2019年6月6日 · Now that we have substantial knowledge about the key proteins with which ASOs interact in plasma and other in vivo sites, we have established an effort to conjugate ligands (LICA) to ASOs to...

2021年8月25日 · Their newer platforms use ligand-conjugated antisense (LICA) technology to deliver ASOs to target cells. Following Alnylam’s lead that resulted in successful delivery of siRNA, Ionis conducted an in-depth investigation of triantennary GalNAc-conjugated ASOs.

2020年12月7日 · AKCEA-TTR-L Rx (ION-682884) is a ligand-conjugated antisense drug designed for receptor-mediated uptake by hepatocytes, the primary source of circulating transthyretin (TTR). Enhanced delivery of the antisense pharmacophore is expected to increase drug potency and support lower, less frequent dosing in treatment.

2019年9月4日 · Antisense oligonucleotides (ASOs) are a novel therapeutic approach to target difficult-to-drug protein classes by targeting their corresponding mRNAs. Significantly enhanced ASO activity has been achieved by the targeted delivery of ASOs to selected tissues.

2024年2月13日 · GalNAc 3 -conjugated 2′- O -methoxyethyl (2′MOE) modified antisense oligonucleotides (ASOs) have demonstrated a higher potency than the unconjugated form to support lower doses for an equivalent pharmacological effect.

Antisense oligonucleotides (ASOs) have emerged in recent years as a potential gene therapy to treat DM1. However, the clinical efficacy of the systemic administration of ASOs is limited by a combination of insufficient potency and poor tissue distribution.

2018年12月7日 · Serial in vivo spectroscopy measurements in mice treated with a C16 fatty acid ligand conjugated antisense (LICA) oligonucleotide reveal a dose-dependent therapeutic response within seven days,...

Today, we have fully validated intravenous (IV), subcutaneous (SC) and intrathecal (IT) administration. We also have encouraging data on aerosol administration and can give antisense...

2017年3月26日 · LICA modified ASOs dramatically improved whole heart activity (80-85% target reduction), which was consistent with increased activity observed in the isolated cardiomyocyte and fibroblasts cell...

Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA ...