WAVE LIFE SCIENCES LTD. (20250066775). OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

Organization Name

WAVE LIFE SCIENCES LTD.

Inventor(s)

Chandra Vargeese of Schwenksville PA (US)

Zhong Zhong of Hingham MA (US)

Naoki Iwamoto of Boston MA (US)

Jason Jingxin Zhang of Walpole MA (US)

Jean-Cosme Dodart of Somerville MA (US)

Yuanjing Liu of Boston MA (US)

Pachamuthu Kandasamy of Lexington MA (US)

Sethumadhavan Divakaramenon of Lexington MA (US)

Genliang Lu of Winchester MA (US)

Subramanian Marappan of Acton MA (US)

OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

This abstract first appeared for US patent application 20250066775 titled 'OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

Original Abstract Submitted

among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., knockdown ability, stability, delivery, etc. in some embodiments, the oligonucleotides decrease the expression, activity and/or level of a c9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof. in some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in treatment of c9orf72-related disorders.