US 11,739,325 B2
Oligonucleotide compositions and methods thereof
Chandra Vargeese, Schwenksville, PA (US); Zhong Zhong, Hingham, MA (US); Naoki Iwamoto, Brighton, MA (US); Jason Jingxin Zhang, Walpole, MA (US); Jean-Cosme Dodart, Boston, MA (US); Yuanjing Liu, Arlington, MA (US); Pachamuthu Kandasamy, Belmont, MA (US); Sethumadhavan Divakaramenon, Lexington, MA (US); Genliang Lu, Winchester, MA (US); and Subramanian Marappan, Acton, MA (US)
Assigned to WAVE LIFE SCIENCES LTD., Singapore (SG)
Appl. No. 16/636,902
Filed by WAVE LIFE SCIENCES LTD., Singapore (SG)
PCT Filed Aug. 7, 2018, PCT No. PCT/US2018/045659
§ 371(c)(1), (2) Date Feb. 5, 2020,
PCT Pub. No. WO2019/032612, PCT Pub. Date Feb. 14, 2019.
Claims priority of provisional application 62/542,778, filed on Aug. 8, 2017.
Prior Publication US 2021/0032620 A1, Feb. 4, 2021
Int. Cl. C12N 15/113 (2010.01); C12N 15/11 (2006.01); C12Q 1/68 (2018.01)
CPC C12N 15/113 (2013.01) [C12N 15/11 (2013.01); C12N 2310/11 (2013.01); C12N 2310/12 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/33 (2013.01); C12N 2310/341 (2013.01); C12N 2310/346 (2013.01)] 23 Claims
 
1. An oligonucleotide composition comprising oligonucleotides of a particular oligonucleotide type characterized by:
a) a common base sequence;
b) a common pattern of backbone linkages;
c) a common pattern of backbone chiral centers;
wherein the composition is enriched, relative to a substantially racemic preparation of oligonucleotides having the same common base sequence, for oligonucleotides of the particular oligonucleotide type; and
wherein each oligonucleotide of the particular oligonucleotide type is independently an oligonucleotide comprising a first wing, a second wing and a core in a format of first wing-core-second wing or second wing-core-first wing, wherein:
(i) the first wing and the second wing comprise different sugar modifications; and
(ii) oligonucleotides of the particular oligonucleotide type comprise (Op)n(Sp)m in the core, wherein:
Sp indicates the S configuration of a chiral linkage phosphorus of a chiral modified internucleotidic linkage;
Op indicates an achiral linkage phosphorus of a natural phosphate linkage, wherein the nucleotidic unit comprising Op comprises a 5′-modification on its sugar; and
each of n and m is independently 1-20.