US 12,257,318 B2
Polynucleotide compositions, related formulations, and methods of use thereof
Mirko Hennig, Mountain View, CA (US); Daniella Ishimaru, Menlo Park, CA (US); David J. Lockhart, Menlo Park, CA (US); Michael Torres, Menlo Park, CA (US); Jackson Eby, Menlo Park, CA (US); Dmitri Boudko, Menlo Park, CA (US); and Brandon A. Wustman, San Diego, CA (US)
Assigned to ReCode Therapeutics, Inc., Menlo Park, CA (US)
Filed by ReCode Therapeutics, Inc., Menlo Park, CA (US)
Filed on Feb. 2, 2024, as Appl. No. 18/431,504.
Application 18/431,504 is a continuation of application No. 18/283,523, previously published as PCT/US2022/021526, filed on Mar. 23, 2022.
Claims priority of provisional application 63/274,912, filed on Nov. 2, 2021.
Claims priority of provisional application 63/208,966, filed on Jun. 9, 2021.
Claims priority of provisional application 63/164,573, filed on Mar. 23, 2021.
Prior Publication US 2024/0269325 A1, Aug. 15, 2024
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 48/00 (2006.01); A61K 9/00 (2006.01); A61K 9/12 (2006.01); A61K 9/127 (2006.01); A61K 9/1271 (2025.01); A61K 9/51 (2006.01); A61K 38/17 (2006.01)
CPC A61K 48/005 (2013.01) [A61K 9/0078 (2013.01); A61K 9/12 (2013.01); A61K 9/1271 (2013.01); A61K 9/5123 (2013.01); A61K 38/1709 (2013.01); A61K 48/0075 (2013.01)] 30 Claims
 
1. A pharmaceutical composition comprising a synthetic polynucleotide assembled with a lipid composition,
wherein said synthetic polynucleotide encodes a cystic fibrosis transmembrane conductance regulator (CFTR) protein that comprises a nucleic acid sequence having at least about 98% sequence identity to SEQ ID NO: 4,
wherein said lipid composition comprises
an ionizable cationic lipid at a molar percentage of about 5% to about 30%;
a selective organ targeting (SORT) lipid separate from said ionizable cationic lipid at a molar percentage of about 5% to about 30%;
a phospholipid at a molar percentage of about 5% to about 25%;
a steroid or steroid derivative at a molar percentage of about 15% to about 46%; and
a polymer-conjugated lipid at a molar percentage of about 0.5% to about 10%.