The U.S. Food and Drug Administration (FDA) is reviewing an application that seeks the approval of exagamglogene autotemcel, a CRISPR-based…
Articles by Margarida Maia, PhD
A protein called NLRP12 appears to act as a switch that turns on inflammation and tissue damage when it senses…
People in Ghana with sickle cell disease (SCD) use prevention, monitoring, diagnosis, and treatment to self-manage their condition, but…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EDIT-301, an experimental cell-based gene-editing therapy given…
Bluebird Bio is seeking approval from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate lovotibeglogene…
AddMedica and Abacus Medicine Pharma Services have partnered to market and distribute the sickle cell disease (SCD) therapy…
People with sickle cell disease (SCD) may see their physical, mental, and social health get better after a…
Bluebird bio will resume its study of lovotibeglogene autotemcel (lovo-cel) in children and adolescents with sickle cell disease…
A first patient has been enrolled in BEACON, a Phase 1/2 clinical trial into the safety and efficacy of BEAM-101,…
Vertex Pharmaceuticals and CRISPR Therapeutics are planning a November launch for a biologics license application (BLA) for their gene-editing therapy…