Skip to main content
Premium Trial:

Request an Annual Quote

Alnylam Begins Phase I Trial of TTR Amyloidosis Drug

Premium

Alnylam Pharmaceuticals announced this week that it has begun dosing patients in a phase I trial of its transthyretin-mediated amyloidosis drug ALN-TTR01.

The study is being conducted in Portugal, Sweden, and the UK and will enroll about 28 patients with the disease.

The primary objective of the trial is to evaluate the safety and tolerability of a single dose of intravenous ALN-TTR01, with patients being enrolled into five sequential cohorts of increasing doses ranging from 0.01 to 0.4 mg/kg, Alnylam said. Secondary objectives include characterization of plasma and urine pharmacokinetics of ALN-TTR01, and assessment of pharmacodynamic activity based on measurements of circulating TTR serum levels.

TTR amyloidosis is a hereditary, systemic disease caused by a mutation in the transthyretin gene. ALN-TTR01 is a systemically delivered siRNA designed to inhibit both mutant and wild-type TTR. It is delivered using partner Tekmira Pharmaceutical's stable nucleic acid-lipid particle technology.

The Scan

Panel Recommends Pfizer-BioNTech Vaccine for Kids

CNN reports that the US Food and Drug Administration advisory panel has voted in favor of authorizing the Pfizer-BioNTech SARS-CoV-2 vaccine for children between 5 and 11 years old.

Sharing How to Make It

Merck had granted a royalty-free license for its COVID-19 treatment to the Medicines Patent Pool, according to the New York Times.

Bring it Back In

Bloomberg reports that a genetic analysis has tied a cluster of melioidosis cases in the US to a now-recalled aromatherapy spray.

Nucleic Acids Research Papers on SomaMutDB, VThunter, SCovid Databases

In Nucleic Acids Research this week: database of somatic mutations in normal tissue, viral receptor-related expression signatures, and more.