Drug Discovery

CEL-031 is in preclinical testing for the nonmuscle invasive form of the disease.

DBV’s Viaskin platform is already undergoing human testing in patients with peanut and milk allergies.

BMS plans to submit an NDA for mavacamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy to the FDA in the first quarter of 2021; based on positive data from the Phase III EXPLORER-HCM trial announced in May.

Firms will codevelop microbiology assays on multiplex platform acquired from Philips.

Four hospitalizations and deaths, or “events,” occurred in patients 29 days after treatment with the bamlanivimab-etesevimab combination, compared with 15 reported in patients given placebo.

A proteomic survey has identified the phosphorylation-driven host cell signaling changes that occur upon infection with SARS-CoV-2. Conspicuous changes include modifications in growth factor receptor (GFR) signaling and downstream pathways. In cell cultures, interruption of these pathways with existing drugs, which happen to be anticancer drugs, was found to prevent viral replication.

Benitec will use ddRNAi Platform to develop follow-on compounds to lead EGFR-AS candidate

Modified liposomes demonstrated thermostable cargo retention as well as effective release upon exposure to clinically relevant doses of near-infrared radiation.

The results of an open-label pilot study found that patients with an advanced form of age-related macular degeneration (AMD) demonstrated improved vision and stabilized disease following treatment using the Parkinson’s disease therapy, levodopa. The study demonstrated that levodopa was safe and well-tolerated, and reduced the need for patients with neovascular AMD (nAMD) to receive expensive, painful anti-VEGF injections. These early findings point to the potential use of levodopa as an effective adjuvant therapy to anti-VEGF injections for nAMD.

A team led by researchers at Sangamo Therapeutics has shown the first, direct, demonstration of allele-selective transcriptional repression at the huntingtin locus. This opens up a novel therapeutic approach to targeting the gene responsible for Huntington's disease.