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Betibeglogene autotemcel - bluebird bio

Drug Profile

Betibeglogene autotemcel - bluebird bio

Alternative Names: Autologous CD34+ cells encoding βA-T87Q-globin gene therapy - bluebird bio; bb1111; BB305-beta-A(T87Q)-globin-gene-therapy-bluebird-bio; Beta-globin-gene-therapy-bluebird-bio; beti-cel; HPV569-beta-A(T87Q)-globin-gene-therapy-bluebird-bio; HPV569-beta-globin-gene-therapy-bluebird-bio; LentiGlobin gene therapy; LentiGlobin-HPV569; LentiGlobin™ bb1111; LentiGlobin™ BB305; Lentiviral beta AT87Q-Globin vector; lovo-cel; Lovotibeglogene autotemcel - bluebird bio; LYFGENIA; ZYNTEGLO; Zynteglo

Latest Information Update: 23 Feb 2024

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At a glance

  • Originator Genetix Pharmaceuticals
  • Developer bluebird bio
  • Class Gene therapies; Haematopoietic stem cells therapies
  • Mechanism of Action Cell replacements; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Beta-thalassaemia; Sickle cell anaemia
  • New Molecular Entity No

Highest Development Phases

  • Marketed Beta-thalassaemia
  • Registered Sickle cell anaemia

Most Recent Events

  • 30 Jan 2024 bluebird bio completes a phase I/II HGB-206 trial in Sickle cell anaemia in the USA (NCT02140554)
  • 11 Dec 2023 Pooled efficacy, adverse events, pharmacodynamics data from the phase I/II HGB-206 and phase III HGB-210 trial in Sickle cell anaemia released by Bluebird Bio
  • 10 Dec 2023 Efficacy and adverse events data from a phase III long-term follow up trial in Beta-thalassaemia released byBluebird Bio
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