SI3237618T1 - RNA inducirana supresija huntingtin gena - Google Patents
RNA inducirana supresija huntingtin genaInfo
- Publication number
- SI3237618T1 SI3237618T1 SI201530791T SI201530791T SI3237618T1 SI 3237618 T1 SI3237618 T1 SI 3237618T1 SI 201530791 T SI201530791 T SI 201530791T SI 201530791 T SI201530791 T SI 201530791T SI 3237618 T1 SI3237618 T1 SI 3237618T1
- Authority
- SI
- Slovenia
- Prior art keywords
- gene suppression
- huntingtin gene
- rnai induced
- induced huntingtin
- rnai
- Prior art date
Links
- 108091030071 RNAI Proteins 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
- 230000001629 suppression Effects 0.000 title 1
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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14200308 | 2014-12-24 | ||
| PCT/EP2015/081157 WO2016102664A1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
| EP15817385.6A EP3237618B1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SI3237618T1 true SI3237618T1 (sl) | 2019-09-30 |
Family
ID=52282540
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI201530791T SI3237618T1 (sl) | 2014-12-24 | 2015-12-23 | RNA inducirana supresija huntingtin gena |
Country Status (25)
| Country | Link |
|---|---|
| US (4) | US10174321B2 (sl) |
| EP (2) | EP3540063A1 (sl) |
| JP (2) | JP6839094B2 (sl) |
| KR (1) | KR102636633B1 (sl) |
| CN (1) | CN108064292B (sl) |
| AU (1) | AU2015370903B2 (sl) |
| CA (1) | CA2971920C (sl) |
| CO (1) | CO2017007335A2 (sl) |
| CY (1) | CY1121733T1 (sl) |
| DK (1) | DK3237618T3 (sl) |
| EA (1) | EA037696B1 (sl) |
| ES (1) | ES2732023T3 (sl) |
| HK (1) | HK1246344B (sl) |
| HR (1) | HRP20190992T1 (sl) |
| HU (1) | HUE043842T2 (sl) |
| IL (1) | IL252990B (sl) |
| LT (1) | LT3237618T (sl) |
| MX (1) | MX381121B (sl) |
| PL (1) | PL3237618T3 (sl) |
| PT (1) | PT3237618T (sl) |
| RS (1) | RS58862B1 (sl) |
| SI (1) | SI3237618T1 (sl) |
| SM (1) | SMT201900347T1 (sl) |
| TR (1) | TR201908969T4 (sl) |
| WO (1) | WO2016102664A1 (sl) |
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| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| JP6128529B2 (ja) | 2011-07-19 | 2017-05-17 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | 官能化核酸の合成のための方法 |
| SG11201500243WA (en) | 2012-07-13 | 2015-04-29 | Shin Nippon Biomedical Lab Ltd | Chiral nucleic acid adjuvant |
| KR101850319B1 (ko) | 2012-07-13 | 2018-04-20 | 웨이브 라이프 사이언시스 리미티드 | 비대칭 보조 그룹 |
| CN112007045A (zh) | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
| 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 |
| 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 |
| JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| EP4137572A1 (en) | 2014-01-16 | 2023-02-22 | Wave Life Sciences Ltd. | Chiral design |
| SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| HK1244299A1 (zh) | 2014-11-14 | 2018-08-03 | Voyager Therapeutics, Inc. | 治療肌萎縮側索硬化(als)的組合物和方法 |
| WO2016102664A1 (en) * | 2014-12-24 | 2016-06-30 | Uniqure Ip B.V. | Rnai induced huntingtin gene suppression |
| US10450563B2 (en) | 2015-02-10 | 2019-10-22 | Genzyme Corporation | Variant RNAi |
| US20160319278A1 (en) | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| EA201800367A1 (ru) | 2015-12-10 | 2019-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способы лечения болезни хантингтона |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| 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 |
| US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| SG11201909868YA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| BR112019025740A2 (pt) | 2017-06-05 | 2020-06-23 | Ptc Therapeutics, Inc. | Compostos para tratar a doença de huntington |
| CA3064590A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | Two-tailed self-delivering sirna and related methods |
| US11382918B2 (en) | 2017-06-28 | 2022-07-12 | Ptc Therapeutics, Inc. | Methods for treating Huntington's Disease |
| MX2019015580A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| MY205041A (en) | 2017-09-22 | 2024-09-29 | Genzyme Corp | Variant rnai |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| CN108384803A (zh) * | 2017-12-19 | 2018-08-10 | 上海百力格生物技术有限公司 | 一种用于miRNA干扰载体构建筛选特异对照的质粒及其构建方法 |
| JP7399870B2 (ja) | 2018-03-27 | 2023-12-18 | ピーティーシー セラピューティクス， インコーポレイテッド | ハンチントン病を処置するための化合物 |
| MA51938B1 (fr) * | 2018-05-15 | 2022-10-31 | Univ Washington | Compositions et procédés pour réduire les anomalies d'épissage et traiter des troubles de dominance arn |
| WO2019222413A1 (en) * | 2018-05-15 | 2019-11-21 | University Of Massachusetts | Modified aav constructs and uses thereof |
| WO2020005873A1 (en) | 2018-06-27 | 2020-01-02 | Ptc Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating huntington's disease |
| AU2019294482B2 (en) | 2018-06-27 | 2022-09-01 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating Huntington's disease |
| JP7565218B2 (ja) | 2018-07-02 | 2024-10-10 | ボイジャー セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症および脊髄に関連する障害の治療 |
| 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 |
| CA3112007A1 (en) | 2018-09-12 | 2020-03-19 | Uniqure Ip B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
| CA3120177A1 (en) * | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | Method and means to deliver mirna to target cells |
| JP2022507283A (ja) | 2018-11-19 | 2022-01-18 | ユニキュアー アイピー ビー．ブイ． | 脊髄小脳失調症３型の治療のためのアタキシン－３のｒｎａｉ誘導低減 |
| CA3119721A1 (en) | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | A companion diagnostic to monitor the effects of gene therapy |
| CN113302302A (zh) * | 2019-01-09 | 2021-08-24 | 科英布拉大学 | 双链rna及其用途 |
| AU2020207935A1 (en) | 2019-01-18 | 2021-08-26 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| US20220251567A1 (en) | 2019-07-10 | 2022-08-11 | Inserm (Institut National De La Santè Et De La Recherche Médicale) | Methods for the treatment of epilepsy |
| BR112022002307A2 (pt) | 2019-08-09 | 2022-06-28 | Univ Massachusetts | Oligonucleotídeos quimicamente modificados que têm como alvo snps |
| WO2021053018A1 (en) | 2019-09-16 | 2021-03-25 | Uniqure Ip B.V. | Targeting misspliced transcripts in genetic disorders |
| CN114650822A (zh) | 2019-11-01 | 2022-06-21 | 诺华股份有限公司 | 剪接调节子用于减慢亨廷顿氏病进展的治疗的用途 |
| KR20220094213A (ko) * | 2019-11-01 | 2022-07-05 | 알닐람 파마슈티칼스 인코포레이티드 | 헌팅틴 (HTT) iRNA 제제 조성물 및 이의 사용 방법 |
| WO2021204872A1 (en) * | 2020-04-07 | 2021-10-14 | Uniqure Ip B.V. | Gene constructs for silencing angiopoietin-like 3 (angptl3) and uses thereof |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| US20220090069A1 (en) * | 2020-08-03 | 2022-03-24 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
| TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
| AU2022246786A1 (en) | 2021-03-29 | 2023-10-05 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| CN115141848A (zh) * | 2021-03-30 | 2022-10-04 | 南京大学 | 一种用于治疗亨廷顿病的rna递送系统 |
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| JP2025503910A (ja) * | 2022-01-27 | 2025-02-06 | アスクレピオス バイオファーマシューティカル， インコーポレイテッド | 神経疾患を処置するための組成物 |
| EP4508213A1 (en) | 2022-04-12 | 2025-02-19 | uniQure biopharma B.V. | Nucleic acid regulation of apoe |
| EP4508214A1 (en) | 2022-04-12 | 2025-02-19 | uniQure biopharma B.V. | Nucleic acid regulation of c9orf72 |
| US20250230437A1 (en) | 2022-04-12 | 2025-07-17 | Uniqure Biopharma B.V. | Novel systems for nucleic acid regulation |
| WO2023198663A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of snca |
| WO2024218204A1 (en) | 2023-04-18 | 2024-10-24 | Uniqure Biopharma B.V. | Gene delivery vehicles comprising rna and antibodies |
| WO2025051805A1 (en) | 2023-09-04 | 2025-03-13 | Uniqure Biopharma B.V. | Novel neurotropic recombinant adeno-associated virus particles |
| WO2025078598A1 (en) | 2023-10-11 | 2025-04-17 | Uniqure Biopharma B.V. | Novel nucleic acid regulation of huntingtin gene |
| WO2025078602A1 (en) | 2023-10-11 | 2025-04-17 | Uniqure Biopharma B.V. | Nucleic acid regulation of msh3 in repeat expansion disorders |
| WO2025078606A1 (en) | 2023-10-11 | 2025-04-17 | Uniqure Biopharma B.V. | Further novel systems for nucleic acid regulation |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2526893C (en) | 2003-05-14 | 2010-10-26 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| EP1735443A2 (en) | 2004-04-14 | 2006-12-27 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| CN101287837B (zh) | 2005-10-20 | 2015-03-18 | 尤尼克尔生物制药股份有限公司 | 昆虫细胞中生产的改进的aav载体 |
| WO2007148971A2 (en) | 2006-06-21 | 2007-12-27 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells |
| CA2662704A1 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
| US20090036395A1 (en) * | 2007-04-26 | 2009-02-05 | Davidson Beverly L | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| PT2164967E (pt) | 2007-05-31 | 2015-10-27 | Univ Iowa Res Found | Redução da toxicidade não dirigida do arn de interferência |
| PL3093345T3 (pl) | 2007-07-26 | 2020-03-31 | Uniqure Ip B.V. | Wektory bakulowirusowe zawierające powtórzone sekwencje kodujące o zróżnicowanej preferencji kodonów |
| EP2250256B1 (en) | 2008-02-19 | 2017-08-02 | uniQure IP B.V. | Optimisation of expression of parvoviral rep and cap proteins in insect cells |
| EP2411505A4 (en) * | 2009-03-26 | 2013-01-30 | Univ California | MESENCHYMAL STEM CELLS FOR PRODUCING HEMDERING RNA FOR SICKNESS MODIFICATION |
| HRP20181423T1 (hr) * | 2009-05-02 | 2018-11-30 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| WO2011122950A1 (en) | 2010-04-01 | 2011-10-06 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Monomeric duplex aav vectors |
| EP2561078B1 (en) * | 2010-04-23 | 2018-09-19 | Cold Spring Harbor Laboratory | NOVEL STRUCTURALLY DESIGNED shRNAs |
| EP2583692A4 (en) * | 2010-06-18 | 2014-11-26 | Lsip Llc | HEMMER OF THE EXPRESSION OF DOMINANT ALLELES |
| KR101961347B1 (ko) | 2011-09-08 | 2019-03-25 | 유니큐어 아이피 비.브이. | Aav 제조물로부터 오염 바이러스의 제거 |
| CN103224556B (zh) * | 2013-01-31 | 2015-04-15 | 清华大学 | 亨廷顿舞蹈症的靶位蛋白质及其编码基因和应用 |
| WO2016102664A1 (en) * | 2014-12-24 | 2016-06-30 | Uniqure Ip B.V. | Rnai induced huntingtin gene suppression |
-
2015
- 2015-12-23 WO PCT/EP2015/081157 patent/WO2016102664A1/en active Application Filing
- 2015-12-23 PL PL15817385T patent/PL3237618T3/pl unknown
- 2015-12-23 TR TR2019/08969T patent/TR201908969T4/tr unknown
- 2015-12-23 PT PT15817385T patent/PT3237618T/pt unknown
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