Clinical research
PepGen’s lead candidate for myotonic dystrophy type 1 barely beat the placebo in a Phase 2 trial in terms of fixing incorrect gene splicing, but the biotech attributed the poor result to an outlier.
Presentations at the 2026 meeting of the American Academy of Dermatology not only demonstrate the therapeutic potential of next-generation skin drugs but also shed light on how they might fare on the market.
Despite hitting its primary endpoint, Viridian’s thyroid eye disease antibody failed to ease eye bulging to the degree that analysts had been hoping for, and the biotech’s stock price fell by one-third.
Merck is eyeing a quick review for its lipid-lowering drug candidate enlicitide, which in December was awarded a Commissioner’s National Priority Voucher.
While tonlamarsen missed one of two co-primary endpoints, Kardigan says the drug has shown a clinically meaningful effect on blood pressure, supporting advancement into Phase 2b.
William Blair hailed a positive readout in cutaneous lupus erythematosus as a turning point for Biogen, while RBC Capital analysts called the results “another derisking step” for the company’s immunology and inflammation pipeline.
The lack of a dose-response effect could be due to the high number of dropouts in the higher-dose Winrevair arm and the relatively small study population, a discussant for Merck explained.
While participants on a lower dose of Wave Life Sciences’ RNA therapy lost 5.3% total fat at the six-month mark, those receiving the higher dose saw a less than 1% drop at three months.
Beam is now in the strongest position of all companies advancing a genetic therapy for alpha-1 antitrypsin deficiency, according to analysts at William Blair.
In this episode of Denatured, you’ll listen to Sergey Jakimov, managing partner at LongeVC and Artem Trotsyuk, operating partner, US, LongeVC. We speak about how developers in the longevity space should stop chasing aging as an abstract target and concentrate on specific mechanisms that can clearly tackle age-related conditions.
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