HRP20191400T1 - Peptidi i nanočestice za unutarstaničnu isporuku molekula - Google Patents

Peptidi i nanočestice za unutarstaničnu isporuku molekula Download PDF

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HRP20191400T1
HRP20191400T1 HRP20191400T HRP20191400T1 HR P20191400 T1 HRP20191400 T1 HR P20191400T1 HR P20191400 T HRP20191400 T HR P20191400T HR P20191400 T1 HRP20191400 T1 HR P20191400T1
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peptide
nanoparticle
sec
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optionally
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Gilles Divita
Neil Desai
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Aadigen Llc
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Claims (21)

1. Peptid koji se ne pojavljuje u prirodi i koji sadrži niz aminokiselina X1KWRSX2X3X4RWRLWRX5X6X7X8SR (ID BR. SEK.: 1), pri čemu je a) X1 bilo koja aminokiselina ili nijedna i pri čemu je X2-X8 bilo koja aminokiselina; ili b) X1 je βA, S, ili nijedna, X2 je A ili V, X3 je G ili L, X4 je W ili Y, X5 je V ili S, X6 je R, V ili A, X7 je S ili L, a X8 je W ili Y, opcijski pri čemu je peptid dugačak 19 ili 20 aminokiselina.
2. Peptid patentnog zahtjeva 1, pri čemu peptid sadrži sekvencu aminokiselina od: a) KWRSAGWRWRLWRVRSWSR (ID BR. SEK.: 2), b) KWRSALYRWRLWRSRSWSR (ID BR. SEK.: 3) ili c) KWRSALYRWRLWRSALYSR (ID BR. SEK.: 4).
3. Peptid patentnog zahtjeva 1 ili 2, nadalje sadrži a) jedan ili više dijelova kovalentno vezanih na N-terminalni kraj peptida, pri čemu je jedan ili više dijelova odabrano iz grupe koja se sastoji od acetilne skupine, stearilne skupine, masne kiseline, kolesterola, polietilen glikola, signala nuklearne lokalizacije, nuklearnog izvoznog signala, antitijela ili njegovog fragmenta antitijela, peptida, polisaharida i ciljne molekule; i/ili b) jedan ili više dijelova kovalentno vezanih na C-terminalni kraj peptida, pri čemu je jedan ili više dijelova odabrano iz grupe koja se sastoji od cisteamidne skupine, cistina, tiola, amida, nitrilotriacetne kiseline, karboksilne skupine, linearne ili razgranata C1-C6 alkilne skupine, primarnog ili sekundarnog amina, oksidnod derivata, lipida, fosfolipida, masne kiseline, kolesterola, polietilen glikola, signala nuklearne lokalizacije, nuklearnog izvoznog signala, antitijela ili njegovog fragmenta antitijela, peptida, polisaharida i ciljne molekule, naznačen time da peptid sadrži acetilnu skupinu kovalentno vezanu za njegov N-terminalni kraj i/ili cisteamidnu skupinu vezanu kovalentnom vezom na njegov C-terminalni kraj.
4. Peptid bilo kojeg patentnog zahtjeva od 1-3, pri čemu peptid sadrži odvojena po tri ili šest ostataka koji su vezani ugljikovodičnom vezom, pri čemu opcijski peptid sadrži aminokiselinski slijed: aa) KWRSsAGWRsWRLWRVRSWSR (ID BR. SEK.: 7), ab) KWRsSAGWRWRsLWRVRSWSR (ID BR. SEK.: 8), ac) KWRSAGWRsWRLWRVRsSWSR (ID BR. SEK.: 9), ba) KWRSsALYRsWRLWRSRSWSR (ID BR. SEK.: 10), bb) KWRsSALYRWRsLWRSRSWSR (ID BR. SEK.: 11), bc) KWRSALYRsWRLWRSRsSWSR (ID BR. SEK.: 12), bd) KWRSALYRWRsLWRSsRSWSR (ID BR. SEK.: 13), be) KWRSALYRWRLWRSsRSWSsR (ID BR. SEK.: 14), ca) KWRsSALYRWRsLWRSALYSR (ID BR. SEK.: 15), cb) KWRSsALYRsWRLWRSALYSR (ID BR. SEK.: 16), cc) KWRSALYRWRsLWRSsALYSR (ID BR. SEK.: 17) ili cd) KWRSALYRWRLWRSsALYSsR (ID BR. SEK.: 18), naznačeni time da su ostaci označeni donjim indeksom „S“ dva ostatka vezana ugljikovodičnom vezom.
5. Kompleksni spoj, koji sadrži peptid bilo kojeg patentnog zahtjeva 1-4 i molekulu tereta.
6. Nanočestica, koja sadrži peptid bilo kojeg patentnog zahtjeva 1-4 i molekulu tereta.
7. Kompleksni spoj prema patentnom zahtjevu 5 ili nanočestica prema patentnom zahtjevu 6, pri čemu je molekula tereta a) nukleinska kiselina; b) siRNK, miRNK, DNK plazmid ili analogna njima; c) oligonukleotid; d) jednolančana RNK, jednolančana DNK, dvolančana RNK, dvolančana DNK, ili njihov derivat, pri čemu je opcijski teret i) duljine oko 2 do oko 40 nukleotida; ii) do 100 nukleotida; ili iii) više od 100 nukleotida.
8. Nanočestica prema patentnom zahtjevu 6 ili 7, pri čemu je a) molarni omjer molekule tereta i peptida u nanočesticama je od oko 1:1 do oko 1:80, pri čemu je molarni omjer molekule tereta i peptida u nanočesticama opcijski od oko 1:5 do oko 1:40; b) molekula tereta je složena sa skupnom molekulom kako bi formirala jezgru nanočestice, pri čemu se skupna molekula opcijski odabire iz skupine koja se sastoji od peptida, proteina, antitijela, lipida, fosfolipida, polimera, aptamera, nanočestica, liposoma, dendrimera, polimerosoma, virusnog vektora i micele; c) nanočestica nadalje sadrži površinski sloj, pri čemu opcijski površinski sloj obuhvaća peptide prema bilo kojem od patentnih zahtjeva 1-4; d) nanočestica sadrži ciljani dio na površini, pri čemu je peptid opcijski kovalentno vezan na ciljnu skupinu; e) prosječni promjer nanočestice je od oko 10 nm do oko 300 nm, pri čemu je prosječni promjer nanočestice opcijski od oko 50 nm do oko 200 nm ili od oko 80 nm do oko 140 nm; i/ili f) apsolutna vrijednost zeta potencijala je manja od oko 30 mV, pri čemu je apsolutna vrijednost zeta potencijala opcijski manja od oko 10 mV.
9. Nanočestica prema patentnom zahtjevu 8, pri čemu nanočestica nadalje sadrži površinski sloj i intermedijarni sloj, pri čemu intermedijarni sloj opcijski sadrži peptid prema bilo kojem od patentnih zahtjeva 1-4.
10. Farmaceutski pripravak, koji sadrži kompleksni spoj prema patentnom zahtjevu 5 ili 7, ili nanočesticu prema bilo kojem od patentnih zahtjeva 6-9, i farmaceutski prihvatljiv nosač.
11. Farmaceutski spoj prema patentnom zahtjevu 10, pri čemu je a) farmaceutski spoj formuliran je za intravensku, intratumornu, intraarterijsku, topičku, intraokularnu, oftalmičku, intrakranijalnu, intratekalnu, intravezikularnu, intradermalnu, subkutanu, intramuskularnu, intranazalnu, intratrahealnu, pulmonarnu, intrakavitalnu ili oralnu primjenu; b) farmaceutski prihvatljiv nosač je šećer ili protein, pri čemu se opcijski odabire šećer iz skupine koja se sastoji od saharoze, glukoze, manitola i njihove kombinacije, a prisutan je u farmaceutskom pripravku u koncentraciji od oko 5 % do oko 20 %; i/ili protein albumin; i/ili c) je farmaceutski spoj liofiliziran.
12. Postupak pripreme nanočestice prema bilo kojem od patentnih zahtjeva 6-9, koji obuhvaća: a) kombiniranje pripravka koji sadrži peptid prema bilo kojem od patentnih zahtjeva 1-4 sa sastavom koji obuhvaća molekulu tereta za formiranje mješavine; i b) inkubaciju mješavine za formiranje nanočestice.
13. Farmaceutski spoj prema patentnom zahtjevu 10 ili 11 za uporabu kao lijek.
14. Farmaceutski spoj prema patentnom zahtevu 10 ili 11 za uporabu u liječenju bolesti, koji je odabran iz skupine koja se sastoji od karcinoma, dijabetesa, upalnih bolesti, fibroze, virusnih upalnih bolesti, nasljednih bolesti te starenja i degenerativnih bolesti.
15. Metoda isporuke molekule u stanicu, koji obuhvaća kontakt stanice s kompleksnim spojem prema patentnom zahtjevu 5 ili 7, ili nanočesticom bilo kojeg od patentnih zahtjeva 6-9, pri čemu kompleksni spoj ili nanočestica sadrži molekulu i pri čemu se dodirivanje stanice s kompleksnim spojem ili nanočesticom provodi ex vivo ili in vitro.
16. Metoda prema patentnom zahtjevu 15, pri čemu je stanica granulocit, mastocit, monocit, dendritička stanica, B stanica, T stanica ili stanica prirodni ubojica.
17. Metoda prema patentnom zahtjevu 16, pri čemu je a) molekula je plazmid koji kodira himerni antigenski receptor, koji sadrži izvanstaničnu domenu vezanja antigena, koja se specifično veže na ciljni antigen, transmembransku domenu i unutarstaničnu signalnu domenu, opcijski je ciljni antigen antigen povezan na karcinom i/ili i) kompleksni spoj ili nanočestica nadalje sadrži siRNK; ili ii) metoda nadalje obuhvaća kontakt stanice s kompleksnim spojem prema patentnom zahtjevu 5 ili 7 ili nanočesticu prema bilo kojem od patentnih zahtjeva 6-9, pri čemu kompleksni spoj ili nanočestica sadrži siRNK; ili b) ili je molekula siRNK.
18. Modificirana stanica koja sadrži kompleksni spoj prema patentnim zahtjevima 5 ili 7 ili nanočesticu prema patentnim zahtjevima 6-9 pripremljena u skladu s metodom bilo kojeg drugog patentnog zahtjeva 15-17 za upotrebu kao lijek, opcijski, pri čemu se modificirana stanica daje intravenski, intraarterijski, intraperitonealno, intravezikularno, subkutano, intratekalno, intrapulmonalno, intramuskularno, intratrahealno, intraokularno, transderrnalno, oralno ili inhalacijskim putem.
19. Modificirana stanica koja sadrži kompleksni spoj prema patentnom zahtjevu 5 ili 7 ili nanočesticu prema patentnim zahtjevima 6-9 pripremljena u skladu s metodom bilo kojeg drugog patentnog zahtjeva 15-17 za upotrebu u liječenju karcinoma, opcijski, pri čemu se modificirana stanica daje intravenski, intraarterijski, intraperitonealno, intravezikularno, subkutano, intratekalno, intrapulmonalno, intramuskularno, intratrahealno, intraokularno, transderrnalno, oralno ili inhalacijskim putem.
20. Postupak stabilizacije nukleinske kiseline koji obuhvaća: a) kombiniranje spoja koji sadrži peptid bilo kojeg patentnog zahtjeva 1-4 sa spojem koji obuhvaća nukleinsku kiselinu za izradu mješavine; i b) inkubaciju mješavine za izradu kompleksnog spoja koji sadrži nukleinsku kiselinu.
21. Komplet koji sadrži spoj koji sadrži peptid prema bilo kojem od patentnih zahtjeva 1-4 i upute za pripremu kompleksnog spoja prema patentnom zahtjevu 5 ili 7 i/ili nanočesticu prema bilo kojem od patentnih zahtjeva 6-9, pri čemu komplet opcijski sadrži spoj koji sadrži skupnu molekulu odabranu iz skupine koja se sastoji od peptida, proteina, antitijela, lipida, fosfolipida, polimera, aptamera, nanočestice, liposoma, dendrimera, polimerosoma, virusnog vektora i micele.
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Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014053880A1 (en) 2012-10-04 2014-04-10 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
WO2014053879A1 (en) 2012-10-04 2014-04-10 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
PL3237436T3 (pl) 2014-12-24 2020-03-31 Aadigen, Llc Peptydy i nanocząsteczki do wewnątrzkomórkowego dostarczania cząsteczek
AU2017268842B2 (en) * 2016-05-27 2022-08-11 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of genome-editing molecules
EP3525804A4 (en) * 2016-10-11 2020-09-09 Minerva Biotechnologies Corporation HUMANIZED ANTI-MUC1 * ANTIBODIES AND USE OF THE CLIVING ENZYME
ES2746856T3 (es) 2016-12-09 2020-03-09 Onk Therapeutics Ltd Células asesinas naturales manipuladas y usos de las mismas
WO2019032917A1 (en) * 2017-08-10 2019-02-14 Aadigen, Llc PEPTIDES AND NANOPARTICLES FOR INTRACELLULAR DELIVERY OF VIRUSES
CN109557067B (zh) * 2017-09-26 2021-07-20 中国科学院烟台海岸带研究所 一种核—卫星结构金纳米颗粒及其制备方法
JP7395483B2 (ja) * 2017-10-16 2023-12-11 アーディジェン， エルエルシー ｍＲＮＡの細胞内送達のためのペプチドおよびナノ粒子
CN108424437B (zh) * 2018-02-08 2021-06-22 青岛农业大学 一种递送分子及纳米颗粒、制备方法、用途
EP3833676A1 (en) * 2018-08-07 2021-06-16 ETH Zürich Polypeptides self-assembling into nanoparticles
JP2022529784A (ja) * 2019-04-17 2022-06-24 アーディジェン， エルエルシー 分子を細胞内送達するためのペプチドおよびナノ粒子
KR102146392B1 (ko) * 2019-06-11 2020-08-21 한림대학교 산학협력단 포스포글리세레이트 뮤테이즈 1 융합단백질 및 이를 포함하는 약학 조성물
CN110540992B (zh) * 2019-08-21 2021-02-09 武汉泽智生物医药有限公司 增强靶基因沉默效率的siRNA分子及其应用
MX2023001049A (es) 2020-07-24 2023-04-11 Aadigen Llc Composiciones y métodos para tratar infecciones virales.
CN113563429A (zh) * 2021-07-19 2021-10-29 天津大学 一种基于烷基化多肽的核酸递送***及制备方法与应用
WO2023215549A1 (en) * 2022-05-05 2023-11-09 Case Western Reserve University Immunostimulatory nanoparticle

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH08500681A (ja) 1992-08-25 1996-01-23 メルク パテント ゲゼルシャフト ミット ベシュレンクテル ハフトング ペーパーホワイトｐｄｌｃシステム
US7514530B2 (en) 2004-04-26 2009-04-07 Centre National De La Recherche Scientifique Peptide carrier for delivering siRNA into mammalian cells
GB0513096D0 (en) 2005-06-28 2005-08-03 Strathclyde Treatment of microbial infections
CA2632451C (en) 2005-12-06 2015-11-03 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
ATE490266T1 (de) 2005-12-06 2010-12-15 Centre Nat Rech Scient In zellen eindringende peptide als träger für moleküle
US7579318B2 (en) 2005-12-06 2009-08-25 Centre De La Recherche De La Scientifique Cell penetrating peptides for intracellular delivery of molecules
WO2008036929A2 (en) 2006-09-21 2008-03-27 Alnylam Pharmaceuticals, Inc. Complex for transferring an anionic substance into a cell
CA2725227A1 (en) 2008-05-06 2009-11-12 Asim Kumar Debnath Antiviral cell penetrating peptides
HRP20211788T1 (hr) 2008-08-26 2022-02-18 City Of Hope Postupak i pripravci za poboljšanje anti-tumorskog efektorskog funkcioniranja t stanica
WO2012137036A1 (en) 2011-04-04 2012-10-11 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
CN104080480A (zh) * 2012-01-01 2014-10-01 奇比艾企业有限公司 用于选择性递送治疗剂和诊断剂的endo180靶向微粒
WO2013150338A1 (en) * 2012-04-04 2013-10-10 Centre National De La Recherche Scientifique Stapled cell penetrating peptides for intracellular delivery of molecules
EP2836239A1 (en) 2012-04-11 2015-02-18 The United States of America, as Represented By the Secretary, Department of Health and Human Services Chimeric antigen receptors targeting b-cell maturation antigen
EP2849762A4 (en) 2012-05-16 2016-02-24 Aadigen Llc MULTI-TARGET MODULATION FOR TREATING FIBROSIS AND INFLAMMATORY DISORDERS
WO2014053881A1 (en) 2012-10-04 2014-04-10 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
WO2014053880A1 (en) 2012-10-04 2014-04-10 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
WO2014053879A1 (en) 2012-10-04 2014-04-10 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
WO2014053882A1 (en) 2012-10-04 2014-04-10 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
US20150329640A1 (en) 2012-12-20 2015-11-19 Bluebird Bio, Inc. Chimeric antigen receptors and immune cells targeting b cell malignancies
PT3300745T (pt) 2013-02-15 2019-11-27 Univ California Recetor de antigénio quimérico e métodos de utilização do mesmo
PL3237436T3 (pl) 2014-12-24 2020-03-31 Aadigen, Llc Peptydy i nanocząsteczki do wewnątrzkomórkowego dostarczania cząsteczek

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