US 12,240,824 B2
Vitamin cationic lipids
Yi Zhang, Lexington, MA (US); Shrirang Karve, Lexington, MA (US); Saswata Karmakar, Lexington, MA (US); Frank DeRosa, Lexington, MA (US); and Michael Heartlein, Lexington, MA (US)
Assigned to TRANSLATE BIO, INC., Waltham, MA (US)
Appl. No. 17/058,574
Filed by Translate Bio, Inc., Lexington, MA (US)
PCT Filed May 29, 2019, PCT No. PCT/US2019/034461
§ 371(c)(1), (2) Date Nov. 24, 2020,
PCT Pub. No. WO2019/232095, PCT Pub. Date Dec. 5, 2019.
Claims priority of provisional application 62/807,671, filed on Feb. 19, 2019.
Claims priority of provisional application 62/807,672, filed on Feb. 19, 2019.
Claims priority of provisional application 62/807,673, filed on Feb. 19, 2019.
Claims priority of provisional application 62/677,818, filed on May 30, 2018.
Claims priority of provisional application 62/677,851, filed on May 30, 2018.
Claims priority of provisional application 62/677,855, filed on May 30, 2018.
Claims priority of provisional application 62/677,828, filed on May 30, 2018.
Prior Publication US 2021/0206739 A1, Jul. 8, 2021
Int. Cl. C07C 401/00 (2006.01); A61K 9/127 (2006.01); C07C 225/14 (2006.01); C07C 229/12 (2006.01); C07C 323/25 (2006.01); C07C 323/27 (2006.01); C07C 403/12 (2006.01); C07D 311/20 (2006.01); A61K 31/592 (2006.01); A61K 31/593 (2006.01)
CPC C07D 311/20 (2013.01) [C07C 225/14 (2013.01); C07C 229/12 (2013.01); C07C 323/25 (2013.01); C07C 323/27 (2013.01); C07C 403/12 (2013.01); C07C 2601/14 (2017.05); C07C 2601/16 (2017.05); C07C 2602/24 (2017.05)] 17 Claims
 
1. A liposome encapsulating an mRNA encoding a peptide or polypeptide, wherein the liposome comprises
one or more cationic lipids, and
optionally one or more non-cationic lipids, optionally one or more cholesterol-based lipids and optionally one or more PEG-modified lipids; and
wherein the liposome comprises at least one cationic lipid having the structure according to Formula D-A:

OG Complex Work Unit Chemistry
wherein
custom character represents a single or double bond;
X1 is NH2, guanidine, amidine, a mono- or dialkylamine, 5- to 6-membered heterocycloalkyl, 5- to 6-membered nitrogen-containing heteroaryl, or

OG Complex Work Unit Chemistry
R3a and R3b are each independently C1-C30-alkyl, C2-C30-alkenyl, or C2-C30-alkynyl;
n is independently an integer having a value between 1 and 6;
X2 is O or S;
Z is O or a covalent bond;
R1 is C1-C30-alkylene or C2-C30-alkenylene; and
R2 is H or C1-C4-alkyl.