Novartis’ offer comes three months after Gyroscope presented at a medical meeting early data from 13 patients who had been dosed with the gene therapy, …

Huntington’s was the first illness ever traced back to changes in a specific chromosome. And yet, almost 40 years since that discovery, no drug has …

The FDA’s acceptance of Bluebird’s application, which comes two months after the company submitted it to the agency, marks the final stretch of a long …

In both hemophilia and Duchenne, unexpected findings have led Pfizer to redraw its development plans. The company recently paused patient screening and dosing in a …

SGS has just completed a significant expansion project at its Glasgow facility, meaning it can provide increased biosafety testing for the biologics, drug development, and …

Boston biotech Vertex is the leading developer of cystic fibrosis medicines. Its four approved drugs can treat most patients. Trikafta, the one most recently approved …

Voyager’s first generation of gene therapies for ALS, Parkinson’s and Huntington’s disease, all now discontinued, relied on a direct infusion into brain tissue, an invasive …

Although Pfizer beat Sarepta to start a Phase 3 trial of its gene therapy, the smaller biotech is claiming a milestone in initiating the study …

While the clinical hold lifting is encouraging news for the companies’ partnership, questions still loom about safety in the genetic medicine field. Earlier this month, …

The theoretical risk that gene therapy may cause cancer in humans has hung over the field for decades. Those worries resurfaced last year when a …

Rocket’s program has been in limbo since early May, when the company surprised investors and analysts with news of the trial’s stoppage. That announcement stalled …

Oxford Genetics, a leading innovator of biopharmaceutical technologies, has signed a licensing agreement with world-class service organization, Aldevron to bring to market a standardized range …