The FDA placed a clinical hold yesterday on two gene therapy trials for sickle cell disease (SCD) after one participant developed acute myeloid leukemia (AML) and another developed myelodysplastic syndrome (MDS). The sponsoring company, bluebird bio, suspended the trials last week upon learning of the cases.

The company has also put the brakes on a treatment for beta thalassemia already approved in the European Union and the United Kingdom, betibeglogene autotemcel (Zynteglo). The treatment hasn't been associated with problems but uses the same gene delivery vector, a lentivirus, as that used in the SCD trials.

Overall, the company has enrolled 47 SCD patients and 63 with beta thalassemia in trials.

The gene therapy "space" is one with spectacular successes — for a form of retinal blindness and spinal muscular atrophy — rising against a backdrop of recent setbacks and failures — for Duchenne muscular dystrophy, lipoprotein lipase deficiency, and myotubular myopathy.

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