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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

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules 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
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules 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
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5176Compounds of unknown constitution, e.g. material from plants or animals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14111Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14111Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141Use of virus, viral particle or viral elements as a vector
    • C12N2710/14144Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Nanotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Optics & Photonics (AREA)
  • Botany (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • Virology (AREA)
  • Biophysics (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
US201862675324P 2018-05-23 2018-05-23
US201862675317P 2018-05-23 2018-05-23
US201862675322P 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 (85)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MY203184A (en) 2017-08-09 2024-06-13 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
CA3091250A1 (fr) * 2018-02-14 2019-08-22 Generation Bio Co. Vecteurs d'adn non viraux et utilisations associees pour la production d'anticorps et de proteines de fusion
EP3796893A1 (fr) * 2018-05-23 2021-03-31 Modernatx, Inc. Administration d'adn
MX2021001599A (es) 2018-08-09 2021-07-02 Bioverativ Therapeutics Inc Moleculas de acido nucleico y sus usos para la terapia genica no viral.
CA3147875A1 (fr) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Compositions a recombinase et leurs methodes d'utilisation
CA3147728A1 (fr) * 2019-08-12 2021-02-18 Generation Bio Co. Methodes et compositions pour reduire des reponses immunitaires associees a une therapie genique ou d'acide nucleique
CA3150452A1 (fr) 2019-09-06 2021-03-11 Generation Bio Co. Compositions de nanoparticules lipidiques comprenant de l'adn a extremites fermees et des lipides clivables et leurs procedes d'utilisation
JP2023502576A (ja) * 2019-11-22 2023-01-25 ジェネレーション バイオ カンパニー イオン化可能な脂質およびそれらのナノ粒子組成物
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
CA3179444A1 (fr) 2020-05-20 2021-11-25 Avak Kahvejian Compositions immunogenes et leurs utilisations
AU2021275223A1 (en) 2020-05-20 2023-02-02 Flagship Pioneering Innovations Vi, Llc. Coronavirus antigen compositions and their uses
KR20230029685A (ko) 2020-05-29 2023-03-03 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Trem 조성물 및 이에 관련된 방법
WO2021243301A2 (fr) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Compositions à base de trem et procédés associés
CN116437964A (zh) * 2020-07-17 2023-07-14 世代生物公司 用于将多核苷酸封装成减小尺寸的脂质纳米颗粒以及其新型调配物的方法
BR112023001648A2 (pt) 2020-07-27 2023-04-04 Anjarium Biosciences Ag Moléculas de dna de fita dupla, veículo de entrega e método para preparar uma molécula de dna com extremidade em grampo
AU2021336976A1 (en) 2020-09-03 2023-03-23 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
GB202014751D0 (en) 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
KR20230135585A (ko) 2020-12-23 2023-09-25 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변형된 trem의 조성물 및 이의 용도
WO2022155195A1 (fr) * 2021-01-12 2022-07-21 Peranteau William Nanoparticules de lipide ionisables destinées à l'administration in utéro d'arnm
JP2024512669A (ja) 2021-03-31 2024-03-19 フラグシップ パイオニアリング イノベーションズ ブイ,インコーポレーテッド タノトランスミッションポリペプチド及び癌の処置におけるそれらの使用
EP4333906A4 (fr) * 2021-05-07 2025-04-02 Generation Bio Co. Compositions de vecteurs d'adn non viraux lyophilisées et leurs utilisations
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
EP4464783A3 (fr) 2021-09-17 2025-01-22 Flagship Pioneering Innovations VI, LLC Compositions et procédés de production de polyribonucléotides circulaires
TW202322826A (zh) 2021-10-18 2023-06-16 美商旗艦先鋒創新有限責任公司 用於純化多核糖核苷酸之組成物及方法
IL313004A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Coronavirus immunogen compositions and their uses
IL312799A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions of varicella-zoster virus and uses thereof
EP4436598A2 (fr) 2021-11-24 2024-10-02 Flagship Pioneering Innovations VI, LLC Compositions immunogènes et leurs utilisations
EP4448758A1 (fr) 2021-12-17 2024-10-23 Flagship Pioneering Innovations VI, LLC Procédés d'enrichissement en arn circulaire dans des conditions de dénaturation
CA3241061A1 (fr) 2021-12-22 2023-06-29 Alexandra Sophie DE BOER Compositions et procedes de purification de polyribonucleotides
MX2024007870A (es) 2021-12-23 2024-08-15 Flagship Pioneering Innovations Vi Llc Polirribonucleotidos circulares que codifican polipeptidos antifusogenicos.
EP4436558B1 (fr) * 2022-02-02 2025-09-24 MSLsolutions GmbH Procédé de production de médicaments et de vaccins
KR20240161965A (ko) * 2022-03-14 2024-11-13 제너레이션 바이오 컴퍼니 이종 프라임 부스트 백신 조성물 및 사용 방법
KR20240166554A (ko) 2022-03-25 2024-11-26 세일 바이오메디슨스, 인크. 신규한 이온화 가능 지질 및 지질 나노입자, 및 이를 사용하는 방법
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
EP4522743A1 (fr) 2022-05-09 2025-03-19 Flagship Pioneering Innovations VI, LLC Compositions de trem et procédés d'utilisation pour traiter des troubles prolifératifs
JP2025516638A (ja) 2022-05-13 2025-05-30 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 二本鎖dna組成物及び関連する方法
JP2025522311A (ja) 2022-05-25 2025-07-15 アカゲラ・メディスンズ,インコーポレイテッド 核酸を送達するための脂質ナノ粒子およびその使用方法
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
EP4569112A1 (fr) 2022-08-12 2025-06-18 Remix Therapeutics Inc. Procédés et compositions pour moduler l'épissage au niveau de sites d'épissage alternatifs
KR20250056256A (ko) 2022-08-31 2025-04-25 세일 바이오메디슨스, 인크. 신규 이온화성 지질 및 지질 나노입자, 그리고 이의 사용 방법
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
AR131008A1 (es) 2022-11-08 2025-02-05 Flagship Pioneering Innovations Vi Llc Composiciones y métodos para producir polirribonucleótidos circulares
TW202430215A (zh) 2022-12-14 2024-08-01 美商旗艦先鋒創新有限責任(Vii)公司 用於將治療劑遞送至骨之組成物和方法
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
WO2024151687A1 (fr) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Commutateurs génétiques et leur utilisation dans le traitement du cancer
US20240238473A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Recombinant nucleic acid molecules and their use in wound healing
EP4648794A2 (fr) 2023-01-09 2025-11-19 Flagship Pioneering Innovations VII, LLC Vaccins et procédés associés
TW202438515A (zh) 2023-02-06 2024-10-01 美商旗艦先鋒創新有限責任(Vii)公司 免疫調節組合物及相關方法
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
IL322201A (en) 2023-02-17 2025-09-01 Flagship Pioneering Innovations Vii Llc DNA structures containing modified uracil
EP4665856A2 (fr) 2023-02-17 2025-12-24 Flagship Pioneering Innovations VII, LLC Compositions d'adn comprenant de la cytosine modifiée
WO2024182707A1 (fr) 2023-03-02 2024-09-06 Krystal Biotech, Inc. Interleukine-2 et interleukine-12 pour le traitement du cancer
AU2024235803A1 (en) 2023-03-15 2025-09-25 Flagship Pioneering Innovations Vi, Llc Compositions comprising polyribonucleotides and uses thereof
WO2024192422A1 (fr) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Compositions immunogènes et leurs utilisations
KR20240149552A (ko) * 2023-04-06 2024-10-15 주식회사 무진메디 수분산성이 우수한 고함량 세라마이드 조성물
AU2024255972A1 (en) 2023-04-12 2025-10-23 Flagship Pioneering Innovations Vi, Llc Modified trems, compositions, and related methods thereof
WO2024216128A1 (fr) 2023-04-12 2024-10-17 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
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
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
WO2025064475A2 (fr) 2023-09-18 2025-03-27 Flagship Pioneering Innovations Vii, Llc Compositions lipidoïdes ionisables et leurs utilisations thérapeutiques
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
TW202535835A (zh) 2023-11-14 2025-09-16 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
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
US20250375499A1 (en) 2024-01-26 2025-12-11 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
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
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
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

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
DE10066104A1 (de) * 2000-09-08 2003-01-09 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
EP2816118B1 (fr) * 2005-05-31 2018-10-17 The Regents of the University of Colorado, a body corporate Procédés pour administrer des gènes
EP1979485A2 (fr) 2006-01-31 2008-10-15 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
US9943574B2 (en) * 2012-06-27 2018-04-17 Arthrogen B.V. Combination for treating an inflammatory disorder
CA2891911C (fr) * 2012-12-07 2023-03-07 Alnylam Pharmaceuticals, Inc. Formulations de particules lipidiques d'acide nucleique ameliorees
MX365409B (es) * 2013-03-14 2019-05-31 Shire Human Genetic Therapies Composiciones de ácido ribonucleico mensajero del regulador transmembrana de fibrosis quística y métodos y usos relacionados.
CN105408486B (zh) * 2013-05-21 2020-07-14 佛罗里达大学研究基金会有限公司 衣壳修饰的raav3载体组合物以及在人肝癌基因治疗中的用途
EP4019506A1 (fr) * 2013-12-19 2022-06-29 Novartis AG Lipides et compositions lipidiques pour l'administration 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
CA3021949C (fr) * 2015-04-24 2023-10-17 University Of Massachusetts Constructions d'aav modifiees et utilisations de celles-ci
ES2913626T5 (en) * 2015-12-22 2025-05-12 Modernatx Inc Compounds and compositions for intracellular delivery of agents
MX2018010633A (es) * 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.

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US20210059953A1 (en) 2021-03-04
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