US 12,478,589 B2
Co-delivery of nucleic acids for simultaneous suppression and expression of target genes
Omid Farokhzad, Waban, MA (US); Xiaoding Xu, Malden, MA (US); and Jinjun Shi, Newton, MA (US)
Assigned to The Brigham and Women's Hospital, Inc., Boston, MA (US)
Filed by The Brigham and Women's Hospital, Inc., Boston, MA (US)
Filed on Jul. 8, 2020, as Appl. No. 16/923,938.
Application 16/923,938 is a continuation of application No. 16/470,506, abandoned, previously published as PCT/US2017/067090, filed on Dec. 18, 2017.
Claims priority of provisional application 62/435,171, filed on Dec. 16, 2016.
Prior Publication US 2021/0299060 A1, Sep. 30, 2021
Int. Cl. A61K 9/51 (2006.01); A61K 31/7088 (2006.01); A61K 38/46 (2006.01); A61K 45/06 (2006.01); A61K 47/60 (2017.01); A61K 47/62 (2017.01); A61K 47/69 (2017.01); A61K 48/00 (2006.01); A61P 35/00 (2006.01); C12N 15/113 (2010.01)
CPC A61K 9/5146 (2013.01) [A61K 31/7088 (2013.01); A61K 38/465 (2013.01); A61K 45/06 (2013.01); A61K 47/60 (2017.08); A61K 47/62 (2017.08); A61K 47/6935 (2017.08); A61K 48/0041 (2013.01); A61P 35/00 (2018.01); C12N 15/113 (2013.01); C12Y 301/03048 (2013.01); C12N 2310/14 (2013.01); C12N 2310/141 (2013.01); C12N 2310/351 (2013.01); C12N 2320/31 (2013.01); C12N 2320/32 (2013.01)] 16 Claims
 
1. A nanoparticle formulation comprising redox-responsive polymeric nanoparticles encapsulating
an inhibitory functional nucleic acid,
wherein the inhibitory functional nucleic acid reduces or suppresses expression of an immune costimulatory molecule or signal, wherein the inhibitory functional nucleic acid is an inhibitory RNA,
and
a stimulatory nucleic acid species comprising an mRNA which enhances or increases an activity in a cell or encodes a protein or peptide,
wherein the stimulatory nucleic acid species encodes an antigen,
wherein the redox-responsive polymeric nanoparticles comprise a poly(disulfide) polymer comprising a disulfide bond linking two L-cysteine residues.