US 12,303,526 B2
DNA compositions and related methods
Alexandra Rachael Sneider, Cambridge, MA (US); Jacob Rosenblum Rubens, Cambridge, MA (US); Narahari Subbanna Pujar, Berwyn, PA (US); Molly Krisann Gibson, Medford, MA (US); Benjamin Andrew Portney, Somerville, MA (US); Geoffrey A. Von Maltzahn, Somerville, MA (US); and Camilo Ayala Breton, Andover, MA (US)
Assigned to FLAGSHIP PIONEERING INNOVATIONS VII, LLC, Cambridge, MA (US)
Filed by FLAGSHIP PIONEERING INNOVATIONS VII, LLC, Cambridge, MA (US)
Filed on Nov. 11, 2022, as Appl. No. 18/054,813.
Application 18/054,813 is a continuation of application No. PCT/US2022/078301, filed on Oct. 18, 2022.
Claims priority of provisional application 63/262,690, filed on Oct. 18, 2021.
Claims priority of provisional application 63/304,913, filed on Jan. 31, 2022.
Claims priority of provisional application 63/373,293, filed on Aug. 23, 2022.
Claims priority of provisional application 63/402,772, filed on Aug. 31, 2022.
Prior Publication US 2023/0255999 A1, Aug. 17, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/11 (2006.01); A61K 9/127 (2006.01); A61K 31/7115 (2006.01); A61K 48/00 (2006.01)
CPC A61K 31/7115 (2013.01) [A61K 9/127 (2013.01); A61K 48/00 (2013.01); C12N 15/11 (2013.01); C12N 2320/30 (2013.01)] 22 Claims
 
1. A pharmaceutical formulation comprising:
a lipid nanoparticle (LNP) comprising a single stranded DNA (ssDNA), wherein the ssDNA:
(a) encodes only one protein, which is a therapeutic protein,
(b) is covalently closed,
(c) does not form a double stranded structure longer than 40 base pairs,
(d) is at least 200 nucleotides in length, and
(e) does not comprise a protelomerase target sequence;
wherein:
(i) at least 95% by mass of total DNA in the composition is the ssDNA that is covalently closed;
(ii) the pharmaceutical formulation is free of polypeptides; and
(iii) the therapeutic protein is expressed when the ssDNA is introduced into a target cell.