AU2021261423A1 - Compositions for treating cancer with KRAS mutations and uses thereof - Google Patents

Compositions for treating cancer with KRAS mutations and uses thereof Download PDF

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Publication number
AU2021261423A1
AU2021261423A1 AU2021261423A AU2021261423A AU2021261423A1 AU 2021261423 A1 AU2021261423 A1 AU 2021261423A1 AU 2021261423 A AU2021261423 A AU 2021261423A AU 2021261423 A AU2021261423 A AU 2021261423A AU 2021261423 A1 AU2021261423 A1 AU 2021261423A1
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cell
genome
seq
peptide
penetrating peptide
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AU2021261423A
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AU2021261423A8 (en
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Neil P. Desai
Gilles Divita
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Aadigen LLC
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Aadigen LLC
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Publication of AU2021261423A1 publication Critical patent/AU2021261423A1/en
Publication of AU2021261423A8 publication Critical patent/AU2021261423A8/en
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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
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    • A61K47/6921Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
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    • A61K47/6929Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
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    • 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
    • A61K48/0041Medicinal 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 the non-active part being polymeric
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AU2021261423A 2020-04-24 2021-04-23 Compositions for treating cancer with KRAS mutations and uses thereof Pending AU2021261423A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
FR2004126 2020-04-24
FRFR2004126 2020-04-24
PCT/US2021/028995 WO2021217100A1 (en) 2020-04-24 2021-04-23 Compositions for treating cancer with kras mutations and uses thereof

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AU2021261423A1 true AU2021261423A1 (en) 2022-12-08
AU2021261423A8 AU2021261423A8 (en) 2022-12-22

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Families Citing this family (9)

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Publication number Priority date Publication date Assignee Title
EP3955967A4 (en) 2019-04-17 2023-06-07 Aadigen, LLC PEPTIDES AND NANOPARTICLES FOR INTRACELLULAR DELIVERY OF MOLECULES
WO2023081893A1 (en) * 2021-11-08 2023-05-11 Entrada Therapeutics, Inc. Intracellular targeting of oligonucleotides
EP4448745A4 (en) * 2021-12-15 2025-12-24 Univ Indiana Trustees CELLULAR TARGETING COMPOSITIONS AND METHODS
JP2023176123A (ja) * 2022-05-31 2023-12-13 株式会社 光バイオ ウイルスベクター及びそれを含むがん細胞増殖抑制剤
CN119384498A (zh) * 2022-06-16 2025-01-28 因特利亚治疗公司 用于对细胞进行遗传修饰的方法和组合物
WO2024187174A2 (en) 2023-03-09 2024-09-12 Aadigen, Llc Compositions for treating cancer with kras mutations and uses thereof
WO2025059215A1 (en) * 2023-09-12 2025-03-20 Aadigen, Llc Methods and compositions for treating or preventing cancer
WO2025193628A2 (en) 2024-03-09 2025-09-18 Aadigen, Llc Compositions for treating cancer with kras mutations and uses thereof
CN120005043A (zh) * 2025-02-17 2025-05-16 山东第一医科大学附属肿瘤医院(山东省肿瘤防治研究院、山东省肿瘤医院) 一种用于抑制PRDX1 O-GlcNAc修饰的短肽及其应用

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Publication number Priority date Publication date Assignee Title
KR100666099B1 (ko) * 2004-04-27 2007-01-10 국립암센터 K―ras 올리고뉴클레오티드 마이크로어레이 및 이를이용하여 K―ras 유전자의 돌연변이를 검사하는 방법
US8889642B2 (en) * 2012-04-19 2014-11-18 Silenseed Ltd. Methods and compositions for RNAi-based cancer treatment
WO2016049024A2 (en) * 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
CA3025523A1 (en) * 2016-05-27 2017-11-30 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of genome-editing molecules
KR20190039115A (ko) * 2016-07-05 2019-04-10 더 존스 홉킨스 유니버시티 암을 치료하기 위한 crispr/cas9-기반 조성물 및 방법
KR101997116B1 (ko) * 2016-10-14 2019-07-05 연세대학교 산학협력단 Kras 유전자에 상보적인 가이드 rna 및 이의 용도
KR101796036B1 (ko) * 2016-12-29 2017-11-10 주식회사 무진메디 Cas9 단백질, KRAS 유전자의 발현을 억제하는 가이드 RNA 및 양이온성 폴리머의 복합체가 봉입된 나노 리포좀 전달체 조성물 또는 이를 함유하는 KRAS 유전자 변이에 따른 항암제 저항성 대장암 치료제
AU2018313940A1 (en) * 2017-08-10 2020-03-05 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of virus
AU2018352221B2 (en) * 2017-10-16 2024-04-18 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of mRNA
CN112533627A (zh) * 2018-03-27 2021-03-19 G+Flas 生命科学公司 序列特异性体内细胞靶向

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EP4139454A4 (en) 2025-07-30
AU2021261423A8 (en) 2022-12-22
US20230167437A1 (en) 2023-06-01
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CA3181170A1 (en) 2021-10-28
WO2021217100A1 (en) 2021-10-28

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Free format text: IN VOL 36 , NO 49 , PAGE(S) 6712 UNDER THE HEADING PCT APPLICATIONS THAT HAVE ENTERED THE NATIONAL PHASE - NAME INDEX UNDER THE NAME AADIGEN, LLC, APPLICATION NO. 2021261423, UNDER INID (54) CORRECT THE TITLE TO READ COMPOSITIONS FOR TREATING CANCER WITH KRAS MUTATIONS AND USES THEREOF