The patient, who was being treated with an investigational gene therapy for limb-girdle muscular dystrophy, died of acute ...

The panelists flagged safety concerns with Blenrep and GSK’s failure to optimize its dosing regimen for the antibody-drug ...

In its Q2 earnings call Thursday, Novartis said it is moving quickly to reshore its drug manufacturing operations, but CEO ...

Sarepta Therapeutics appears to have right-sized itself after laying off over a third of its staff, announcing a significant ...

The rollercoaster week for Sarepta Therapeutics continued, with shares of the embattled gene therapy-turned-siRNA biotech down 37% Friday afternoon as media outlets reported FDA plans to request a ...

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...

Participants in trials of BrainStorm Cell Therapeutics' NurOwn filed a Citizens’ Petition with the FDA earlier this month ...

In advance of CMS’ negotiated price for the blood thinner taking effect next year, partners Bristol Myers Squibb and Pfizer ...

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...

The molecule, developed in collaboration with Massachusetts-based Kailera Therapeutics, is headed for a new drug application ...

Seven biotech unicorns are advancing AI-powered drug discovery and development—but must contend with a difficult investing ...

Takeda’s oveporexton improved wakefulness, attention and other key narcolepsy endpoints “with a high degree of statistical ...