As Wall Street ponders how pricing and reimbursement for gene therapies might shake out, companies in the field march intrepidly on, among them Orchard Therapeutics Inc., which early this month rolled out heartening results from an ongoing proof-of-concept trial evaluating the safety and efficacy of OTL-203 for mucopolysaccharidosis type I (MPS-I), a space that's heating up.

Findings disclosed from Synlogic Inc.'s phase Ib/IIa trial with SYNB-1020 in hyperammonemia surprised the company and Wall Street, dealing a blow to the compound but leaving in place the Synthetic Biotic platform, which genetically engineers probiotic microbes.

Abbott Laboratories has kicked off a pivotal study to evaluate its catheter-based Triclip transcatheter tricuspid valve repair system in patients with severe tricuspid regurgitation (TR). The first enrollments in the U.S. FDA-approved Triluminate study were performed at Abbott Northwestern Hospital in Minneapolis by a cardiac team led by Paul Sorajja, director of the Center for Valve and Structural Heart Disease at the Minneapolis Heart Institute and co-primary investigator for the trial.

Following its phase II stumble of AR-105, Aridis Pharmaceuticals Inc. is dropping its development of the IgG1 monoclonal antibody. The study failed to meet its primary endpoint of showing superiority in clinical cure rates compared to placebo for treating ventilator-associated pneumonia caused by gram-negative Pseudomonas aeruginosa.

DUBLIN – Shares in Pharnext SA plunged by more than 40% Friday on news that the FDA requires the company to undertake a second phase III trial of its Charcot-Marie-Tooth disease type 1A (CMT1A) therapy PXT-3003.

HONG KONG – Following a regulatory committee's ruling that three of four deaths previously reported during a phase II trial were unrelated to the study drug, South Korea's Ministry of Food and Drug Safety (MFDS) cleared Yuhan Corp. to continue testing lazertinib (YH-25448) in the non-small-cell lung cancer (NSCLC) study.

PERTH, Australia – San Diego-based Okogen Inc. is taking its clinical trial program to Australia where it will test its lead compound OKG-0301 for acute adenoviral conjunctivitis in the phase II (RUBY) trial.

PERTH, Australia – Telix Pharmaceuticals Ltd. raised AU$40 million as it prepares regulatory packages and commercial strategies in Europe, the U.S. and Australia.

What the phase III multiple sclerosis (MS) win scored by Biogen Inc. and partner Alkermes plc might mean in the marketplace for diroximel fumarate – tested against their approved MS therapy Tecfidera (dimethyl fumarate) – became fodder for speculation as investors weighed the fine points of side-effect profiles and would-be reimbursement hitches.