Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid, pemphigus, e.g., pemphigus vulgaris (PV) and pemphigus foliaceus (PF), and C3 glomerulopathy.

Abstract: The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting carbonic anhydrase 2 (CA2), and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of carbonic anhydrase 2.

Abstract: The disclosure relates to RNA agents targeting LRP1 receptor modified for targeted delivery to the brain and/or the eye. The present invention provides modified double stranded ribonucleic acid (dsRNAi) agents conjugated to a peptide ligand, as well as methods of modulating the expression of a target gene in a CNS cell or tissue and/or an ocular cell or tissue and methods of treating subjects having a CNS and/or an ocular disease or disorder using such dsRNAi agents.

Abstract: The present invention relates, in general to agents that modulate the pharmacological activity of conjugated siRNAs.

Abstract: The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the CIDEB gene, as well as methods of inhibiting expression of CIDEB, and methods of treating subjects that would benefit from reduction in expression of CIDEB, such as subjects having a CIDEB-associated disease, disorder, or condition, using such dsRNA compositions.

Abstract: The present invention relates, in general to agents that modulate the pharmacological activity of siRNAs. In addition, the invention relates generally to methods and systems for use in assessing the efficacy and safety of a pharmaceutical composition for use in the treatment or prophylaxis of a disease.

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the myosin regulatory light chain interacting protein (MYLIP) gene, as well as methods of inhibiting expression of MYLIP, and methods of treating subjects that would benefit from reduction in expression of MYLIP, such as subjects having a MYLIP-associated disease, disorder, or condition, using such dsRNA compositions.

Abstract: The present invention relates, in general to, compounds, compositions and methods useful for modulating gene expression of multiple target nucleic acids by a single chemical entity.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the CIDEB gene, as well as methods of inhibiting expression of CIDEB, and methods of treating subjects that would benefit from reduction in expression of CIDEB, such as subjects having a CIDEB-associated disease, disorder, or condition, using such dsRNA compositions.

Abstract: The present disclosure relates monomers and methods for synthesizing oligonucleotides comprising 2,6-diaminopurine (DAP) and 2-aminoadenine conjugates. The methodology employs simple aromatic nucleophilic substitution of halogen atom at the 2-position in 2-haloadenosine derivatives with amines or alkali earth hydroxides obviating the need for any protecting group on adenine.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.

Abstract: The disclosure relates to use of double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an amyloid precursor protein (APP) gene, including methods of inhibiting expression of an APP gene and methods of treating subjects having a disease or disorder characterized by enlarged endosomes, e.g., Alzheimer's disease (AD) and Down syndrome (DS), particularly occurrences of such neurodegenerative diseases associated with one or more mutations in presenilin 1 (PSEN1), using such dsRNAi agents and compositions.

Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the KISS1 gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a KISS1 gene and to methods of preventing and treating a deficiency in glycemic control, e.g., type 2 diabetes mellitus (T2DM).

Abstract: One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.

Abstract: The present invention relates to ligand conjugates of oligonucleotides (e.g., iRNA agents) and methods for their preparation. The ligands are derived primarily from monosaccharides These conjugates are useful for the in vivo delivery of oligonucleotides.

Abstract: The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.

Abstract: One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2?-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.

Abstract: The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting ANGPTL7. The invention also relates to methods of using such dsRNA compositions to inhibit expression of ANGPTL7 and to methods of treating ANGPTL7-associated disorders, e.g., glaucoma, using such dsRNA compositions.