Synthesis and bioactivity of readily hydrolysable novel cationic lipids for potential lung delivery application of mRNAs.


Journal

Chemistry and physics of lipids
ISSN: 1873-2941
Titre abrégé: Chem Phys Lipids
Pays: Ireland
ID NLM: 0067206

Informations de publication

Date de publication:
03 2022
Historique:
received: 20 08 2021
revised: 27 01 2022
accepted: 31 01 2022
pubmed: 6 2 2022
medline: 3 3 2022
entrez: 5 2 2022
Statut: ppublish

Résumé

Lipid nanoparticles (LNPs) mediated mRNA delivery has gained prominence due to the success of mRNA vaccines against Covid-19, without which it would not have been possible. However, there is little clinical validation of this technology for other mRNA-based therapeutic approaches. Systemic administration of LNPs predominantly targets the liver, but delivery to other organs remains a challenge. Local approaches remain a viable option for some disease indications, such as Cystic Fibrosis, where aerosolized delivery to airway epithelium is the preferred route of administration. With this in mind, novel cationic lipids (L1-L4) have been designed, synthesized and co-formulated with a proprietary ionizable lipid. These LNPs were further nebulized, along with baseline control DOTAP-based LNP (DOTAP

Identifiants

pubmed: 35122738
pii: S0009-3084(22)00006-8
doi: 10.1016/j.chemphyslip.2022.105178
pmc: PMC9749011
pii:
doi:

Substances chimiques

Lipid Nanoparticles 0
Liposomes 0
RNA, Messenger 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

105178

Informations de copyright

Copyright © 2022 Elsevier B.V. All rights reserved.

Références

Science. 1989 Sep 8;245(4922):1066-73
pubmed: 2475911
Mol Pharm. 2014 Jun 2;11(6):1808-22
pubmed: 24811243
J Control Release. 2012 Jan 10;157(1):141-8
pubmed: 21864597
J Phys Chem B. 2018 Feb 22;122(7):2073-2081
pubmed: 29332384
FASEB J. 1990 Jul;4(10):2718-25
pubmed: 1695593
Am J Respir Cell Mol Biol. 2002 Aug;27(2):151-9
pubmed: 12151306
Nat Nanotechnol. 2020 Apr;15(4):313-320
pubmed: 32251383
J Phys Chem C Nanomater Interfaces. 2012 Aug 30;116(34):18440-18450
pubmed: 22962627
Nat Biotechnol. 2010 Feb;28(2):172-6
pubmed: 20081866
Biochemistry. 2007 Sep 11;46(36):10365-72
pubmed: 17685634
Hum Gene Ther. 1996 Sep 10;7(14):1701-17
pubmed: 8886841
AAPS J. 2009 Sep;11(3):511-25
pubmed: 19593675
Int J Pharm. 2012 Jul 1;430(1-2):328-34
pubmed: 22525086
Neuron. 1992 May;8(5):821-9
pubmed: 1375035
ACS Nano. 2018 May 22;12(5):4787-4795
pubmed: 29614232
Mol Ther. 2010 Jul;18(7):1357-64
pubmed: 20461061
Int J Pharm. 2011 Jan 17;403(1-2):237-44
pubmed: 20974237
Proc Natl Acad Sci U S A. 2017 Mar 7;114(10):E1941-E1950
pubmed: 28202722
Nat Biomed Eng. 2021 Sep;5(9):1059-1068
pubmed: 34616046
Cancer Res. 2013 Apr 15;73(8):2412-7
pubmed: 23423979
J Med Chem. 2020 Nov 12;63(21):12992-13012
pubmed: 33119286
J Diabetes Sci Technol. 2017 Jan;11(1):148-156
pubmed: 27378794
Biochim Biophys Acta. 2001 Feb 9;1510(1-2):152-66
pubmed: 11342155
Mol Ther. 2013 Aug;21(8):1570-8
pubmed: 23799535
J Control Release. 2012 Jun 10;160(2):217-24
pubmed: 22079949
Angew Chem Int Ed Engl. 2012 Aug 20;51(34):8529-33
pubmed: 22782619
ACS Nano. 2013 Sep 24;7(9):7534-41
pubmed: 23909689
Langmuir. 2005 Oct 11;21(21):9426-31
pubmed: 16207017
Biochim Biophys Acta. 1990 Mar 30;1023(1):124-32
pubmed: 2317491
Mol Ther. 2018 Jun 6;26(6):1509-1519
pubmed: 29653760
Nanomedicine. 2019 Jun;18:31-43
pubmed: 30831275
Mol Ther. 2018 Aug 1;26(8):2034-2046
pubmed: 29910178
Gene Ther. 1999 Dec;6(12):1995-2004
pubmed: 10637451
J Control Release. 2010 Feb 25;142(1):138-45
pubmed: 19840823
Mol Ther. 2001 Mar;3(3):337-50
pubmed: 11273776
Adv Drug Deliv Rev. 2014 Aug;75:112-28
pubmed: 24907426
Nano Lett. 2017 Sep 13;17(9):5711-5718
pubmed: 28836442
Nat Genet. 1993 Feb;3(2):151-6
pubmed: 7684646
J Med Chem. 2004 Nov 18;47(24):6082-99
pubmed: 15537362
J Med Chem. 2006 Jan 12;49(1):349-57
pubmed: 16392819
Nanomaterials (Basel). 2018 Apr 24;8(5):
pubmed: 29695068
Sci Rep. 2016 Feb 17;6:20019
pubmed: 26883577
Naunyn Schmiedebergs Arch Pharmacol. 1984 Dec;328(2):111-8
pubmed: 6527701
Biochem Biophys Res Commun. 2005 Mar 4;328(1):57-62
pubmed: 15670750
Proc Natl Acad Sci U S A. 1996 Oct 15;93(21):11493-8
pubmed: 8876163
Proc Natl Acad Sci U S A. 1996 Oct 15;93(21):11454-9
pubmed: 8876156
Angew Chem Int Ed Engl. 2016 Oct 24;55(44):13719-13723
pubmed: 27699963

Auteurs

Yihua Pei (Y)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Yanjie Bao (Y)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Cristiano Sacchetti (C)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Juthamart Brady (J)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Kyra Gillard (K)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Hailong Yu (H)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Scott Roberts (S)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Kumar Rajappan (K)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA. Electronic address: kumar@arcturusrx.com.

Steven P Tanis (SP)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA. Electronic address: steve@arcturusrx.com.

Carlos G Perez-Garcia (CG)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Padmanabh Chivukula (P)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Priya P Karmali (PP)

Arcturus Therapeutics, 10628 Science Center Drive, Suite 250, San Diego, CA 92121, USA.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH