Safety Concerns Hit Bluebird Sickle Cell Trial, Again
The FDA placed a partial hold on its clinical program for lovo-cel due to an adolescent patient experiencing anemia 18 months after treatment, raising uncertainty over filing plans.
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The company built on its case for FDA approval with new beta-thalassemia data at ASH, but investor jitters linger after the pullout of Zynteglo from Europe amid reimbursement woes for the gene therapy.
The company has initiated the first pivotal double-blind gene therapy trial in DMD in the US in a tight race with Pfizer.
Lack of reference standards inhibits ability to make comparisons between products or set caps for total vector genome dose or total capsid dose, advisory committee says; panelists suggest other approaches to prevent and mitigate toxicities, including running longer-term animal studies and investigating the efficacy of immunosuppression prophylaxis and treatment strategies.