Takeda Pharmaceutical Co. Ltd. is exiting all work in cell therapies and will seek an external partner to advance the company’s research and clinic-ready cell therapy programs, the company said, noting that it is not currently running any clinical trials using cell therapy technology.
Synendos Therapeutics AG has delivered initial clinical data demonstrating the potential of its lead compound to selectively modulate the endocannabinoid system in the brain and to relieve anxiety symptoms without triggering a psychoactive response.
Phase IIb data of Metsera Inc.’s lead GLP-1 receptor agonist, MET-097i, showed significant weight loss and good tolerability, supporting a phase III start later this year, and validating Pfizer Inc.’s decision a week ago to buy the obesity-focused company for $7.3 billion.
Opus Genetics Inc. will be sitting down with the U.S. FDA to talk about positive three-month data from the pediatric cohort of its ongoing phase I/II trial called OPGx-LCA5-1001 – partially funded by the agency – evaluating OPGx-LCA5, a gene augmentation therapy for ultra-rare Leber congenital amaurosis type 5 (LCA5). The affliction is a severe form of retinal dystrophy that renders babies blind in the first year of life.
Up to 71% of people carry at least one pathogenic variant that could contribute to development of a heritable disorder in offspring, but until now, prospective parents often had to undergo multiple tests to understand their risks. Pacific Biosciences of California Inc. (Pacbio)’s expanded Puretarget portfolio provides a quicker and more streamlined solution as it covers all challenging tier 3 genes identified in the American College of Medical Genetics technical standard.
Anaphylaxis rates caused Larimar Therapeutics Inc.’s stock (NASDAQ:LRMR) to take a hit on the latest data from an open-label study with nomlabofusp in the neuromuscular disease Friedreich’s ataxia (FA), but the company is targeting a BLA submission to seek accelerated approval in the second quarter of next year.
Enanta Pharmaceuticals Inc.’s phase IIb study of zelicapavir missed its primary endpoint in treating respiratory syncytial virus (RSV) but mined enough positive results to get the company to talk about advancing the once-daily oral treatment into further, larger studies.
Bar what it described as a “placebo wobble,” Moonlake Therapeutics AG turned in positive results from the phase III trials of sonelokimab in treating hidradenitis suppurativa (HS), only to see its share price take a complete battering. The stock (Nasdaq:MLTX) fell 90%, or $55.75, to close Sept. 29 at $6.24, after the Zug Switzerland-based company published 16-week data from two identical trials, Vela-1 and Vela-2.
A month after raising $55 million to advance its AI-enhanced ultrasound diagnostics for cardiovascular disease, Ultromics Ltd. presented strong results for its U.S. FDA-cleared AI analysis of echocardiograms at the American Society of Echocardiography's 2025 Scientific Sessions in Nashville, Tenn. Ultromics' Echogo Amyloidosis was able to detect cardiac amyloidosis earlier and more accurately than traditional methods, missing fewer cases without increasing unnecessary testing.
Xeltis BV reported successful clinical data from the Axess EU pivotal trial which demonstrated the transformative potential of its restorative vascular access conduit, Axess, in hemodialysis treatment.