Bluebird Bio's Shares Drop To 52-Week Low After Another Gene Therapy Trial Placed On FDA Hold

Shares for Bluebird Bio Inc BLUE is hovering at a 52-week low after the FDA placed a clinical hold on clinical studies of elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for cerebral adrenoleukodystrophy (CALD).
The company reported an adverse reaction to myelodysplastic syndrome (MDS), which is likely mediated by Lenti-D lentiviral vector (LVV) insertion, in a patient treated with eli-cel for CALD over one year ago in Phase 3 ALD-104 study.
Evidence currently available suggests that specific design features of Lenti-D LVV likely contributed to this event.
The company does not anticipate the clinical hold to impact its sickle cell disease (SCD) programs, β-thalassemia, or oncology.
Cash, cash equivalents, and marketable securities as of June 30 were $941.6 million.
In July, the European Commission approved Skysona (eli-cel), one-time gene therapy for early CALD in patients less than 18 years of age with an ABCD1 genetic mutation.
In June, the FDA lifted the clinical hold on sickle cell disease and transfusion-dependent β-thalassemia trials of betibeglogene autotemcel gene therapy.
The programs were placed on clinical hold after they reported an incidence of acute myeloid leukemia in a patient treated more than five years ago.
Price Action: BLUE shares are down 23.3% at $19.21 during the market session on the last check Monday.
Related content: Benzinga's Full FDA Calendar.

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