C12N2310/335

Amphiphilic polynucleotides

Compositions and methods disclosed herein can help provide improved delivery of non-natural therapeutic nucleotides for the treatment of diseases such as cancer. An example composition includes an assembly of amphiphilic polynucleotides, where each amphiphilic polynucleotide includes an aptamer portion, a first nucleotide portion, and a second nucleotide portion.

COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE LECT2 GENE
20220372487 · 2022-11-24 · ·

The invention relates to antisense polynucleotide agents targeting the LECT2 gene, and methods of using such antisense polynucleotide agents to inhibit expression of LECT2 and to treat subjects having a LECT2-associated disease, e.g., amyloidosis.

COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE LECT2 GENE
20220372487 · 2022-11-24 · ·

The invention relates to antisense polynucleotide agents targeting the LECT2 gene, and methods of using such antisense polynucleotide agents to inhibit expression of LECT2 and to treat subjects having a LECT2-associated disease, e.g., amyloidosis.

MODIFIED RIBONUCLEIC ACIDS AND USES THEREOF

Disclosed herein is a modified ribonucleotide comprising a nucleoside comprising N4-acetylcytidine and/or 5-hydroxymethyluridine, and polyribonucleotides comprising the same. Also provided herein are compositions comprising a polyribonucleotide of the present disclosure and methods of making and using the same.

ANTI-CANCER IMMUNOTHERAPEUTIC COMPOSITION FOR TREATING CANCER
20230055235 · 2023-02-23 ·

The present invention relates to an aptamer or a functional fragment thereof, capable of specifically binding to PD-L1 or capable of inhibiting interaction between PD-1 and PD-L1 by specifically binding to PD-L1, which includes a nucleic acid sequence of SEQ ID NO. 1, 2, 10 or 18.

ANTI-CANCER IMMUNOTHERAPEUTIC COMPOSITION FOR TREATING CANCER
20230055235 · 2023-02-23 ·

The present invention relates to an aptamer or a functional fragment thereof, capable of specifically binding to PD-L1 or capable of inhibiting interaction between PD-1 and PD-L1 by specifically binding to PD-L1, which includes a nucleic acid sequence of SEQ ID NO. 1, 2, 10 or 18.

5-halouracil-modified microRNAs and their use in the treatment of cancer

The present disclosure provides nucleic acid compositions that incorporate one or more halouracil molecules. More specifically, the present disclosure reveals that the replacement of uracil nucleotides within a microRNA nucleotide sequence with a 5-halouracil increases the ability of the micro-RNA to inhibit cancer progression and tumorigenesis. As such, the present disclosure provides various nucleic acid (e.g., microRNA) compositions having 5-halouracil molecules incorporated in their nucleic acid sequences and methods for using the same. The present disclosure further provides pharmaceutical compositions comprising the modified nucleic acid compositions, and methods for treating cancers using the same.

5-halouracil-modified microRNAs and their use in the treatment of cancer

The present disclosure provides nucleic acid compositions that incorporate one or more halouracil molecules. More specifically, the present disclosure reveals that the replacement of uracil nucleotides within a microRNA nucleotide sequence with a 5-halouracil increases the ability of the micro-RNA to inhibit cancer progression and tumorigenesis. As such, the present disclosure provides various nucleic acid (e.g., microRNA) compositions having 5-halouracil molecules incorporated in their nucleic acid sequences and methods for using the same. The present disclosure further provides pharmaceutical compositions comprising the modified nucleic acid compositions, and methods for treating cancers using the same.

Antisense compounds and uses thereof

The present disclosure provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.

STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
20230093948 · 2023-03-30 ·

The invention relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the SCAP gene, as well as methods of inhibiting expression of a SCAP gene and methods of treating subjects having a SCAP-associated disorder, such as nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH), using such dsRNAi agents and compositions.