Skip to main content
Premium Trial:

Request an Annual Quote

Alnylam Data Shows 'Predictable Correlation' of Clinical, Preclinical Findings on Cancer Drug

Premium

Alnylam Pharmaceuticals this week released new data from an ongoing phase I study of its liver cancer drug ALN-VSP02.

The drug comprises two siRNAs, one targeting vascular endothelial growth factor and one targeting kinesin spindle protein, both of which the firm said are critical for the growth and survival of cancer cells. It is formulated in a first-generation lipid nanoparticle developed by partner Tekmira Pharmaceuticals.

According to Alnylam, new human pharmacokinetic data show "a clear relationship of human and preclinical animal research findings."

Previous studies indicated that human pharmacokinetic parameters for the drug were dose proportional with no evidence of cumulative effects upon patient re-dosing. "Based on new comparative analyses, human [pharmacokinetics] of ALN-VSP02 were found to correlate within a factor of only one- to two-fold with … results from pre-clinical animal research studies," Alnylam said.

"These new data thus demonstrate a predictable level of human exposure for RNAi therapeutics" using Tekmira's technology, it added.

The Scan

Study Points to Tuberculosis Protection by Gaucher Disease Mutation

A mutation linked to Gaucher disease in the Ashkenazi Jewish population appears to boost Mycobacterium tuberculosis resistance in a zebrafish model of the lysosomal storage condition, a new PNAS study finds.

SpliceVault Portal Provides Look at RNA Splicing Changes Linked to Genetic Variants

The portal, described in Nature Genetics, houses variant-related messenger RNA splicing insights drawn from RNA sequencing data in nearly 335,700 samples — a set known as the 300K-RNA resource.

Automated Sequencing Pipeline Appears to Allow Rapid SARS-CoV-2 Lineage Detection in Nevada Study

Researchers in the Journal of Molecular Diagnostics describe and assess a Clear Labs Dx automated workflow, sequencing, and bioinformatic analysis method for quickly identifying SARS-CoV-2 lineages.

UK Team Presents Genetic, Epigenetic Sequencing Method

Using enzymatic DNA preparation steps, researchers in Nature Biotechnology develop a strategy for sequencing DNA, along with 5-methylcytosine and 5-hydroxymethylcytosine, on existing sequencers.