JP2024511463A5 - - Google Patents

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Publication number
JP2024511463A5
JP2024511463A5 JP2023558515A JP2023558515A JP2024511463A5 JP 2024511463 A5 JP2024511463 A5 JP 2024511463A5 JP 2023558515 A JP2023558515 A JP 2023558515A JP 2023558515 A JP2023558515 A JP 2023558515A JP 2024511463 A5 JP2024511463 A5 JP 2024511463A5
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JP
Japan
Prior art keywords
approximately
lipid
aerosol
composition according
aerosol composition
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
JP2023558515A
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English (en)
Japanese (ja)
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JP2024511463A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/021442 external-priority patent/WO2022204219A1/en
Publication of JP2024511463A publication Critical patent/JP2024511463A/ja
Publication of JP2024511463A5 publication Critical patent/JP2024511463A5/ja
Pending legal-status Critical Current

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JP2023558515A 2021-03-22 2022-03-22 細胞への標的送達のための組成物および方法 Pending JP2024511463A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163164523P 2021-03-22 2021-03-22
US63/164,523 2021-03-22
US202163229497P 2021-08-04 2021-08-04
US63/229,497 2021-08-04
US202263305426P 2022-02-01 2022-02-01
US63/305,426 2022-02-01
PCT/US2022/021442 WO2022204219A1 (en) 2021-03-22 2022-03-22 Compositions and methods for targeted delivery to cells

Publications (2)

Publication Number Publication Date
JP2024511463A JP2024511463A (ja) 2024-03-13
JP2024511463A5 true JP2024511463A5 (OSRAM) 2025-04-01

Family

ID=83396024

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023558515A Pending JP2024511463A (ja) 2021-03-22 2022-03-22 細胞への標的送達のための組成物および方法

Country Status (10)

Country Link
US (10) US20240207178A1 (OSRAM)
EP (1) EP4312987A4 (OSRAM)
JP (1) JP2024511463A (OSRAM)
KR (1) KR20240005692A (OSRAM)
AU (1) AU2022246065A1 (OSRAM)
BR (1) BR112023019229A2 (OSRAM)
CA (1) CA3213299A1 (OSRAM)
IL (1) IL305782A (OSRAM)
MX (1) MX2023011227A (OSRAM)
WO (1) WO2022204219A1 (OSRAM)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108271360B (zh) * 2015-09-14 2023-01-24 得克萨斯州大学系统董事会 亲脂阳离子树枝状聚合物及其用途
MX2023011104A (es) * 2021-03-22 2024-03-01 Recode Therapeutics Inc Composiciones de polinucleotidos, formulaciones relacionadas y metodos de uso de las mismas.
WO2022204053A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204219A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Compositions and methods for targeted delivery to cells
EP4313001A4 (en) * 2021-03-22 2025-11-26 Univ Texas Compositions and methods for targeted administration to cells
KR20230175196A (ko) 2021-03-23 2023-12-29 리코드 테라퓨틱스, 인크. 폴리뉴클레오티드 조성물, 관련된 제제, 및 이의 사용 방법
MX2023011230A (es) * 2021-03-23 2023-12-06 Recode Therapeutics Inc Composiciones y metodos para administracion sistemica dirigida a celulas.
WO2022226344A1 (en) * 2021-04-22 2022-10-27 The Board Of Regents Of The University Of Texas System All-in-one dendrimer-based lipid nanoparticles enable precise hdr-mediated gene editing in vivo
EP4630057A1 (en) 2022-12-08 2025-10-15 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
CN116211831A (zh) * 2022-12-14 2023-06-06 北京剂泰医药科技有限公司 脂质基质局部注射制剂
EP4385523A1 (en) * 2022-12-14 2024-06-19 Beijing Jitai Pharmaceutical Technology Co., Ltd. Lipid-based topical injection formulations
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
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
CN116983280B (zh) * 2023-07-19 2025-09-30 南通大学 一种具有肺选择性的脂质纳米颗粒及其制备方法与应用
WO2025075396A1 (ko) * 2023-10-06 2025-04-10 한국과학기술연구원 타겟 핵산 검출물질이 담지된 지질 나노입자 및 이를 이용한 막융합 기반의 타겟 핵산 검출 방법
TW202525812A (zh) * 2023-10-24 2025-07-01 南韓商綠十字股份有限公司 可離子化脂質及其用途
US12553042B2 (en) 2023-12-01 2026-02-17 Recode Therapeutics, Inc. Method for quantifying an amount of capped messenger RNA
WO2025117816A1 (en) * 2023-12-01 2025-06-05 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
US12364773B2 (en) * 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
WO2025137646A1 (en) * 2023-12-22 2025-06-26 Recode Therapeutics, Inc. Gene editing methods and compositions for treating cystic fibrosis
CN117919200B (zh) * 2024-01-19 2025-09-05 晟迪生物医药(苏州)有限公司 含有可电离脂质的肺部递送系统、其制备方法和应用
CN117942406B (zh) * 2024-01-19 2025-07-11 晟迪生物医药(苏州)有限公司 预制载体在制备用于免疫细胞和干细胞的体外基因递送的产品中的应用
WO2025240355A1 (en) * 2024-05-14 2025-11-20 Recode Therapeutics, Inc. Lipid nanoparticles for extrahepatic delivery

Family Cites Families (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
CA2222328C (en) * 1995-06-07 2012-01-10 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
PT941066E (pt) 1996-08-26 2004-03-31 Transgene Sa Complexos de lipido cationico - acido nucleico
US5962429A (en) 1996-11-22 1999-10-05 University Of Iowa Complexes of adenovirus with cationic molecules
EP1903054A3 (de) 2001-06-05 2008-07-23 CureVac GmbH Stabilisierte mRNA mit erhöhtem G/C-Gehalt und optimierter Codon Usage für die Gentherapie
HUE043492T2 (hu) 2005-08-23 2019-08-28 Univ Pennsylvania Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására
MX2011004859A (es) 2008-11-07 2011-08-03 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
TR201811076T4 (tr) 2009-06-10 2018-08-27 Arbutus Biopharma Corp Geliştirilmiş lipit formulasyonu.
PL3338765T3 (pl) 2009-12-01 2019-06-28 Translate Bio, Inc. Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
ES2762873T3 (es) 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
EP3865123A1 (en) 2012-03-29 2021-08-18 Translate Bio, Inc. Lipid-derived neutral nanoparticles
WO2013185069A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Pulmonary delivery of mrna to non-lung target cells
WO2015061467A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Lipid formulations for delivery of messenger rna
PT3083556T (pt) 2013-12-19 2020-03-05 Novartis Ag Lípidos e composições lipídicas para a entrega de agentes ativos
IL316479A (en) 2014-06-25 2024-12-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle compositions for nucleic acid delivery
WO2016004202A1 (en) 2014-07-02 2016-01-07 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
WO2016005004A1 (en) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
EP3229842B1 (en) 2014-12-08 2022-07-06 The Board of Regents of The University of Texas System Lipocationic polymers and uses thereof
WO2016118724A1 (en) 2015-01-21 2016-07-28 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
EP3247398A4 (en) 2015-01-23 2018-09-26 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
ES2949540T3 (es) 2015-06-19 2023-09-29 Massachusetts Inst Technology 2,5-piperazinadionas sustituidas con alquenilo y su uso en composiciones para suministrar un agente a un sujeto o una célula
HUE067372T2 (hu) 2015-06-29 2024-10-28 Acuitas Therapeutics Inc Lipidek és nanoszemcsés lipid formulázások nukleinsavak beadására
WO2017001570A2 (en) 2015-06-30 2017-01-05 Ethris Gmbh Atp-binding cassette family coding polyribonucleotides and formulations thereof
AU2016291228B2 (en) 2015-07-09 2020-04-09 Insmed Incorporated Compositions and methods for treating lung diseases and lung injury
CN108271360B (zh) 2015-09-14 2023-01-24 得克萨斯州大学系统董事会 亲脂阳离子树枝状聚合物及其用途
RS63986B1 (sr) 2015-10-28 2023-03-31 Acuitas Therapeutics Inc Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
EP3397613A1 (en) 2015-12-30 2018-11-07 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
SI3436077T1 (sl) 2016-03-30 2025-07-31 Intellia Therapeutics, Inc. Formulacije lipidnih nanodelcev za komponente crispr/cas
US20190167811A1 (en) * 2016-04-13 2019-06-06 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
AU2017267634C1 (en) 2016-05-16 2022-05-26 The Board Of Regents Of The University Of Texas System Cationic sulfonamide amino lipids and amphiphilic zwitterionic amino lipids
US20170354672A1 (en) 2016-05-16 2017-12-14 The Board Of Regents Of The University Of Texas System Compositions for the delivery of trna as nanoparticles and methods of use therewith
PT3463483T (pt) 2016-05-27 2024-03-04 Transcriptx Inc Tratamento da discinesia ciliar primária com rna do mensageiro sintético
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
EP3810148A4 (en) 2018-06-19 2022-06-08 The Board Of Regents Of The University Of Texas System LIPIDAN NOPARTICLE COMPOSITIONS FOR DELIVERY OF MRNA AND LONG NUCLEIC ACIDS
EP3846822A4 (en) 2018-09-04 2022-07-06 The Board Of Regents Of The University Of Texas System COMPOSITIONS AND METHODS FOR ORGAN-SPECIFIC DELIVERY OF NUCLEIC ACIDS
GB2592504B (en) * 2018-09-04 2023-02-15 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
EP3883592A1 (en) 2018-11-21 2021-09-29 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
CN113412273B (zh) 2019-01-07 2025-08-22 川斯勒佰尔公司 用于治疗原发性纤毛运动障碍的组合物和方法
US12521355B2 (en) * 2019-07-12 2026-01-13 Duke University Nanoparticle systems for targeted delivery of CRISPR/Cas13 and methods of using same
WO2021155323A1 (en) 2020-01-31 2021-08-05 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for preventing and treating coronavirus infection-sars-cov-2 vaccines
WO2021173840A1 (en) * 2020-02-25 2021-09-02 Translate Bio, Inc. Improved processes of preparing mrna-loaded lipid nanoparticles
US12618067B2 (en) 2020-04-09 2026-05-05 Technische Universität München Targeted delivery of an inhibitor of miR-21 to macrophages for the treatment of pulmonary fibrosis
US20240293317A1 (en) 2020-04-20 2024-09-05 Board Of Regents, The University Of Texas System Lipid nanoparticle (lnp) delivery systems and uses thereof
CA3176844A1 (en) * 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
EP4146680A1 (en) 2020-05-07 2023-03-15 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
JP2023538260A (ja) 2020-08-06 2023-09-07 モダーナティエックス・インコーポレイテッド ペイロード分子を気道上皮に送達するための組成物
AU2021327782A1 (en) * 2020-08-21 2023-03-23 The Board Of Regents Of The University Of Texas System Functional ionizable phospholipids
CN117157101A (zh) 2021-02-08 2023-12-01 德克萨斯大学系统董事会 不饱和的树枝状聚合物组合物、有关的制剂、及其使用方法
US20240123087A1 (en) 2021-03-19 2024-04-18 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204053A1 (en) 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
MX2023011104A (es) 2021-03-22 2024-03-01 Recode Therapeutics Inc Composiciones de polinucleotidos, formulaciones relacionadas y metodos de uso de las mismas.
WO2022204219A1 (en) 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Compositions and methods for targeted delivery to cells
MX2023011230A (es) 2021-03-23 2023-12-06 Recode Therapeutics Inc Composiciones y metodos para administracion sistemica dirigida a celulas.
KR20230175196A (ko) 2021-03-23 2023-12-29 리코드 테라퓨틱스, 인크. 폴리뉴클레오티드 조성물, 관련된 제제, 및 이의 사용 방법
CA3213308A1 (en) 2021-04-05 2022-10-13 Daniel J. Siegwart Compositions, methods and uses for treating cystic fibrosis and related disorders
WO2022226344A1 (en) 2021-04-22 2022-10-27 The Board Of Regents Of The University Of Texas System All-in-one dendrimer-based lipid nanoparticles enable precise hdr-mediated gene editing in vivo
EP4352240A4 (en) 2021-06-09 2025-07-09 Recode Therapeutics Inc POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS AND METHODS OF USE THEREOF

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