US 10,889,817 B2
Oligonucleotide therapy for Leber Congenital Amaurosis
Patricia Coromoto Biasutto, The Hague (NL); and Hee Lam Chan, The Hague (NL)
Assigned to ProQR Therapeutics II B.V., Leiden (NL)
Filed by ProQR Therapeutics II B.V., Leiden (NL)
Filed on Sep. 20, 2019, as Appl. No. 16/576,853.
Application 16/576,853 is a continuation of application No. 15/551,026, granted, now 10,421,963, previously published as PCT/EP2016/054164, filed on Feb. 26, 2016.
Claims priority of application No. 1503408.5 (GB), filed on Feb. 27, 2015.
Prior Publication US 2020/0063134 A1, Feb. 27, 2020
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/113 (2010.01); A61K 31/7125 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 31/7125 (2013.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/346 (2013.01); C12N 2320/33 (2013.01); C12N 2330/51 (2013.01)] 6 Claims
 
1. An oligonucleotide consisting of the nucleotide sequence of SEQ NO: 5, 6, or 7, wherein the oligonucleotide comprises a modified ribose that is substituted at the 2′ position with a substituent selected from the group consisting of:
(i) OH;
(ii) F;
(iii) substituted or unsubstituted, linear or branched lower (C1-C10) alkyl, alkenyl, alkynl, alkaryl, allyl, or aralkyl, that may be interrupted by one or more heteroatoms,
(iv) O-, S-, or N-alkyl,
(v) O-, S-, or N-alkenyl,
(vi) O-, S-, or N-alkynyl,
(vii) O-, S-, or N-allyl,
(viii) O-alkyl-O-alkyl, -methoxy, or aminopropoxy,
(ix) methoxyethoxy,
(x) dimethylaminooxyethoxy, and
(xi) dimethylaminoethoxyethoxy.