JP2018533954A5 - - Google Patents

Download PDF

Info

Publication number
JP2018533954A5
JP2018533954A5 JP2018524194A JP2018524194A JP2018533954A5 JP 2018533954 A5 JP2018533954 A5 JP 2018533954A5 JP 2018524194 A JP2018524194 A JP 2018524194A JP 2018524194 A JP2018524194 A JP 2018524194A JP 2018533954 A5 JP2018533954 A5 JP 2018533954A5
Authority
JP
Japan
Prior art keywords
pharmaceutical composition
composition according
pharmaceutically acceptable
expression
salt
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.)
Granted
Application number
JP2018524194A
Other languages
English (en)
Japanese (ja)
Other versions
JP6751142B2 (ja
JP2018533954A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2016/077383 external-priority patent/WO2017081223A1/en
Publication of JP2018533954A publication Critical patent/JP2018533954A/ja
Publication of JP2018533954A5 publication Critical patent/JP2018533954A5/ja
Application granted granted Critical
Publication of JP6751142B2 publication Critical patent/JP6751142B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2018524194A 2015-11-12 2016-11-11 父方ube3aの発現を誘導するオリゴヌクレオチド Active JP6751142B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP15194367 2015-11-12
EP15194367.7 2015-11-12
EP16189502.4 2016-09-19
EP16189502 2016-09-19
PCT/EP2016/077383 WO2017081223A1 (en) 2015-11-12 2016-11-11 Oligonucleotides for inducing paternal ube3a expression

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2020136397A Division JP7279000B2 (ja) 2015-11-12 2020-08-12 父方ube3aの発現を誘導するオリゴヌクレオチド

Publications (3)

Publication Number Publication Date
JP2018533954A JP2018533954A (ja) 2018-11-22
JP2018533954A5 true JP2018533954A5 (enExample) 2020-04-09
JP6751142B2 JP6751142B2 (ja) 2020-09-02

Family

ID=57345910

Family Applications (7)

Application Number Title Priority Date Filing Date
JP2018521433A Expired - Fee Related JP7066609B2 (ja) 2015-11-12 2016-11-11 霊長類種に由来する標準化された神経細胞アッセイ
JP2018524194A Active JP6751142B2 (ja) 2015-11-12 2016-11-11 父方ube3aの発現を誘導するオリゴヌクレオチド
JP2018521350A Pending JP2018533362A (ja) 2015-11-12 2016-11-11 薬物候補の有効性プロファイルを決定するための方法
JP2020136397A Active JP7279000B2 (ja) 2015-11-12 2020-08-12 父方ube3aの発現を誘導するオリゴヌクレオチド
JP2021113496A Pending JP2021191262A (ja) 2015-11-12 2021-07-08 薬物候補の有効性プロファイルを決定するための方法
JP2023009553A Pending JP2023052630A (ja) 2015-11-12 2023-01-25 父方ube3aの発現を誘導するオリゴヌクレオチド
JP2024196762A Pending JP2025024060A (ja) 2015-11-12 2024-11-11 父方ube3aの発現を誘導するオリゴヌクレオチド

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2018521433A Expired - Fee Related JP7066609B2 (ja) 2015-11-12 2016-11-11 霊長類種に由来する標準化された神経細胞アッセイ

Family Applications After (5)

Application Number Title Priority Date Filing Date
JP2018521350A Pending JP2018533362A (ja) 2015-11-12 2016-11-11 薬物候補の有効性プロファイルを決定するための方法
JP2020136397A Active JP7279000B2 (ja) 2015-11-12 2020-08-12 父方ube3aの発現を誘導するオリゴヌクレオチド
JP2021113496A Pending JP2021191262A (ja) 2015-11-12 2021-07-08 薬物候補の有効性プロファイルを決定するための方法
JP2023009553A Pending JP2023052630A (ja) 2015-11-12 2023-01-25 父方ube3aの発現を誘導するオリゴヌクレオチド
JP2024196762A Pending JP2025024060A (ja) 2015-11-12 2024-11-11 父方ube3aの発現を誘導するオリゴヌクレオチド

Country Status (30)

Country Link
US (10) US10494633B2 (enExample)
EP (5) EP3374499A1 (enExample)
JP (7) JP7066609B2 (enExample)
KR (4) KR20250121139A (enExample)
CN (5) CN116064540A (enExample)
AU (4) AU2016352836B2 (enExample)
CA (1) CA3004799C (enExample)
CL (3) CL2018001189A1 (enExample)
CO (1) CO2018004550A2 (enExample)
CR (3) CR20180264A (enExample)
DK (2) DK3374509T3 (enExample)
ES (2) ES2852998T4 (enExample)
FI (1) FI4220360T3 (enExample)
HK (2) HK1258309A1 (enExample)
HR (2) HRP20241138T1 (enExample)
HU (2) HUE067905T2 (enExample)
IL (4) IL281211B2 (enExample)
LT (2) LT3374509T (enExample)
MA (1) MA67580B1 (enExample)
MX (4) MX2018004978A (enExample)
PE (2) PE20210451A1 (enExample)
PH (1) PH12018501005A1 (enExample)
PL (2) PL4220360T3 (enExample)
RS (2) RS61529B9 (enExample)
RU (1) RU2742007C2 (enExample)
SG (1) SG11201803951QA (enExample)
SI (2) SI3374509T1 (enExample)
UA (1) UA125963C2 (enExample)
WO (3) WO2017081223A1 (enExample)
ZA (1) ZA201802494B (enExample)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
NZ599032A (en) 2009-09-11 2014-09-26 Isis Pharmaceuticals Inc Modulation of huntingtin expression
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
ES2809199T3 (es) 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
KR20240148947A (ko) 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
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
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
US10400243B2 (en) 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
IL281211B2 (en) 2015-11-12 2024-01-01 Hoffmann La Roche Oligonucleotides for inducing paternal ube3a expression
HRP20250804T1 (hr) 2017-12-01 2025-09-12 The Texas A&M University System Antisense liječenje angelmanovog sindroma
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
CN108795935B (zh) * 2018-05-23 2022-06-21 中国农业科学院上海兽医研究所(中国动物卫生与流行病学中心上海分中心) 日本血吸虫SjELAV-like 1基因的siRNA及其应用
CA3113648A1 (en) * 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
EP3911957A1 (en) 2019-01-17 2021-11-24 F. Hoffmann-La Roche AG E3 ubiquitin ligase (ube3a) protein targets
CN113490742A (zh) * 2019-02-20 2021-10-08 罗氏创新中心哥本哈根有限公司 膦酰基乙酸酯缺口聚物寡核苷酸
MD3947684T2 (ro) * 2019-03-29 2025-10-31 Ionis Pharmaceuticals Inc Compuși și metode pentru modularea UBE3A-ATS
AU2020279387A1 (en) * 2019-05-22 2021-12-09 The University Of North Carolina At Chapel Hill UBE3A genes and expression cassettes and their use
CN113874494A (zh) 2019-05-28 2021-12-31 豪夫迈·罗氏有限公司 产生单核细胞祖细胞的方法
TW202204607A (zh) * 2020-04-16 2022-02-01 英商羅斯林科技有限公司 誘導性多功能幹細胞(iPSC)之誘導
US20230212572A1 (en) * 2020-06-09 2023-07-06 Roche Innovation Center Copenhagen A/S Guanosine Analogues for Use in Therapeutics Polynucleotides
EP4282651A4 (en) 2021-01-21 2024-12-18 Fujimori Kogyo Co., Ltd. MULTILAYER FILM AND PACKAGING BAGS
CN119173632A (zh) 2022-05-10 2024-12-20 豪夫迈·罗氏有限公司 靶向cfp-elk1基因间区域的反义寡核苷酸
EP4705457A2 (en) 2023-05-04 2026-03-11 F. Hoffmann-La Roche AG Oligonucleotides capable of upregulating glucocerebrosidase expression
KR20250068540A (ko) * 2023-11-08 2025-05-16 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
WO2025125542A1 (en) 2023-12-15 2025-06-19 F. Hoffmann-La Roche Ag Intrathecal dosage method
WO2025213123A1 (en) * 2024-04-05 2025-10-09 Encoded Therapeutics, Inc. Methods and compositions for increasing expression of ube3a
TW202548015A (zh) * 2024-04-11 2025-12-16 美商奧崔基尼克斯製藥公司 天使症候群反義治療的給藥方案

Family Cites Families (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4373084A (en) 1981-07-07 1983-02-08 E. I. Du Pont De Nemours And Company Sulfur-modified polypropylene ether glycols, a method for preparing them, and polyurethanes prepared therefrom
ATE239484T1 (de) 1991-10-24 2003-05-15 Isis Pharmaceuticals Inc Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6617162B2 (en) 2001-12-18 2003-09-09 Isis Pharmaceuticals, Inc. Antisense modulation of estrogen receptor alpha expression
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
US6238921B1 (en) 1998-03-26 2001-05-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of human mdm2 expression
WO2000040749A2 (en) * 1999-01-06 2000-07-13 Genenews Inc. Method for the detection of gene transcripts in blood and uses thereof
KR100573231B1 (ko) 1999-02-12 2006-04-24 상꾜 가부시키가이샤 신규 뉴클레오시드 및 올리고뉴클레오티드 유사체
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
EP1234174A1 (en) 1999-12-02 2002-08-28 Myriad Genetics, Inc. Protein-protein interactions
US6300132B1 (en) 1999-12-17 2001-10-09 Isis Pharmaceuticals, Inc. Antisense inhibition of telomeric repeat binding factor 2 expression
AU2001268163A1 (en) * 2000-06-01 2001-12-11 Genaissance Pharmaceuticals, Inc. Haplotypes of the ube3a gene
US20030087855A1 (en) 2001-09-13 2003-05-08 Isis Pharmaceuticals Inc. Antisense modulation of protein kinase R expression
CA2451643C (en) * 2001-06-21 2012-11-13 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US20050124572A1 (en) 2002-06-17 2005-06-09 Freier Susan M. Compositions and their uses directed to signal tranducers
WO2004016754A2 (en) 2002-08-14 2004-02-26 Pharmacia Corporation ANTISENSE MODULATION OF Nav1.3 EXPRESSION
WO2004028458A2 (en) * 2002-09-25 2004-04-08 Pharmacia Corporation Antisense modulation of microsomal prostaglandin e2 synthase expression
EP2752488B1 (en) 2002-11-18 2020-02-12 Roche Innovation Center Copenhagen A/S Antisense design
KR20060115351A (ko) 2003-08-29 2006-11-08 위스콘신 얼럼나이 리서어치 화운데이션 영장류 배아 줄기 세포 기원의 신경 줄기 세포, 운동 뉴런및 도파민 뉴런의 시험관내 분화 방법
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
CN103301475B (zh) 2005-12-28 2016-08-03 斯克里普斯研究所 药物组合物和表达载体以及调节基因表达的方法和核酸分子的应用
CA2640171C (en) 2006-01-27 2014-10-28 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
CA3044969A1 (en) 2006-05-05 2007-12-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gene expression
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
JP5441688B2 (ja) 2006-05-11 2014-03-12 アイシス ファーマシューティカルズ, インコーポレーテッド 5’修飾二環式核酸類似体
US8580756B2 (en) 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
CN101796062B (zh) 2007-07-05 2014-07-30 Isis制药公司 6-双取代双环核酸类似物
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
WO2009090182A1 (en) 2008-01-14 2009-07-23 Santaris Pharma A/S C4'-substituted - dna nucleotide gapmer oligonucleotides
WO2009124238A1 (en) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
DK2356129T3 (da) 2008-09-24 2013-05-13 Isis Pharmaceuticals Inc Substituerede alpha-L-bicykliske nukleosider
WO2010096496A2 (en) * 2009-02-17 2010-08-26 Memorial Sloan-Kettering Cancer Center Methods of neural conversion of human embryonic stem cells
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
AU2010306377B2 (en) 2009-10-13 2015-01-22 Stemcell Technologies Canada Inc. Manipulation of osmolality for differentiating stem cells
KR101891352B1 (ko) 2009-12-23 2018-08-24 큐알엔에이, 인크. 간세포 성장 인자(hgf)에 대한 천연 안티센스 전사체의 억제에 의한 hgf 관련 질환의 치료
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
CA2805318A1 (en) 2010-07-14 2012-01-19 Curna, Inc. Treatment of discs large homolog (dlg) related diseases by inhibition of natural antisense transcript to dlg
US20130225659A1 (en) 2010-07-19 2013-08-29 Isis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
KR20160033798A (ko) * 2010-08-19 2016-03-28 에프. 호프만-라 로슈 아게 체세포의 유도된 재프로그래밍된 신경 줄기 세포 (irnscs) 로의 전환
WO2012064806A2 (en) 2010-11-11 2012-05-18 The University Of North Carolina At Chapel Hill Methods and compositions for unsilencing imprinted genes
WO2012068405A2 (en) 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
WO2012135621A2 (en) 2011-03-30 2012-10-04 Cellular Dynamics International. Inc Priming of pluripotent stem cells for neural differentiation
KR20140031217A (ko) 2011-04-20 2014-03-12 로슈 글리카트 아게 혈액-뇌 차단벽의 pH 의존적 통과를 위한 방법 및 구축물
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CA2863253A1 (en) 2011-09-07 2013-03-14 Marina Biotech, Inc. Synthesis and uses of nucleic acid compounds with conformationally restricted monomers
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
ES2809199T3 (es) * 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
JP6452614B2 (ja) 2012-11-15 2019-01-16 ロシュ イノベーション センター コペンハーゲン エーエス オリゴヌクレオチドコンジュゲート
WO2014077693A1 (en) 2012-11-16 2014-05-22 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Means and methods for reducing an effect of aging in a mammalian cell
US9694036B2 (en) 2014-03-21 2017-07-04 Cellular Dynamics International, Inc. Production of midbrain dopaminergic neurons and methods for the use thereof
IL281211B2 (en) 2015-11-12 2024-01-01 Hoffmann La Roche Oligonucleotides for inducing paternal ube3a expression
RU2019143627A (ru) 2017-06-28 2021-07-28 Юниверсити Оф Соус Флорида Модифицированный ген ube3a для генной терапии синдрома ангельмана
HRP20250804T1 (hr) 2017-12-01 2025-09-12 The Texas A&M University System Antisense liječenje angelmanovog sindroma
JP7383618B2 (ja) 2018-01-26 2023-11-20 エフ. ホフマン-ラ ロシュ アーゲー 放射標識オリゴヌクレオチドおよびその調製のための方法
MD3947684T2 (ro) 2019-03-29 2025-10-31 Ionis Pharmaceuticals Inc Compuși și metode pentru modularea UBE3A-ATS

Similar Documents

Publication Publication Date Title
JP2018533954A5 (enExample)
HRP20210227T1 (hr) Oligonukleotidi za indukciju ekspresije paternalnog gena ube3a
MX2023010958A (es) Agentes de arn modificados con efecto reducido fuera de la diana.
WO2015106128A3 (en) Rnai agents modified at the 4'-c-position
MX2024005653A (es) Agentes de acido ribonucleico (arn) de cadena doble modificados.
WO2013074974A3 (en) Modified rnai agents
JP2018519835A5 (enExample)
BR112013010525A2 (pt) tratamento de hbv
JP2015516989A5 (enExample)
BR112015022876A2 (pt) agente, composição farmacêutica e método para inibição da expressão do componente do complemento c5, uso do referido agente, vetor e célula.
RU2016123086A (ru) Способы и композиции для лечения болезни хантингтона
JP2018530530A5 (enExample)
BR112015025766A2 (pt) derivado de nucleosídeo altamente ativo para o tratamento de hcv
BR112015021470A2 (pt) conjugado de polímero para administração de um agente bioativo
PE20210668A1 (es) Analogos de carba-nucleosidos sustituidos con 2'-fluoro antivirales
BR112014019584A8 (pt) Composto da fórmula (i), sua forma tautomérica, seu isômero, ou seu sal farmaceuticamente aceitável, composição farmacêutica, método de inibição da replicação de um vírus contendo rna, método de tratamento ou prevenção de infecção causada por um vírus contendo rna, e, método de tratamento de infecção por hepatite c
MX2020005680A (es) Tratamiento antisentido del sindrome de angelman.
CL2013003105A1 (es) Compuestos de microarn que comprende secuencias de nucleósidos modificados, inhibidores de mir-21; usos de los compuestos para modular la actividad de mir -21 y para preparar un medicamento para tratar fibrosis, heridas o cancer
JP2016520310A5 (enExample)
EA201000085A1 (ru) dsPHK, ПРЕДНАЗНАЧЕННАЯ ДЛЯ ЛЕЧЕНИЯ ВИРУСНОЙ ИНФЕКЦИИ
JP2021500016A5 (enExample)
JP2011515357A5 (enExample)
WO2013162350A3 (en) Circular antiviral rna
JP2011519365A5 (enExample)
BR112018010736A2 (pt) oligonucleotídeos antissenso contra il-34 e métodos de uso dos mesmos