Skip to main content
Premium Trial:

Request an Annual Quote

Alnylam Presents New Preclinical Data on Complement-mediated Disease Drug


NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals last week released new preclinical data on ALN-CC5, its siRNA-based treatment for complement-mediated diseases, showing that the drug is effective in silencing its target and may offer therapeutic benefit in an associated kidney disorder.

In a study of non-human primates, ALN-CC5 was administered subcutaneously either monthly or twice a month. At 5 mg/kg, either dosing regimen led to as much as 98.7 percent clamped knockdown of serum levels of the C5 component of the complement pathway. It also inhibited complement activity by up to 91.3 percent.

Based on translational data of its second-generation GalNAc delivery conjugates, which will be used with the drug, Alnylam said that doses less than 1 mg/kg and 1 mL/injection are expected to have similar effects in humans.

Meanwhile, in a rat model of primary membranous nephropathy — a complement-mediated progressive disease of the kidney — ALN-CC5 treatment led to a greater than 70 percent reduction in proteinuria associated with complement-mediated renal damage.

The data were presented at the 25th International Complement Workshop in Brazil.

Alnylam said that it remains on track to file a regulatory application to begin testing ALN-CC5 in humans before the end of this year, with initial clinical data available by mid-2015.

The Scan

NFTs for Genome Sharing

Nature News writes that non-fungible tokens could be a way for people to profit from sharing genomic data.

Wastewater Warning System

Time magazine writes that cities and college campuses are monitoring sewage for SARS-CoV-2, an approach officials hope lasts beyond COVID-19.

Networks to Boost Surveillance

Scientific American writes that new organizations and networks aim to improve the ability of developing countries to conduct SARS-CoV-2 genomic surveillance.

Genome Biology Papers on Gastric Cancer Epimutations, BUTTERFLY, GUNC Tool

In Genome Biology this week: recurrent epigenetic mutations in gastric cancer, correction tool for unique molecular identifier-based assays, and more.