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Author

Thazha P. Prakash

2 papers indexed here

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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. · 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. · 0 citations

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