US 11,883,541 B2
Nonviral gene transfer to the suprachoroidal space
Peter A. Campochiaro, Baltimore, MD (US); Jordan Green, Nottingham, MD (US); Jayoung Kim, Baltimore, MD (US); and Jikui Shen, Dundalk, MD (US)
Assigned to THE JOHNS HOPKINS UNIVERSITY, Baltimore, MD (US)
Appl. No. 16/753,245
Filed by The Johns Hopkins University, Baltimore, MD (US)
PCT Filed Oct. 2, 2018, PCT No. PCT/US2018/053990
§ 371(c)(1), (2) Date Apr. 2, 2020,
PCT Pub. No. WO2019/070727, PCT Pub. Date Apr. 11, 2019.
Claims priority of provisional application 62/567,043, filed on Oct. 2, 2017.
Prior Publication US 2020/0330396 A1, Oct. 22, 2020
Int. Cl. A61K 9/51 (2006.01); A61K 9/00 (2006.01); A61K 9/50 (2006.01); A61K 45/06 (2006.01); B82Y 5/00 (2011.01)
CPC A61K 9/5153 (2013.01) [A61K 9/0051 (2013.01); A61K 9/5031 (2013.01); A61K 45/06 (2013.01); B82Y 5/00 (2013.01)] 18 Claims
 
1. A method for treating a disease or condition of the eye, the method comprising non-virally transfecting one or more cells in a suprachoroidal space by administering to a suprachoroidal space of an eye of a subject a composition comprising a nanoparticle or a microparticle comprising a poly(beta-amino ester) (PBAE) and a therapeutic agent, wherein the therapeutic agent is selected from an expression construct, a therapeutic transgene, a therapeutic protein, a viral vector, a plasmid, a gene, a nucleic acid, DNA, RNA, and mRNA, and wherein the PBAE is

OG Complex Work Unit Chemistry
wherein:
n is an integer from 1 to 10,000;
R1, R2, R3, R4, R5, R6, R7, R8, and R9 are each hydrogen;
R1 is absent and when present the compound of formula (I) further comprises a counter ion selected from the group consisting of chloride, fluoride, bromide, iodide, sulfate, nitrate, fumarate, acetate, carbonate, stearate, laurate, and oleate; and

OG Complex Work Unit Chemistry