US 12,290,572 B2
Gene editing to improve joint function
Peter J. Millett, Edwards, CO (US); Iain Alasdair Russell, Cambridge (GB); and Matthew J. Allen, Swaffham Bulbeck (GB)
Assigned to ORTHOBIO THERAPEUTICS, INC., Vail, CO (US)
Filed by ORTHOBIO THERAPEUTICS, INC., Edwards, CO (US)
Filed on Apr. 27, 2021, as Appl. No. 17/241,839.
Application 17/241,839 is a continuation of application No. 17/166,343, filed on Feb. 3, 2021, granted, now 11,324,838.
Application 17/166,343 is a continuation of application No. 16/916,014, filed on Jun. 29, 2020, granted, now 11,033,590.
Application 16/916,014 is a continuation of application No. PCT/US2020/014139, filed on Jan. 17, 2020.
Claims priority of provisional application 62/794,340, filed on Jan. 18, 2019.
Claims priority of provisional application 62/894,184, filed on Aug. 30, 2019.
Claims priority of provisional application 62/914,635, filed on Oct. 14, 2019.
Prior Publication US 2021/0261956 A1, Aug. 26, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 48/00 (2006.01); A61K 9/00 (2006.01); A61K 9/127 (2006.01); A61K 35/76 (2015.01); A61P 19/02 (2006.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/113 (2010.01); C12N 15/85 (2006.01); C12N 15/86 (2006.01)
CPC A61K 48/00 (2013.01) [A61K 48/0075 (2013.01); A61P 19/02 (2018.01); C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 15/1136 (2013.01); C12N 15/85 (2013.01); C12N 15/86 (2013.01); A61K 9/0019 (2013.01); A61K 9/127 (2013.01); A61K 35/76 (2013.01); C12N 2310/20 (2017.05); C12N 2750/14143 (2013.01)] 20 Claims
 
1. A pharmaceutical composition for the treatment or prevention of a joint disease or condition, comprising:
a therapeutically effective amount of a Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) gene-editing system, the system comprising:
(i) a nucleic acid encoding a CRISPR Associated Protein 9 (Cas9) protein; and
(ii) at least one guide RNA targeting an IL-1α gene, wherein:
the at least one guide RNA comprises a crRNA sequence that is complementary to a target sequence in exon 4 of the IL-1α gene, and
the target sequence is adjacent to a protospacer adjacent motif (PAM) sequence for the Cas9 protein.