Skip to main content
Premium Trial:

Request an Annual Quote

Investigational RNAi Drug Lowers Blood Pressure in Phase I Study

An RNA interference therapy for hypertension may decrease serum angiotensinogen and blood pressure levels, findings from a Phase I study suggest. In a multi-part, multicenter study funded by Alnylam Pharmaceuticals, researchers from the US and UK aimed to assess the safety, study the pharmacokinetic and pharmacodynamic characteristics, and examine the antihypertensive efficacy of zilebesiran, an investigation RNAi agent designed to bind to and reduce the levels of hepatic angiotensinogen mRNA. They enrolled 107 patients with hypertension into the study, with patients in part A receiving a single ascending dose of zilebesiran or placebo, and patients in parts B and E receiving zilebesiran alongside a certain diet or another hypertension treatment, respectively. As the researchers report in the New England Journal of Medicine, patients receiving zilebesiran experienced declines in serum angiotensinogen levels as well as in blood pressure, and only reported mild injection site reactions. The researchers further note that patients in parts B and E — part D is ongoing and part C was removed from the protocol — indicated that the treatment could be attenuated by a high-salt diet and augmented by simultaneous irbesartan treatment. "Overall, these preliminary data regarding efficacy and safety support the potential for further study of quarterly or twice-yearly administration of zilebesiran as a treatment for patients with hypertension," the researchers report.

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.