France-based clinical research company Aixial Group is consolidating clinical data from one of its business units into Medidata’s digital platform, according to a Sept. 3 release.

After escaping a clinical hold several years back, Dyne Therapeutics has revealed new phase 1/2 data for its Duchenne muscular dystrophy (DMD) therapy DYNE-251. The readout notes several serious treatment-emergent adverse events (TEAEs), including acute kidney injury.

Arrowhead Pharmaceuticals has shown its hand ahead of a potential showdown with Ionis, publishing phase 3 data on a rare metabolic disease treatment that is racing toward regulators.

Sanofi is still set on taking its multiple sclerosis (MS) med tolebrutinib to the FDA, executives have told Fierce Biotech, despite the BTK inhibitor falling short in two of three phase 3 trials that read out Monday.

Bayer suspended the phase 3 trial for its factor XIa inhibitor asundexian late last year after the drug showed “inferior efficacy” at preventing strokes in patients with atrial fibrillation compared to Bristol Myers Squibb and Pfizer’s Eliquis. The full picture of what that “inferior efficacy” looks like has now come into focus: Patients receiving asundexian actually suffered strokes or systemic embolisms at a higher rate than those receiving Eliquis.

Many microbes are extremophiles, capable of surviving conditions that would spell instant death for you and me. From deep-sea hydrothermal vents to boiling hot springs, these single-celled creatures have evolved special tools to help them thrive in tough places. Researchers have long wanted to harness these tools for human applications.

In 2022, on the alpine shores of Lake Maggiore in northern Italy, longtime friends Samuel Bakhoum, M.D., Ph.D., and Stefano Santaguida, Ph.D., were catching up. Pandemic restrictions had prevented the two from seeing each other for years, but, at a small conference in the lakeside town of Stresa, they seized the opportunity to share what they’d been working on recently—the collapse of micronuclei in cancer cells.

Qiagen and AstraZeneca are expanding their diagnostics partnership, with plans to explore the use of genomic profiling in chronic diseases beyond cancer.

Gain Therapeutics has set its sights on proving the effectiveness of its Parkinson’s disease therapy next year after the brain-penetrant small molecule demonstrated “peripheral target engagement” in a phase 1 trial.

Johnson & Johnson has taken another step toward realizing a return on its $6.5 billion nipocalimab bet, filing for FDA approval to challenge argenx and UCB for the generalized myasthenia gravis (gMG) market.