US 11,969,506 B2
Lipid nanoparticle formulation
Siddharth Patel, Corvallis, OR (US); Emily Robinson, Corvallis, OR (US); Anna Brown, Corvallis, OR (US); Orn Almarsson, Cambridge, MA (US); Kerry E. Benenato, Sudbury, MA (US); Staci Sabnis, Cambridge, MA (US); Gaurav Sahay, Portland, OR (US); and Ashwani Kumar Narayana, Portland, OR (US)
Assigned to ModernaTX, Inc., Cambridge, MA (US); and Oregon State University, Corvallis, OR (US)
Appl. No. 16/493,814
Filed by ModernaTX, Inc., Cambridge, MA (US); and Oregon State University, Corvallis, OR (US)
PCT Filed Mar. 15, 2018, PCT No. PCT/US2018/022756
§ 371(c)(1), (2) Date Sep. 13, 2019,
PCT Pub. No. WO2018/170336, PCT Pub. Date Sep. 20, 2018.
Claims priority of provisional application 62/475,166, filed on Mar. 22, 2017.
Claims priority of provisional application 62/471,949, filed on Mar. 15, 2017.
Prior Publication US 2020/0129445 A1, Apr. 30, 2020
Int. Cl. A61K 9/51 (2006.01); A61K 31/7088 (2006.01); A61K 45/06 (2006.01); A61K 48/00 (2006.01); C12N 15/113 (2010.01)
CPC A61K 9/5123 (2013.01) [A61K 9/5146 (2013.01); A61K 31/7088 (2013.01); A61K 45/06 (2013.01); A61K 48/0033 (2013.01); C12N 15/113 (2013.01); C12N 2320/32 (2013.01)] 13 Claims
 
1. A nanoparticle composition comprising a lipid component comprising
an ionizable lipid,
a phospholipid,
a cholesterol, and
a cholesterol derivative; wherein the cholesterol derivative is
(a) β-sitosterol or campesterol; or
(b) a compound of the formula

OG Complex Work Unit Chemistry
or a salt thereof, wherein Ra is C1-6 alkyl optionally substituted with OH or halo; Rb is C1-6 alkyl optionally substituted with OH or halo; and D1 is H or F,
wherein the molar ratio between the cholesterol and cholesterol derivative is between about 1:100 and 100:1.