FDA Hits Bluebird Bio’s Sickle Cell Gene Therapy With Clinical Hold

The FDA has placed a partial clinical hold on Bluebird Bio’s gene therapy for sickle cell disease (SCD), lovotibeglogene autotemcel (lovo-cel), for trial participants under the age of 18 after a volunteer contracted persistent anemia.

The company said the patient, who was diagnosed 18 months following treatment with lovo-cel, is “clinically well” and that enrollment and dosing of SCD patients 18 years and older in three separate clinical studies will continue “as planned.” Follow-ups for treated patients of any age will also still occur.

Bluebird Bio pledged to work with the FDA to resolve the hold and said it expects to receive written questions from the agency on the program early next year.