synuclein |
| JP7627042B2 (ja) | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | Ｏ－メチルリッチ完全安定化オリゴヌクレオチド |
| EP4461814A3 (en) * | 2018-10-12 | 2025-02-26 | Genzyme Corporation | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
| AU2019385506A1 (en) * | 2018-11-21 | 2021-06-03 | Takeda Pharmaceutical Company Limited | Recombinant viral vectors and nucleic acids for producing the same |
| CA3125310A1 (en) * | 2019-01-09 | 2020-07-16 | Universidade De Coimbra | Double stranded rna and uses thereof |
| WO2020150636A1 (en) | 2019-01-18 | 2020-07-23 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| EP3983077A4 (en) * | 2019-06-17 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM |
| CA3149835A1 (en) | 2019-08-09 | 2021-02-18 | University Of Massachusetts | Chemically modified oligonucleotides targeting snps |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| JP2023522701A (ja) * | 2020-04-20 | 2023-05-31 | ユニバーシティー オブ マサチューセッツ | Ｍｓｈ３調節のためのオリゴヌクレオチド |
| EP4157289A4 (en) | 2020-05-26 | 2024-06-26 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| CA3238867A1 (en) | 2021-11-29 | 2023-06-01 | Shanghai Regenelead Therapies Co., Ltd. | Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease |
| CN118660913A (zh) | 2022-02-21 | 2024-09-17 | 上海瑞宏迪医药有限公司 | Vegf结合分子及其医药用途 |
| WO2024187106A2 (en) * | 2023-03-09 | 2024-09-12 | Leal Therapeutics, Inc. | Compositions and methods for modulating grin2a |
| TW202508610A (zh) | 2023-08-23 | 2025-03-01 | 大陸商上海瑞宏迪醫藥有限公司 | 醫藥組成物及其用途 |
| KR20250129583A (ko) * | 2024-02-21 | 2025-08-29 | 서울대학교산학협력단 | Drosha 절단을 촉진시키는 구조적 모티프를 포함하는 rna 간섭 유도 핵산 분자의 변형 전구체 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | 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 | Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| EP2369002A1 (en) | 1999-08-09 | 2011-09-28 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| US20080274989A1 (en) * | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| EP1556402B1 (en) | 2002-09-25 | 2011-06-22 | University of Massachusetts | In vivo gene silencing by chemically modified and stable sirna |
| WO2004101787A1 (ja) * | 2003-05-14 | 2004-11-25 | Japan Science And Technology Agency | ハンチンチン遺伝子の発現抑制 |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| US7902352B2 (en) * | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| CA2688514A1 (en) | 2007-04-26 | 2008-11-06 | University Of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| WO2008150897A2 (en) * | 2007-05-31 | 2008-12-11 | University Of Iowa Research Foundation | Reduction of off-target rna interference toxicity |
| JP2011517339A (ja) * | 2008-02-04 | 2011-06-02 | ガラパゴス・ナムローゼ・フェンノートシャップ | 神経変性疾患の治療に有用な分子標的及び化合物並びにこれらの同定方法 |
| US8318924B2 (en) * | 2008-02-14 | 2012-11-27 | Michael Paul Marie Gantier | Immunostimulatory siRNA molecules |
| FR2929292A1 (fr) | 2008-03-28 | 2009-10-02 | Exonhit Therapeutics S A Sa | Procede et methodes de diagnostic de la maladie d'alzheimer |
| US8796238B2 (en) * | 2009-09-17 | 2014-08-05 | Sigma-Aldrich Co. Llc | Short RNA mimetics |
| EP3567106A1 (en) | 2010-04-23 | 2019-11-13 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| SG10202007803QA (en) | 2011-02-17 | 2020-09-29 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| NZ629427A (en) | 2012-02-29 | 2016-04-29 | Sangamo Biosciences Inc | Methods and compositions for treating huntington’s disease |
| US9550989B2 (en) * | 2013-10-03 | 2017-01-24 | Washington University | Rational design of microRNA-siRNA chimeras for multi-functional target suppression |
| DK3628334T5 (da) | 2014-03-21 | 2024-09-02 | Genzyme Corp | Genterapi til behandling af retinitis pigmentosa |
| EP3137497B2 (en) | 2014-05-02 | 2025-03-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| EP3237618B1 (en) | 2014-12-24 | 2019-05-22 | uniQure IP B.V. | Rnai induced huntingtin gene suppression |
| US10450563B2 (en) | 2015-02-10 | 2019-10-22 | Genzyme Corporation | Variant RNAi |
| IL273394B2 (en) | 2017-09-22 | 2025-04-01 | Genzyme Corp | RNAI variant |
-
2016
- 2016-02-09 US US15/549,895 patent/US10450563B2/en active Active
- 2016-02-09 HK HK18107116.4A patent/HK1247641A1/zh unknown
- 2016-02-09 SG SG11201706444TA patent/SG11201706444TA/en unknown
- 2016-02-09 KR KR1020177025286A patent/KR102670852B1/ko active Active
- 2016-02-09 JP JP2017560478A patent/JP2018506304A/ja active Pending
- 2016-02-09 TN TNP/2017/000354A patent/TN2017000354A1/en unknown
- 2016-02-09 AU AU2016219396A patent/AU2016219396B2/en active Active
- 2016-02-09 CN CN201680020699.5A patent/CN107438671B/zh active Active
- 2016-02-09 MY MYPI2017702831A patent/MY181458A/en unknown
- 2016-02-09 EP EP16710035.3A patent/EP3256588A2/en active Pending
- 2016-02-09 MX MX2017010369A patent/MX2017010369A/es unknown
- 2016-02-09 WO PCT/US2016/017207 patent/WO2016130589A2/en not_active Ceased
- 2016-02-09 MY MYPI2020002819A patent/MY194175A/en unknown
- 2016-02-09 EA EA201791805A patent/EA201791805A1/ru unknown
- 2016-02-09 PE PE2017001371A patent/PE20171382A1/es unknown
- 2016-02-09 CA CA2976075A patent/CA2976075C/en active Active
- 2016-02-09 CR CR20170406A patent/CR20170406A/es unknown
- 2016-02-09 CA CA3255657A patent/CA3255657A1/en active Pending
- 2016-02-09 SG SG10201907396XA patent/SG10201907396XA/en unknown
- 2016-02-09 MA MA40819A patent/MA40819B1/fr unknown
- 2016-02-10 AR ARP160100356A patent/AR103646A1/es unknown
- 2016-02-10 SG SG10201912942YA patent/SG10201912942YA/en unknown
- 2016-02-10 UY UY0001036554A patent/UY36554A/es not_active Application Discontinuation
- 2016-02-15 TW TW105104339A patent/TWI781079B/zh active
-
2017
- 2017-08-08 IL IL253893A patent/IL253893B/en unknown
- 2017-08-08 CL CL2017002027A patent/CL2017002027A1/es unknown
- 2017-08-09 PH PH12017501432A patent/PH12017501432A1/en unknown
- 2017-08-10 MX MX2022013005A patent/MX2022013005A/es unknown
- 2017-09-05 CO CONC2017/0009083A patent/CO2017009083A2/es unknown
- 2017-09-08 EC ECIEPI201759343A patent/ECSP17059343A/es unknown
-
2019
- 2019-09-10 US US16/566,565 patent/US10760079B2/en active Active
-
2020
- 2020-07-28 US US16/941,303 patent/US11781137B2/en active Active
-
2021
- 2021-05-06 JP JP2021078464A patent/JP7526710B2/ja active Active
-
2022
- 2022-06-17 AU AU2022204274A patent/AU2022204274B2/en active Active
-
2023
- 2023-10-09 US US18/483,474 patent/US20240182896A1/en active Pending
-
2025
- 2025-07-16 AU AU2025205501A patent/AU2025205501A1/en active Pending