MA50096A - Formulations de nanoparticules lipidiques de vecteurs d'adn exempts de capside non viraux - Google Patents

Formulations de nanoparticules lipidiques de vecteurs d'adn exempts de capside non viraux

Info

Publication number
MA50096A
MA50096A MA050096A MA50096A MA50096A MA 50096 A MA50096 A MA 50096A MA 050096 A MA050096 A MA 050096A MA 50096 A MA50096 A MA 50096A MA 50096 A MA50096 A MA 50096A
Authority
MA
Morocco
Prior art keywords
free dna
lipid nanoparticle
dna vectors
nanoparticle formulations
viral capside
Prior art date
Application number
MA050096A
Other languages
English (en)
Inventor
Ozan Alkan
Douglas Anthony Kerr
Robert Michael Kotin
Ara Karl Malakian
Matthew John Simmons
Matthew G Stanton
Jie Su
Teresa L Wright
Original Assignee
Generation Bio Co
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 Generation Bio Co filed Critical Generation Bio Co
Publication of MA50096A publication Critical patent/MA50096A/fr

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Nanotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Optics & Photonics (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Botany (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
MA050096A 2017-09-08 2018-09-07 Formulations de nanoparticules lipidiques de vecteurs d'adn exempts de capside non viraux MA50096A (fr)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762556334P 2017-09-08 2017-09-08
US201762556333P 2017-09-08 2017-09-08
US201762556381P 2017-09-09 2017-09-09
US201862675322P 2018-05-23 2018-05-23
US201862675317P 2018-05-23 2018-05-23
US201862675324P 2018-05-23 2018-05-23
US201862675327P 2018-05-23 2018-05-23

Publications (1)

Publication Number Publication Date
MA50096A true MA50096A (fr) 2020-07-15

Family

ID=65635269

Family Applications (1)

Application Number Title Priority Date Filing Date
MA050096A MA50096A (fr) 2017-09-08 2018-09-07 Formulations de nanoparticules lipidiques de vecteurs d'adn exempts de capside non viraux

Country Status (16)

Country Link
US (1) US20210059953A1 (fr)
EP (1) EP3679148A4 (fr)
JP (3) JP2020537493A (fr)
KR (1) KR102696307B1 (fr)
CN (1) CN111295448A (fr)
AU (1) AU2018330208B2 (fr)
BR (1) BR112020004219A2 (fr)
CA (1) CA3075180A1 (fr)
CO (1) CO2020002262A2 (fr)
IL (1) IL272799B2 (fr)
MA (1) MA50096A (fr)
MX (1) MX2020002501A (fr)
MY (1) MY209906A (fr)
PH (1) PH12020500466A1 (fr)
SG (1) SG11202000765PA (fr)
WO (1) WO2019051289A1 (fr)

Families Citing this family (95)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020002394A2 (pt) 2017-08-09 2020-07-28 Bioverativ Therapeutics Inc. moléculas de ácidos nucleicos e usos das mesmas
MA51842A (fr) * 2018-02-14 2020-12-23 Generation Bio Co Vecteurs d'adn non viraux et utilisations associées pour la production d'anticorps et de protéines de fusion
US20210346306A1 (en) * 2018-05-23 2021-11-11 Modernatx, Inc. Delivery of dna
KR102925832B1 (ko) 2018-08-09 2026-02-11 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 비바이러스 유전자 치료를 위한 이의 용도
CA3147875A1 (fr) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Compositions a recombinase et leurs methodes d'utilisation
AU2020327960A1 (en) * 2019-08-12 2022-02-17 Generation Bio Co. Methods and compositions for reducing gene or nucleic acid therapy-related immune responses
AU2020342668B2 (en) * 2019-09-06 2025-10-09 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended DNA and cleavable lipids and methods of use thereof
US20220370357A1 (en) * 2019-11-22 2022-11-24 Generation Bio Co. Ionizable lipids and nanoparticle compositions thereof
CN110974954B (zh) * 2019-12-24 2021-03-16 珠海丽凡达生物技术有限公司 一种用于增强核酸疫苗免疫效果的脂质纳米颗粒及其制备方法
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
TW202206097A (zh) 2020-05-20 2022-02-16 美商旗艦先鋒創新有限責任公司 冠狀病毒抗原組成物及其用途
EP4153223A1 (fr) 2020-05-20 2023-03-29 Flagship Pioneering Innovations VI, LLC Compositions immunogènes et leurs utilisations
IL298539A (en) 2020-05-29 2023-01-01 Flagship Pioneering Innovations Vi Llc Trem compositions and methods relating thereto
CA3182026A1 (fr) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Compositions a base de trem et procedes associes
EP4181948A4 (fr) * 2020-07-17 2024-12-04 Generation Bio Co. Procédés d'encapsulation de polynucléotides dans nanoparticules lipidiques de taille réduite et nouvelle formulation associée
WO2022023284A1 (fr) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions de molécules d'adn, leurs procédés de fabrication et leurs procédés d'utilisation
EP4208196A1 (fr) 2020-09-03 2023-07-12 Flagship Pioneering Innovations VI, LLC Compositions immunogènes et leurs utilisations
GB202014751D0 (en) 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
CZ310613B6 (cs) 2020-09-23 2026-01-28 Ústav organické chemie a biochemie AV ČR, v. v. i. Lipidoidy pro transfekci nukleových kyselin a jejich použití
CA3200234A1 (fr) 2020-11-25 2022-06-02 Daryl C. Drummond Nanoparticules lipidiques utilisees pour l'administration d'acides nucleiques, et methodes d'utilisation associees
BR112023012377A2 (pt) 2020-12-23 2023-10-24 Flagship Pioneering Innovations Vi Llc Composições de trems modificadas e usos das mesmas
WO2022155195A1 (fr) * 2021-01-12 2022-07-21 Peranteau William Nanoparticules de lipide ionisables destinées à l'administration in utéro d'arnm
US20220325287A1 (en) 2021-03-31 2022-10-13 Flagship Pioneering Innovations V, Inc. Thanotransmission polypeptides and their use in treating cancer
US20240261395A1 (en) * 2021-05-07 2024-08-08 Generation Bio Co. Lyophilized non-viral dna vector compositions and uses thereof
CZ310443B6 (cs) 2021-07-19 2025-06-25 Ústav organické chemie a biochemie AV ČR, v. v. i. Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití
EP4377457A1 (fr) 2021-07-26 2024-06-05 Flagship Pioneering Innovations VI, LLC Compositions de trem et leurs utilisations
EP4124348A1 (fr) * 2021-07-30 2023-02-01 4basebio UK Ltd Nanoparticules pour l'administration de cellules
CN116179605B (zh) * 2021-08-12 2025-11-11 蓝图生物医药(广州)有限公司 一种重组腺相关病毒载体及其应用
DK4271818T3 (da) 2021-09-17 2024-11-04 Flagship Pioneering Innovations Vi Llc Sammensætninger og fremgangsmåder til fremstilling af cirkulære polyribonukleotider
US20240417714A1 (en) 2021-10-18 2024-12-19 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
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
CA3238370A1 (fr) 2021-11-24 2023-06-01 Flagship Pioneering Innovations Vi, Llc Compositions d'immunogenes du virus varicelle-zona et leurs utilisations
IL312965A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and their uses
MX2024007307A (es) 2021-12-17 2024-08-06 Flagship Pioneering Innovations Vi Llc Métodos de enriquecimiento de arn circular en condiciones desnaturalizantes.
AU2022417517A1 (en) 2021-12-22 2024-06-27 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
MX2024007870A (es) 2021-12-23 2024-08-15 Flagship Pioneering Innovations Vi Llc Polirribonucleotidos circulares que codifican polipeptidos antifusogenicos.
US20250134825A1 (en) * 2022-02-02 2025-05-01 Mslsolutions Gmbh Method for producing medications and vaccines
AU2023235112A1 (en) * 2022-03-14 2024-10-17 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
EP4499607A1 (fr) 2022-03-25 2025-02-05 Sail Biomedicines, Inc. Nouveaux lipides et nanoparticules lipidiques ionisables et leurs procédés d'utilisation
US20250228928A1 (en) * 2022-04-04 2025-07-17 Spark Therapeutics, Inc. Immune enhancement and infectious disease treatment
WO2023196634A2 (fr) 2022-04-08 2023-10-12 Flagship Pioneering Innovations Vii, Llc Vaccins et procédés associés
US20250295687A1 (en) 2022-05-09 2025-09-25 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use for treating proliferative disorders
TW202409283A (zh) 2022-05-13 2024-03-01 美商旗艦先鋒創新有限責任(Vii)公司 雙股dna組合物及相關方法
EP4531819A2 (fr) 2022-05-25 2025-04-09 Akagera Medicines, Inc. Nanoparticules lipidiques pour l'administration d'acides nucléiques et leurs procédés d'utilisation
WO2023250112A1 (fr) 2022-06-22 2023-12-28 Flagship Pioneering Innovations Vi, Llc Compositions de trem modifiées et leurs utilisations
WO2024015428A1 (fr) * 2022-07-12 2024-01-18 Trustees Of Boston University Voies de signalisation cellulaire synthétiques et leurs utilisations
AU2023320333A1 (en) 2022-08-01 2025-01-16 Flagship Pioneering Innovations Vii, Llc Immunomodulatory proteins and related methods
KR102544632B1 (ko) * 2022-08-05 2023-06-16 주식회사 무진메디 장쇄 세라마이드가 포함된 지질 나노입자 및 이를 포함하는 세포 사멸용 조성물
WO2024035952A1 (fr) 2022-08-12 2024-02-15 Remix Therapeutics Inc. Procédés et compositions pour moduler l'épissage au niveau de sites d'épissage alternatifs
EP4581012A2 (fr) 2022-08-31 2025-07-09 Sail Biomedicines, Inc. Nouveaux lipides ionisables et nanoparticules lipidiques, ainsi que procédés d'utilisation associés
WO2024077191A1 (fr) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Molécules d'acide nucléique codant pour des trif et des polypeptides supplémentaires et leur utilisation dans le traitement du cancer
EP4612296A1 (fr) 2022-10-31 2025-09-10 Flagship Pioneering Innovations VI, LLC Compositions et procédés de purification de polyribonucléotides
TW202434728A (zh) 2022-11-08 2024-09-01 美商旗艦先鋒創新有限責任公司 用於產生環狀多核糖核苷酸的組成物和方法
WO2024129988A1 (fr) 2022-12-14 2024-06-20 Flagship Pioneering Innovations Vii, Llc Compositions et procédés d'administration d'agents thérapeutiques à un os
WO2024151673A2 (fr) 2023-01-09 2024-07-18 President And Fellows Of Harvard College Molécules d'acide nucléique recombinant et leur utilisation dans la cicatrisation de plaies
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
US20240269251A1 (en) 2023-01-09 2024-08-15 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
WO2024151685A1 (fr) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Molécules d'acide nucléique recombinant et leur utilisation dans la cicatrisation des plaies
WO2024167885A1 (fr) 2023-02-06 2024-08-15 Flagship Pioneering Innovations Vii, Llc Compositions immunomodulatrices et procédés associés
EP4665736A2 (fr) 2023-02-13 2025-12-24 Flagship Pioneering Innovations VII, LLC Lipides ionisables contenant un lieur clivable et supports lipidiques pour compositions thérapeutiques
AU2024222598A1 (en) 2023-02-17 2025-07-31 Flagship Pioneering Innovations Vii, Llc Dna compositions comprising modified uracil
JP2026507514A (ja) 2023-02-17 2026-03-04 フラッグシップ パイオニアリング イノベーションズ セブン,エルエルシー 修飾されたシトシンを含むdna組成物
AU2024229184A1 (en) 2023-03-02 2025-08-28 Krystal Biotech, Inc. Interleukin-2 and interleukin-12 for cancer therapy
WO2024192422A1 (fr) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Compositions immunogènes et leurs utilisations
AU2024235803A1 (en) 2023-03-15 2025-09-25 Flagship Pioneering Innovations Vi, Llc Compositions comprising polyribonucleotides and uses thereof
KR102938591B1 (ko) * 2023-04-06 2026-03-12 주식회사 무진메디 수분산성이 우수한 고함량 세라마이드 조성물
JP2026514004A (ja) 2023-04-12 2026-05-01 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 修飾されたtrem、組成物及びその関連方法
EP4695391A1 (fr) 2023-04-12 2026-02-18 Flagship Pioneering Innovations VI, LLC Trems destinés à être utilisés dans la correction de mutations faux-sens
WO2024220746A2 (fr) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Agents d'arni ciblant la synthase d'acides gras et procédés associés
WO2024258829A1 (fr) 2023-06-12 2024-12-19 Flagship Pioneering Innovations Vii, Llc Compositions de vaccin contre le sars-cov-2 et procédés associés
WO2025006684A1 (fr) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Polyribonucléotides circulaires codant pour des polypeptides antifusogènes
US20250092426A1 (en) 2023-07-25 2025-03-20 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
WO2025042786A1 (fr) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprenant des polyribonucléotides circulaires et leurs utilisations
WO2025049632A1 (fr) * 2023-08-29 2025-03-06 Rejuvenation Technologies Inc. Compositions et méthodes pour l'administration large d'arn dans un tissu
WO2025054236A2 (fr) 2023-09-06 2025-03-13 Flagship Pioneering Innovations Vii, Llc Compositions de vaccin contre le sars-cov-2 et procédés associés
WO2025052180A2 (fr) 2023-09-07 2025-03-13 Axelyf ehf. Lipides et nanoparticules lipidiques
TW202525266A (zh) 2023-09-18 2025-07-01 美商旗艦先鋒創新有限責任(Vii)公司 可電離類脂質組成物及其治療用途
WO2025072331A1 (fr) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Nucléases cas et procédés ou méthodes associés
WO2025096807A2 (fr) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Nouvelles formes d'adn thérapeutique
WO2025106670A1 (fr) 2023-11-14 2025-05-22 Flagship Pioneering Innovations Vii, Llc Compositions lipidoïdes ionisables et leurs utilisations thérapeutiques
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
TW202545974A (zh) 2024-01-26 2025-12-01 美商旗艦先鋒創新有限責任(Vii)公司 免疫受體抑制蛋白及相關方法
WO2025194019A1 (fr) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Méthodes de traitement de la fibrose hépatique et de la stéatose hépatique non alcoolique
WO2025217275A2 (fr) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Compositions ciblées sur des cellules immunitaires et procédés associés
WO2025240680A1 (fr) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Protéines inhibitrices d'immunorécepteurs et procédés associés
WO2025245188A2 (fr) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Méthodes de traitement de la stéatose hépatique et d'une stéatose hépatique non alcoolique
WO2025245111A1 (fr) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Protéines de ciblage d'immunorécepteurs et procédés associés
US20260000702A1 (en) 2024-06-26 2026-01-01 Flagship Pioneering Innovations Vii, Llc Therapeutic circular dna forms
WO2026003582A2 (fr) 2024-06-27 2026-01-02 Axelyf ehf. Lipides et nanoparticules lipidiques
WO2026055547A1 (fr) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Compositions d'adn et procédés associés
WO2026055543A1 (fr) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Compositions d'adn modifiées et procédés associés
WO2026064313A1 (fr) 2024-09-17 2026-03-26 Flagship Pioneering Innovations Vii, Llc Compositions d'arn et procédés associés
WO2026085300A1 (fr) 2024-10-16 2026-04-23 Flagship Pioneering Innovations Vii, Llc Procédés et compositions
WO2026085495A1 (fr) 2024-10-18 2026-04-23 Generation Bio Co. Compositions d'acides nucléiques synthétiques partiellement simples brin, utilisations et procédés associés
WO2026096919A1 (fr) 2024-11-01 2026-05-07 Sail Biomedicines, Inc. Nouveaux lipides à cœur dipeptidique, vecteurs lipidiques et méthodes d'utilisation de ceux-ci

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) * 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869305A (en) * 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
DE10044384A1 (de) * 2000-09-08 2002-04-18 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
EP1838853A2 (fr) * 2005-01-06 2007-10-03 Benitec, Inc. Agents arni pour l'entretien de cellules souches
DK2816118T3 (en) * 2005-05-31 2018-12-03 Univ Colorado Regents Methods for delivery of genes
WO2008016391A2 (fr) * 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Vecteurs parvoviraux auto-complémentaires et leurs procédés de fabrication et d'utilisation
AU2016203992A1 (en) * 2009-05-11 2016-06-30 Berg Llc Methods for the diagnosis of metabolic disorders using epimetabolic shifters, multidimensional intracellular molecules, or environmental influencers
EP2500434A1 (fr) * 2011-03-12 2012-09-19 Association Institut de Myologie Vecteurs AAV sans capside, compositions et procédés pour la production des vecteurs et la thérapie génique
AU2013281328B2 (en) * 2012-06-27 2017-11-23 MeiraGTx Gene Regulation Limited Combination for treating an inflammatory disorder
US9687448B2 (en) * 2012-12-07 2017-06-27 Alnylam Pharmaceuticals, Inc. Nucleic acid lipid particle formulations
EP2968586B1 (fr) * 2013-03-14 2018-07-25 Translate Bio, Inc. Composition à base d'arnm du gène cftr et procédés et utilisations associés
KR102298579B1 (ko) * 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
WO2015095346A1 (fr) * 2013-12-19 2015-06-25 Novartis Ag Lipides et compositions lipidiques destinés à la libération d'agents actifs
EP3242945B1 (fr) * 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Construction génique à vecteur unique comprenant des gènes de l'insuline et de la glucokinase
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
JP7114465B2 (ja) * 2015-12-22 2022-08-08 モデルナティエックス インコーポレイテッド 薬剤の細胞内送達のための化合物および組成物
SG10201913688TA (en) * 2016-03-03 2020-03-30 Univ Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

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