Bluebird Bio's Shares Drop To 52-Week Low After Another Gene Therapy Trial Placed On FDA Hold
- Shares for Bluebird Bio Inc (NASDAQ: 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.
© 2025 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Posted-In: Biotech Earnings News Short Ideas Health Care Small Cap FDA Movers Best of Benzinga
