The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Mar. 07, 2023

Filed:

Oct. 12, 2018
Applicant:

Wave Life Sciences Ltd., Singapore, SG;

Inventors:

Jason Jingxin Zhang, Walpole, MA (US);

Chikdu Shakti Shivalila, Somerville, MA (US);

Chandra Vargeese, Schwenksville, PA (US);

Naoki Iwamoto, Brighton, MA (US);

Assignee:

WAVE LIFE SCIENCES LTD., Singapore, SG;

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); A61K 31/7125 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
A61K 31/7125 (2013.01); C12N 15/113 (2013.01); C12N 2310/31 (2013.01); C12N 2320/33 (2013.01);
Abstract

Among other things, the present disclosure provides technologies for altering splicing, particularly for increasing inclusion of exons in splicing products. In some embodiments, the present disclosure provides SMN2 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides chirally controlled SMN2 oligonucleotide compositions. In some embodiments, provided oligonucleotides and compositions can increase level of an exon 7-containing SMN2 splicing product and/or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of splicing-related conditions, disorders and diseases. In some embodiments, the present disclosure provides methods for treating SMN2-related conditions, disorders and diseases such as SMA (spinal muscular atrophy) and ALS (amyotrophic lateral sclerosis).


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