US 12,241,064 B2
REVERSIR™ compounds
Vasant Jadhav, Cambridge, MA (US); John Maraganore, Cambridge, MA (US); Martin Maier, Cambridge, MA (US); Kallanthottathil G. Rajeev, Cambridge, MA (US); Muthiah Manoharan, Cambridge, MA (US); Akin Akinc, Cambridge, MA (US); and Ivan Zlatev, Cambridge, MA (US)
Assigned to ALNYLAM PHARMACEUTICALS, INC., Cambridge, MA (US)
Filed by ALNYLAM PHARMACEUTICALS, INC., Cambridge, MA (US)
Filed on Aug. 10, 2021, as Appl. No. 17/398,923.
Application 17/398,923 is a continuation of application No. 15/537,083, abandoned, previously published as PCT/US2015/066465, filed on Dec. 17, 2015.
Claims priority of provisional application 62/238,467, filed on Oct. 7, 2015.
Claims priority of provisional application 62/093,906, filed on Dec. 18, 2014.
Prior Publication US 2022/0127604 A1, Apr. 28, 2022
Prior Publication US 2022/0259589 A9, Aug. 18, 2022
Int. Cl. C12N 15/11 (2006.01); C12N 15/113 (2010.01)
CPC C12N 15/111 (2013.01) [C12N 15/113 (2013.01); C12N 2310/113 (2013.01); C12N 2310/315 (2013.01); C12N 2310/3183 (2013.01); C12N 2310/319 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/3341 (2013.01); C12N 2310/346 (2013.01); C12N 2310/351 (2013.01)] 25 Claims
OG exemplary drawing
 
1. A modified oligonucleotide consisting of 6 to 20 linked nucleotides and having a nucleobase sequence substantially complementary to positions 2-10, counting from 5′-end of antisense strand of a siRNA, wherein the siRNA comprises a sense strand and an antisense strand, wherein the sense and antisense strand hybridize to form a duplex structure comprising from 14 to 30 basepairs, wherein the antisense strand is complementary to at least a part of a target sequence, wherein the modified oligonucleotide is at least 4 nucleotides shorter than the antisense strand, wherein the modified oligonucleotide comprises a 2′-deoxy nucleoside only at position 1, counting from the 3′-end, and wherein the modified oligonucleotide comprises at least 2 locked nucleic acid (LNA) modifications, and two of the LNA modifications in the modified oligonucleotide are: (i) at position 2, counting from 3′-end, and at position 1, counting from 5′-end; or (ii) at position 2, counting from 3′-end, and at position 2, counting from 5′-end).