CN104411338A - 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 - Google Patents

用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 Download PDF

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Publication number
CN104411338A
CN104411338A CN201380028773.4A CN201380028773A CN104411338A CN 104411338 A CN104411338 A CN 104411338A CN 201380028773 A CN201380028773 A CN 201380028773A CN 104411338 A CN104411338 A CN 104411338A
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China
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optionally substituted
independently
alkyl
polypeptide
isolated polynucleotide
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Pending
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CN201380028773.4A
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Chinese (zh)
Inventor
斯蒂芬·邦塞尔
提尔塔·柴可拉博提
安东宁·德富热罗勒
S·M·伊巴希尔
马蒂亚斯·约翰
阿坦恩·洛依
苏珊·沃利斯基
K·M·伍德
保罗·哈塔拉
杰森·P·斯洛姆
凯内齐·伊杰贝
杰夫·L·埃尔斯沃思
贾斯汀·基尔德
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Moderna Inc
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Moderna Therapeutics Inc
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Priority to CN201810507242.8A priority Critical patent/CN108949772A/zh
Publication of CN104411338A publication Critical patent/CN104411338A/zh
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
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    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
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    • A61P11/00Drugs for disorders of the respiratory system
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    • A61P7/06Antianaemics
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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CN201380028773.4A 2012-04-02 2013-03-09 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 Pending CN104411338A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN201810507242.8A CN108949772A (zh) 2012-04-02 2013-03-09 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸

Applications Claiming Priority (101)

Application Number Priority Date Filing Date Title
US201261618878P 2012-04-02 2012-04-02
US201261618945P 2012-04-02 2012-04-02
US201261618862P 2012-04-02 2012-04-02
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PCT/US2013/030062 WO2013151666A2 (en) 2012-04-02 2013-03-09 Modified polynucleotides for the production of biologics and proteins associated with human disease

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CN201380028773.4A Pending CN104411338A (zh) 2012-04-02 2013-03-09 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
CN201810507242.8A Pending CN108949772A (zh) 2012-04-02 2013-03-09 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸

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Country Status (6)

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JP (6) JP2015518705A (2)
CN (2) CN104411338A (2)
AU (4) AU2013243949A1 (2)
CA (1) CA2868391A1 (2)
HK (1) HK1206612A1 (2)
WO (1) WO2013151666A2 (2)

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CN108430456A (zh) * 2015-10-22 2018-08-21 摩登纳特斯有限公司 癌症疫苗
CN109423514A (zh) * 2017-08-24 2019-03-05 上海市计划生育科学研究所 反复自然流产相关微小rna及其应用
CN109640962A (zh) * 2016-05-18 2019-04-16 摩登纳特斯有限公司 编码松弛素的多核苷酸
CN111164207A (zh) * 2017-07-27 2020-05-15 优卡瑞斯 新型嵌合酶及其应用
CN112334489A (zh) * 2018-05-16 2021-02-05 星火治疗有限公司 密码子优化的酸性α-葡萄糖苷酶表达盒及其使用方法
CN112567032A (zh) * 2018-04-18 2021-03-26 利甘达尔股份有限公司 基因组编辑的方法和组合物
CN112996519A (zh) * 2018-09-04 2021-06-18 德克萨斯大学系统董事会 用于核酸的器官特异性递送的组合物和方法
CN114222590A (zh) * 2019-03-27 2022-03-22 西吉隆医疗股份有限公司 用于因子vii疗法的组合物、装置和方法

Families Citing this family (287)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
ES2646669T3 (es) 2010-07-06 2017-12-14 Glaxosmithkline Biologicals Sa Procedimientos de aumento de una respuesta inmunitaria mediante el suministro de ARN
EP2591114B1 (en) 2010-07-06 2016-06-08 GlaxoSmithKline Biologicals SA Immunisation of large mammals with low doses of rna
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
LT4226941T (lt) 2010-08-31 2025-01-10 Glaxosmithkline Biologicals Sa Pegilintos liposomos, skirtos imunogeną koduojančios rnr pristatymui
EP2625189B1 (en) 2010-10-01 2018-06-27 ModernaTX, Inc. Engineered nucleic acids and methods of use thereof
WO2012051211A2 (en) 2010-10-11 2012-04-19 Novartis Ag Antigen delivery platforms
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
EP2729165B1 (en) 2011-07-06 2017-11-08 GlaxoSmithKline Biologicals SA Immunogenic combination compositions and uses thereof
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EP2858679B2 (en) 2012-06-08 2024-06-05 Translate Bio, Inc. Pulmonary delivery of mrna to non-lung target cells
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2014028429A2 (en) 2012-08-14 2014-02-20 Moderna Therapeutics, Inc. Enzymes and polymerases for the synthesis of rna
UA116217C2 (uk) 2012-10-09 2018-02-26 Санофі Пептидна сполука як подвійний агоніст рецепторів glp1-1 та глюкагону
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
WO2014093574A1 (en) * 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
AU2013366692B2 (en) 2012-12-21 2017-11-23 Sanofi Dual GLP1/GIP or trigonal GLP1/GIP/Glucagon agonists
AU2013374345A1 (en) * 2013-01-17 2015-08-06 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
BR112015022855A2 (pt) 2013-03-14 2017-11-07 Shire Human Genetic Therapies composições e método para produção de um anticorpo in vitro
JP6586075B2 (ja) 2013-03-14 2019-10-02 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの精製方法
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
RS57739B1 (sr) 2013-03-14 2018-12-31 Translate Bio Inc Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
EP3388834B1 (en) 2013-03-15 2020-04-15 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
WO2014152031A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Ribonucleic acid purification
US10590161B2 (en) 2013-03-15 2020-03-17 Modernatx, Inc. Ion exchange purification of mRNA
WO2014152027A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Manufacturing methods for production of rna transcripts
ES2644468T3 (es) 2013-03-15 2017-11-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Transgén sintético de la metilmalonil-CoA mutasa para el tratamiento de la acidemia metilmalónica (MMA) de clase MUT
US9944918B2 (en) 2013-03-15 2018-04-17 The United States Of America, As Represented By The Secretary, Dept. Of Health & Human Services Synthetic methylmalonyl-CoA mutase transgene for the treatment of MUT class methylmalonic acidemia (MMA)
EP2971165A4 (en) 2013-03-15 2016-11-23 Moderna Therapeutics Inc DISSOLUTION OF DNA FRAGMENTS IN MRNA MANUFACTURING METHODS
CA2917348A1 (en) 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
EP3041934A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
EP3052521A1 (en) * 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
AU2014337156A1 (en) * 2013-10-18 2016-05-12 Modernatx, Inc. Compositions and methods for tolerizing cellular systems
CN106413811A (zh) * 2013-10-22 2017-02-15 夏尔人类遗传性治疗公司 精氨基琥珀酸合成酶缺乏症的mrna疗法
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
AU2014340149B2 (en) 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
JP6506749B2 (ja) 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのmRNA療法
EP3080152A1 (en) 2013-12-13 2016-10-19 Sanofi Non-acylated exendin-4 peptide analogues
EP3080154B1 (en) 2013-12-13 2018-02-07 Sanofi Dual glp-1/gip receptor agonists
WO2015086728A1 (en) 2013-12-13 2015-06-18 Sanofi Exendin-4 peptide analogues as dual glp-1/gip receptor agonists
EP3053585A1 (en) * 2013-12-13 2016-08-10 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
WO2015086733A1 (en) 2013-12-13 2015-06-18 Sanofi Dual glp-1/glucagon receptor agonists
US20170002060A1 (en) * 2014-01-08 2017-01-05 Moderna Therapeutics, Inc. Polynucleotides for the in vivo production of antibodies
EP3092250A4 (en) 2014-01-08 2017-05-24 Moderna Therapeutics, Inc. Polynucleotides for the in vivo production of antibodies
SMT202000531T1 (it) 2014-03-09 2020-11-10 Univ Pennsylvania Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc)
TW201625670A (zh) 2014-04-07 2016-07-16 賽諾菲公司 衍生自exendin-4之雙重glp-1/升糖素受體促效劑
TW201625669A (zh) 2014-04-07 2016-07-16 賽諾菲公司 衍生自艾塞那肽-4(Exendin-4)之肽類雙重GLP-1/升糖素受體促效劑
TW201625668A (zh) 2014-04-07 2016-07-16 賽諾菲公司 作為胜肽性雙重glp-1/昇糖素受體激動劑之艾塞那肽-4衍生物
PL4023249T3 (pl) 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
EP3134546A4 (en) * 2014-04-24 2017-12-06 Dana-Farber Cancer Institute, Inc. Tumor suppressor and oncogene biomarkers predictive of anti-immune checkpoint inhibitor response
US9850269B2 (en) 2014-04-25 2017-12-26 Translate Bio, Inc. Methods for purification of messenger RNA
CN114395559A (zh) 2014-04-25 2022-04-26 吉尼松公司 高胆红素血症的治疗
JP6557722B2 (ja) 2014-05-30 2019-08-07 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 核酸の送達のための生分解性脂質
US9932381B2 (en) 2014-06-18 2018-04-03 Sanofi Exendin-4 derivatives as selective glucagon receptor agonists
WO2015196128A2 (en) 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
KR102559979B1 (ko) 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
US20170204152A1 (en) 2014-07-16 2017-07-20 Moderna Therapeutics, Inc. Chimeric polynucleotides
US10407683B2 (en) 2014-07-16 2019-09-10 Modernatx, Inc. Circular polynucleotides
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
KR20230145241A (ko) 2014-10-01 2023-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 항원 및 어쥬번트로서 인터류킨-21을 갖는 백신
WO2016077123A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Multiparametric nucleic acid optimization
JP6767976B2 (ja) 2014-12-05 2020-10-14 トランスレイト バイオ, インコーポレイテッド 関節疾患の治療のためのメッセンジャーrna治療法
US11147887B2 (en) 2014-12-17 2021-10-19 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson disease
US11118238B2 (en) * 2014-12-17 2021-09-14 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions
WO2016149508A1 (en) 2015-03-19 2016-09-22 Shire Human Genetic Therapies, Inc. Mrna therapy for pompe disease
WO2016155809A1 (en) * 2015-03-31 2016-10-06 Biontech Rna Pharmaceuticals Gmbh Lipid particle formulations for delivery of rna and water-soluble therapeutically effective compounds to a target cell
SI3286209T1 (sl) * 2015-04-24 2021-01-29 Ferring Bv Postopek izdelave gonadotropina
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
BR102015012334A2 (pt) * 2015-05-27 2016-11-29 Fundação Hemoct De Ribeirão Preto Fundherp processo de produção do fator vii de coagulação sanguínea e fator vii de coagulação sanguínea
AR105319A1 (es) 2015-06-05 2017-09-27 Sanofi Sa Profármacos que comprenden un conjugado agonista dual de glp-1 / glucagón conector ácido hialurónico
EP3101125A1 (en) 2015-06-05 2016-12-07 Laboratorios Del Dr. Esteve, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
TW201706291A (zh) 2015-07-10 2017-02-16 賽諾菲公司 作為選擇性肽雙重glp-1/升糖素受體促效劑之新毒蜥外泌肽(exendin-4)衍生物
MA42502A (fr) 2015-07-21 2018-05-30 Modernatx Inc Vaccins contre une maladie infectieuse
US10808242B2 (en) * 2015-08-28 2020-10-20 Biontech Rna Pharmaceuticals Gmbh Method for reducing immunogenicity of RNA
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
EP3350333B2 (en) 2015-09-17 2025-08-06 ModernaTX, Inc. Polynucleotides containing a stabilizing tail region
EP3359670B2 (en) 2015-10-05 2024-02-14 ModernaTX, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
JP6997704B2 (ja) 2015-10-14 2022-02-04 トランスレイト バイオ, インコーポレイテッド 生産性向上のためのrna関連酵素の修飾
ES2879400T3 (es) * 2015-10-21 2021-11-22 Us Health ADNc de ATP7A de tamaño reducido con codones optimizados y usos para el tratamiento de trastornos del transporte de cobre
AU2016342045A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Human cytomegalovirus vaccine
CA3005565A1 (en) * 2015-11-13 2017-05-18 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EA202190827A1 (ru) 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
MA43415A (fr) * 2015-12-09 2018-10-17 Modernatx Inc Arnm modifié codant pour une uridine diphosphate glucuronosyltransférase et utilisations associées
JP7061067B2 (ja) 2015-12-14 2022-04-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クリグラー・ナジャー症候群の処置のための組成物
MA43568A (fr) 2015-12-17 2018-11-14 Modernatx Inc Polynucléotides codant pour la méthylmalonyl-coa mutase
PL3394093T3 (pl) 2015-12-23 2022-05-16 Modernatx, Inc. Metody stosowania polinukleotydów kodujących ligand ox40
MA43587A (fr) 2016-01-10 2018-11-14 Modernatx Inc Arnm thérapeutiques codant pour des anticorps anti-ctla-4
US10717774B2 (en) 2016-03-14 2020-07-21 Kiromic, Inc. Compositions and methods for treating cancers
EP3825400B1 (en) 2016-04-08 2024-12-25 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
WO2017201342A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
US11001861B2 (en) 2016-05-18 2021-05-11 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
JP2019519601A (ja) 2016-05-18 2019-07-11 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド
EP3458108A4 (en) 2016-05-18 2020-04-22 ModernaTX, Inc. POLYNUCLEOTIDES ENCODING A CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS
MA45036A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2
US12123030B2 (en) * 2016-05-18 2024-10-22 Modernatx, Inc. Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia
EP3469074B1 (en) 2016-06-13 2020-12-09 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
WO2017223176A1 (en) 2016-06-24 2017-12-28 Modernatx, Inc. Methods and apparatus for filtration
MX2019000205A (es) 2016-07-07 2019-09-23 Rubius Therapeutics Inc Composiciones y metodos relacionados con sistemas celulares terapeuticos que expresan arn exogeno.
CN113481294B (zh) 2016-07-08 2024-03-12 花王株式会社 核酸样品的制备方法
EP3293259A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
MA46761A (fr) * 2016-11-10 2019-09-18 Translate Bio Inc Administration sous-cutanée d'arn messager
MA46766A (fr) 2016-11-11 2019-09-18 Modernatx Inc Vaccin antigrippal
WO2018104540A1 (en) * 2016-12-08 2018-06-14 Curevac Ag Rnas for wound healing
WO2018127382A1 (en) * 2017-01-03 2018-07-12 Ethris Gmbh Ornithine transcarbamylase coding polyribonucleotides and formulations thereof
US11993645B2 (en) 2017-01-11 2024-05-28 The Board Of Trustees Of The Leland Stanford Junior University Compositions comprising R-Spondin (RSPO) surrogate molecules
WO2018140821A1 (en) 2017-01-26 2018-08-02 Surrozen, Inc. Tissue-specific wnt signal enhancing molecules and uses thereof
BR112019015797A2 (pt) 2017-02-01 2020-03-17 Modernatx, Inc. Composições de mrna terapêuticas imunomoduladoras que codificam peptídeos de mutação de oncogene de ativação
EP3585895A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Compositions and methods for gene editing
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
KR102746901B1 (ko) 2017-03-03 2024-12-26 옵시디안 테라퓨틱스, 인크. Cd19 조성물 및 면역요법을 위한 방법
CA3055200A1 (en) 2017-03-03 2018-09-07 Obsidian Therapeutics, Inc. Compositions and methods for immunotherapy
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
JP7285220B2 (ja) 2017-05-18 2023-06-01 モデルナティエックス インコーポレイテッド 連結したインターロイキン-12(il12)ポリペプチドをコードするポリヌクレオチドを含む脂質ナノ粒子
EP3630200A4 (en) * 2017-05-31 2021-02-24 Arcturus Therapeutics, Inc. THERAPEUTICS FOR PHENYLKETONURIA
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
EP3638292A1 (en) 2017-06-14 2020-04-22 ModernaTX, Inc. Polynucleotides encoding coagulation factor viii
MA49421A (fr) * 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
US10034951B1 (en) 2017-06-21 2018-07-31 New England Biolabs, Inc. Use of thermostable RNA polymerases to produce RNAs having reduced immunogenicity
GB201710973D0 (en) 2017-07-07 2017-08-23 Avacta Life Sciences Ltd Scaffold proteins
EP3675817A1 (en) 2017-08-31 2020-07-08 Modernatx, Inc. Methods of making lipid nanoparticles
WO2019048632A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE
US20200208152A1 (en) 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
CA3079428A1 (en) * 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
EP3714045A1 (en) * 2017-11-22 2020-09-30 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
CN111819185A (zh) 2017-12-15 2020-10-23 旗舰创业创新第六有限责任公司 包含环状多核糖核苷酸的组合物及其用途
US11167043B2 (en) 2017-12-20 2021-11-09 Translate Bio, Inc. Composition and methods for treatment of ornithine transcarbamylase deficiency
IT201800003279A1 (it) * 2018-03-05 2019-09-05 Consiglio Nazionale Ricerche Peptide neurotrofico per il trattamento terapeutico di patologie neurodegenerative e/o infiammatorie.
EP3762495A4 (en) * 2018-03-07 2021-12-22 Icahn School of Medicine at Mount Sinai SPHINGOLIPID-METABOLIZING PROTEINS CODING FOR MODRNA
EP3775211B1 (en) 2018-04-12 2023-04-05 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
TW202016314A (zh) 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法
AU2019271132B2 (en) * 2018-05-15 2026-02-05 Translate Bio, Inc. Subcutaneous delivery of messenger RNA
AU2019293286A1 (en) 2018-06-28 2021-01-07 Crispr Therapeutics Ag Compositions and methods for genomic editing by insertion of donor polynucleotides
CA3104868A1 (en) 2018-07-09 2020-01-16 Surrozen, Inc. Tissue-specific wnt signal enhancing molecules and uses thereof
US12091675B2 (en) 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
KR102950141B1 (ko) 2018-08-24 2026-04-07 트랜슬레이트 바이오 인코포레이티드 전령 rna의 정제 방법
WO2020047201A1 (en) 2018-09-02 2020-03-05 Modernatx, Inc. Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency
WO2020056155A2 (en) 2018-09-13 2020-03-19 Modernatx, Inc. Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex e1-alpha, e1-beta, and e2 subunits for the treatment of maple syrup urine disease
MX2021003015A (es) 2018-09-13 2021-07-15 Modernatx Inc Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis.
JP2022500444A (ja) 2018-09-14 2022-01-04 モダーナティエックス・インコーポレイテッドModernaTX, Inc. クリグラー−ナジャー症候群の治療のためのウリジン二リン酸グリコシルトランスフェラーゼ1ファミリー、ポリペプチドa1をコードするポリヌクレオチド
EP3849617A1 (en) * 2018-09-14 2021-07-21 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
EP3856233A1 (en) * 2018-09-27 2021-08-04 Modernatx, Inc. Polynucleotides encoding arginase 1 for the treatment of arginase deficiency
AU2019355177A1 (en) 2018-10-04 2021-05-06 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed RNA
US11072808B2 (en) 2018-10-04 2021-07-27 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed RNA
JP7301326B2 (ja) * 2018-10-16 2023-07-03 公立大学法人横浜市立大学 新生児期~小児期発症の脳小血管病又はその保因者の検出方法
MX2021004684A (es) 2018-10-23 2021-06-04 Glycardial Diagnostics S L Anticuerpos especificos para apoj glucosilada y usos de los mismos.
AU2019375450A1 (en) 2018-11-07 2021-06-17 Vivet Therapeutics Codon-optimized ABCB11 transgene for the treatment of progressive familial intrahepatic cholestasis type 2 (PFIC2)
WO2020106946A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
GB201821089D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
TWI851647B (zh) 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
CN109734791B (zh) * 2019-01-17 2022-07-12 武汉明德生物科技股份有限公司 人nf186抗原、人nf186抗体检测试剂盒及其制备方法与应用
WO2020198403A2 (en) 2019-03-25 2020-10-01 Flagship Pioneering Innovations Vi, Llc Compositions comprising modified circular polyribonucleotides and uses thereof
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
JP2022532078A (ja) 2019-05-08 2022-07-13 アストラゼネカ アクチボラグ 皮膚及び創傷のための組成物並びにその使用の方法
BR112021023656A2 (pt) * 2019-05-24 2022-02-01 Ultragenyx Pharmaceutical Inc Composições e métodos para tratamento de hemocromatose
US20220267751A1 (en) * 2019-07-12 2022-08-25 Monash University Methods for making recombinant protein
US20220281911A1 (en) 2019-08-19 2022-09-08 Mina Therapeutics Limited Oligonucleotide conjugate compositions and methods of use
KR20220051401A (ko) 2019-09-06 2022-04-26 크리스퍼 테라퓨틱스 아게 배양 중 개선된 지속성을 갖는 유전적으로 조작된 t 세포
EP4031662A1 (en) 2019-09-20 2022-07-27 Translate Bio, Inc. Mrna encoding engineered cftr
WO2021074695A1 (en) 2019-10-16 2021-04-22 Avacta Life Sciences Limited PD-L1 INHIBITOR - TGFβ INHIBITOR BISPECIFIC DRUG MOIETIES.
US20230242655A1 (en) 2019-12-03 2023-08-03 Evotec International Gmbh Interferon-associated antigen binding proteins and uses thereof
CN111041025B (zh) 2019-12-17 2021-06-18 深圳市瑞吉生物科技有限公司 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法
GB201918586D0 (en) 2019-12-17 2020-01-29 Patterson James Engineered platelets for targeted delivery of a therapeutic agent
CN112111524B (zh) * 2020-01-10 2024-02-27 深圳瑞吉生物科技有限公司 mRNA-GalNAc靶向分子的制备方法及其体内递送系统和应用
EP4107176A4 (en) * 2020-02-20 2024-03-20 Gritstone bio, Inc. ANTIGEN-BINDING PROTEINS TARGETING THE SHARED ANTIGEN KKLC-1
KR102462990B1 (ko) 2020-03-17 2022-11-07 에이비온 주식회사 인비트로 트랜스크립트 mRNA 및 이를 함유하는 약학조성물
CA3176844A1 (en) * 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
US12188060B2 (en) 2020-05-15 2025-01-07 Crispr Therapeutics Ag Messenger RNA encoding Cas9 for use in genome-editing systems
GB202101299D0 (en) 2020-06-09 2021-03-17 Avacta Life Sciences Ltd Diagnostic polypetides and methods
CN111744019B (zh) 2020-07-01 2023-08-04 深圳瑞吉生物科技有限公司 基于甘露糖的mRNA靶向递送系统及其应用
BR112023001456A2 (pt) * 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
US12419948B2 (en) 2020-08-31 2025-09-23 The Broad Institute, Inc. Immunogenic compositions and use thereof
CN116322716A (zh) 2020-09-23 2023-06-23 克里斯珀医疗股份公司 Regnase-1和/或TGFBRII被破坏的基因工程化T细胞具有改善的功能性和持久性
WO2022104131A1 (en) 2020-11-13 2022-05-19 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
JP2023549854A (ja) 2020-11-16 2023-11-29 スロゼン オペレーティング, インコーポレイテッド 肝臓特異的Wntシグナル増強分子およびその使用
WO2022137181A1 (en) 2020-12-23 2022-06-30 Crispr Therapeutics Ag Co-use of lenalidomide with car-t cells
EP4251193A4 (en) * 2020-12-26 2025-03-19 Duke University Compositions and methods for treating and/or preventing glycogen storage diseases
WO2022147044A1 (en) * 2020-12-28 2022-07-07 Gjerde Doulas T Temperature stable nucleic acid method for preparing vaccine
EP4284922A1 (en) 2021-01-27 2023-12-06 New England Biolabs, Inc. Faustovirus capping enzyme, mrna capping enzyme compositions, methods and kits
US11028379B1 (en) 2021-01-27 2021-06-08 New England Biolabs, Inc. FCE mRNA capping enzyme compositions, methods and kits
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
CN113209124B (zh) * 2021-02-24 2022-04-15 四川大学 Dna四面体在制备预防和治疗1型糖尿病的药物中的用途
EP4305153A1 (en) 2021-03-09 2024-01-17 CRISPR Therapeutics AG Genetically engineered t cells with ptpn2 knockout have improved functionality and anti-tumor activity
TW202305133A (zh) 2021-03-26 2023-02-01 英商米納治療有限公司 Tmem173 sarna組合物及其使用方法
WO2022234003A1 (en) 2021-05-07 2022-11-10 Avacta Life Sciences Limited Cd33 binding polypeptides with stefin a protein
GB202108585D0 (en) 2021-06-16 2021-07-28 Rockend Ltd Methods and compositions
US20230042860A1 (en) * 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Polycistronic expression vectors
US20240398940A1 (en) 2021-09-03 2024-12-05 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
US20230128917A1 (en) 2021-09-14 2023-04-27 Crispr Therapeutics Ag Genetically engineered immune cells having a disrupted cd83 gene
EP4413038A1 (en) 2021-10-07 2024-08-14 Avacta Life Sciences Limited Pd-l1 binding affimers
US20240409638A1 (en) 2021-10-07 2024-12-12 Avacta Life Sciences Limited Serum half-life extended pd-l1 binding polypeptides
US20250295755A1 (en) 2021-10-22 2025-09-25 Sail Biomedicines, Inc. Mrna vaccine composition
EP4422698A1 (en) 2021-10-29 2024-09-04 CureVac SE Improved circular rna for expressing therapeutic proteins
WO2023084399A1 (en) 2021-11-09 2023-05-19 Crispr Therapeutics Ag Genetically engineered immune cells expressing masked chimeric antigen receptors specific to protein tyrosine kinase 7
CA3238764A1 (en) 2021-11-23 2023-06-01 Siddharth Patel A bacteria-derived lipid composition and use thereof
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
MX2024006828A (es) * 2021-12-06 2024-06-19 Empirico Inc Tratamiento de enfermedades y trastornos relacionados con fgg.
WO2023111913A1 (en) 2021-12-15 2023-06-22 Crispr Therapeutics Ag Engineered anti-liv1 cell with regnase-1 and/or tgfbrii disruption
EP4452239A1 (en) 2021-12-20 2024-10-30 Sail Biomedicines, Inc. Compositions comprising mrna and lipid reconstructed plant messenger packs
US20230346836A1 (en) 2021-12-22 2023-11-02 Crispr Therapeutics Ag Genetically engineered t cells with disrupted casitas b-lineage lymphoma proto-oncogene-b (cblb) and uses thereof
US20250099614A1 (en) 2022-01-28 2025-03-27 CureVac SE Nucleic acid encoded transcription factor inhibitors
CN119213021A (zh) 2022-02-10 2024-12-27 艾菲赛尔治疗株式会社 与CD40L特异性结合的Stefin A蛋白变体及其用途
EP4238988A1 (en) 2022-03-01 2023-09-06 Consejo Superior De Investigaciones Científicas Antibodies against sars-cov-2 and uses thereof
WO2023166425A1 (en) 2022-03-01 2023-09-07 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions
CN114685586B (zh) * 2022-03-05 2025-07-18 武汉瑞佶生物科技有限公司 一种mRNA-脂肪酸靶向化合物及其制备方法和应用
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
WO2023177904A1 (en) 2022-03-18 2023-09-21 Modernatx, Inc. Sterile filtration of lipid nanoparticles and filtration analysis thereof for biological applications
US20230293646A1 (en) 2022-03-21 2023-09-21 Crispr Therapeutics Ag Methods and compositions for treating lipoprotein-related diseases
US20230331841A1 (en) 2022-03-23 2023-10-19 Crispr Therapeutics Ag Anti-cd83 car-t cells with regnase-1 and/or tgfbrii disruption
US20230303713A1 (en) 2022-03-23 2023-09-28 Crispr Therapeutics Ag Anti-cd19 car-t cells with multiple gene edits and therapeutic uses thereof
EP4504958A1 (en) 2022-04-07 2025-02-12 New England Biolabs, Inc. Methods of higher fidelity rna synthesis
WO2023218243A1 (en) 2022-05-12 2023-11-16 Avacta Life Sciences Limited Lag-3/pd-l1 binding fusion proteins
TW202400784A (zh) * 2022-05-13 2024-01-01 大陸商上海瑞宏迪醫藥有限公司 包含utr的核酸構建體及其應用
WO2023223183A1 (en) 2022-05-16 2023-11-23 Crispr Therapeutics Ag Picornaviral vectors for gene editing
CN114632092B (zh) * 2022-05-17 2022-08-23 北京第一生物化学药业有限公司 睾丸片在制备具有降糖活性的药物中的应用
US20250345407A1 (en) 2022-05-25 2025-11-13 CureVac SE Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
JP2025522474A (ja) 2022-06-17 2025-07-15 クリスパー・セラピューティクス・アクチェンゲゼルシャフト 脂質ナノ粒子(lnp)ベースの眼内デリバリー
EP4540378A1 (en) 2022-06-20 2025-04-23 CRISPR Therapeutics AG Base editing proteins and uses thereof
US20250381304A1 (en) 2022-06-21 2025-12-18 Crispr Therapeutics Ag Methods and compositions for in vivo editing of stem cells
WO2023248145A1 (en) 2022-06-21 2023-12-28 Crispr Therapeutics Ag Compositions and methods for treating human immunodeficiency virus
WO2024003786A1 (en) 2022-06-29 2024-01-04 Crispr Therapeutics Ag Chimeric antigen receptor targeting gpc-3 and immune cells expressing such for therapeutic uses
WO2024023801A2 (en) 2022-07-29 2024-02-01 Crispr Therapeutics Ag Genetically engineered immune cells having disrupted transporter associated with antigen processing-1 (tap-1) gene
WO2024023802A2 (en) 2022-07-29 2024-02-01 Crispr Therapeutics Ag Genetically engineered immune cells having disrupted transporter associated with antigen processing-2 (tap-2) gene
WO2024023804A2 (en) 2022-07-29 2024-02-01 Crispr Therapeutics Ag Genetically engineered immune cells having disrupted transporter associated with antigen processing binding protein (tapbp) gene
WO2024044147A1 (en) 2022-08-23 2024-02-29 Modernatx, Inc. Methods for purification of ionizable lipids
WO2024062388A2 (en) 2022-09-20 2024-03-28 Crispr Therapeutics Ag Genetically engineered immune cells expressing chimeric antigen receptor targeting cd20
CN118715316B (zh) * 2022-10-08 2025-09-19 凌意(杭州)生物科技有限公司 用于治疗GCase缺乏相关疾病的多核苷酸
EP4608442A1 (en) 2022-10-28 2025-09-03 GlaxoSmithKline Biologicals S.A. Nucleic acid based vaccine
WO2024102434A1 (en) 2022-11-10 2024-05-16 Senda Biosciences, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
WO2024157194A1 (en) 2023-01-25 2024-08-02 Crispr Therapeutics Ag Methods and assays for off-target analysis
CN121057577A (zh) 2023-01-27 2025-12-02 赛欧生物医药股份有限公司 经修饰的脂质组合物及其用途
WO2024166905A1 (ja) * 2023-02-07 2024-08-15 ExoEarth株式会社 薬剤流通サービスシステム、無針注射デバイス、管理システム
WO2024184500A1 (en) 2023-03-08 2024-09-12 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
WO2024192108A1 (en) 2023-03-14 2024-09-19 Evolveimmune Therapeutics, Inc. Genetically modified car t cells and methods of making and using the same
EP4434534A1 (en) * 2023-03-22 2024-09-25 ADvantage Therapeutics, Inc. Klotho mrna
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
EP4704816A1 (en) 2023-05-03 2026-03-11 ModernaTX, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
WO2024238723A1 (en) 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
EP4713027A1 (en) 2023-05-16 2026-03-25 Omega Therapeutics, Inc. Methods and compositions for modulating methylation of a target gene
WO2025019742A1 (en) 2023-07-19 2025-01-23 Omega Therapeutics, Inc. Methods and compositions for modulating ctnnb1 expression
WO2025019023A1 (en) 2023-07-20 2025-01-23 New England Biolabs, Inc. Polynucleotide error recognition methods and compositions
AU2024301706A1 (en) 2023-07-21 2026-02-05 Crispr Therapeutics Ag Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene
CN120882865A (zh) * 2023-07-28 2025-10-31 凌意(杭州)生物科技有限公司 用于治疗威尔逊病的多核苷酸
WO2025059215A1 (en) 2023-09-12 2025-03-20 Aadigen, Llc Methods and compositions for treating or preventing cancer
WO2025059500A1 (en) 2023-09-14 2025-03-20 New England Biolabs, Inc. Rna polymerases
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025072696A1 (en) 2023-09-29 2025-04-03 New England Biolabs, Inc. Compositions, methods, and cells for making 2-aminoadenine (dz) dna
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
CN117517658B (zh) * 2023-11-14 2024-04-12 北京大学 检测组蛋白烟酰化修饰试剂的新用途
WO2025126049A2 (en) 2023-12-11 2025-06-19 Crispr Therapeutics Ag Multiplex gene editing
WO2025126242A1 (en) * 2023-12-11 2025-06-19 Dr. Reddy's Institute Of Life Sciences Nucleic acid sequence(s) and construct(s) for treating methylmalonic acidemia and method(s) thereof
WO2025137453A1 (en) 2023-12-20 2025-06-26 New England Biolabs, Inc. Compositions, methods, kits, and instruments for analyzing rna structure
US20250269059A1 (en) 2024-02-27 2025-08-28 Crispr Therapeutics Ag Rt editing compositions and methods
WO2025186726A1 (en) 2024-03-05 2025-09-12 Crispr Therapeutics Ag Modulating expression of agt (angiotensinogen) gene
WO2025190864A1 (en) * 2024-03-11 2025-09-18 Sartorius Stedim Biotech Gmbh Improved cell lysis methods, reagents, and kits for viral vector release and production
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
US12492420B2 (en) 2024-03-29 2025-12-09 New England Biolabs, Inc. Compositions, kits, and methods for in vitro transcription
WO2025213179A1 (en) 2024-04-05 2025-10-09 New England Biolabs, Inc. Compositions and methods for characterizing rna
WO2025227034A1 (en) 2024-04-26 2025-10-30 New England Biolabs, Inc. Salt active nuclease compositions and methods
WO2025235563A1 (en) 2024-05-07 2025-11-13 Omega Therapeutics, Inc. Epigenetic modulation for upregulation of genes
WO2026010863A2 (en) 2024-07-01 2026-01-08 New England Biolabs, Inc. Programmable cleavage of rna
US12281325B1 (en) * 2024-07-25 2025-04-22 Wyvern Pharmaceuticals Inc. Composition for regulating production of proteins
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use
WO2026036000A1 (en) 2024-08-08 2026-02-12 Invaio Sciences, Inc. Nature-derived lipid particles for use in agriculture
CN119185350B (zh) * 2024-08-15 2025-09-09 安徽农业大学 靶向lrp6的抑制剂在调节动物剩余采食量中的应用
US20260092310A1 (en) 2024-09-27 2026-04-02 New England Biolabs, Inc. Cas12a compositions and methods

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011071931A2 (en) * 2009-12-07 2011-06-16 Katalin Kariko Rna preparations comprising purified modified rna for reprogramming cells
WO2011090965A1 (en) * 2010-01-22 2011-07-28 Merck Sharp & Dohme Corp. Novel cationic lipids for oligonucleotide delivery
WO2012019168A2 (en) * 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof

Family Cites Families (329)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
CA1327358C (en) 1987-11-17 1994-03-01 Morio Fujiu Fluoro cytidine derivatives
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5221732A (en) 1988-12-06 1993-06-22 The United States Of America As Represented By The Department Of Health And Human Services Antimicrobial magainin modified peptides
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
DE69034150T2 (de) 1989-10-24 2005-08-25 Isis Pharmaceuticals, Inc., Carlsbad 2'-Modifizierte Oligonukleotide
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US7037646B1 (en) 1990-01-11 2006-05-02 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
US6783931B1 (en) 1990-01-11 2004-08-31 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
CA2042448A1 (en) 1990-06-05 1991-12-06 Jonathan P. Duvick Antimicrobial peptides and plant disease resistance based thereon
US5447914A (en) 1990-06-21 1995-09-05 Emory University Antimicrobial peptides
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
KR930702373A (ko) 1990-11-08 1993-09-08 안토니 제이. 페이네 합성 올리고누클레오티드에 대한 다중 리포터(Reporter)그룹의 첨합
AU648140B2 (en) 1991-02-01 1994-04-14 Virtual Drug Development, Inc. Reverse antimicrobial peptides and antimicrobial compositions
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
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
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
EP0538194B1 (de) 1991-10-17 1997-06-04 Novartis AG Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
US5607914A (en) 1993-01-13 1997-03-04 Pioneer Hi-Bred International, Inc. Synthetic antimicrobial peptides
US5459235A (en) 1993-03-19 1995-10-17 The Regents Of The University Of California Antimicrobial peptides antibodies and nucleic acid molecules from bovine neutrophils
CA2159631A1 (en) 1993-03-30 1994-10-13 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
US6294664B1 (en) 1993-07-29 2001-09-25 Isis Pharmaceuticals, Inc. Synthesis of oligonucleotides
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5550109A (en) 1994-05-24 1996-08-27 Magainin Pharmaceuticals Inc. Inducible defensin peptide from mammalian epithelia
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5585108A (en) 1994-12-30 1996-12-17 Nanosystems L.L.C. Formulations of oral gastrointestinal therapeutic agents in combination with pharmaceutically acceptable clays
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US6057291A (en) 1995-06-02 2000-05-02 University Of British Columbia Antimicrobial cationic peptides
US5734015A (en) 1995-06-19 1998-03-31 Magainin Pharmaceuticals Inc. Family of linear antimicrobial peptides from hagfish intestine
DE69637731D1 (de) 1995-08-23 2008-12-11 Univ British Columbia Antimikrobielle kationische peptide und methoden zu ihrer identifizierung
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US6605698B1 (en) 1995-12-13 2003-08-12 Syngenta Limited Antifungal peptides and composition thereof
US5714577A (en) 1996-01-26 1998-02-03 University Of Pittsburgh Antimicrobial peptides
US5994308A (en) 1996-02-28 1999-11-30 Board Of Trustees Of Southern Illinois University Broad spectrum antimicrobial peptides containing a tryptophan triplet and methods of use
US5773696A (en) 1996-03-29 1998-06-30 Monsanto Company Antifungal polypeptide and methods for controlling plant pathogenic fungi
US5856127A (en) 1996-07-26 1999-01-05 The Research Foundation Of State University Of New York Antimicrobial peptides
WO1998007833A1 (en) 1996-08-22 1998-02-26 The Trustees Of The University Of Pennsylvania Compositions and methods for use of defensin
US6121436A (en) 1996-12-13 2000-09-19 Monsanto Company Antifungal polypeptide and methods for controlling plant pathogenic fungi
US6576752B1 (en) 1997-02-14 2003-06-10 Isis Pharmaceuticals, Inc. Aminooxy functionalized oligomers
US5998374A (en) 1997-02-28 1999-12-07 The Regents Of University Of California Clavaspirins
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
NL1006164C2 (nl) 1997-05-29 1998-12-01 Univ Leiden Antimicrobiële peptiden.
FR2766191B1 (fr) 1997-07-21 2000-11-10 Ifremer Peptides anti-microbiens de crustaces
US20030073640A1 (en) 1997-07-23 2003-04-17 Ribozyme Pharmaceuticals, Inc. Novel compositions for the delivery of negatively charged molecules
EP0988314B1 (en) 1997-07-31 2006-07-19 Philip Richard Abraham Synthetic peptides with antimicrobial and endotoxin neutralizing properties for management of the sepsis syndrome
US6576755B1 (en) 1997-09-10 2003-06-10 Zymogenetics, Inc. Beta-defensins
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US20030083272A1 (en) 1997-09-19 2003-05-01 Lahive & Cockfield, Llp Sense mrna therapy
WO1999015548A2 (en) 1997-09-25 1999-04-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Isolated and recombinant antimicrobial peptides thrombocidin-1 (tc-1) and thrombocidin-2 (tc-2) or variants thereof
US6004573A (en) 1997-10-03 1999-12-21 Macromed, Inc. Biodegradable low molecular weight triblock poly(lactide-co-glycolide) polyethylene glycol copolymers having reverse thermal gelation properties
US6548633B1 (en) 1998-12-22 2003-04-15 Genset, S.A. Complementary DNA's encoding proteins with signal peptides
US7655777B2 (en) * 1997-11-24 2010-02-02 Monsanto Technology Llc Nucleic acid molecules associated with the tocopherol pathway
AU1819499A (en) 1997-12-12 1999-06-28 Samyang Corporation Positively-charged poly{alpha-(omega-aminoalkyl)glycolic acid} for the delivery of a bioactive agent via tissue and cellular uptake
US6517869B1 (en) 1997-12-12 2003-02-11 Expression Genetics, Inc. Positively charged poly(alpha-(omega-aminoalkyl)lycolic acid) for the delivery of a bioactive agent via tissue and cellular uptake
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
DE69941447D1 (de) 1998-01-05 2009-11-05 Univ Washington Erhöhter transport unter benutzung membranzerstörender stoffe
NL1008139C2 (nl) 1998-01-27 1999-07-28 Stichting Tech Wetenschapp Antimicrobiële peptiden.
CA2320429C (en) 1998-02-18 2011-04-19 Harbor-Ucla Research And Education Institute Antimicrobial peptides and derived metapeptides
EP0953641A3 (en) * 1998-03-26 2002-03-13 Pfizer Products Inc. Polynucleotide molecules encoding neospora proteins
JPH11326617A (ja) * 1998-05-13 1999-11-26 Olympus Optical Co Ltd 回折光学素子を含む光学系及びその設計方法
DE69939740D1 (de) 1998-05-20 2008-11-27 Expression Genetics Inc Laktose oder galaktose-polyethylen glykol-gepfropfte poly-l-lysin als genträger
EP1117720A4 (en) 1998-07-13 2001-11-14 Expression Genetics Inc POLYESTER POLY-L-LYSINE ANALOG USED AS A BIODEGRADABLE SOLUBLE GENE CONVEYOR
US6476189B1 (en) 1998-08-12 2002-11-05 National Institute Of Agrobiological Sciences Antibacterial peptides and antibacterial agents containing such peptides as an effective ingredient
US6288212B1 (en) 1998-08-28 2001-09-11 The University Of British Columbia Anti-endotoxic, antimicrobial cationic peptides and methods of use therefor
US6107460A (en) 1999-03-01 2000-08-22 The Board Of Regents Of The University Of Oklahoma Antimicrobial peptides and methods of use thereof
US8410248B2 (en) 1999-03-12 2013-04-02 Human Genome Sciences Inc. HWBAO62 polypeptides
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7244710B2 (en) 2002-05-21 2007-07-17 Zengen, Inc. Treatment of ophthalmic infections using antimicrobial peptides
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
EP1179012B2 (en) 1999-05-17 2009-07-15 ConjuChem Biotechnologies Inc. Long lasting fusion peptide inhibitors of viral infection
FR2796072B1 (fr) 1999-07-08 2003-09-19 Centre Nat Rech Scient Peptides anti-microbiens de mollusques
CA2311201A1 (en) 1999-08-05 2001-02-05 Genset S.A. Ests and encoded human proteins
US7071293B1 (en) 1999-08-18 2006-07-04 The University Of Iowa Research Foundation Alpha helical peptides with broad spectrum antimicrobial activity that are insensitive to salt
EP1101771A1 (en) 1999-11-15 2001-05-23 Korea Kumho Petrochemical Co. Ltd. Pathogen-induced genes from Capsicum annuum
US6573361B1 (en) 1999-12-06 2003-06-03 Monsanto Technology Llc Antifungal proteins and methods for their use
MXPA02006028A (es) 1999-12-15 2004-08-23 Cubist Pharm Inc Lipopeptidos novedosos como agentes antibacterianos.
US7737108B1 (en) 2000-01-07 2010-06-15 University Of Washington Enhanced transport using membrane disruptive agents
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
EP1130091A3 (en) 2000-03-01 2001-11-14 Message Pharmaceuticals, Inc. Bacterial RNaseP Proteins and their use in identifying antibacterial compounds
US6337317B1 (en) 2000-06-27 2002-01-08 The University Of British Columbia Antimicrobial peptides and methods of use thereof
US6492328B2 (en) 2000-06-28 2002-12-10 The University Of Iowa Research Foundation Novispirins: antimicrobial peptides
AU2001279073B2 (en) 2000-07-28 2006-08-24 Christopher J. Murphy Transplant media
US6727066B2 (en) 2000-07-28 2004-04-27 Incyte Corporation Genes expressed in treated human C3A liver cell cultures
US6875907B2 (en) 2000-09-13 2005-04-05 Pioneer Hi-Bred International, Inc. Antimicrobial peptides and methods of use
US20040142474A1 (en) 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
US6696038B1 (en) 2000-09-14 2004-02-24 Expression Genetics, Inc. Cationic lipopolymer as biocompatible gene delivery agent
JP4413493B2 (ja) 2000-10-04 2010-02-10 サンタリス ファーマ アー/エス プリンlna類似体の改善された合成方法
US6998115B2 (en) 2000-10-10 2006-02-14 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US6649138B2 (en) 2000-10-13 2003-11-18 Quantum Dot Corporation Surface-modified semiconductive and metallic nanoparticles having enhanced dispersibility in aqueous media
GB0026924D0 (en) 2000-11-03 2000-12-20 Univ Cambridge Tech Antibacterial agents
US6835713B2 (en) 2001-02-16 2004-12-28 University Of Pittsburgh Virus derived antimicrobial peptides
US6652886B2 (en) 2001-02-16 2003-11-25 Expression Genetics Biodegradable cationic copolymers of poly (alkylenimine) and poly (ethylene glycol) for the delivery of bioactive agents
US6887847B2 (en) 2001-02-16 2005-05-03 University Of Pittsburgh Virus derived antimicrobial peptides
US7314858B2 (en) 2001-04-18 2008-01-01 The Regents Of The University Of California Retrocyclins: antiviral and antimicrobial peptides
ES2340499T3 (es) 2001-06-05 2010-06-04 Curevac Gmbh Arnm de antigeno tumoral estabilizado con un contenido de g/c aumentado.
US6872705B2 (en) 2001-07-13 2005-03-29 Allergan, Inc. Use of antimicrobial peptides as preservatives in ophthalmic preparations, including solutions, emulsions, and suspensions
US6586524B2 (en) 2001-07-19 2003-07-01 Expression Genetics, Inc. Cellular targeting poly(ethylene glycol)-grafted polymeric gene carrier
WO2003014078A2 (en) 2001-08-08 2003-02-20 The Research Foundation Of The State University Of New York Muc7d1 peptides as antifungal and antibacterial agents
US6743598B2 (en) 2001-08-10 2004-06-01 Mycologics, Inc. Methods for the identification of fungal glucose utilization inhibitors and antifungal agents
US6835536B2 (en) 2001-08-21 2004-12-28 Micrologix Biotech Inc. Antimicrobial cationic peptides and formulations thereof
AU2002336746A1 (en) 2001-09-21 2003-04-01 Message Pharmaceuticals, Inc. Inhibitors of rnase p proteins as antibacterial compounds
EP2447370B1 (en) 2001-09-28 2018-07-18 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
WO2003028657A2 (en) 2001-10-03 2003-04-10 The Johns Hopkins University Compositions for oral gene therapy and methods of using same
US6478825B1 (en) 2001-11-28 2002-11-12 Osteotech, Inc. Implant, method of making same and use of the implant for the treatment of bone defects
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
EP1474109B1 (en) 2001-12-21 2010-08-25 Alcon, Inc. Use of synthetic inorganic nanoparticles as carriers for ophthalmic drugs
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
WO2003092665A2 (en) 2002-05-02 2003-11-13 Massachusetts Eye And Ear Infirmary Ocular drug delivery systems and use thereof
US7374930B2 (en) 2002-05-21 2008-05-20 Expression Genetics, Inc. GLP-1 gene delivery for the treatment of type 2 diabetes
AU2003252136A1 (en) 2002-07-24 2004-02-09 Ptc Therapeutics, Inc. METHODS FOR IDENTIFYING SMALL MOLEDULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY
WO2004041203A2 (en) 2002-11-04 2004-05-21 Xenoport, Inc. Gemcitabine prodrugs, pharmaceutical compositions and uses thereof
US20080227085A1 (en) 2003-01-17 2008-09-18 Pellegrini Matthew C Methods and Systems for the Identification of Rna Regulatory Sequences and Compounds that Modulate their Function
US8460864B2 (en) 2003-01-21 2013-06-11 Ptc Therapeutics, Inc. Methods for identifying compounds that modulate untranslated region-dependent gene expression and methods of using same
US8426194B2 (en) 2003-01-21 2013-04-23 Ptc Therapeutics, Inc. Methods and agents for screening for compounds capable of modulating VEGF expression
US9068234B2 (en) 2003-01-21 2015-06-30 Ptc Therapeutics, Inc. Methods and agents for screening for compounds capable of modulating gene expression
US7091185B2 (en) 2003-02-24 2006-08-15 Dow Global Technologies Inc. Periodic antimicrobial peptides
GB0316089D0 (en) 2003-07-09 2003-08-13 Xo Bioscience Ltd Differentiation method
KR100992646B1 (ko) 2003-07-09 2010-11-05 제이에스알 가부시끼가이샤 파장판
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
CL2004001996A1 (es) * 2003-08-08 2005-05-06 Eyetech Pharmaceuticals Inc Aptameros anti-vegf (factor de crecimiento endotelial vascular) con bloqueo nucleotidico 5'-5' o 3'-3' invertido, composicion que lo contiene, util para trastornos de neovascularizacion.
JP2005179268A (ja) 2003-12-19 2005-07-07 Gc Corp 口腔用組成物
US8372966B2 (en) 2003-12-19 2013-02-12 University Of Cincinnati Oligonucleotide decoys and methods of use
EP1713514B1 (en) 2004-01-28 2021-11-24 Johns Hopkins University Drugs and gene carrier particles that rapidly move through mucous barriers
CA2563533C (en) 2004-04-15 2013-10-01 Shmuel A. Ben-Sasson Compositions capable of facilitating penetration across a biological barrier
US7527947B2 (en) 2004-06-14 2009-05-05 Novozymes A/S Signal peptide for producing a polypeptide
DE102004042546A1 (de) * 2004-09-02 2006-03-09 Curevac Gmbh Kombinationstherapie zur Immunstimulation
AU2005286640A1 (en) * 2004-09-21 2006-03-30 Anesiva, Inc. Delivery of polynucleotides
EP2402323A3 (en) 2004-10-13 2012-05-30 PTC Therapeutics, Inc. Pyrazole or triazole compounds and their use for the manufacture of a medicament for treating somatic mutation-related diseases
US8057821B2 (en) 2004-11-03 2011-11-15 Egen, Inc. Biodegradable cross-linked cationic multi-block copolymers for gene delivery and methods of making thereof
WO2006058088A2 (en) 2004-11-23 2006-06-01 Ptc Therapeutics, Inc. Carbazole, carboline and indole derivatives useful in the inhibition of vegf production
WO2006063249A2 (en) 2004-12-10 2006-06-15 Justin Hanes Functionalized poly (ether-anhydride) block copolymers
US9068969B2 (en) 2004-12-28 2015-06-30 Ptc Therapeutics, Inc. Cell based methods and systems for the identification of RNA regulatory sequences and compounds that modulate their functions
US8192718B1 (en) 2005-01-04 2012-06-05 Gp Medical, Inc. Pharmaceutical composition of nanoparticles
US8187570B1 (en) 2005-01-04 2012-05-29 Gp Medical, Inc. Nanoparticles for protein drug delivery
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2007086881A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
MX2007012157A (es) 2005-04-01 2008-03-14 Intezyne Technologies Llc Micelas polimericas para suministro de farmacos.
US8246995B2 (en) 2005-05-10 2012-08-21 The Board Of Trustees Of The Leland Stanford Junior University Hydrophobic nanotubes and nanoparticles as transporters for the delivery of drugs into cells
DE102005023170A1 (de) 2005-05-19 2006-11-23 Curevac Gmbh Optimierte Formulierung für mRNA
CN102016814B (zh) 2005-06-17 2013-10-23 北卡罗来纳大学查珀尔希尔分校 纳米粒子制备方法、系统及材料
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
CA2613442C (en) 2005-06-30 2016-08-23 Archemix Corp. Materials and methods for the generation of fully 2'-modified nucleic acid transcripts
PL2573114T3 (pl) 2005-08-10 2016-10-31 Identyfikacja i inżynieria przeciwciał z wariantami regionów FC oraz sposoby ich stosowania
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
US8603457B2 (en) 2005-12-02 2013-12-10 University Of Rochester Nonsense suppression and genetic codon alteration by targeted modification
JP2009523864A (ja) 2006-01-12 2009-06-25 マサチューセッツ インスティテュート オブ テクノロジー 生分解性エラストマー
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
EP2061433B1 (en) 2006-09-08 2011-02-16 Johns Hopkins University Compositions for enhancing transport through mucus
US8313777B2 (en) 2006-10-05 2012-11-20 The Johns Hopkins University Water-dispersible oral, parenteral, and topical formulations for poorly water soluble drugs using smart polymeric nanoparticles
DE102006051516A1 (de) 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
WO2008140615A2 (en) 2006-12-21 2008-11-20 Novozymes, Inc. Modified messenger rna stabilizing sequences for expressing genes in bacterial cells
WO2008078180A2 (en) 2006-12-22 2008-07-03 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
DE102007001370A1 (de) 2007-01-09 2008-07-10 Curevac Gmbh RNA-kodierte Antikörper
CA2689042A1 (en) 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
US8039214B2 (en) 2007-06-29 2011-10-18 Cellscript, Inc. Synthesis of tagged nucleic acids
US20090042825A1 (en) 2007-08-06 2009-02-12 Majed Matar Composition, method of preparation & application of concentrated formulations of condensed nucleic acids with a cationic lipopolymer
CA2715078C (en) 2007-09-26 2019-07-23 Intrexon Corporation Synthetic 5'utrs, expression vectors, and methods for increasing transgene expression
EP2436376B1 (en) 2007-09-28 2014-07-09 BIND Therapeutics, Inc. Cancer cell targeting using nanoparticles
SG10201408162PA (en) 2007-12-11 2015-01-29 Scripps Research Inst Compositions and methods related to mrna translational enhancer elements
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
WO2009108891A2 (en) 2008-02-29 2009-09-03 Egen, Inc. Modified poloxamers for gene expression and associated methods
WO2009114854A1 (en) 2008-03-14 2009-09-17 Egen, Inc. Biodegradable cross-linked branched poly (alkylene imines)
CA2721333C (en) * 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
US8318211B2 (en) 2008-06-16 2012-11-27 Bind Biosciences, Inc. Therapeutic polymeric nanoparticles comprising vinca alkaloids and methods of making and using same
WO2010005726A2 (en) 2008-06-16 2010-01-14 Bind Biosciences Inc. Therapeutic polymeric nanoparticles with mtor inhibitors and methods of making and using same
EA020954B1 (ru) 2008-06-16 2015-03-31 Бинд Терапьютикс, Инк. Загруженные лекарственным средством полимерные наночастицы, фармацевтическая композиция и способ лечения рака
JP2011525180A (ja) 2008-06-16 2011-09-15 バインド バイオサイエンシズ インコーポレイテッド 治療的標的化ナノ粒子の製作に用いるためのジブロックコポリマーで官能化された標的薬の製造方法
WO2010001381A1 (en) 2008-06-30 2010-01-07 Association For Public Health Services Air quality monitor
US20100009424A1 (en) 2008-07-14 2010-01-14 Natasha Forde Sonoporation systems and methods
WO2010021865A1 (en) 2008-08-18 2010-02-25 Merck Sharp & Dohme Corp. Novel lipid nanoparticles and novel components for delivery of nucleic acids
US20100087337A1 (en) 2008-09-10 2010-04-08 Bind Biosciences, Inc. High Throughput Fabrication of Nanoparticles
CA2740000C (en) * 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
CN111808084A (zh) * 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
JP2012512175A (ja) 2008-12-15 2012-05-31 バインド バイオサイエンシズ インコーポレイテッド 治療薬を徐放するための長時間循環性ナノ粒子
WO2010080724A1 (en) 2009-01-12 2010-07-15 Merck Sharp & Dohme Corp. Novel lipid nanoparticles and novel components for delivery of nucleic acids
US8669085B2 (en) 2009-02-05 2014-03-11 Ut-Battelle, Llc Transformation of gram positive bacteria by sonoporation
KR20110126717A (ko) 2009-02-24 2011-11-23 더 스크립스 리서치 인스티튜트 단백질 생성 증강을 위한 mrna 1차 구조의 재구조화
EP2406095B1 (en) 2009-03-12 2013-11-06 Illinois Tool Works Inc. Mis-fuel inhibitor
US8460696B2 (en) 2009-03-20 2013-06-11 Egen, Inc. Polyamine derivatives
JP5766178B2 (ja) 2009-03-24 2015-08-19 ザ・ユニバーシティ・オブ・シカゴThe University Of Chicago SlipChip装置および方法
JP5622254B2 (ja) 2009-03-31 2014-11-12 国立大学法人東京大学 二本鎖リボ核酸ポリイオンコンプレックス
JP2012524780A (ja) 2009-04-21 2012-10-18 セレクタ バイオサイエンシーズ インコーポレーテッド Th1バイアス応答をもたらす免疫ナノ治療薬(Immunonanotherapeutics)
US8287910B2 (en) 2009-04-30 2012-10-16 Intezyne Technologies, Inc. Polymeric micelles for polynucleotide encapsulation
SG10201911942UA (en) 2009-05-05 2020-02-27 Muthiah Manoharan Lipid compositions
BRPI1012036A2 (pt) 2009-05-27 2017-10-10 Selecta Biosciences Inc nanocarreadores que possuem componentes com diferentes taxas de liberação
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2011000107A1 (en) * 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
JP2013500667A (ja) 2009-07-30 2013-01-07 ティーピー ビジョン ホールディング ビー ヴィ 分散イメージリターゲティング
CA2769670C (en) 2009-07-31 2018-10-02 Ethris Gmbh Rna with a combination of unmodified and modified nucleotides for protein expression
ES2527684T3 (es) 2009-08-01 2015-01-28 F.Hoffmann-La Roche Ag Detección mejorada de contaminación bacteriana (mollicutes)
ES2579936T3 (es) 2009-08-20 2016-08-17 Sirna Therapeutics, Inc. Nuevos lípidos catiónicos con diversos grupos de cabeza para el suministro oligonucleotídico
EP2485770A4 (en) 2009-10-08 2013-04-10 Merck Sharp & Dohme Novel cationic lipids with short lipid chains for oligonucleotide delivery
WO2011060250A1 (en) 2009-11-13 2011-05-19 Bend Research, Inc. Cationic dextran polymer derivatives
US9415113B2 (en) 2009-11-18 2016-08-16 University Of Washington Targeting monomers and polymers having targeting blocks
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
DK2509634T3 (en) 2009-12-11 2019-04-23 Pfizer Stable formulations for lyophilization of therapeutic particles
EA201290497A1 (ru) 2009-12-15 2013-01-30 Байнд Байосайенсиз, Инк. Терапевтические полимерные наночастицы, включающие кортикостероиды, и способы получения таковых
EP2515942B1 (en) 2009-12-15 2020-02-12 Pfizer Inc. Therapeutic polymeric nanoparticle compositions with high glass transition temperature or high molecular weight copolymers
WO2011084521A2 (en) 2009-12-15 2011-07-14 Bind Biosciences, Inc. Therapeutic polymeric nanoparticles comprising epothilone and methods of making and using same
CA3009891C (en) 2009-12-23 2020-09-15 Novartis Ag Lipids, lipid compositions, and methods of using them
WO2011085231A2 (en) 2010-01-08 2011-07-14 Selecta Biosciences, Inc. Synthetic virus-like particles conjugated to human papillomavirus capsid peptides for use as vaccines
EP2525815B1 (en) 2010-01-24 2015-02-25 Novartis AG Irradiated biodegradable polymer microparticles
WO2011106702A2 (en) 2010-02-25 2011-09-01 The Johns Hopkins University Sustained delivery of therapeutic agents to an eye compartment
US20130005797A1 (en) 2010-03-18 2013-01-03 Merck Sharp & Dohme Corp. Endosomolytic poly(amidoamine) disulfide polymers for the delivery of oligonucleotides
US9149432B2 (en) 2010-03-19 2015-10-06 Massachusetts Institute Of Technology Lipid vesicle compositions and methods of use
EP2553019A1 (en) 2010-03-26 2013-02-06 Mersana Therapeutics, Inc. Modified polymers for delivery of polynucleotides, method of manufacture, and methods of use thereof
US20130037977A1 (en) 2010-04-08 2013-02-14 Paul A. Burke Preparation of Lipid Nanoparticles
US20110262491A1 (en) 2010-04-12 2011-10-27 Selecta Biosciences, Inc. Emulsions and methods of making nanocarriers
US8802438B2 (en) 2010-04-16 2014-08-12 Children's Medical Center Corporation Compositions, kits, and methods for making induced pluripotent stem cells using synthetic modified RNAs
US8802863B2 (en) 2010-05-24 2014-08-12 Sirna Therapeutics, Inc. Amino alcohol cationic lipids for oligonucleotide delivery
CA2798739A1 (en) 2010-05-26 2011-12-01 Selecta Biosciences, Inc. Nanocarrier compositions with uncoupled adjuvant
DK2575767T3 (en) 2010-06-04 2017-03-13 Sirna Therapeutics Inc HOWEVER UNKNOWN LOW MOLECULAR CATIONIC LIPIDS TO PROCESS OIGONUCLEOTIDES
KR101130137B1 (ko) 2010-07-02 2012-03-28 연세대학교 산학협력단 발광다이오드 모듈
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
MX342608B (es) 2010-07-06 2016-10-06 Novartis Ag * Particulas de suministro similares a viriones para moleculas de arn de autorreplicacion.
NZ606591A (en) 2010-07-06 2015-02-27 Novartis Ag Cationic oil-in-water emulsions
GB2495418B (en) 2010-07-28 2017-05-24 Murata Manufacturing Co Antenna apparatus and communication terminal instrument
DE102010032758B4 (de) 2010-07-29 2012-02-23 Fujitsu Technology Solutions Intellectual Property Gmbh Computersystem, Verfahren zum Programmieren einer Echtzeituhr und Computerprogrammprodukt
KR101761388B1 (ko) 2010-07-30 2017-07-25 큐어백 아게 트랜스펙션 및 면역 자극을 위한 이황화-크로스링크된 양이온 성분 및 핵산의 복합체
US20130190626A1 (en) 2010-08-02 2013-07-25 Curtin University Of Technology Determining location of, and imaging, a subsurface boundary
US8829329B2 (en) 2010-08-18 2014-09-09 International Business Machines Corporation Solar cell and battery 3D integration
WO2012024621A2 (en) 2010-08-20 2012-02-23 Selecta Biosciences, Inc. Synthetic nanocarrier vaccines comprising peptides obtained or derived from human influenza a virus hemagglutinin
MX2013002332A (es) 2010-08-31 2013-03-18 Novartis Ag Lipidos apropiados para suministro liposomal del arn que codifica la proteina.
LT4226941T (lt) 2010-08-31 2025-01-10 Glaxosmithkline Biologicals Sa Pegilintos liposomos, skirtos imunogeną koduojančios rnr pristatymui
SI4043040T1 (sl) 2010-08-31 2023-04-28 Glaxosmithkline Biologicals Sa Mali liposomi za dostavo imunogen-kodirajoče RNA
CA2809858C (en) 2010-09-20 2019-11-12 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
CA2811430A1 (en) 2010-09-30 2012-04-05 Merck Sharp & Dohme Corp. Low molecular weight cationic lipids for oligonucleotide delivery
US9782342B2 (en) 2010-10-11 2017-10-10 Wichita State University Composite magnetic nanoparticle drug delivery system
EP3485913A1 (en) 2010-10-21 2019-05-22 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
US20150056300A1 (en) 2010-10-22 2015-02-26 Bind Therapeutics, Inc. Therapeutic nanoparticles with high molecular weight copolymers
WO2012061703A1 (en) 2010-11-05 2012-05-10 The Johns Hopkins University Compositions and methods relating to reduced mucoadhesion
WO2012061259A2 (en) 2010-11-05 2012-05-10 Merck Sharp & Dohme Corp. Novel low molecular weight cyclic amine containing cationic lipids for oligonucleotide delivery
WO2012068187A1 (en) 2010-11-19 2012-05-24 Merck Sharp & Dohme Corp. Poly(amide) polymers for the delivery of oligonucleotides
WO2012082574A1 (en) 2010-12-17 2012-06-21 Merck Sharp & Dohme Corp. Membrane lytic poly(amido amine) polymers for the delivery of oligonucleotides
US20120171229A1 (en) 2010-12-30 2012-07-05 Selecta Biosciences, Inc. Synthetic nanocarriers with reactive groups that release biologically active agents
WO2012094304A1 (en) 2011-01-04 2012-07-12 Brown University Nanotubes as carriers of nucleic acids into cells
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
DE102011002640B4 (de) 2011-01-13 2021-10-07 Evonik Operations Gmbh Verfahren zur Aufreinigung von Biphephos
WO2012099805A2 (en) 2011-01-19 2012-07-26 Ocean Nanotech, Llc Nanoparticle based immunological stimulation
WO2012109121A1 (en) 2011-02-07 2012-08-16 Purdue Research Foundation Carbohydrate nanoparticles for prolonged efficacy of antimicrobial peptide
EP2489371A1 (en) 2011-02-18 2012-08-22 Instituto Nacional de Investigacion y Tecnologia Agraria y Alimentaria Carrier peptides for drug delivery
US20120237565A1 (en) 2011-03-14 2012-09-20 Intezyne Technologies, Incorporated Pegylated polyplexes containing two or more different polymers for polynucleotide delivery
WO2012125987A2 (en) 2011-03-17 2012-09-20 Massachusetts Institute Of Technology Delivery system
WO2012129648A1 (en) 2011-03-25 2012-10-04 University Of Guelph Enhancing protein expression of adeno-associated virus vectors
MX366228B (es) 2011-03-25 2019-07-03 Selecta Biosciences Inc Nanoportadores sinteticos de liberacion mediada por osmosis.
CA2831471C (en) 2011-03-31 2020-02-25 Ingell Technologies Holding B.V. Biodegradable compositions suitable for controlled release
DK2691079T3 (da) 2011-03-31 2020-09-28 Ingell Tech Holding B V Bionedbrydelige sammensætninger, der er egnet til kontrolleret udløsning
US10647959B2 (en) 2011-04-27 2020-05-12 President And Fellows Of Harvard College Cell-friendly inverse opal hydrogels for cell encapsulation, drug and protein delivery, and functional nanoparticle encapsulation
US20120283503A1 (en) 2011-04-29 2012-11-08 The Johns Hopkins University Nanoparticle loaded stem cells and their use in mri guided hyperthermia
MX374963B (es) 2011-04-29 2025-03-06 Selecta Biosciences Inc Nanoportadores sintéticos tolerogénicos para reducir las respuestas de anticuerpos.
EP3409112B1 (en) 2011-05-04 2022-07-27 The University of Nottingham Novel polymers which resist bacterial attachment
US9327029B2 (en) 2011-05-05 2016-05-03 Celacare Technologies, Llc Antimicrobial silver hydrogel composition for the treatment of burns and wounds
US20120302940A1 (en) 2011-05-26 2012-11-29 Jackson State University Popcorn Shape Gold Nanoparticle For Targeted Diagnosis, Photothermal Treatment and In-Situ Monitoring Therapy Response for Cancer and Multiple Drug Resistance Bacteria
US20140308363A1 (en) 2011-05-31 2014-10-16 Bind Therapeutics, Inc. Drug loaded polymeric nanoparticles and methods of making and using same
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
ES2795110T3 (es) 2011-06-08 2020-11-20 Translate Bio Inc Lípidos escindibles
EP4115875A1 (en) 2011-07-06 2023-01-11 GlaxoSmithKline Biologicals S.A. Liposomes having useful n:p ratio for delivery of rna molecules
US9617392B2 (en) 2011-07-10 2017-04-11 President And Fellows Of Harvard College Compositions and methods for self-assembly of polymers with complementary macroscopic and microscopic scale units
WO2013012476A2 (en) 2011-07-21 2013-01-24 Arizona Chemical Company, Llc Branched polyether-polyamide block copolymers and methods of making and using the same
KR20140050698A (ko) 2011-07-29 2014-04-29 셀렉타 바이오사이언시즈, 인크. 체액성 및 세포독성 t 림프구(ctl) 면역 반응을 발생시키는 합성 나노운반체
EP2755986A4 (en) * 2011-09-12 2015-05-20 Moderna Therapeutics Inc MANIPULATED NUCLEIC ACIDS AND METHOD OF APPLICATION THEREFOR
GB201117279D0 (en) * 2011-10-06 2011-11-16 Nexeon Ltd Etched silicon structures, method of forming etched silicon structures and uses thereof
US8673184B2 (en) * 2011-10-13 2014-03-18 Flexcon Company, Inc. Systems and methods for providing overcharge protection in capacitive coupled biomedical electrodes
US20140315170A1 (en) * 2011-11-23 2014-10-23 Proteus Digital Health, Inc. Apparatus, System, and Method to Promote Behavior Change Based on Mindfulness Methodologies
US8930541B2 (en) * 2011-11-25 2015-01-06 International Business Machines Corporation System, method and program product for cost-aware selection of templates for provisioning shared resources
CA2856887C (en) * 2011-11-28 2021-06-15 Expanergy, Llc Energy search engine with autonomous control
CN103136247B (zh) * 2011-11-29 2015-12-02 阿里巴巴集团控股有限公司 属性数据区间划分方法及装置
DE102011087509A1 (de) * 2011-12-01 2013-06-06 Robert Bosch Gmbh Sensorübertragungsvorrichtung und Verfahren zur Übertragung von Nutzdaten von einer Mehrzahl von Sensoren an eine Bussteuervorrichtung für ein Fahrzeug

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011071931A2 (en) * 2009-12-07 2011-06-16 Katalin Kariko Rna preparations comprising purified modified rna for reprogramming cells
WO2011090965A1 (en) * 2010-01-22 2011-07-28 Merck Sharp & Dohme Corp. Novel cationic lipids for oligonucleotide delivery
WO2012019168A2 (en) * 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
FUMIO ENDO 等: "A Nonsense Mutation in the 4-Hydroxyphenylpyruvic Acid Dioxygenase Gene (If@) Causes Skipping of the Constitutive Exon and Hypertyrosinemia in Mouse Strain III", 《GENOMICS》 *
ULLA RÜETSCHI等: "Human 4-Hydroxyphenylpyruvate Dioxygenase Gene (HPD)", 《GENOMICS》 *
ULLA RÜETSCHI等: "Human 4-hydroxyphenylpyruvate dioxygenase Primary structure and chromosomal localization of the gene", 《EUR.J.BIOCHEM》 *

Cited By (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108430456A (zh) * 2015-10-22 2018-08-21 摩登纳特斯有限公司 癌症疫苗
CN108430456B (zh) * 2015-10-22 2022-01-18 摩登纳特斯有限公司 癌症疫苗
CN114404581A (zh) * 2015-10-22 2022-04-29 摩登纳特斯有限公司 癌症疫苗
CN109640962A (zh) * 2016-05-18 2019-04-16 摩登纳特斯有限公司 编码松弛素的多核苷酸
CN111164207A (zh) * 2017-07-27 2020-05-15 优卡瑞斯 新型嵌合酶及其应用
CN109423514A (zh) * 2017-08-24 2019-03-05 上海市计划生育科学研究所 反复自然流产相关微小rna及其应用
CN112567032A (zh) * 2018-04-18 2021-03-26 利甘达尔股份有限公司 基因组编辑的方法和组合物
CN112334489A (zh) * 2018-05-16 2021-02-05 星火治疗有限公司 密码子优化的酸性α-葡萄糖苷酶表达盒及其使用方法
CN112996519A (zh) * 2018-09-04 2021-06-18 德克萨斯大学系统董事会 用于核酸的器官特异性递送的组合物和方法
CN114222590A (zh) * 2019-03-27 2022-03-22 西吉隆医疗股份有限公司 用于因子vii疗法的组合物、装置和方法

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