PL3237618T3 - Supresja genu huntingtyny indukowana rnai - Google Patents

Supresja genu huntingtyny indukowana rnai

Info

Publication number
PL3237618T3
PL3237618T3 PL15817385T PL15817385T PL3237618T3 PL 3237618 T3 PL3237618 T3 PL 3237618T3 PL 15817385 T PL15817385 T PL 15817385T PL 15817385 T PL15817385 T PL 15817385T PL 3237618 T3 PL3237618 T3 PL 3237618T3
Authority
PL
Poland
Prior art keywords
gene suppression
huntingtin gene
rnai induced
induced huntingtin
rnai
Prior art date
Application number
PL15817385T
Other languages
English (en)
Inventor
Pavlina Stefanova KONSTANTINOVA
Jana Miniarikova
Original Assignee
Uniqure Ip B.V.
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Uniqure Ip B.V. filed Critical Uniqure Ip B.V.
Publication of PL3237618T3 publication Critical patent/PL3237618T3/pl

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
PL15817385T 2014-12-24 2015-12-23 Supresja genu huntingtyny indukowana rnai PL3237618T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP14200308 2014-12-24
EP15817385.6A EP3237618B1 (en) 2014-12-24 2015-12-23 Rnai induced huntingtin gene suppression
PCT/EP2015/081157 WO2016102664A1 (en) 2014-12-24 2015-12-23 Rnai induced huntingtin gene suppression

Publications (1)

Publication Number Publication Date
PL3237618T3 true PL3237618T3 (pl) 2019-09-30

Family

ID=52282540

Family Applications (1)

Application Number Title Priority Date Filing Date
PL15817385T PL3237618T3 (pl) 2014-12-24 2015-12-23 Supresja genu huntingtyny indukowana rnai

Country Status (25)

Country Link
US (4) US10174321B2 (pl)
EP (2) EP3237618B1 (pl)
JP (2) JP6839094B2 (pl)
KR (1) KR102636633B1 (pl)
CN (1) CN108064292B (pl)
AU (1) AU2015370903B2 (pl)
BR (1) BR112017013573A2 (pl)
CA (1) CA2971920A1 (pl)
CO (1) CO2017007335A2 (pl)
CY (1) CY1121733T1 (pl)
DK (1) DK3237618T3 (pl)
EA (1) EA037696B1 (pl)
ES (1) ES2732023T3 (pl)
HK (1) HK1246344B (pl)
HR (1) HRP20190992T1 (pl)
HU (1) HUE043842T2 (pl)
IL (1) IL252990B (pl)
LT (1) LT3237618T (pl)
MX (1) MX2017008500A (pl)
PL (1) PL3237618T3 (pl)
PT (1) PT3237618T (pl)
RS (1) RS58862B1 (pl)
SI (1) SI3237618T1 (pl)
TR (1) TR201908969T4 (pl)
WO (1) WO2016102664A1 (pl)

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101881596B1 (ko) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 인 원자 변형된 핵산의 합성 방법
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
DK2872485T3 (da) 2012-07-13 2021-03-08 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
CA2878945A1 (en) 2012-07-13 2014-01-16 Wave Life Sciences Pte. Ltd. Chiral control
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
WO2015108046A1 (ja) 2014-01-15 2015-07-23 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
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
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
US10160969B2 (en) 2014-01-16 2018-12-25 Wave Life Sciences Ltd. Chiral design
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
AU2015370903B2 (en) * 2014-12-24 2021-06-17 Uniqure Ip B.V. RNAi induced huntingtin gene suppression
EP3256588A2 (en) 2015-02-10 2017-12-20 Genzyme Corporation VARIANT RNAi
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
KR102488323B1 (ko) 2015-12-10 2023-01-12 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Preparations and methods for treating Huntington's disease
MX2018014154A (es) 2016-05-18 2019-05-06 Voyager Therapeutics Inc Polinucleotidos moduladores.
US10457940B2 (en) * 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
MX2019014514A (es) 2017-06-05 2020-07-20 Ptc Therapeutics Inc Compuestos para tratar la enfermedad de huntington.
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CN111356763B (zh) * 2017-09-22 2024-03-12 建新公司 变体RNAi
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JP2021502060A (ja) 2017-10-16 2021-01-28 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)の治療
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
WO2019191092A1 (en) 2018-03-27 2019-10-03 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
US20210222196A1 (en) * 2018-05-15 2021-07-22 University Of Massachusetts Modified aav constructs and uses thereof
BR112020023298A2 (pt) * 2018-05-15 2021-03-09 University Of Washington composições e métodos para reduzir a spliceopatia e tratamento de distúrbios de dominância de rna
KR20210042265A (ko) 2018-06-27 2021-04-19 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료를 위한 헤테로사이클릭 및 헤테로아릴 화합물
CA3103963A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020053258A1 (en) 2018-09-12 2020-03-19 Uniqure Ip B.V. Rnai induced c9orf72 suppression for the treatment of als/ftd
CA3121010A1 (en) 2018-11-19 2020-05-28 Uniqure Ip B.V. Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
AU2019382824A1 (en) 2018-11-19 2021-06-03 Uniqure Ip B.V. Method and means to deliver miRNA to target cells
CA3119721A1 (en) 2018-11-19 2020-05-28 Uniqure Ip B.V. A companion diagnostic to monitor the effects of gene therapy
EP3908658A1 (en) * 2019-01-09 2021-11-17 Universidade de Coimbra Double stranded rna and uses thereof
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
WO2021053018A1 (en) 2019-09-16 2021-03-25 Uniqure Ip B.V. Targeting misspliced transcripts in genetic disorders
AR120341A1 (es) * 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
MX2022005254A (es) 2019-11-01 2022-06-29 Novartis Ag El uso de un modulador de empalme para un tratamiento que retrasa la progresion de la enfermedad de huntington.
EP4133074A1 (en) * 2020-04-07 2023-02-15 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
WO2022031591A2 (en) * 2020-08-03 2022-02-10 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
EP4317434A1 (en) * 2021-03-30 2024-02-07 Nanjing University Rna delivery system for treatment of huntington's disease
WO2022268835A1 (en) 2021-06-21 2022-12-29 Uniqure Biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
WO2023147058A2 (en) * 2022-01-27 2023-08-03 Asklepios Biopharmaceutical, Inc. Compositions for treating neurological disease
WO2023198702A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of c9orf72
WO2023198745A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of apoe
WO2023198662A1 (en) 2022-04-12 2023-10-19 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

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2004239114B2 (en) * 2003-05-14 2008-03-13 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
WO2005105995A2 (en) 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2007046703A2 (en) 2005-10-20 2007-04-26 Amsterdam Molecular Therapeutics B.V. Improved aav vectors produced in insect cells
CN101506369B (zh) 2006-06-21 2014-02-12 尤尼克尔生物制药股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav-rep78翻译起始密码子的载体
EP2046993A4 (en) 2006-07-07 2010-11-17 Univ Massachusetts RNA INTERFERENCE COMPOSITIONS AND METHOD FOR TREATING MORBUS HUNTINGTON
WO2008134646A2 (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
CN101868547B (zh) 2007-07-26 2016-03-16 尤尼克尔Ip股份有限公司 包含具有差别密码子偏性的重复编码序列的杆状病毒载体
ES2929031T3 (es) 2008-02-19 2022-11-24 Uniqure Ip Bv Optimización de la expresión de las proteínas rep y cap parvovirales en células de insecto
EP2411505A4 (en) * 2009-03-26 2013-01-30 Univ California MESENCHYMAL STEM CELLS PRODUCING INHIBITORY RNA WHICH CAN BE USED TO ACT IN THE COURSE OF A DISEASE
KR101835490B1 (ko) * 2009-05-02 2018-03-08 젠자임 코포레이션 신경변성 질환에 대한 유전자 요법
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
LT2561078T (lt) * 2010-04-23 2019-01-10 Cold Spring Harbor Laboratory Naujos struktūriškai sukonstruotos shrnr
JP5941842B2 (ja) * 2010-06-18 2016-06-29 Lsipファンド運営合同会社 優性アレル発現抑制剤
US9840694B2 (en) 2011-09-08 2017-12-12 Uniqure Ip B.V. Removal of contaminating viruses from AAV preparations
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
AU2015370903B2 (en) * 2014-12-24 2021-06-17 Uniqure Ip B.V. RNAi induced huntingtin gene suppression

Also Published As

Publication number Publication date
KR102636633B1 (ko) 2024-02-13
AU2015370903A1 (en) 2017-07-20
SI3237618T1 (sl) 2019-09-30
AU2015370903B2 (en) 2021-06-17
DK3237618T3 (da) 2019-06-24
HK1246344B (zh) 2020-01-10
CN108064292A (zh) 2018-05-22
CA2971920A1 (en) 2016-06-30
IL252990B (en) 2021-09-30
EA201791417A1 (ru) 2017-12-29
CY1121733T1 (el) 2020-07-31
JP2021000136A (ja) 2021-01-07
US11371044B2 (en) 2022-06-28
JP6839094B2 (ja) 2021-03-03
WO2016102664A1 (en) 2016-06-30
PT3237618T (pt) 2019-07-04
JP2018502601A (ja) 2018-02-01
EP3540063A1 (en) 2019-09-18
CN108064292B (zh) 2021-05-04
CO2017007335A2 (es) 2018-01-05
KR20170120572A (ko) 2017-10-31
RS58862B1 (sr) 2019-07-31
MX2017008500A (es) 2018-02-01
US10767180B2 (en) 2020-09-08
EP3237618A1 (en) 2017-11-01
US20170355989A1 (en) 2017-12-14
HUE043842T4 (hu) 2019-08-28
HUE043842T2 (hu) 2019-09-30
US20200339992A1 (en) 2020-10-29
EP3237618B1 (en) 2019-05-22
ES2732023T3 (es) 2019-11-20
HRP20190992T1 (hr) 2019-09-20
EA037696B1 (ru) 2021-05-12
US10174321B2 (en) 2019-01-08
LT3237618T (lt) 2019-07-10
TR201908969T4 (tr) 2019-07-22
US20230119344A1 (en) 2023-04-20
NZ733296A (en) 2021-10-29
US20190144860A1 (en) 2019-05-16
BR112017013573A2 (pt) 2018-03-06
IL252990A0 (en) 2017-08-31

Similar Documents

Publication Publication Date Title
HK1246344B (zh) Rnai誘發的亨廷頓基因抑制
IL280941A (en) Modified double helix RNA materials
HK1247641A1 (zh) 變體RNAi
ZA201607100B (en) Frequency response
SG11201606379UA (en) Aptamer construct
IL249254A0 (en) Gene expression system
GB201408823D0 (en) Demulsifiers
IL257500A (en) Altered cullin1 gene
GB201517285D0 (en) Stabilizer arrangement
GB201621449D0 (en) Frequency-domain denoising
IL248812A0 (en) Very effective stabilizer
GB2547614B (en) Stabilizer
SG11201702844VA (en) Photobioreactor
SG11201700652PA (en) Modified antimir-138 oligonucleotides
GB201608944D0 (en) Gene Tharapy
AU5592P (en) Lanmichigan Mandevilla boliviensis x sanderi
GB201410599D0 (en) Sensitizers
GB201407865D0 (en) Nucleases
GB201403353D0 (en) Novel oligonucleotides
GB201403087D0 (en) Novel oligonucleotides
IL230329A0 (en) croc sponge