US 11,753,638 B2
Conjugated oligonucleotides
Anastasia Khvorova, Westborough, MA (US); Mehran Nikan, Boston, MA (US); Matthew Hassler, Worcester, MA (US); Maire Osborn, Worcester, MA (US); Reka Haraszti, Shrewsbury, MA (US); Andrew Coles, Auburn, MA (US); Anton Turanov, Boston, MA (US); Neil Aronin, Newtonville, MA (US); and Annabelle Biscans, Cambridge, MA (US)
Assigned to UNIVERSITY OF MASSACHUSETTS, Boston, MA (US)
Appl. No. 16/322,212
Filed by UNIVERSITY OF MASSACHUSETTS, Boston, MA (US)
PCT Filed Aug. 11, 2017, PCT No. PCT/US2017/046593
§ 371(c)(1), (2) Date Jan. 31, 2019,
PCT Pub. No. WO2018/031933, PCT Pub. Date Feb. 15, 2018.
Claims priority of provisional application 62/374,499, filed on Aug. 12, 2016.
Claims priority of provisional application 62/461,529, filed on Feb. 21, 2017.
Prior Publication US 2019/0185855 A1, Jun. 20, 2019
Int. Cl. A61K 31/7088 (2006.01); C12N 15/113 (2010.01); A61K 47/54 (2017.01); C07H 21/02 (2006.01); C07H 21/04 (2006.01); C07H 21/00 (2006.01); C12N 15/11 (2006.01); A61K 47/55 (2017.01); A61P 1/16 (2006.01); A61P 9/00 (2006.01); A61P 13/12 (2006.01); A61P 1/00 (2006.01); A61P 5/00 (2006.01); A61P 11/00 (2006.01); A61K 9/00 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 9/0019 (2013.01); A61K 31/7088 (2013.01); A61K 47/54 (2017.08); A61K 47/543 (2017.08); A61K 47/549 (2017.08); A61K 47/551 (2017.08); A61K 47/554 (2017.08); A61P 1/00 (2018.01); A61P 1/16 (2018.01); A61P 5/00 (2018.01); A61P 9/00 (2018.01); A61P 11/00 (2018.01); A61P 13/12 (2018.01); C07H 21/00 (2013.01); C07H 21/02 (2013.01); C07H 21/04 (2013.01); C12N 15/111 (2013.01); C12N 2310/14 (2013.01); C12N 2310/312 (2013.01); C12N 2310/315 (2013.01); C12N 2310/346 (2013.01); C12N 2310/351 (2013.01); C12N 2310/3515 (2013.01); C12N 2320/32 (2013.01)] 18 Claims
 
1. A compound of formula (I):

OG Complex Work Unit Chemistry
wherein:
O is an oligonucleotide;
L is a linker selected from:

OG Complex Work Unit Chemistry
Xc is a cholesterol, Lithocholic acid, Retinoic acid or α-tocopheryl succinate; and
Zc is a phosphodiester or phosphodiester derivative selected from

OG Complex Work Unit Chemistry
wherein X is O, S or BH3.