EP4680595A2 - Lipidnanopartikel mit codierenden rna-molekülen zur verwendung bei der geneditierung und als impfstoffe und therapeutika - Google Patents

Lipidnanopartikel mit codierenden rna-molekülen zur verwendung bei der geneditierung und als impfstoffe und therapeutika

Info

Publication number
EP4680595A2
EP4680595A2 EP24719912.8A EP24719912A EP4680595A2 EP 4680595 A2 EP4680595 A2 EP 4680595A2 EP 24719912 A EP24719912 A EP 24719912A EP 4680595 A2 EP4680595 A2 EP 4680595A2
Authority
EP
European Patent Office
Prior art keywords
optionally substituted
certain embodiments
formula
compound
glycero
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP24719912.8A
Other languages
English (en)
French (fr)
Inventor
Muthusamy Jayaraman
Ganapathy Subramanian SANKARAN
Karolina Anna KOSAKOWSKA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Renagade Therapeutics Management Inc
Original Assignee
Renagade Therapeutics Management Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Renagade Therapeutics Management Inc filed Critical Renagade Therapeutics Management Inc
Publication of EP4680595A2 publication Critical patent/EP4680595A2/de
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D211/00Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/08Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
    • C07D211/18Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D211/34Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D207/00Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D207/02Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D207/30Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members
    • C07D207/32Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached to ring carbon atoms
    • C07D207/325Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached to ring carbon atoms with substituted hydrocarbon radicals directly attached to the ring nitrogen atom
    • C07D207/327Radicals substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D405/00Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/02Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
    • C07D405/12Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
EP24719912.8A 2023-03-15 2024-03-14 Lipidnanopartikel mit codierenden rna-molekülen zur verwendung bei der geneditierung und als impfstoffe und therapeutika Pending EP4680595A2 (de)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US202363490298P 2023-03-15 2023-03-15
US202363490305P 2023-03-15 2023-03-15
US202363452293P 2023-03-15 2023-03-15
US202363490381P 2023-03-15 2023-03-15
US202363490385P 2023-03-15 2023-03-15
US202363452292P 2023-03-15 2023-03-15
US202363490311P 2023-03-15 2023-03-15
US202363490384P 2023-03-15 2023-03-15
US202363490312P 2023-03-15 2023-03-15
US202363490387P 2023-03-15 2023-03-15
US202463552484P 2024-02-12 2024-02-12
PCT/US2024/019990 WO2024192277A2 (en) 2023-03-15 2024-03-14 Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Publications (1)

Publication Number Publication Date
EP4680595A2 true EP4680595A2 (de) 2026-01-21

Family

ID=90789684

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24719912.8A Pending EP4680595A2 (de) 2023-03-15 2024-03-14 Lipidnanopartikel mit codierenden rna-molekülen zur verwendung bei der geneditierung und als impfstoffe und therapeutika

Country Status (4)

Country Link
EP (1) EP4680595A2 (de)
CN (1) CN121263402A (de)
AU (1) AU2024234874A1 (de)
WO (1) WO2024192277A2 (de)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025166238A1 (en) 2024-01-31 2025-08-07 Orna Therapeutics, Inc. Fast-shedding polyethylene glycol lipids
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026008743A1 (en) * 2024-07-02 2026-01-08 Sanofi Pasteur Inc. Water-soluble polyanionic polymer as adjuvant for carrier-formulated nucleic acid

Family Cites Families (109)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5773244A (en) 1993-05-19 1998-06-30 Regents Of The University Of California Methods of making circular RNA
US5766903A (en) 1995-08-23 1998-06-16 University Technology Corporation Circular RNA and uses thereof
ES2340499T3 (es) 2001-06-05 2010-06-04 Curevac Gmbh Arnm de antigeno tumoral estabilizado con un contenido de g/c aumentado.
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
DE10229872A1 (de) 2002-07-03 2004-01-29 Curevac Gmbh Immunstimulation durch chemisch modifizierte RNA
DE10335833A1 (de) 2003-08-05 2005-03-03 Curevac Gmbh Transfektion von Blutzellen mit mRNA zur Immunstimulation und Gentherapie
DE102004042546A1 (de) 2004-09-02 2006-03-09 Curevac Gmbh Kombinationstherapie zur Immunstimulation
DE102005023170A1 (de) 2005-05-19 2006-11-23 Curevac Gmbh Optimierte Formulierung für mRNA
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
DE102006007433A1 (de) 2006-02-17 2007-08-23 Curevac Gmbh Adjuvanz in Form einer Lipid-modifizierten Nukleinsäure
EP2046954A2 (de) 2006-07-31 2009-04-15 Curevac GmbH Nukleinsäure der formel (i): gixngn, oder (ii): cixmcn, insbesondere als immunstimulierendes mittel/adjuvans
DE102006051516A1 (de) 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
DE102006061015A1 (de) 2006-12-22 2008-06-26 Curevac Gmbh Verfahren zur Reinigung von RNA im präparativen Maßstab mittels HPLC
DE102007001370A1 (de) 2007-01-09 2008-07-10 Curevac Gmbh RNA-kodierte Antikörper
US20090163705A1 (en) * 2007-05-21 2009-06-25 Alnylam Pharmaceuticals, Inc. Cationic lipids
CN101977510A (zh) 2007-06-29 2011-02-16 联邦科学技术研究组织 降解毒性化合物的方法
WO2009030254A1 (en) 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
EP2535419A3 (de) 2007-09-26 2013-05-29 Intrexon Corporation Synthetische 5'UTRs, Expressionsvektoren und Verfahren zur Erhöhung der Transgenexpression
CN101939428B (zh) 2007-12-11 2014-05-07 斯克利普斯研究所 涉及mRNA翻译增强因子的组合物和方法
WO2009120247A2 (en) 2007-12-27 2009-10-01 The Ohio State University Research Foundation Lipid nanoparticle compositions and methods of making and using the same
KR101483715B1 (ko) 2008-01-31 2015-01-19 큐어백 게엠바하 면역증강제/애주번트인 화학식(NuGlXmGnNv)a를 포함하는 핵산 및 이의 유도체
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation 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
WO2010037408A1 (en) 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
WO2010088927A1 (en) 2009-02-09 2010-08-12 Curevac Gmbh Use of pei for the improvement of endosomal release and expression of transfected nucleic acids, complexed with cationic or polycationic compounds
US20110053829A1 (en) 2009-09-03 2011-03-03 Curevac Gmbh Disulfide-linked polyethyleneglycol/peptide conjugates for the transfection of nucleic acids
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
US9670487B2 (en) 2010-01-22 2017-06-06 Sirna Therapeutics, Inc. Cationic lipids for oligonucleotide delivery
JP5902616B2 (ja) 2010-04-28 2016-04-13 協和発酵キリン株式会社 カチオン性脂質
EP2387999A1 (de) 2010-05-21 2011-11-23 CureVac GmbH Histidin enthaltende Lösung zur Transfektion und/oder Injektion von Nukleinsäuren und deren Verwendungen
ES2558106T3 (es) 2010-07-30 2016-02-02 Curevac Ag Formación de complejos de ácidos nucleicos con componentes catiónicos disulfuro-reticulados para la transfección e inmunoestimulación
WO2012019630A1 (en) 2010-08-13 2012-02-16 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded protein
WO2012089225A1 (en) 2010-12-29 2012-07-05 Curevac Gmbh Combination of vaccination and inhibition of mhc class i restricted antigen presentation
EP3202760B1 (de) 2011-01-11 2019-08-21 Alnylam Pharmaceuticals, Inc. Pegylierte lipide und deren verwendung zur wirkstofffreisetzung
WO2012116715A1 (en) 2011-03-02 2012-09-07 Curevac Gmbh Vaccination in newborns and infants
WO2012113413A1 (en) 2011-02-21 2012-08-30 Curevac Gmbh Vaccine composition comprising complexed immunostimulatory nucleic acids and antigens packaged with disulfide-linked polyethyleneglycol/peptide conjugates
WO2012116714A1 (en) 2011-03-02 2012-09-07 Curevac Gmbh Vaccination in elderly patients
BR112013031553A2 (pt) 2011-06-08 2020-11-10 Shire Human Genetic Therapies, Inc. composições, mrna que codifica para uma hgla e seu uso, uso de pelo menos uma molécula de mrna e um veículo de transferência e uso de um mrna que codifica para proteína exógena
JP6250543B2 (ja) 2011-09-27 2017-12-20 アルニラム・ファーマシューティカルズ・インコーポレーテッド ジ脂肪族置換peg化脂質
ES2921724T1 (es) 2011-12-07 2022-08-31 Alnylam Pharmaceuticals Inc Lípidos biodegradables para la administración de agentes activos
JP6305343B2 (ja) 2011-12-07 2018-04-04 アルニラム・ファーマシューティカルズ・インコーポレーテッド 活性薬剤の送達のための分岐アルキルおよびシクロアルキルを末端とする生分解性脂質
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
EP2623121A1 (de) 2012-01-31 2013-08-07 Bayer Innovation GmbH Pharmazeutische Zusammensetzung mit einem Polymerträger-Cargo-Komplex und einem Antigen
WO2013113326A1 (en) 2012-01-31 2013-08-08 Curevac Gmbh Pharmaceutical composition comprising a polymeric carrier cargo complex and at least one protein or peptide antigen
WO2013113325A1 (en) 2012-01-31 2013-08-08 Curevac Gmbh Negatively charged nucleic acid comprising complexes for immunostimulation
WO2013120499A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly (a) sequence or a polyadenylation signal for increasing the expression of an encoded pathogenic antigen
WO2013120500A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded tumour antigen
WO2013120497A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
WO2013120498A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded allergenic antigen or an autoimmune self-antigen
ES2660129T3 (es) 2012-03-27 2018-03-20 Curevac Ag Moléculas de ácido nucleico artificiales que comprenden un 5'UTR-TOP
MX362981B (es) 2012-03-27 2019-02-28 Curevac Ag Moleculas artificiales de acido nucleico para la expresion mejorada de proteina o peptido.
CA2866955A1 (en) 2012-03-27 2013-10-03 Curevac Gmbh Artificial nucleic acid molecules
EP3498267A1 (de) 2012-05-25 2019-06-19 CureVac AG Reversible immobilisierung und/oder kontrollierte freisetzung von nucleinsäuren mit nanopartikeln durch (biologisch abbaubare) polymerbeschichtungen
CA2876155C (en) 2012-06-08 2022-12-13 Ethris Gmbh Pulmonary delivery of mrna to non-lung target cells
US20150299702A1 (en) 2012-11-30 2015-10-22 Aarhus Universitet Circular rna for inhibition of microrna
MX385338B (es) 2013-02-22 2025-03-18 CureVac SE Combinación de vacunación e inhibición de la trayectoria pd-1.
US20160145348A1 (en) 2013-03-14 2016-05-26 Fred Hutchinson Cancer Research Center Compositions and methods to modify cells for therapeutic objectives
ES2795249T3 (es) 2013-03-15 2020-11-23 Translate Bio Inc Mejora sinérgica de la administración de ácidos nucleicos a través de formulaciones mezcladas
AU2014310930A1 (en) 2013-08-21 2016-01-21 Curevac Ag Composition and vaccine for treating prostate cancer
CN105517569A (zh) 2013-08-21 2016-04-20 库瑞瓦格股份公司 狂犬病疫苗
SG11201510748PA (en) 2013-08-21 2016-03-30 Curevac Ag Composition and vaccine for treating lung cancer
CN105451779A (zh) 2013-08-21 2016-03-30 库瑞瓦格股份公司 增加rna编码蛋白表达的方法
CN105473157A (zh) 2013-08-21 2016-04-06 库瑞瓦格股份公司 组合疫苗
SG11201510746WA (en) 2013-08-21 2016-03-30 Curevac Ag Respiratory syncytial virus (rsv) vaccine
WO2015062738A1 (en) 2013-11-01 2015-05-07 Curevac Gmbh Modified rna with decreased immunostimulatory properties
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CA2927254C (en) 2013-12-30 2023-10-24 Curevac Ag Artificial nucleic acid molecules
AU2014375404C1 (en) 2013-12-30 2020-11-19 CureVac Manufacturing GmbH Methods for RNA analysis
KR102399799B1 (ko) 2013-12-30 2022-05-18 큐어백 아게 인공 핵산 분자
PL3981437T3 (pl) 2014-04-23 2025-02-24 Modernatx, Inc. Szczepionki z kwasem nukleinowym
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
EP3298170B1 (de) 2015-05-21 2021-12-29 Cofactor Genomics, Inc. Verfahren zur erzeugung von zirkulärer dna aus zirkulärer rna
ES3031941T3 (en) 2015-05-26 2025-07-14 Univ Ramot Targeted lipid particles for systemic delivery of nucleic acid molecules to leukocytes
US11000547B2 (en) 2015-06-05 2021-05-11 Dana-Farber Cancer Institute, Inc. Compositions related to rna in circularized form
ES2969082T3 (es) 2015-09-17 2024-05-16 Modernatx Inc Compuestos y composiciones para la administración intracelular de agentes terapéuticos
US10167457B2 (en) 2015-10-23 2019-01-01 President And Fellows Of Harvard College Nucleobase editors and uses thereof
FI3368507T3 (fi) 2015-10-28 2023-03-21 Acuitas Therapeutics Inc Uusia lipidejä ja lipidinanopartikkeliformulaatioita nukleiinihappojen annostelemiseksi
NZ749593A (en) 2016-06-20 2025-10-31 Univ Leland Stanford Junior Circular rnas and their use in immunomodulation
JP7086870B2 (ja) 2016-06-30 2022-06-20 アルブータス・バイオファーマー・コーポレイション メッセンジャーrnaを送達するための組成物及び方法
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
EP3707257A4 (de) 2017-11-07 2021-12-08 The University of North Carolina at Chapel Hill Verfahren und zusammensetzungen für zirkuläre rna-moleküle
IL275205B2 (en) 2017-12-15 2025-03-01 Flagship Pioneering Innovations Vi Llc Preparations containing circular polyribonucleotides and uses thereof
AU2019216307A1 (en) 2018-01-30 2020-07-09 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
EP3802507A1 (de) 2018-05-30 2021-04-14 Translate Bio, Inc. Kationische vitamin-lipide
IL319091A (en) 2018-06-06 2025-04-01 Massachusetts Inst Technology Circular RNA for translation in eukaryotic cells
GB2596224B (en) 2018-06-19 2022-09-07 Univ Texas Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
US12419965B2 (en) 2018-10-01 2025-09-23 BioNTech SE RNA particles comprising polysarcosine
EP3972653A1 (de) 2019-05-22 2022-03-30 Massachusetts Institute of Technology Zirkuläre rns-zusammensetzungen und verfahren
JP7638972B2 (ja) 2019-09-19 2025-03-04 モデルナティエックス インコーポレイテッド 治療薬の細胞内送達のための分岐状尾部脂質化合物及び組成物
CA3155075A1 (en) 2019-10-18 2021-04-22 Michael Mitchell Lipid nanoparticles and formulations thereof for car mrna delivery
TW202204622A (zh) 2020-04-09 2022-02-01 大陸商蘇州艾博生物科技有限公司 針對冠狀病毒之核酸疫苗
WO2021226597A2 (en) 2020-05-08 2021-11-11 Orna Therapeutics, Inc. Circular rna compositions and methods
AU2021328980A1 (en) 2020-08-20 2023-03-09 Suzhou Abogen Biosciences Co., Ltd. Lipid compounds and lipid nanoparticle compositions
US20230320994A1 (en) 2020-08-21 2023-10-12 The Board Of Regents Of The University Of Texas System Functional ionizable phospholipids
WO2022081694A1 (en) 2020-10-13 2022-04-21 The Trustees Of The University Of Pennsylvania In vivo targeting of fibrosis by anti-cd5-targeted fap-car t mrna-lnp
MX2023004300A (es) 2020-10-13 2023-06-22 Univ Pennsylvania Direccionamiento in vivo de células t para terapia con ácido ribonucleico mensajero (arnm).
KR20230144574A (ko) 2021-02-09 2023-10-16 세리나 쎄라퓨틱스, 인코포레이티드 폴리옥사졸린-지질 접합체, 및 이를 포함하는 지질 나노입자 및 제약 조성물
WO2023044343A1 (en) 2021-09-14 2023-03-23 Renagade Therapeutics Management Inc. Acyclic lipids and methods of use thereof
JP2024533865A (ja) * 2021-09-14 2024-09-12 レナゲード セラピューティクス マネージメント インコーポレイテッド 環状脂質及びその使用方法
WO2023141470A2 (en) 2022-01-19 2023-07-27 President And Fellows Of Harvard College Immunomodulatory lipids and uses thereof
CN118871134A (zh) 2022-03-21 2024-10-29 生物探险有限公司 用于增强蛋白质表达的内部核糖体进入位点(IRES)、质粒载体和环状mRNA
IL315577A (en) 2022-04-05 2024-11-01 Capstan Therapeutics Inc Ionizable cationic lipids and lipid nanoparticles
CA3255619A1 (en) * 2022-04-07 2023-10-12 Renagade Therapeutics Management Inc. CYCLIC LIPIDS AND LIPID NANOPARTICLES (LPNs) FOR THE SUPPLY OF NUCLEIC ACIDS OR PEPTIDES INTENDED FOR USE IN VACCINATION AGAINST INFECTIOUS AGENTS
WO2023240261A1 (en) * 2022-06-10 2023-12-14 Renagade Therapeutics Management Inc. Nucleobase editing system and method of using same for modifying nucleic acid sequences
WO2024020346A2 (en) 2022-07-18 2024-01-25 Renagade Therapeutics Management Inc. Gene editing components, systems, and methods of use
EP4577520A1 (de) 2022-08-26 2025-07-02 Renagade Therapeutics Management Inc. Pegylierte lipidverbindungen und verfahren zur verwendung davon

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