The present invention provides an aptamer that specifically hinds CD44, composition comprising the aptamer, as well as methods for detecting CD44 and for targeted delivery to CD44-expressing cells.

The present invention concerns the use of antisense oligonucleotides (AON) capable of inhibiting expression of dynamin 2, advantageously human dynamin 2, for use in the treatment of Charcot-Marie-Tooth disease (CMT) and centronuclear myopathies (CNM).

A chimeric complex comprising a microRNA in combination with an aptamer for AXL receptor tyrosine kinase is provided. Use of the chimeric complex for targeted treatment of a tumor disease, in particular in a therapy affecting onset and/or progression of tumor metastasis is also provided.

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SOD1 gene, as well as methods of inhibiting expression of a SOD1 gene and methods of treating subjects having a SOD1-associated neurodegenerative disease or disorder, e.g., Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), and Down's syndrome (DS), using such dsRNAi agents and compositions.

Described herein are compositions and methods for inhibition of Asialoglycoprotein receptor 1 (ASGR1) gene expression. RNA interference (RNAi) agents, e.g., double stranded RNAi agents, and RNAi agent-targeting ligand conjugates for inhibiting the expression of an ASGR1 gene are described. Pharmaceutical compositions comprising one or more ASGR1 RNAi agents, optionally with one or more additional therapeutics, are also described. The ASGR1 RNAi agents can be used in methods of treatment of various diseases and conditions, such as cardiometabolic diseases related to elevated non-HDL cholesterol (non-HDL-C) levels, elevated LDL cholesterol (LDL-C) levels, elevated total cholesterol levels, and/or elevated triglyceride (TG) levels.

Therapeutic oligonucleotides comprising pharmacokinetic (PK)-modifying anchors are provided. Methods for treating diseases or disorders comprising administering to a subject a therapeutic oligonucleotide comprising one or more PK-modifying anchors are provided.

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.

The present disclosure provides antisense compounds, methods, and compositions for silencing ADAM33 mRNA. The present disclosure provides antisense compounds, methods, and compositions for the treatment, prevention, or amelioration of diseases, disorders, and conditions associated with ADAM33 in a subject in need thereof. Also contemplated are antisense compounds and methods for the preparation of a medicament for the treatment, prevention, or amelioration of a disease, disorder, or condition associated with ADAM33.

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).

A three-dimensional star-shaped α-helix polypeptide having a high-efficiency gene delivery capability, and a preparation method and an application thereof. A dendrimer is used as an initiator and dichloromethane is used as a reaction solvent to initiate high-speed ring-opening polymerization of different types of N-carboxylic anhydride monomers, and groups having different electrical properties are introduced at the ends via click chemistry reactions. The abundant amino groups on the surface of the dendrimer provide enough polymerization sites to enable the polypeptide to form a three-dimensional spherical topological structure, and the topological structure provides an opportunity for initial acceleration of the ring-opening polymerization reaction. The higher positive charge density caused by polypeptide side chain modified guanidine/amino groups etc. achieves a high-efficiency gene loading capability by the electrostatic effect between positive and negative charges, and the α-helix rigid structure on the secondary structure thus enables the polypeptide to have stronger membrane penetration capability.