JP2012528572A5 - - Google Patents

Download PDF

Info

Publication number
JP2012528572A5
JP2012528572A5 JP2012513363A JP2012513363A JP2012528572A5 JP 2012528572 A5 JP2012528572 A5 JP 2012528572A5 JP 2012513363 A JP2012513363 A JP 2012513363A JP 2012513363 A JP2012513363 A JP 2012513363A JP 2012528572 A5 JP2012528572 A5 JP 2012528572A5
Authority
JP
Japan
Prior art keywords
region
polynucleotide
complementary
self
target
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
JP2012513363A
Other languages
English (en)
Japanese (ja)
Other versions
JP2012528572A (ja
JP5875976B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2010/036962 external-priority patent/WO2010141511A2/en
Publication of JP2012528572A publication Critical patent/JP2012528572A/ja
Publication of JP2012528572A5 publication Critical patent/JP2012528572A5/ja
Application granted granted Critical
Publication of JP5875976B2 publication Critical patent/JP5875976B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2012513363A 2009-06-01 2010-06-01 多価ｒｎａ干渉のためのポリヌクレオチド、組成物およびそれらの使用方法 Expired - Fee Related JP5875976B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US18301109P 2009-06-01 2009-06-01
US61/183,011 2009-06-01
PCT/US2010/036962 WO2010141511A2 (en) 2009-06-01 2010-06-01 Polynucleotides for multivalent rna interference, compositions and methods of use thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2015151507A Division JP2015192674A (ja) 2009-06-01 2015-07-31 多価ｒｎａ干渉のためのポリヌクレオチド、組成物およびそれらの使用方法

Publications (3)

Publication Number Publication Date
JP2012528572A JP2012528572A (ja) 2012-11-15
JP2012528572A5 true JP2012528572A5 (enExample) 2013-05-30
JP5875976B2 JP5875976B2 (ja) 2016-03-02

Family

ID=43298453

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2012513363A Expired - Fee Related JP5875976B2 (ja) 2009-06-01 2010-06-01 多価ｒｎａ干渉のためのポリヌクレオチド、組成物およびそれらの使用方法
JP2015151507A Pending JP2015192674A (ja) 2009-06-01 2015-07-31 多価ｒｎａ干渉のためのポリヌクレオチド、組成物およびそれらの使用方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2015151507A Pending JP2015192674A (ja) 2009-06-01 2015-07-31 多価ｒｎａ干渉のためのポリヌクレオチド、組成物およびそれらの使用方法

Country Status (7)

Country Link
US (3) US9200276B2 (enExample)
EP (1) EP2438168B1 (enExample)
JP (2) JP5875976B2 (enExample)
CN (1) CN102575252B (enExample)
CA (1) CA2764158A1 (enExample)
ES (1) ES2804764T3 (enExample)
WO (1) WO2010141511A2 (enExample)

Families Citing this family (152)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010141511A2 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
US9115167B2 (en) * 2011-03-10 2015-08-25 Biomics Biotechnologies Co., Ltd. Multi-targets interfering RNA molecules and their applications
WO2013075035A1 (en) 2011-11-18 2013-05-23 Alnylam Pharmaceuticals Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
HK1200191A1 (en) 2011-11-18 2015-07-31 Alnylam Pharmaceuticals, Inc. Modified rnai agents
WO2013103693A1 (en) * 2012-01-04 2013-07-11 Protiva Biotherapeutics Inc. Nicked polynucleotide complexes for multivalent rna interference
WO2013180038A1 (ja) * 2012-05-26 2013-12-05 株式会社ボナック デリバリー機能を有する遺伝子発現制御用の一本鎖核酸分子
CA2879683C (en) 2012-08-03 2023-02-14 Alnylam Pharmaceuticals, Inc. Modified rnai agents
PT2929031T (pt) 2012-12-05 2018-01-19 Alnylam Pharmaceuticals Inc Composições de irna de pcsk9 e métodos de seu uso
CN114015692B (zh) 2013-03-14 2024-12-31 阿尔尼拉姆医药品有限公司 补体组分C5 iRNA组合物及其使用方法
PE20152002A1 (es) 2013-05-01 2016-01-21 Isis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de ttr y vhb
EP3412774A1 (en) 2013-05-22 2018-12-12 Alnylam Pharmaceuticals, Inc. Serpina1 irna compositions and methods of use thereof
TWI727917B (zh) 2013-05-22 2021-05-21 美商阿尼拉製藥公司 ＴＭＰＲＳＳ６ｉＲＮＡ 組成物及其使用方法
TW201610151A (zh) 2013-09-23 2016-03-16 阿尼拉製藥公司 治療或預防與甲狀腺素運載蛋白相關疾病之方法
IL314045A (en) 2013-12-12 2024-09-01 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
US10612020B2 (en) 2013-12-26 2020-04-07 Tokyo Medical University Artificial mimic miRNA for controlling gene expression, and use of same
CA2935022A1 (en) 2013-12-27 2015-07-02 Bonac Corporation Artificial match-type mirna for controlling gene expression and use therefor
US10119136B2 (en) 2014-01-09 2018-11-06 Alnylam Pharmaceuticals, Inc. RNAi agents modified at the 4′-C position
KR102592370B1 (ko) 2014-02-11 2023-10-25 알닐람 파마슈티칼스 인코포레이티드 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법
WO2015134528A1 (en) * 2014-03-04 2015-09-11 Donald Danforth Plant Science Center New generation of artificial micrornas
CN103820442B (zh) * 2014-03-07 2015-10-21 中国农业科学院植物保护研究所 水稻黑条矮缩病毒RNAi多价靶基因序列及应用
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療ｓｅｒｐｉｎｃ１相關疾患之方法和組成物
MX382901B (es) 2014-05-22 2025-03-13 Alnylam Pharmaceuticals Inc Composiciones de angiotensinogeno (agt) arni y métodos de uso de las mismas.
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
TWI755351B (zh) 2014-10-10 2022-02-21 美商艾爾妮蘭製藥公司 用於抑制ｈａｏ１（羥酸氧化酶１（乙醇酸鹽氧化酶））基因表現的組合物及方法
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
CN107250362B (zh) 2014-11-17 2021-10-22 阿尔尼拉姆医药品有限公司 载脂蛋白C3(APOC3)iRNA组合物及其使用方法
US20170369872A1 (en) 2014-12-18 2017-12-28 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
CA2971827A1 (en) 2014-12-27 2016-06-30 Bonac Corporation Naturally occuring mirna for controlling gene expression, and use of same
JP2018510621A (ja) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
US11142769B2 (en) 2015-03-27 2021-10-12 Bonac Corporation Single-stranded nucleic acid molecule having delivery function and gene expression regulating ability
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
EP3340967B1 (en) * 2015-08-24 2024-05-22 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
SG10202007937SA (en) 2015-09-02 2020-09-29 Alnylam Pharmaceuticals Inc PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
KR20250078597A (ko) 2015-11-06 2025-06-02 아이오니스 파마수티컬즈, 인코포레이티드 아포리포프로테인 (a) 발현 조정
KR20210157474A (ko) 2015-12-07 2021-12-28 젠자임 코포레이션 Serpinc1-연관 장애의 치료를 위한 방법 및 조성물
EP3469083A1 (en) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
TW202313978A (zh) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
SG10201913552UA (en) 2016-12-16 2020-03-30 Alnylam Pharmaceuticals Inc Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
CN108342386B (zh) * 2017-01-22 2022-04-15 广州市锐博生物科技有限公司 一种多聚寡核酸分子及其在多靶标干扰中的应用
US20200163987A1 (en) 2017-07-10 2020-05-28 Genzyme Corporation Methods and compositions for treating a bleeding event in a subject having hemophilia
JP7277432B2 (ja) 2017-07-13 2023-05-19 アルナイラム ファーマシューティカルズ， インコーポレイテッド 乳酸脱水素酵素Ａ（ＬＤＨＡ）ｉＲＮＡ組成物及びその使用方法
US20230151370A1 (en) * 2017-07-14 2023-05-18 Halo-Bio Rnai Therapeutics, Inc. Methods and compositions for aptamer-driven surface formulation of self-forming polynucleotide nanoparticles
US20200385719A1 (en) 2017-11-16 2020-12-10 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
BR112020012088A2 (pt) 2017-12-18 2020-11-17 Alnylam Pharmaceuticals, Inc. composições de irna do grupo de elevada mobilidade box-1 (hmgb1) e métodos de uso dos mesmos
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
AU2018433363B2 (en) * 2018-07-20 2022-12-08 Guangzhou Ribobio Co., Ltd. Nucleic acid unit and polymeric nucleic acid and application thereof
CN113227371A (zh) * 2018-08-27 2021-08-06 圣诺制药公司 微型发夹RNAi触发物(mxRNA)和其使用方法
US20210332367A1 (en) 2018-09-18 2021-10-28 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
KR20210093851A (ko) * 2018-09-28 2021-07-28 서나오믹스, 인크. 표적과의 상보적 상호 작용을 통해 유전자 발현을 조절하는 다수의 올리고뉴클레오티드로 구성된 다중-표적화 핵산 구조체
WO2020069055A1 (en) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
WO2020232430A1 (en) * 2019-05-16 2020-11-19 University Of South Florida Methods of downregulating ccl20 genes for treatment of traumatic brain injuries
AU2020279101B2 (en) 2019-05-17 2025-07-24 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021022109A1 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. SERPIN FAMILY F MEMBER 2 (SERPINF2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
US12486512B2 (en) 2019-10-18 2025-12-02 Alnylam Pharmaceuticals, Inc. Solute carrier family member iRNA compositions and methods of use thereof
AU2020369515A1 (en) 2019-10-22 2022-04-21 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
CN119499394A (zh) 2019-11-01 2025-02-25 阿尔尼拉姆医药品有限公司 亨廷顿（HTT）iRNA药剂组合物及其使用方法
EP4055166A2 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
TWI877247B (zh) 2019-11-13 2025-03-21 美商阿尼拉製藥公司 用於治療血管收縮素原相關病症之方法及組成物
WO2021102373A1 (en) 2019-11-22 2021-05-27 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
PH12022551394A1 (en) 2019-12-13 2023-09-11 Alnylam Pharmaceuticals Inc Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
TW202138559A (zh) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）ｉＲＮＡ組成物及其使用方法
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
AU2021220765A1 (en) 2020-02-10 2022-09-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing VEGF-A expression
JP7735288B2 (ja) 2020-02-18 2025-09-08 アルナイラム ファーマシューティカルズ， インコーポレイテッド アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用法
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
WO2021178736A1 (en) 2020-03-06 2021-09-10 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
US20210363524A1 (en) * 2020-03-18 2021-11-25 University Of Massachusetts Oligonucleotides for snca modulation
AU2021238319A1 (en) * 2020-03-18 2022-10-06 University Of Massachusetts Oligonucleotides for MAPT modulation
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
EP4127134A4 (en) 2020-04-01 2024-07-31 Alnylam Pharmaceuticals, Inc. COMPOSITIONS OF ALPHA-2A ADRENERGIC RECEPTOR (ADRA2A) ARNI AGENTS AND METHODS OF USE THEREOF
AR121769A1 (es) 2020-04-06 2022-07-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para el silenciamiento de la expresión de myoc
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
CA3179678A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
KR20230018377A (ko) 2020-04-27 2023-02-07 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 e (apoe) irna 제제 조성물 및 이의 사용 방법
BR112022021136A2 (pt) 2020-04-30 2022-11-29 Alnylam Pharmaceuticals Inc Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas
EP4153746A1 (en) 2020-05-21 2023-03-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
JP2023529903A (ja) 2020-06-09 2023-07-12 アルナイラム ファーマシューティカルズ， インコーポレイテッド ＧＰＡＭ（グリセロール－３－リン酸アシルトランスフェラーゼ１、ミトコンドリア）発現をサイレンシングするためのｓｉＲＮＡ組成物および方法
TW202214856A (zh) 2020-06-18 2022-04-16 美商阿尼拉製藥公司 黃嘌呤脫氫酶（ＸＤＨ）ｉＲＮＡ組成物及其使用之方法
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
IL302709A (en) 2020-11-13 2023-07-01 Alnylam Pharmaceuticals Inc Coagulation factor iRNA compositions (F5) and methods of using them
AU2021393417A1 (en) 2020-12-01 2023-06-29 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
CN114606196A (zh) * 2020-12-04 2022-06-10 南京大学 一种进行siRNA表达和体内递送的细胞疗法
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
WO2022147223A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
AU2021411579A1 (en) 2020-12-31 2023-07-13 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
EP4288542A4 (en) * 2021-02-08 2025-08-20 Ractigen Therapeutics MULTIVALENT OLIGONUCLEOTIDE AGENT AND METHODS OF USE THEREOF
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
JP2024509783A (ja) 2021-02-25 2024-03-05 アルナイラム ファーマシューティカルズ， インコーポレイテッド プリオンタンパク質（ｐｒｎｐ）ｉｒｎａ組成物およびその使用方法
AU2022226098A1 (en) 2021-02-26 2023-08-24 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
IL305418A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
MX2023011466A (es) 2021-03-29 2024-02-01 Alnylam Pharmaceuticals Inc Composiciones de agentes de ácido ribonucleico de interferencia (arni) de huntingtina (htt) y métodos de uso de estas.
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
TW202309280A (zh) 2021-04-26 2023-03-01 美商艾拉倫製藥股份有限公司 跨膜絲胺酸蛋白酶６（ＴＭＰＲＳＳ６）ｉＲＮＡ組成物及其使用方法
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4341401A1 (en) 2021-05-18 2024-03-27 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
TW202308663A (zh) 2021-06-04 2023-03-01 美商艾拉倫製藥股份有限公司 人類染色體９開讀框７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ劑組成物及其使用方法
EP4351541A2 (en) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
EP4359536A4 (en) * 2021-06-21 2025-12-03 Univ North Carolina Chapel Hill INVERTED CHIMERIC RNA S1 MOLECULES AND THEIR METHODS OF USE
AU2022303164A1 (en) 2021-06-30 2024-01-18 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
JP2024527584A (ja) 2021-07-09 2024-07-25 アルナイラム ファーマシューティカルズ， インコーポレイテッド ＣＮＳ送達のためのＢｉｓ－ＲＮＡｉ化合物
JP2024528659A (ja) 2021-07-19 2024-07-30 アルナイラム ファーマシューティカルズ， インコーポレイテッド 非原発性高シュウ酸尿症疾患または障害を有するまたは発症するリスクがある対象を治療するための方法および組成物
CA3226887A1 (en) 2021-07-21 2023-01-26 Alnylam Pharmaceuticals, Inc. Metabolic disorder-associated target gene irna compositions and methods of use thereof
TW202325312A (zh) 2021-07-23 2023-07-01 美商艾拉倫製藥股份有限公司 β－鏈蛋白（ＣＴＮＮＢ１）ｉＲＮＡ組成物及其使用方法
EP4377458A1 (en) 2021-07-29 2024-06-05 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
CA3227852A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
AU2022324003A1 (en) 2021-08-04 2024-02-08 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
MX2024001573A (es) 2021-08-13 2024-02-14 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) contra el factor xii (f12) y sus metodos de uso.
JP2024538859A (ja) 2021-08-31 2024-10-24 アルナイラム ファーマシューティカルズ， インコーポレイテッド 細胞死誘導ＤＦＦＡ様エフェクターＢ（ＣＩＤＥＢ）ｉＲＮＡ組成物およびその使用方法
JP2024531728A (ja) * 2021-09-14 2024-08-29 アルゴノート アールエヌエー リミテッド 心血管疾患の処置
JP2024535850A (ja) 2021-09-17 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド 補体成分（Ｃ３）をサイレンシングするためのｉＲＮＡ組成物および方法
JP2024535888A (ja) 2021-09-20 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド インヒビンサブユニットベータｅ（ｉｎｈｂｅ）モジュレーター組成物およびその使用方法
MX2024003519A (es) 2021-09-24 2024-04-01 Alnylam Pharmaceuticals Inc Composiciones de agentes de acido ribonucleico de interferencia (arni) de proteina tau asociada a microtubulos (mapt) y sus metodos de uso.
KR20240101590A9 (ko) 2021-10-15 2025-12-10 알닐람 파마슈티칼스 인코포레이티드 간외 전달 irna 조성물 및 이를 이용하는 방법
JP2024539512A (ja) 2021-10-22 2024-10-28 セイル バイオメディシンズ インコーポレイテッド Ｍｒｎａワクチン組成物
JP2024541974A (ja) 2021-10-29 2024-11-13 アルナイラム ファーマシューティカルズ， インコーポレイテッド ハンチンチン（ＨＴＴ）ｉＲＮＡ剤組成物およびその使用方法
CA3234636A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
CN118488834A (zh) 2021-11-23 2024-08-13 赛欧生物医药股份有限公司 细菌源性脂质组合物和其用途
EP4452239A1 (en) 2021-12-20 2024-10-30 Sail Biomedicines, Inc. Compositions comprising mrna and lipid reconstructed plant messenger packs
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
EP4547683A2 (en) 2022-06-30 2025-05-07 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
EP4569113A1 (en) 2022-09-15 2025-06-18 Regeneron Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
JP2025532985A (ja) 2022-09-30 2025-10-03 アルナイラム ファーマシューティカルズ， インコーポレイテッド 修飾二本鎖ｒｎａ剤
WO2024102434A1 (en) 2022-11-10 2024-05-16 Senda Biosciences, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs
WO2024159172A1 (en) 2023-01-27 2024-08-02 Senda Biosciences, Inc. A modified lipid composition and uses thereof
WO2024216155A1 (en) 2023-04-12 2024-10-17 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
WO2024220625A1 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids
WO2024220712A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Vaccine compositions
WO2024228030A2 (en) 2023-05-04 2024-11-07 Argonaute RNA Limited Dual silencing
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
WO2025076031A2 (en) 2023-10-03 2025-04-10 Alnylam Pharmaceuticals, Inc. Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof
US20260043035A1 (en) 2024-08-08 2026-02-12 Invaio Sciences, Inc. Nature-derived lipid particles for use in agriculture

Family Cites Families (124)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
AU5661386A (en) 1985-03-15 1986-10-13 Stirchak, E. Stereoregular polynucleotide-binding polymers
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
EP0705334A1 (en) 1993-06-14 1996-04-10 Basf Aktiengesellschaft Tight control of gene expression in eucaryotic cells by tetracycline-responsive promoters
US5856174A (en) 1995-06-29 1999-01-05 Affymetrix, Inc. Integrated nucleic acid diagnostic device
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US20040171031A1 (en) 1996-06-06 2004-09-02 Baker Brenda F. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US5874554A (en) 1996-12-13 1999-02-23 Incyte Pharmaceuticals, Inc. Methods and solvent vehicles for reagent delivery in oligonucleotide synthesis using automated pulse jetting devices
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6218181B1 (en) 1998-03-18 2001-04-17 The Salk Institute For Biological Studies Retroviral packaging cell line
WO2000043507A1 (en) 1999-01-19 2000-07-27 Unilever Plc Method for producing antibody fragments
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
KR100909681B1 (ko) 2000-12-01 2009-07-29 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US7423142B2 (en) 2001-01-09 2008-09-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US20070270579A1 (en) 2001-05-18 2007-11-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20050191618A1 (en) * 2001-05-18 2005-09-01 Sirna Therapeutics, Inc. RNA interference mediated inhibition of human immunodeficiency virus (HIV) gene expression using short interfering nucleic acid (siNA)
US20070173473A1 (en) * 2001-05-18 2007-07-26 Sirna Therapeutics, Inc. RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
US20050196781A1 (en) * 2001-05-18 2005-09-08 Sirna Therapeutics, Inc. RNA interference mediated inhibition of STAT3 gene expression using short interfering nucleic acid (siNA)
US10590418B2 (en) 2001-07-23 2020-03-17 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for RNAi mediated inhibition of gene expression in mammals
ES2546829T3 (es) 2001-07-23 2015-09-29 The Board Of Trustees Of The Leland Stanford Junior University Métodos y composiciones para la inhibición mediada por iARN de la expresión génica en mamíferos
CA2459347C (en) 2001-09-04 2012-10-09 Exiqon A/S Locked nucleic acid (lna) compositions and uses thereof
DE10163098B4 (de) 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
US7745418B2 (en) 2001-10-12 2010-06-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting viral replication
DE10202419A1 (de) 2002-01-22 2003-08-07 Ribopharma Ag Verfahren zur Hemmung der Expression eines durch eine Chromosomen-Aberration entstandenen Zielgens
KR100464261B1 (ko) 2002-01-24 2005-01-03 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US20100240730A1 (en) 2002-02-20 2010-09-23 Merck Sharp And Dohme Corp. RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA)
KR20030084444A (ko) 2002-04-26 2003-11-01 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
WO2004030634A2 (en) 2002-10-02 2004-04-15 Alnylam Pharmaceuticals Inc. Therapeutic compositions
WO2004035765A2 (en) 2002-10-18 2004-04-29 Nucleonics, Inc. Double-stranded rna structures and constructs, and methods for generating and using the same
US20080188429A1 (en) * 2002-12-27 2008-08-07 Iyer Radhakrishnan P Synthetic siRNA compounds and methods for the downregulation of gene expression
JP2004261002A (ja) 2003-01-08 2004-09-24 Tsutomu Suzuki ｓｉＲＮＡの製造方法
WO2004064737A2 (en) 2003-01-17 2004-08-05 Alnylam Pharmaceuticals Therapeutics compositions
AU2004220556B2 (en) 2003-03-07 2009-05-07 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
US20050233342A1 (en) 2003-03-07 2005-10-20 Muthiah Manoharan Methods of preventing off-target gene silencing
EP1608735A4 (en) 2003-04-03 2008-11-05 Alnylam Pharmaceuticals RNAI CONJUGATES
WO2004091572A2 (en) 2003-04-09 2004-10-28 Biodelivery Sciences International, Inc. Cochleate compositions directed against expression of proteins
US20070179100A1 (en) 2003-04-09 2007-08-02 Muthiah Manoharan Protected monomers
CA2521464C (en) 2003-04-09 2013-02-05 Alnylam Pharmaceuticals, Inc. Irna conjugates
CA2522349A1 (en) 2003-04-17 2004-11-04 Alnylam Pharmaceuticals, Inc. Protected monomers
EP2669377A3 (en) 2003-04-17 2015-10-14 Alnylam Pharmaceuticals Inc. Modified iRNA agents
WO2004104199A2 (en) * 2003-05-15 2004-12-02 Oligo Engine, Inc. Modulation of gene expression using dna-dna hybrids
US7595306B2 (en) 2003-06-09 2009-09-29 Alnylam Pharmaceuticals Inc Method of treating neurodegenerative disease
CA2542232A1 (en) 2003-06-09 2005-01-20 Alnylam Pharmaceuticals, Inc. Method for treating neurodegenerative disease by inhibiting alpha-synuclein
EP1633770B1 (en) 2003-06-13 2015-04-29 Alnylam Europe AG Double-stranded ribonucleic acid with increased effectiveness in an organism
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
WO2005027979A2 (en) 2003-09-17 2005-03-31 Let There Be Hope Medical Research Institute Targeted lipid-drug formulations for delivery of drugs to myeloid and lymphoid immune cells
WO2005089224A2 (en) 2004-03-12 2005-09-29 Alnylam Pharmaceuticals, Inc. iRNA AGENTS TARGETING VEGF
JP2007531794A (ja) 2004-04-05 2007-11-08 アルニラム ファーマスーティカルズ インコーポレイテッド オリゴヌクレオチドの合成および精製に使用する方法および反応試薬
EP1768998A2 (en) 2004-04-27 2007-04-04 Alnylam Pharmaceuticals Inc. Single-stranded and double-stranded oligonucleotides comprising a 2-arylpropyl moiety
EP3034510A1 (en) 2004-04-30 2016-06-22 Alnylam Pharmaceuticals Inc. Oligonucleotides comprising a c5-modified pyrimidine
AU2005247509C1 (en) 2004-05-27 2012-09-20 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
EP1789553B1 (en) 2004-06-30 2014-03-26 Alnylam Pharmaceuticals Inc. Oligonucleotides comprising a non-phosphate backbone linkage
US20080213891A1 (en) 2004-07-21 2008-09-04 Alnylam Pharmaceuticals, Inc. RNAi Agents Comprising Universal Nucleobases
WO2006093526A2 (en) 2004-07-21 2006-09-08 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a modified or non-natural nucleobase
AU2005330637B2 (en) 2004-08-04 2012-09-20 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
EP1791567B1 (en) 2004-08-10 2015-07-29 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
CN101133074B (zh) 2004-09-24 2012-05-30 阿尔尼拉姆医药品有限公司 APOB的RNAi调节及其用途
AU2005323303A1 (en) 2004-11-24 2006-07-13 Alnylam Pharmaceuticals, Inc. RNAi modulation of the Bcr-Abl fusion gene and uses thereof
AU2005313883B2 (en) 2004-12-09 2011-03-31 Alnylam Pharmaceuticals, Inc. Compositions and methods for inducing an immune response in a mammal and methods of avoiding an immune response to oligonucleotide agents such as short interfering RNAs
ATE544774T1 (de) 2004-12-14 2012-02-15 Alnylam Pharmaceuticals Inc Rnai-modulation von mll-af4 und verwendungen dafür
EP2765189A1 (en) 2004-12-21 2014-08-13 Monsanto Technology LLC Recombinant DNA constructs and methods for controlling gene expression
US7169561B2 (en) 2004-12-29 2007-01-30 Applera Corporation Methods, compositions, and kits for forming self-complementary polynucleotides
US20090005332A1 (en) * 2004-12-30 2009-01-01 Hauser Todd M Compositions and Methods for Modulating Gene Expression Using Self-Protected Oligonucleotides
US7507809B2 (en) 2005-01-07 2009-03-24 Alnylam Pharmaceuticals, Inc. RNAi modulation of RSV and therapeutic uses thereof
US20060217324A1 (en) 2005-01-24 2006-09-28 Juergen Soutschek RNAi modulation of the Nogo-L or Nogo-R gene and uses thereof
US7718625B2 (en) * 2005-01-27 2010-05-18 University Of South Florida Polynucleotides targeted against the extended 5′-UTR region of argininosuccinate synthase and uses thereof
WO2006084209A2 (en) 2005-02-03 2006-08-10 Benitec, Inc. Rnai expression constructs
US7737265B2 (en) * 2005-06-27 2010-06-15 Alnylam Pharmaceuticals, Inc. RNAi modulation of HIF-1 and therapeutic uses thereof
AU2006272808C1 (en) 2005-07-21 2010-10-21 Alnylam Pharmaceuticals, Inc. RNAi modulation of the Rho-A gene and uses thereof
EP1917357A2 (en) * 2005-08-01 2008-05-07 Purdue Research Foundation Multivalent rna nanoparticles for delivery of active agents to a cell
WO2007021896A2 (en) 2005-08-10 2007-02-22 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides for use in modulating micro rna and uses thereof
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US20070148246A1 (en) * 2005-08-11 2007-06-28 Dan Luo Nucleic Acid-Based Matrixes
WO2007022470A2 (en) 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
CN101365801B (zh) 2005-10-28 2013-03-27 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
CA2627585A1 (en) 2005-11-01 2007-05-10 Alnylam Pharmaceuticals, Inc. Rnai inhibition of influenza virus replication
JP4929288B2 (ja) 2005-11-04 2012-05-09 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｎａｖ１．８遺伝子の発現を抑制するための組成物および方法
CA2626690A1 (en) 2005-11-09 2007-05-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor v leiden mutant gene
CA2527144C (en) 2005-11-15 2014-04-29 Queen's University At Kingston Reversibly switchable surfactants and methods of use thereof
BRPI0618907A2 (pt) 2005-11-25 2012-04-17 Mologen Ag c0nstructo de expressão de dna, método de produção de vetores, kit para produção de vetores e para produção de constructo de expressão
EP1989307B1 (en) 2006-02-08 2012-08-08 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
EP1993611A4 (en) 2006-03-16 2013-05-22 Alnylam Pharmaceuticals Inc RNAi MODULATION OF TGF-BETA AND THERAPEUTIC USES
JP5704741B2 (ja) 2006-03-31 2015-04-22 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｅｇ５遺伝子発現の抑制のための組成物および方法
JP4812874B2 (ja) 2006-04-28 2011-11-09 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｊｃウイルス遺伝子の発現を抑制するための組成物および方法
ES2657400T3 (es) 2006-05-10 2018-03-05 Sarepta Therapeutics, Inc. Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos
EP2194128B1 (en) 2006-05-11 2012-08-01 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
EP2016173A4 (en) * 2006-05-15 2010-06-09 Immunomedics Inc METHOD AND COMPOSITIONS FOR TREATING HUMAN IMMUNODEFECT VIRUS INFECTIONS WITH CONJUGATED ANTIBODIES OR ANTIBODY FRAGMENTS
BRPI0711950A2 (pt) 2006-05-19 2011-12-13 Scripps Research Inst tratamento para o dobramento inadequado da proteìna
JP2009537153A (ja) 2006-05-19 2009-10-29 アルニラム ファーマシューティカルズ， インコーポレイテッド ＡｈａのＲＮＡｉ調節およびその治療上の使用
AU2007253677B2 (en) 2006-05-22 2011-02-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of IKK-B gene
US8124752B2 (en) 2006-07-10 2012-02-28 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the MYC gene
US20080039415A1 (en) 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
US7737266B2 (en) 2006-09-18 2010-06-15 Board Of Regents, The University Of Texas System RNAi modulation of SCAP and therapeutics uses thereof
US7906484B2 (en) 2006-09-21 2011-03-15 Alnylam Pharmaceuticals, Inc. Complex for transferring an anionic substance into a cell
US20090209478A1 (en) 2006-09-21 2009-08-20 Tomoko Nakayama Compositions and methods for inhibiting expression of the hamp gene
EP2695608B1 (en) 2006-10-03 2016-11-23 Arbutus Biopharma Corporation Lipid containing formulations
US8034921B2 (en) 2006-11-21 2011-10-11 Alnylam Pharmaceuticals, Inc. IRNA agents targeting CCR5 expressing cells and uses thereof
AP3018A (en) 2007-03-29 2014-10-31 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expressionof a gene from the ebola
US8877917B2 (en) 2007-04-23 2014-11-04 Alnylam Pharmaceuticals, Inc. Glycoconjugates of RNA interference agents
WO2009018332A1 (en) 2007-08-01 2009-02-05 Alnylam Pharmaceuticals, Inc. Single-stranded and double-stranded oligonucleotides comprising a metal-chelating ligand
CA2884340C (en) 2007-11-15 2017-07-25 Sarepta Therapeutics, Inc. Method of synthesis of morpholino oligomers
CA2910760C (en) 2007-12-04 2019-07-09 Muthiah Manoharan Targeting lipids
WO2010078516A2 (en) * 2009-01-01 2010-07-08 Cornell University Multifunctional nucleic acid nano-structures
WO2010090452A2 (ko) * 2009-02-04 2010-08-12 성균관대학교산학협력단 세포 내 전달능이 증가된 작은 간섭 rna 복합체
US8841438B2 (en) 2009-05-22 2014-09-23 The Research Foundation Of The University Of New York Trans-acting RNA switches
WO2010141511A2 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof

Similar Documents

Publication Publication Date Title
JP2012528572A5 (enExample)
JP5875976B2 (ja) 多価ｒｎａ干渉のためのポリヌクレオチド、組成物およびそれらの使用方法
US10731157B2 (en) Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
JP2022526455A (ja) Ｒｎａを編集する方法および組成物
JP2016537990A5 (enExample)
HRP20190995T1 (hr) LIJEČENJE HBV-a
JP2019522461A5 (enExample)
JP2021500064A5 (enExample)
JP2018512110A5 (enExample)
CN108883201A (zh) Rna指导的治疗hiv感染的方法和组合物
JP2018537955A5 (enExample)
JP2012505657A5 (enExample)
JP2021500016A5 (enExample)
US20220348909A1 (en) Allosteric Conditional Guide RNAs for Cell-Selective Regulation of CRISPR/Cas
NZ604094A (en) Double stranded rna compounds to rhoa and use thereof
JP2014527823A5 (enExample)
Pandey RNA-based regulation in human health and disease
JP2020537514A5 (enExample)
JP2020146066A5 (enExample)
JP2020524495A5 (enExample)
US20230183705A1 (en) Products for suppressing or reducing the expression or activity of a snorna and uses thereof in the treatment of cancer
Piganeau et al. A yeast RNA-hybrid system for the detection of RNA–RNA interactions in vivo
JP2014003946A5 (enExample)
JP2016533190A5 (enExample)
JP2013513359A5 (enExample)