KR102255108B1 - 활성제의 전달을 위한 지질 및 지질 조성물 - Google Patents

활성제의 전달을 위한 지질 및 지질 조성물 Download PDF

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KR102255108B1
KR102255108B1 KR1020157027468A KR20157027468A KR102255108B1 KR 102255108 B1 KR102255108 B1 KR 102255108B1 KR 1020157027468 A KR1020157027468 A KR 1020157027468A KR 20157027468 A KR20157027468 A KR 20157027468A KR 102255108 B1 KR102255108 B1 KR 102255108B1
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bis
lipid
mmol
methyl
dimethylamino
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KR20150126015A (ko
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제레미 리 바리자
로한 에릭 존 벡크위드
키이스 바우만
크리스탈 바이어스
탄지나 파잘
가브리엘 그랜트 갬버
카메론 척-문 리
리테시 바누다스지 티츠쿨레
찬드라 바기스
수앙시 왕
로라 웨스트
토마스 자바와
준핑 자오
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노파르티스 아게
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Families Citing this family (209)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6352950B2 (ja) * 2013-03-08 2018-07-04 ノバルティス アーゲー 活性薬物の送達のための脂質と脂質組成物
CN105683163B (zh) 2013-10-04 2018-11-09 诺华股份有限公司 RNA干扰中使用的RNAi剂的3’端帽
WO2015051366A2 (en) 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
JP6546161B2 (ja) 2013-10-04 2019-07-17 ノバルティス アーゲー Ｂ型肝炎ウイルスを治療するための有機化合物
EP3082760A1 (en) * 2013-12-19 2016-10-26 Novartis AG LEPTIN mRNA COMPOSITIONS AND FORMULATIONS
HUE057800T2 (hu) 2014-04-23 2022-06-28 Modernatx Inc Nukleinsav vakcinák
EP4223285A3 (en) * 2014-07-16 2023-11-22 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
WO2016054237A2 (en) * 2014-10-03 2016-04-07 Institute For Cancer Research D/B/A The Research Institute Of Fox Chase Cancer Center Poly (adp-ribose) polymerase 1 inhibitors structurally unrelated to nad
US10792299B2 (en) 2014-12-26 2020-10-06 Nitto Denko Corporation Methods and compositions for treating malignant tumors associated with kras mutation
US20180002702A1 (en) 2014-12-26 2018-01-04 Nitto Denko Corporation Methods and compositions for treating malignant tumors associated with kras mutation
US10264976B2 (en) * 2014-12-26 2019-04-23 The University Of Akron Biocompatible flavonoid compounds for organelle and cell imaging
US11007260B2 (en) 2015-07-21 2021-05-18 Modernatx, Inc. Infectious disease vaccines
US12150980B2 (en) 2015-07-30 2024-11-26 Modernatx, Inc. Concatemeric peptide epitope RNAs
KR102630720B1 (ko) * 2015-08-28 2024-01-29 세키스이 메디칼 가부시키가이샤 벤질 화합물
GB201518172D0 (en) * 2015-10-14 2015-11-25 Cantab Biopharmaceuticals Patents Ltd Colloidal particles for use in medicine
ES2922760T3 (es) 2015-10-22 2022-09-20 Modernatx Inc Vacunas de virus respiratorios
TN2018000152A1 (en) * 2015-10-22 2019-10-04 Modernatx Inc Nucleic acid vaccines for varicella zoster virus (vzv)
MY192848A (en) 2015-12-28 2022-09-12 Intellia Therapeutics Inc Compositions and methods for the treatment of hemoglobinopathies
US20190136231A1 (en) 2016-03-30 2019-05-09 Intellia Therapeutics, Inc. Lipid nanoparticle formulations for crispr/cas components
CA3021647A1 (en) 2016-04-22 2017-10-26 Intellia Therapeutics, Inc. Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four
JP7246930B2 (ja) 2016-05-18 2023-03-28 モデルナティエックス インコーポレイテッド インターロイキン－１２（ｉｌ１２）をコードするポリヌクレオチドおよびその使用
CA3024509A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Mrna combination therapy for the treatment of cancer
WO2017208191A1 (en) 2016-06-02 2017-12-07 Glaxosmithkline Biologicals Sa Zika viral antigen constructs
JP2019528312A (ja) 2016-08-07 2019-10-10 ノバルティス アーゲー ｍＲＮＡ媒介性の免疫化方法
BE1024774B1 (fr) 2016-09-29 2018-07-02 Glaxosmithkline Biologicals Sa Compositions et procedes de traitement
GB201616904D0 (en) 2016-10-05 2016-11-16 Glaxosmithkline Biologicals Sa Vaccine
EP3532103B1 (en) * 2016-10-26 2025-12-03 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
MA46766A (fr) 2016-11-11 2019-09-18 Modernatx Inc Vaccin antigrippal
EP4043031A3 (en) 2016-11-17 2022-11-23 GlaxoSmithKline Biologicals SA Zika viral antigen constructs
JP2019535795A (ja) * 2016-11-28 2019-12-12 セリックス バイオ プライヴェート リミテッドＣｅｌｌｉｘ Ｂｉｏ Ｐｒｉｖａｔｅ Ｌｉｍｉｔｅｄ 口腔感染症の処置のための組成物及び方法
AU2017379073B2 (en) * 2016-12-22 2023-12-14 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
BR112019013192A2 (pt) * 2016-12-26 2019-12-10 Cellix Bio Private Ltd composto e composição farmacêutica
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
US11576961B2 (en) 2017-03-15 2023-02-14 Modernatx, Inc. Broad spectrum influenza virus vaccine
US11464848B2 (en) 2017-03-15 2022-10-11 Modernatx, Inc. Respiratory syncytial virus vaccine
EP3607074A4 (en) 2017-04-05 2021-07-07 Modernatx, Inc. REDUCTION OR ELIMINATION OF IMMUNE RESPONSES TO NON-INTRAVENOUS THERAPEUTIC PROTEINS, FOR EXAMPLE SUBCUTANEOUSLY
EP3461497A1 (en) 2017-09-27 2019-04-03 GlaxoSmithKline Biologicals S.A. Viral antigens
KR20250093420A (ko) 2017-09-29 2025-06-24 인텔리아 테라퓨틱스, 인크. 게놈 편집을 위한 폴리뉴클레오티드, 조성물 및 방법
EP3688161A1 (en) 2017-09-29 2020-08-05 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating attr amyloidosis
AU2018338967B2 (en) 2017-09-29 2025-04-24 Intellia Therapeutics, Inc. In vitro method of mRNA delivery using lipid nanoparticles
HUE066630T2 (hu) 2017-09-29 2024-08-28 Intellia Therapeutics Inc Készítmények
MA54676A (fr) 2018-01-29 2021-11-17 Modernatx Inc Vaccins à base d'arn contre le vrs
CA3109165A1 (en) 2018-08-17 2020-02-20 Glaxosmithkline Biologicals Sa Immunogenic compositions and uses thereof
KR20210093871A (ko) * 2018-10-02 2021-07-28 인텔리아 테라퓨틱스, 인크. 이온화 가능한 아민 지질
WO2020081613A1 (en) 2018-10-16 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for immunotherapy
MX2021004276A (es) 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para tratar deficiencia de alfa-1 antitripsina.
US20200268906A1 (en) 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Nucleic acid constructs and methods of use
TWI860300B (zh) 2018-10-18 2024-11-01 美商英特利亞醫療公司 用於從白蛋白基因座表現轉殖基因的組成物及方法
US12214023B2 (en) 2018-10-18 2025-02-04 Intellia Therapeutics, Inc. Compositions and methods for expressing factor IX
BR112021010853A2 (pt) 2018-12-05 2021-08-31 Intellia Therapeutics, Inc. Lipídios de amina modificados
WO2020190750A1 (en) 2019-03-15 2020-09-24 Modernatx, Inc. Hiv rna vaccines
JP7636338B2 (ja) 2019-03-28 2025-02-26 インテリア セラピューティクス，インコーポレイテッド コルチコステロイドを含む、ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスを治療するための組成物および方法、またはその使用
AU2020248470A1 (en) 2019-03-28 2021-11-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for polypeptide expression
PH12021552301A1 (en) 2019-03-28 2022-07-04 Intellia Therapeutics Inc Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent
MX2021012934A (es) 2019-04-25 2022-04-06 Intellia Therapeutics Inc Lipidos de amina ionizables y nanoparticulas lipidicas.
US20220259617A1 (en) 2019-06-13 2022-08-18 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
WO2021016075A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
MX2022000879A (es) 2019-07-21 2022-05-02 Glaxosmithkline Biologicals Sa Vacuna viral terapeutica.
EP4021415B1 (en) 2019-09-01 2025-05-14 Nextage Therapeutics Ltd. Cannabinoid containing targeting liposomes
AU2020397956A1 (en) 2019-12-04 2022-07-07 Orna Therapeutics, Inc. Circular RNA compositions and methods
US20230279394A1 (en) 2019-12-18 2023-09-07 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
WO2021141969A1 (en) 2020-01-09 2021-07-15 Guide Therapeutics, Inc. Nanomaterials
KR20230057487A (ko) 2020-03-04 2023-04-28 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈 조정을 위한 방법 및 조성물
EP4135761A1 (en) 2020-04-16 2023-02-22 GlaxoSmithKline Biologicals S.A. Sars cov-2 spike protein construct
JP2023524666A (ja) 2020-04-28 2023-06-13 インテリア セラピューティクス，インコーポレイテッド インビトロ細胞送達の方法
WO2021236930A1 (en) 2020-05-20 2021-11-25 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
EP4153224A1 (en) 2020-05-20 2023-03-29 Flagship Pioneering Innovations VI, LLC Coronavirus antigen compositions and their uses
MX2022014606A (es) 2020-05-22 2023-03-08 Wave Life Sciences Ltd Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas.
IL298539A (en) 2020-05-29 2023-01-01 Flagship Pioneering Innovations Vi Llc Trem compositions and methods relating thereto
EP4158032A2 (en) 2020-05-29 2023-04-05 Flagship Pioneering Innovations VI, LLC Trem compositions and methods relating thereto
US20230234992A1 (en) 2020-06-05 2023-07-27 Glaxosmithkline Biologicals Sa Modified betacoronavirus spike proteins
US20230256090A1 (en) 2020-06-29 2023-08-17 Glaxosmithkline Biologicals Sa Adjuvants
JP7798855B2 (ja) 2020-07-16 2026-01-14 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子に使用するためのカチオン性脂質
CN116670154A (zh) 2020-07-24 2023-08-29 总医院公司 增强的病毒样颗粒及使用其递送至细胞的方法
WO2022051629A1 (en) 2020-09-03 2022-03-10 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
CA3189073A1 (en) * 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Lipid conjugates for the delivery of therapeutic agents
EP4213882A4 (en) 2020-09-15 2025-02-26 Verve Therapeutics, Inc. LIPID FORMULATIONS FOR GENE EDITING
CZ2020529A3 (cs) * 2020-09-23 2022-03-30 Ăšstav organickĂ© chemie a biochemie AV ÄŚR, v. v. i. Lipidoidy pro transfekci nukleových kyselin a jejich použití
US11591544B2 (en) 2020-11-25 2023-02-28 Akagera Medicines, Inc. Ionizable cationic lipids
CN113185421B (zh) * 2020-11-27 2022-01-25 广州市锐博生物科技有限公司 脂质化合物及其组合物
IL303506A (en) 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
MX2023007630A (es) 2020-12-23 2023-08-25 Flagship Pioneering Innovations Vi Llc Composiciones de trem modificadas y usos de las mismas.
US20240301006A1 (en) 2020-12-23 2024-09-12 Glaxosmithkline Biologicals Sa Self-amplifying messenger rna
IL304069A (en) 2020-12-30 2023-08-01 Intellia Therapeutics Inc Engineered T cells
IL303845A (en) 2021-01-20 2023-08-01 Beam Therapeutics Inc Nanomaterials include a degradable property
JP2024502954A (ja) * 2021-01-20 2024-01-24 ビーム セラピューティクス インク． ナノ材料
CN116963729A (zh) * 2021-01-20 2023-10-27 比姆医疗股份有限公司 包括可电离脂质的纳米材料
EP4032546A1 (en) 2021-01-20 2022-07-27 GlaxoSmithKline Biologicals S.A. Therapeutic viral vaccine
CN118026871A (zh) * 2021-02-05 2024-05-14 嘉晨西海(杭州)生物技术有限公司 一种可离子化脂质分子、其制备方法及其在制备脂质纳米颗粒中的应用
CN112961065B (zh) * 2021-02-05 2023-03-14 嘉晨西海(杭州)生物技术有限公司 一种可电离脂质分子及其制备方法及其在制备脂质纳米颗粒的应用
US20240181037A1 (en) 2021-03-26 2024-06-06 Glaxosmithkline Biologicals Sa Immunogenic compositions
KR20230165276A (ko) 2021-03-31 2023-12-05 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 타노트랜스미션 폴리펩티드 및 암의 치료에서의 이의 용도
JP7796311B2 (ja) 2021-04-09 2026-01-09 相互薬工株式会社 脂質および組成物
TW202308597A (zh) 2021-04-17 2023-03-01 美商英特利亞醫療公司 脂質奈米顆粒組合物
CR20230535A (es) 2021-04-17 2024-02-16 Intellia Therapeutics Inc Inhibidores de proteína cinasa dependiente de adn y composiciones y usos de estos
KR20240017793A (ko) 2021-04-17 2024-02-08 인텔리아 테라퓨틱스, 인크. 지질 나노입자 조성물
WO2022248353A1 (en) 2021-05-24 2022-12-01 Glaxosmithkline Biologicals Sa Adjuvants
EP4352247A1 (en) 2021-06-09 2024-04-17 GlaxoSmithKline Biologicals s.a. Release assay for determining potency of self-amplifying rna drug product and methods for using
KR20240032013A (ko) 2021-06-10 2024-03-08 인텔리아 테라퓨틱스, 인크. 유전자 편집을 위한 내부 링커를 포함하는 변형된 가이드 rna
US20250127809A1 (en) 2021-06-23 2025-04-24 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
WO2023009547A1 (en) 2021-07-26 2023-02-02 Flagship Pioneering Innovations Vi, Llc Trem compositions and uses thereof
EP4387597A1 (en) 2021-08-16 2024-06-26 GlaxoSmithKline Biologicals SA Freeze-drying of lipid nanoparticles (lnps) encapsulating rna and formulations thereof
WO2023020994A1 (en) 2021-08-16 2023-02-23 Glaxosmithkline Biologicals Sa Novel methods
US20250134824A1 (en) 2021-08-16 2025-05-01 Glaxosmithkline Biologicals Sa Low-dose lyophilized rna vaccines and methods for preparing and using the same
WO2023020992A1 (en) 2021-08-16 2023-02-23 Glaxosmithkline Biologicals Sa Novel methods
WO2023020993A1 (en) 2021-08-16 2023-02-23 Glaxosmithkline Biologicals Sa Novel methods
JP2024534915A (ja) 2021-09-03 2024-09-26 グラクソスミスクライン バイオロジカルズ ソシエテ アノニム 自己増幅性メッセンジャーリボ核酸におけるヌクレオチド塩基の置換
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
JP2024534428A (ja) 2021-09-17 2024-09-20 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 環状ポリリボヌクレオチドを生成するための組成物及び方法
MX2024004618A (es) 2021-10-18 2024-07-12 Flagship Pioneering Innovations Vi Llc Composiciones y metodos para purificar polirribonucleotidos.
US20240415980A1 (en) 2021-10-28 2024-12-19 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
WO2023081689A2 (en) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
CN118541346A (zh) 2021-11-08 2024-08-23 奥纳治疗公司 用于递送环状多核苷酸的脂质纳米颗粒组合物
EP4436983A1 (en) 2021-11-24 2024-10-02 Flagship Pioneering Innovations VI, LLC Varicella-zoster virus immunogen compositions and their uses
CN118900845A (zh) 2021-11-24 2024-11-05 旗舰创业创新六公司 冠状病毒免疫原组合物及其用途
JP2024541465A (ja) 2021-11-24 2024-11-08 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 免疫原性組成物及びその使用
WO2023102364A1 (en) * 2021-12-02 2023-06-08 Ecole Polytechnique Federale De Lausanne (Epfl) Antiviral compounds, compositions and uses thereof
CA3239381A1 (en) 2021-12-03 2023-06-08 David R. Liu Compositions and methods for efficient in vivo delivery
KR20240117149A (ko) 2021-12-22 2024-07-31 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 폴리리보뉴클레오티드를 정제하기 위한 조성물 및 방법
AU2022420620A1 (en) 2021-12-23 2024-07-04 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
US20250134825A1 (en) * 2022-02-02 2025-05-01 Mslsolutions Gmbh Method for producing medications and vaccines
CA3242731A1 (en) 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
JP2023130120A (ja) * 2022-03-07 2023-09-20 スペラファーマ株式会社 ペプチド合成方法
WO2023178065A2 (en) * 2022-03-15 2023-09-21 Broadpharm Inc. Lipid compounds and lipid nanoparticles
CN119072464A (zh) * 2022-03-25 2024-12-03 赛欧生物医药股份有限公司 新型可电离脂质和脂质纳米颗粒以及其使用方法
EP4504252A2 (en) 2022-04-08 2025-02-12 Flagship Pioneering Innovations VII, LLC Vaccines and related methods
US20250302998A1 (en) 2022-05-09 2025-10-02 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
US20250295687A1 (en) 2022-05-09 2025-09-25 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use for treating proliferative disorders
WO2023220729A2 (en) 2022-05-13 2023-11-16 Flagship Pioneering Innovations Vii, Llc Double stranded dna compositions and related methods
CN120303407A (zh) 2022-05-17 2025-07-11 恩维洛普治疗有限责任公司 用于有效体内递送的组合物和方法
AU2023295529A1 (en) 2022-06-16 2024-12-12 Intellia Therapeutics, Inc. Methods and compositions for genetically modifying a cell
WO2023242817A2 (en) 2022-06-18 2023-12-21 Glaxosmithkline Biologicals Sa Recombinant rna molecules comprising untranslated regions or segments encoding spike protein from the omicron strain of severe acute respiratory coronavirus-2
EP4544043A1 (en) 2022-06-22 2025-04-30 Flagship Pioneering Innovations VI, LLC Compositions of modified trems and uses thereof
CN119585419A (zh) 2022-06-29 2025-03-07 因特利亚治疗公司 工程化t细胞
CN117486738B (zh) * 2022-07-15 2025-08-12 中国科学院基础医学与肿瘤研究所(筹) 一种脂质分子及其组合物
AU2023311822A1 (en) 2022-07-20 2025-01-09 Beam Therapeutics Inc. Nanomaterials comprising triols
IL318625A (en) 2022-07-29 2025-03-01 Regeneron Pharma Compositions and methods for transferrin receptor (TFR)-mediated delivery to brain and muscle
IL318711A (en) 2022-08-01 2025-03-01 Flagship Pioneering Innovations Vii Llc Immune modulator proteins and related methods
EP4569112A1 (en) 2022-08-12 2025-06-18 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
AU2023353931A1 (en) 2022-09-26 2025-03-20 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer
AU2023372397A1 (en) 2022-10-31 2025-05-08 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
WO2024098002A1 (en) 2022-11-04 2024-05-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
EP4615517A1 (en) 2022-11-08 2025-09-17 Orna Therapeutics, Inc. Circular rna compositions
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
AR131008A1 (es) 2022-11-08 2025-02-05 Flagship Pioneering Innovations Vi Llc Composiciones y métodos para producir polirribonucleótidos circulares
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024129988A1 (en) 2022-12-14 2024-06-20 Flagship Pioneering Innovations Vii, Llc Compositions and methods for delivery of therapeutic agents to bone
WO2024129982A2 (en) 2022-12-15 2024-06-20 Orna Therapeutics, Inc. Circular rna compositions and methods
EP4637812A1 (en) 2022-12-19 2025-10-29 GlaxoSmithKline Biologicals S.A. Hepatitis b compositions
EP4638783A2 (en) 2022-12-22 2025-10-29 Intellia Therapeutics, Inc. Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies
CN120418427A (zh) 2022-12-23 2025-08-01 因特利亚治疗公司 用于基因组编辑的系统和方法
TW202438673A (zh) 2023-01-09 2024-10-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 疫苗及相關方法
WO2024151687A1 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
WO2024151685A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Recombinant nucleic acid molecules and their use in wound healing
WO2024151673A2 (en) 2023-01-09 2024-07-18 President And Fellows Of Harvard College Recombinant nucleic acid molecules and their use in wound healing
WO2024160936A1 (en) 2023-02-03 2024-08-08 Glaxosmithkline Biologicals Sa Rna formulation
WO2024167885A1 (en) 2023-02-06 2024-08-15 Flagship Pioneering Innovations Vii, Llc Immunomodulatory compositions and related methods
KR20250151427A (ko) 2023-02-13 2025-10-21 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 치료적 조성물을 위한 절단성 링커 함유 이온화 가능한 지질 및 지질 담체
GB202302092D0 (en) 2023-02-14 2023-03-29 Glaxosmithkline Biologicals Sa Analytical method
TW202446956A (zh) 2023-02-17 2024-12-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 包含經修飾的胞嘧啶的ｄｎａ組成物
US20240285805A1 (en) 2023-02-17 2024-08-29 Flagship Pioneering Innovations Vii, Llc Dna compositions comprising modified uracil
KR20250162610A (ko) 2023-03-15 2025-11-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 폴리리보뉴클레오티드를 포함하는 조성물 및 이의 용도
WO2024192422A1 (en) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024216191A1 (en) 2023-04-12 2024-10-17 Flagship Pioneering Innovations Vi, Llc Modified trems, compositions, and related methods thereof
KR20250171373A (ko) 2023-04-12 2025-12-08 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 미스센스 돌연변이 교정용 trem
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024223724A1 (en) 2023-04-27 2024-10-31 Glaxosmithkline Biologicals Sa Influenza virus vaccines
AU2024260120A1 (en) 2023-04-27 2025-11-06 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
AU2024275548A1 (en) 2023-05-19 2026-01-08 Intellia Therapeutics, Inc. Ionizable amine lipids
WO2024258829A1 (en) 2023-06-12 2024-12-19 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
CN116535381B (zh) * 2023-07-06 2023-10-17 北京悦康科创医药科技股份有限公司 具有五元环缩醛结构的阳离子脂质化合物、包含其的组合物及用途
WO2025011529A2 (en) 2023-07-07 2025-01-16 Shanghai Circode Biomed Co., Ltd. Circular rna vaccines for seasonal flu and methods of uses
TW202516001A (zh) 2023-07-25 2025-04-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 Ｃａｓ內切酶及相關方法
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025049481A1 (en) 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods of editing an hla-a gene in vitro
WO2025045142A1 (en) 2023-08-29 2025-03-06 Shanghai Circode Biomed Co., Ltd. Circular rna encoding vegf polypeptides, formulations, and methods of uses
WO2025054236A2 (en) 2023-09-06 2025-03-13 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
US20250268826A1 (en) 2023-09-18 2025-08-28 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
US20250162981A1 (en) 2023-11-14 2025-05-22 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
TW202523695A (zh) 2023-11-22 2025-06-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於治療非酒精性脂肪性肝病之方法及組成物
WO2025117732A1 (en) * 2023-11-29 2025-06-05 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025137301A1 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Methods for rapid engineering of cells
WO2025137439A2 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Engineered t cells
WO2025132839A1 (en) 2023-12-21 2025-06-26 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2025160340A2 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Combination immunosuppression for inhibiting an immune response and enabling immunogen administration and re-administration
WO2025160334A1 (en) 2024-01-26 2025-07-31 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025194019A1 (en) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Methods for treating liver fibrosis and non-alcoholic fatty liver disease
GB202404607D0 (en) 2024-03-29 2024-05-15 Glaxosmithkline Biologicals Sa RNA formulation
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
WO2025229572A1 (en) 2024-05-01 2025-11-06 Glaxosmithkline Biologicals Sa Epstein-barr virus antigen-encoding messenger ribonucleic acid and antigen protein vaccines
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025240946A1 (en) 2024-05-17 2025-11-20 Intellia Therapeutics, Inc. Lipid nanoparticles and lipid nanoparticle compositions
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease
WO2025245111A1 (en) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Immunoreceptor targeting proteins and related methods
WO2025265017A1 (en) 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i
WO2026006577A1 (en) 2024-06-26 2026-01-02 Flagship Pioneering Innovations Vii, Llc Therapeutic circular dna forms
CN119462481B (zh) * 2025-01-17 2025-04-25 北京悦康科创医药科技股份有限公司 Sting激活型佐剂脂质、包含其的组合物及用途

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4438131A (en) 1981-04-10 1984-03-20 Basf Aktiengesellschaft Ω-Cyano-1, Ω-diphenyl-azaalkane derivatives, their preparation and drugs containing them
US4723039A (en) 1985-12-05 1988-02-02 Basf Aktiengesellschaft Phenylacetaldehydes substituted by basic groups, their preparation and drugs containing these compounds
JP2008105961A (ja) * 2006-10-23 2008-05-08 Japan Science & Technology Agency アリールメチルアミン誘導体およびその塩ならびにそれを構成成分とする薬物担体

Family Cites Families (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
JPH08507203A (ja) 1992-12-04 1996-08-06 イノーバー ラボラトリーズ，インコーポレイテッド 調節可能な核酸治療およびそれらの使用方法
DE69331911T2 (de) 1992-12-04 2002-11-21 Ribozyme Pharmaceuticals, Inc. Diagnose mittels signalverstärkung durch ein ribozym
US5871914A (en) 1993-06-03 1999-02-16 Intelligene Ltd. Method for detecting a nucleic acid involving the production of a triggering RNA and transcription amplification
WO1995008986A1 (en) 1993-09-27 1995-04-06 Smithkline Beecham Corporation Camptothecin formulations
US5989912A (en) 1996-11-21 1999-11-23 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US5837282A (en) 1996-10-30 1998-11-17 University Of British Columbia Ionophore-mediated liposome loading
EP0958303A4 (en) 1996-12-19 2004-03-31 Univ Yale BIOREACTIVE ALLOSTERIC POLYNUCLEOTIDES
JP2002506612A (ja) 1997-12-05 2002-03-05 デューク・ユニバーシティー 核酸仲介ｒｎａタグ付けおよびｒｎａ修復
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
IL126731A0 (en) 1998-10-23 1999-08-17 Intelligene Ltd A method of detection
JP2002528109A (ja) 1998-11-03 2002-09-03 エール ユニバーシティ マルチドメインポリヌクレオチド分子センサ
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
EP1235842A4 (en) 1999-10-15 2003-04-23 Univ Massachusetts Rna interference pathway genes as tools for targeted genetic interference
WO2002002606A2 (en) 2000-07-03 2002-01-10 Chiron S.P.A. Immunisation against chlamydia pneumoniae
WO2002034771A2 (en) 2000-10-27 2002-05-02 Chiron Srl Nucleic acids and proteins from streptococcus groups a & b
US20050175629A1 (en) 2001-08-31 2005-08-11 Giuseppe Del Giudice Helicobacter pylori vaccination
US20040006061A1 (en) 2001-10-04 2004-01-08 Wolfgang Haap Alkoxybenzylamine
WO2005002619A2 (en) 2003-06-26 2005-01-13 Chiron Corporation Immunogenic compositions for chlamydia trachomatis
JP4360854B2 (ja) * 2003-07-08 2009-11-11 テルモ株式会社 グアニジン誘導体およびそれを構成成分とする薬物担体
JP4875490B2 (ja) 2003-07-31 2012-02-15 ノバルティス バクシンズ アンド ダイアグノスティックス，インコーポレーテッド ＳｔｒｅｐｔｏｃｏｃｃｕｓＰｙｏｇｅｎｅｓについての免疫原組成物
WO2005030835A1 (ja) * 2003-09-25 2005-04-07 Terumo Kabushiki Kaisha ポリエチレングリコール誘導体及びこれを膜構成成分とする薬物担体
GB0410866D0 (en) 2004-05-14 2004-06-16 Chiron Srl Haemophilius influenzae
WO2005120461A2 (en) 2004-05-17 2005-12-22 Inex Pharmaceuticals Corporation Liposomal formulations comprising dihydrosphingomyelin and methods of use thereof
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
US7404969B2 (en) * 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
PT2351772T (pt) 2005-02-18 2016-09-22 Glaxosmithkline Biologicals Sa Proteínas e ácidos nucleicos de escherichia coli associada a meningite/septicemia
NZ560929A (en) 2005-02-18 2009-12-24 Novartis Vaccines & Diagnostic Immunogens from uropathogenic escherichia coli
NZ591415A (en) 2005-03-30 2012-10-26 Novartis Vaccines & Diagnostic Haemophilus influenzae type B
CN101238146A (zh) 2005-05-12 2008-08-06 诺华疫苗和诊断有限公司 砂眼衣原体的免疫原性组合物
AU2006307602A1 (en) 2005-10-25 2007-05-03 Novartis Vaccines And Diagnostics Srl Compositions comprising Yersinia pestis antigens
DK2003104T3 (en) * 2006-03-24 2015-08-10 Jitsubo Co Ltd Reagents for organic synthesis and method for organic synthesis reaction with the reagent
US20100166788A1 (en) 2006-08-16 2010-07-01 Novartis Vaccines And Diagnostics Immunogens from uropathogenic escherichia coli
EP2131848A4 (en) * 2007-02-16 2012-06-27 Merck Sharp & Dohme COMPOSITIONS AND METHODS FOR REINFORCED ACTIVITY OF BIOLOGICAL ACTIVE MOLECULES
GB0714963D0 (en) 2007-08-01 2007-09-12 Novartis Ag Compositions comprising antigens
GB0717187D0 (en) 2007-09-04 2007-10-17 Novartis Ag Compositions comprising yersinia pestis antigens
ITMI20081249A1 (it) 2008-07-09 2010-01-09 Novartis Vaccines & Diagnostic Immunogeni di escherichia coli con solubilità migliorata.
EP2268659A2 (en) 2008-03-06 2011-01-05 Novartis AG Mutant forms of chlamydia htra
US20110224447A1 (en) 2008-08-18 2011-09-15 Bowman Keith A Novel Lipid Nanoparticles and Novel Components for Delivery of Nucleic Acids
JP5747282B2 (ja) * 2008-11-10 2015-07-15 テクミラ ファーマシューティカルズ コーポレイションＴｅｋｍｉｒａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｃｏｒｐｏｒａｔｉｏｎ 治療薬を送達するための新規な脂質及び組成物
NZ595689A (en) 2009-04-14 2014-03-28 Novartis Ag Compositions for immunising against staphylococcus aureus
CA2766907A1 (en) 2009-07-06 2011-01-13 Novartis Ag Self replicating rna molecules and uses thereof
AU2010334911A1 (en) 2009-12-23 2012-07-12 Novartis Ag Lipids, lipid compositions, and methods of using them
WO2011136368A1 (ja) * 2010-04-28 2011-11-03 協和発酵キリン株式会社 カチオン性脂質
US20120004310A1 (en) 2010-05-28 2012-01-05 Longo Frank M Non-peptide bdnf neurotrophin mimetics
EP2590626B1 (en) 2010-07-06 2015-10-28 GlaxoSmithKline Biologicals SA Liposomes with lipids having an advantageous pka-value for rna delivery
US9770463B2 (en) 2010-07-06 2017-09-26 Glaxosmithkline Biologicals Sa Delivery of RNA to different cell types
EP4066857B1 (en) 2010-08-31 2022-12-21 GlaxoSmithKline Biologicals SA Pegylated liposomes for delivery of immunogen-encoding rna
US9504747B2 (en) 2013-03-08 2016-11-29 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP6352950B2 (ja) * 2013-03-08 2018-07-04 ノバルティス アーゲー 活性薬物の送達のための脂質と脂質組成物

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4438131A (en) 1981-04-10 1984-03-20 Basf Aktiengesellschaft Ω-Cyano-1, Ω-diphenyl-azaalkane derivatives, their preparation and drugs containing them
US4723039A (en) 1985-12-05 1988-02-02 Basf Aktiengesellschaft Phenylacetaldehydes substituted by basic groups, their preparation and drugs containing these compounds
JP2008105961A (ja) * 2006-10-23 2008-05-08 Japan Science & Technology Agency アリールメチルアミン誘導体およびその塩ならびにそれを構成成分とする薬物担体

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Bulletin of the Chemical Society of Japan, 2012, vol.85, no.3, pp.354-359*

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