#news #biotech Expanding RNA interference (RNAi) therapeutics from the lab to the clinic

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Expanding RNA interference (RNAi) therapeutics from the lab to the clinic .It is now possible to deliver therapeutics based on short interfering RNAs to hepatocytes; however, new delivery solutions are necessary to target additional organs. In a new report now published in Nature Biotechnology, a team of researchers including Kirk M. Brown, Jayaprakash K. Nair, and Maja M. Manas, led by Vasant Jadhav at Alnylam Pharmaceuticals Cambridge MA, U.S., discussed the safe conjugation of 2′-O-hexadecyl (C16) to small interfering RNAs (siRNAs) for potent and durable silencing in target organs of rodents and non-human primates, with broad cell specificity. The experiments delivered sustained RNA interference activity for at least three months. The team observed intravitreal and intranasal administration, which relied on potent and durable knockdown. They investigated the preclinical efficacy of the siRNA targeting amyloid precursor protein via intracerebroventricular dosing in a mouse model of Alzheimer’s disease, which ameliorated physiological and behavioral defects of the disease. The team showed the safety of C16 conjugation of siRNAs for therapeutic silencing of target genes outside the liver.

from Biotechnology News – Biology News https://ift.tt/pvEJBXG