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'Not Dead Yet'

At The New York Times' Prescriptions blog, Andrew Pollack says RNA interference "might not be dead yet." That Alnylam Pharmaceuticals recently announced it had established proof of concept for the efficacy of an RNAi-based drug in a phase 1 clinical trial "at least points to the possibility that the approach can be used in people," Pollack says. Alnylam CEO John Maraganore tells the Times that "this is actually the first time that we or anybody has shown the ability of silencing a disease-causing gene in humans with RNAi." This particular RNAi drug is being tested to treat a rare disease caused by mutations in TTR, which encodes transthyretin. "The mutation causes TTR-mediated amyloidosis, in which abnormal amyloid proteins build up in the body. In one version of the disease, the buildup of proteins damages nerves, and people eventually lose the ability to walk and then to breathe," Pollack says. Alnylam, he adds, reported results from 31 patients – 23 that received a single infusion of the drug designed to inactivate TTR and 8 controls – showing that the RNAi drug worked to depress TTR levels for at least 24 days.

The Scan

Drug Response Variants May Be Distinct in Somatic, Germline Samples

Based on variants from across 21 drug response genes, researchers in The Pharmacogenomics Journal suspect that tumor-only DNA sequences may miss drug response clues found in the germline.

Breast Cancer Risk Gene Candidates Found by Multi-Ancestry Low-Frequency Variant Analysis

Researchers narrowed in on new and known risk gene candidates with variant profiles for almost 83,500 individuals with breast cancer and 59,199 unaffected controls in Genome Medicine.

Health-Related Quality of Life Gets Boost After Microbiome-Based Treatment for Recurrent C. Diff

A secondary analysis of Phase 3 clinical trial data in JAMA Network Open suggests an investigational oral microbiome-based drug may lead to enhanced quality of life measures.

Study Follows Consequences of Early Confirmatory Trials for Accelerated Approval Indications

Time to traditional approval or withdrawal was shorter when confirmatory trials started prior to accelerated approval, though overall regulatory outcomes remained similar, a JAMA study finds.