US 11,730,826 B2
Amphiphilic nanoparticles for delivery of CRISPR based therapy
Paiman Peter Ghoroghchian, Boston, MA (US); Haihua Xiao, Cambridge, MA (US); Ruogu Qi, Houston, TX (US); and Ting Li, Xi'an (CN)
Assigned to Massachusetts Institute of Technology, Cambridge, MA (US)
Filed by Massachusetts Institute of Technology, Cambridge, MA (US)
Filed on May 2, 2017, as Appl. No. 15/584,309.
Claims priority of provisional application 62/489,111, filed on Apr. 24, 2017.
Claims priority of provisional application 62/330,684, filed on May 2, 2016.
Claims priority of provisional application 62/330,697, filed on May 2, 2016.
Prior Publication US 2018/0078657 A1, Mar. 22, 2018
Int. Cl. A61K 9/00 (2006.01); A61K 48/00 (2006.01); A61K 47/60 (2017.01); A61K 47/64 (2017.01); C12N 15/113 (2010.01); A61K 33/243 (2019.01); A61K 47/69 (2017.01); A61K 31/555 (2006.01); C08G 81/00 (2006.01); C12N 15/11 (2006.01); A61K 9/107 (2006.01); A61K 31/282 (2006.01); A61K 47/34 (2017.01); C07K 2/00 (2006.01); C08G 69/10 (2006.01); C12N 15/90 (2006.01); A61K 47/10 (2017.01); A61K 39/00 (2006.01); C12N 15/09 (2006.01); G01N 21/47 (2006.01)
CPC A61K 48/0041 (2013.01) [A61K 9/107 (2013.01); A61K 31/282 (2013.01); A61K 31/555 (2013.01); A61K 33/243 (2019.01); A61K 47/10 (2013.01); A61K 47/34 (2013.01); A61K 47/60 (2017.08); A61K 47/646 (2017.08); A61K 47/6907 (2017.08); A61K 47/6935 (2017.08); A61K 48/005 (2013.01); C07K 2/00 (2013.01); C08G 69/10 (2013.01); C08G 81/00 (2013.01); C12N 15/11 (2013.01); C12N 15/113 (2013.01); C12N 15/90 (2013.01); A61K 2039/585 (2013.01); C12N 15/09 (2013.01); C12N 2310/20 (2017.05); G01N 21/47 (2013.01)] 28 Claims
OG exemplary drawing
 
1. A composition comprising:
an agent, wherein the agent is (a) a nuclease selected from the group consisting of Cas9, TALEN, and zinc finger, or (b) a nucleic acid encoding a nuclease selected from the group consisting of Cas9, TALEN, and zinc finger;
an optional pharmaceutically acceptable carrier;
an optional DNA editing template; and
a block copolymer comprising:
(i) a first block comprising a plurality of first monomers, wherein each first monomer is hydrophilic;
(ii) a second block comprising a plurality of second monomers, wherein each second monomer has the structure of Formula II:

OG Complex Work Unit Chemistry
wherein R2 is C1-C6alkyl, C3-C6alkenyl, C5-C22carbocyclyl-substituted C1-C6alkyl, or 5-22-membered heterocyclyl-substituted C1-C6alkyl; and
(iii) a third block comprising a plurality of third monomers, wherein each third monomer is positively charged at a pH from about 6.8 to about 7.4,
wherein
the agent is non-covalently associated with the block copolymer; and
the DNA editing template, when present, is non-covalently associated with the block copolymer.