The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

The present invention relates to an asymmetric siRNA that may inhibit expression of programmed cell death protein 1 (PD-1), and a use thereof, and more specifically, to: an asymmetric siRNA including an antisense strand including a sequence complementary to mRNA encoding PD-1, and a sense strand forming complementary bonds with the antisense strand; a pharmaceutical composition for preventing or treating cancer, including the asymmetric siRNA; an immune cell in which expression of PD-1 is inhibited, obtained by treatment with the siRNA; and an immune cell therapeutic agent for treating cancer, including the immune cell.

Compositions related to spherical nucleic acids (SNAs) with antisense oligonucleotides and methods of treatment of diseases and disorders are disclosed therein. In particular, the antisense oligonucleotides are targeted to a region in a pre-mRNA of interest to regulate pre-mRNA splicing.

The present invention is directed to a method of treating cancer using interfering RNA duplexes to mediate gene silencing. The present invention is also directed to interfering RNA duplexes and vectors encoding such interfering RNA duplexes.

This disclosure relates to novel targets for angiogenic disorders. Novel oligonucleotides are also provided. Methods of using the novel oligonucleotides for the treatment of angiogenic disorders (e.g., preeclampsia) are also provided.

In certain aspects, provided herein are RNA complexes (e.g., asymmetric RNA complexes, such as asiRNAs or cell penetrating asiRNAs) that inhibit CTGF expression and are therefore useful for treating idiopathic pulmonary fibrosis.

The present invention contemplates a method for the treatment of cancer comprising L-nucieic acid. The present invention contemplates a method for the treatment of cancer comprising guanosine-rich L-oligonucleotides. The present invention also relates to Polycomb Repressive Complex 2 (PRC 2) 1, -oligonucleotide inhibitors and their use for the treatment of cancer and other conditions associated with aberrant PRC2 methyl transferase activity.

Provided herein are therapeutic agents having specificity for having inhibitory activity against cancer cells that overexpress human epidermal growth factor receptor (HER) genes, including therapeutic agents comprising one or more HER-targeting peptides, pharmaceutical compositions comprising such therapeutic agents, and methods of using such compositions to treat or prevent a cancer or other disease condition associated with HER overexpression.

This disclosure relates to novel targets for angiogenic disorders. Novel oligonucleotides are also provided. Methods of using the novel oligonucleotides for the treatment of angiogenic disorders (e.g., preeclampsia) are also provided.

Among other things, the present disclosure relates to chirally controlled oligonucleotides of select designs, chirally controlled oligonucleotide compositions, and methods of making and using the same. In some embodiments, a provided chirally controlled oligonucleotide composition provides different cleavage patterns of a nucleic acid polymer than a reference oligonucleotide composition. In some embodiments, a provided chirally controlled oligonucleotide composition provides single site cleavage within a complementary sequence of a nucleic acid polymer. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and/or pattern or base modifications, sugar modifications, backbone modifications and/or stereochemistry, or combination of these elements, described herein.