Potential Cure for Sickle Cell Clears Hurdle for Approval
An FDA advisory committee meeting on Tuesday did nothing to dispel expectations that the investigational agent exagamglogene autotemcel (exa-cel) will become the first approved gene therapy for sickle cell disease (SCD). Members of the Cellular, Tissue, and Gene Therapies Advisory Committee were not asked to vote on the agent’s efficacy or safety, but instead directed […]