Author

Thazha P. Prakash

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Open access Oct 2026

ION775, a long-acting siRNA targeting APOC3: single-dose, 12-month results in participants with moderate hypertriglyceridemia

Reduction in apolipoprotein C-III (apoC-III) is an established strategy to lower plasma triglycerides (TGs) and TG-rich lipoproteins. To evaluate the safety, tolerability, and pharmacodynamics of ION775, an investigational GalNAc-conjugated small interfering RNA targeting APOC3 messenger RNA, in otherwise...

S. Tsimikas, E. Morgan, J. Sharma et al. · European Heart Journal · 0 citations

Open access Aug 2026

Single-dose ION775, a GalNAc-conjugated siRNA targeting APOC3, lowers triglycerides for up to 12 months in moderate hypertriglyceridemia.

BACKGROUND Apolipoprotein C-III (apoC-III) regulates triglyceride-rich lipoprotein metabolism. ION775 is a GalNAc-conjugated small interfering RNA designed to silence APOC3 mRNA and enable infrequent dosing. OBJECTIVE To evaluate the safety, tolerability, and lipid effects of single-dose ION775 in adults with moderat...

S. Tsimikas, Erin S. Morgan, Jyoti Sharma et al. · Journal of Clinical Lipidolo... · 0 citations

Aug 2026

Delivery of small interfering RNA and antisense oligonucleotides across the blood-brain barrier with monovalent transferrin receptor 1 binding VHH-Fc fusion proteins

A novel monovalent TfR1-binding VHH-Fc is described for the delivery of oligonucleotide cargo, including antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) across the BBB in rodents and non-human primates (NHPs), supporting the translational potential of the VHH-antisense RMT platform for the treatme...

Ian J. Huggins, Michele Carrer, Jinro A. Santos et al. · bioRxiv · 0 citations