The death in Pfizer’s Phase I study could reflect wider safety issues for AAV-based gene therapy and could spur intervention from regulators in 2022.
A trial of Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD) has been halted after the death of a young male.
The company announced on 20 December that it had paused screening and dosing in the Phase I study and it will now work with the trial site investigator to understand the cause of death, and if it was linked to the administration of
In this week's episode: AstraZeneca CEO cautions Europe; Pfizer CEO optimistic on US tariffs, pricing; Merck & Co reassures on pipeline; Merck KGaA buys SpringWorks; and Akeso explains ivonescimab overall survival data.
Vertex is in need of diversification, but in addition to its lower-than-expected Q1 revenue, sales of the company’s newest CF drug Alyftrek and pain medicine Journavx disappointed.
The biotech hopes a third time will be the charm after two FDA complete responses, with plans to position its eye drop as offering quicker onset of action.
The pharma company is helping to validate the biotech’s model by developing a novel amyotrophic lateral sclerosis candidate, based on its novel approach that ‘turns drug discovery on its head’.
Vertex is in need of diversification, but in addition to its lower-than-expected Q1 revenue, sales of the company’s newest CF drug Alyftrek and pain medicine Journavx disappointed.
The biotech hopes a third time will be the charm after two FDA complete responses, with plans to position its eye drop as offering quicker onset of action.
The biotech said it will have a type A meeting with the agency to resubmit for the cell therapy’s approval in EBV+ PTLD.
