KR20240005692A - 세포로의 표적화 전달을 위한 조성물 및 방법 - Google Patents

세포로의 표적화 전달을 위한 조성물 및 방법 Download PDF

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Publication number
KR20240005692A
KR20240005692A KR1020237035962A KR20237035962A KR20240005692A KR 20240005692 A KR20240005692 A KR 20240005692A KR 1020237035962 A KR1020237035962 A KR 1020237035962A KR 20237035962 A KR20237035962 A KR 20237035962A KR 20240005692 A KR20240005692 A KR 20240005692A
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South Korea
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lipid
cells
lung
subject
sort
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Korean (ko)
Inventor
미르코 헨니그
블라디미르 카리토노프
브랜든 우스트만
잭슨 에비
룸파 바타차르지
다니엘 시그와트
Original Assignee
리코드 테라퓨틱스, 인크.
더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템
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Publication of KR20240005692A publication Critical patent/KR20240005692A/ko
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KR1020237035962A 2021-03-22 2022-03-22 세포로의 표적화 전달을 위한 조성물 및 방법 Pending KR20240005692A (ko)

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

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KR20240005692A true KR20240005692A (ko) 2024-01-12

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US (10) US20240207178A1 (cg-RX-API-DMAC7.html)
EP (1) EP4312987A4 (cg-RX-API-DMAC7.html)
JP (1) JP2024511463A (cg-RX-API-DMAC7.html)
KR (1) KR20240005692A (cg-RX-API-DMAC7.html)
AU (1) AU2022246065A1 (cg-RX-API-DMAC7.html)
BR (1) BR112023019229A2 (cg-RX-API-DMAC7.html)
CA (1) CA3213299A1 (cg-RX-API-DMAC7.html)
IL (1) IL305782A (cg-RX-API-DMAC7.html)
MX (1) MX2023011227A (cg-RX-API-DMAC7.html)
WO (1) WO2022204219A1 (cg-RX-API-DMAC7.html)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025150930A1 (ko) 2024-01-12 2025-07-17 주식회사 엘지에너지솔루션 권취기

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102764250B1 (ko) * 2015-09-14 2025-02-07 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 지질양이온성 덴드리머 및 이의 용도
WO2022204043A1 (en) * 2021-03-22 2022-09-29 The Board Of Regents Of The University Of Texas System Compositions and methods for targeted delivery to cells
MX2023011227A (es) 2021-03-22 2023-12-06 Recode Therapeutics Inc Composiciones y metodos para administracion dirigida a celulas.
WO2022204053A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
IL305958A (en) * 2021-03-22 2023-11-01 Recode Therapeutics Inc Polynucleotide preparations, related formulations and methods of using them
KR20240024041A (ko) * 2021-03-23 2024-02-23 리코드 테라퓨틱스, 인크. 세포로의 표적화 전신 전달을 위한 조성물 및 방법
MX2023011243A (es) 2021-03-23 2023-12-14 Recode Therapeutics Inc Composiciones de polinucleotidos, formulaciones relacionadas y metodos de uso de los mismos.
JP2024516164A (ja) * 2021-04-22 2024-04-12 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム 正確なインビボｈｄｒ介在性遺伝子編集を可能にするオールインワンデンドリマーベース脂質ナノ粒子
IL321026A (en) 2022-12-08 2025-07-01 Recode Therapeutics Inc Nanoparticle formulations and their uses
EP4385523A1 (en) * 2022-12-14 2024-06-19 Beijing Jitai Pharmaceutical Technology Co., Ltd. Lipid-based topical injection formulations
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
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
EP4719352A1 (en) 2023-05-31 2026-04-08 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
AR134146A1 (es) * 2023-10-24 2025-12-03 Green Cross Corp Lípidos ionizables y usos de los mismos
WO2025117816A1 (en) * 2023-12-01 2025-06-05 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
US12553042B2 (en) 2023-12-01 2026-02-17 Recode Therapeutics, Inc. Method for quantifying an amount of capped messenger RNA
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
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
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
WO2025240355A1 (en) * 2024-05-14 2025-11-20 Recode Therapeutics, Inc. Lipid nanoparticles for extrahepatic delivery

Family Cites Families (63)

* 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
JP4335310B2 (ja) * 1995-06-07 2009-09-30 ザ ユニバーシティ オブ ブリティッシュ コロンビア 疎水性脂質−核酸複合中間体を通して調製される脂質−核酸粒子、及び遺伝子移送のための使用
EP0941066B1 (en) 1996-08-26 2003-10-29 Transgene S.A. Cationic lipid-nucleic acid complexes
US5962429A (en) 1996-11-22 1999-10-05 University Of Iowa Complexes of adenovirus with cationic molecules
ATE291925T1 (de) 2001-06-05 2005-04-15 Curevac Gmbh Stabilisierte mrna mit erhöhtem g/c-gehalt und optimierter codon usage für die gentherapie
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
SI2506857T1 (en) 2009-12-01 2018-08-31 Translate Bio, Inc. Delivery of mRNA for the enrichment of proteins and enzymes in human genetic diseases
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
HK1206644A1 (en) 2012-03-29 2016-01-15 Shire Human Genetic Therapies, Inc. Lipid-derived neutral nanoparticles
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
CA2953341C (en) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
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
AU2015360794B2 (en) 2014-12-08 2021-07-08 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
RS64331B1 (sr) 2015-06-19 2023-08-31 Massachusetts Inst Technology Alkenil supstituisani 2,5-piperazindioni i njihova primena u sastavima za isporuku agensa u organizam ili ćeliju subjekta
HUE067372T2 (hu) 2015-06-29 2024-10-28 Acuitas Therapeutics Inc Lipidek és nanoszemcsés lipid formulázások nukleinsavak beadására
CA2990883A1 (en) 2015-06-30 2017-01-05 Ethris Gmbh Atp-binding cassette family coding polyribonucleotides and formulations thereof
ES2898340T3 (es) 2015-07-09 2022-03-07 Insmed Inc Composiciones y métodos para tratar neumopatías y lesión pulmonar
KR102764250B1 (ko) 2015-09-14 2025-02-07 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 지질양이온성 덴드리머 및 이의 용도
CN108368028B (zh) 2015-10-28 2021-09-03 爱康泰生治疗公司 用于递送核酸的新型脂质和脂质纳米颗粒制剂
US12678515B2 (en) 2015-12-30 2026-07-14 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
ES3030658T3 (en) 2016-03-30 2025-07-01 Intellia Therapeutics Inc Lipid nanoparticle formulations for crispr/cas components
EP3442590A2 (en) * 2016-04-13 2019-02-20 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
PL3458074T3 (pl) 2016-05-16 2024-11-12 Board Of Regents Of The University Of Texas System KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA
CN114656378A (zh) 2016-05-16 2022-06-24 得克萨斯州大学系统董事会 阳离子磺酰胺氨基脂质和两亲性两性离子氨基脂质
CA3025626A1 (en) 2016-05-27 2017-11-30 Transcriptx, Inc. Treatment of primary ciliary dyskinesia with synthetic messenger rna
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
IL270631B2 (en) 2017-05-16 2024-03-01 Translate Bio Inc Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
GB2596224B (en) 2018-06-19 2022-09-07 Univ Texas Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
US20200040560A1 (en) * 2018-08-01 2020-02-06 Anthony Branton Apparatus for an adjustable p-trap
GB2606038B (en) 2018-09-04 2023-05-03 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
GB2600800B (en) * 2018-09-04 2023-08-16 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
WO2020146344A1 (en) 2019-01-07 2020-07-16 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
EP3999117A4 (en) * 2019-07-12 2023-07-26 Duke University NANOPARTICLE SYSTEMS FOR TARGETED CRISPR/CAS13 DELIVERY AND METHODS OF USE THEREOF
TW202204380A (zh) 2020-01-31 2022-02-01 美商詹森藥物公司 用於預防及治療冠狀病毒感染之組合物及方法－sars-cov-2疫苗
CN115427021A (zh) * 2020-02-25 2022-12-02 翻译生物公司 制备负载mrna的脂质纳米颗粒的改进方法
MX2022012544A (es) 2020-04-09 2022-11-07 Univ Muenchen Tech Liberacion dirigida de un inhibidor de mir-21 a macrofagos para el tratamiento de la fibrosis pulmonar.
US20240293317A1 (en) 2020-04-20 2024-09-05 Board Of Regents, The University Of Texas System Lipid nanoparticle (lnp) delivery systems and uses thereof
WO2021222801A2 (en) * 2020-05-01 2021-11-04 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
WO2021226463A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
WO2022032154A2 (en) 2020-08-06 2022-02-10 Modernatx, Inc. Compositions for the delivery of payload molecules to airway epithelium
US20230320994A1 (en) * 2020-08-21 2023-10-12 The Board Of Regents Of The University Of Texas System Functional ionizable phospholipids
US20240123076A1 (en) 2021-02-08 2024-04-18 The Board Of Regents Of The University Of Texas System Unsaturated dendrimers compositions, related formulations, and methods of use thereof
EP4308703A4 (en) 2021-03-19 2025-06-04 Recode Therapeutics, Inc. Polynucleotide compositions, associated formulations and methods of using the same
MX2023011227A (es) 2021-03-22 2023-12-06 Recode Therapeutics Inc Composiciones y metodos para administracion dirigida a celulas.
WO2022204053A1 (en) 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
IL305958A (en) 2021-03-22 2023-11-01 Recode Therapeutics Inc Polynucleotide preparations, related formulations and methods of using them
MX2023011243A (es) 2021-03-23 2023-12-14 Recode Therapeutics Inc Composiciones de polinucleotidos, formulaciones relacionadas y metodos de uso de los mismos.
KR20240024041A (ko) 2021-03-23 2024-02-23 리코드 테라퓨틱스, 인크. 세포로의 표적화 전신 전달을 위한 조성물 및 방법
AU2022253004A1 (en) 2021-04-05 2023-10-12 Recode Therapeutics, Inc. Compositions, methods and uses for treating cystic fibrosis and related disorders
JP2024516164A (ja) 2021-04-22 2024-04-12 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム 正確なインビボｈｄｒ介在性遺伝子編集を可能にするオールインワンデンドリマーベース脂質ナノ粒子
JP2024520834A (ja) 2021-06-09 2024-05-24 レコード・セラピューティクス・インコーポレイテッド ポリヌクレオチド組成物、関連する製剤及びその使用方法
IL321026A (en) * 2022-12-08 2025-07-01 Recode Therapeutics Inc Nanoparticle formulations and their uses
US12364773B2 (en) * 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025150930A1 (ko) 2024-01-12 2025-07-17 주식회사 엘지에너지솔루션 권취기

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