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Betibeglogene autotemcel - bluebird bio

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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: 21 Aug 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 - Sickle cell anaemia; Beta-thalassaemia
  • New Molecular Entity No

Highest Development Phases

  • Marketed Beta-thalassaemia; Sickle cell anaemia

Most Recent Events

  • 14 Aug 2024 Launched for Beta-thalassaemia (In adolescents, In children, In infants, In neonates, In adults) in USA (IV) prior to August 2024
  • 14 Aug 2024 Launched for Sickle cell anaemia (In adolescents, In children, In adults) in USA (IV) prior to August 2024
  • 30 Jan 2024 bluebird bio completes a phase I/II HGB-206 trial in Sickle cell anaemia in the USA (NCT02140554)

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