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Clinical Trial Updates (SCD)

Risto-cel

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  • Product Information

    Product Information

    Scientific name: Ristoglogene autogetemcel (risto-cel, BEAM-101)
    Brand name: Ν/Α
    RESPONSIBLE: Beam Therapeutics Inc.

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Trial Name: BEACON
    Code: NCT05456880
    Phase: 1/2
    Eligible patient diagnosis: Adults and adolescents (12 – 35 years) with sickle cell disease (SCD) experiencing at least 4 severe vaso-occlusive crises (VOC) in 24 months before screening
    No. of Patients enrolled: 15 [anticipated]
    Study Sites: 19 Sites per country

    Completion date: February 2028
    Scope of the Study / Aim: Evaluation of the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD.

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

     

    Additional notable points:

    • EMA: N/A
    • FDA: Orphan drug designation (2025); Regenerative Medicine Advanced Therapy (2025)
    • MHRA: N/A
Update: 30 June 2026

Ristoglogene autogetemcel (risto-cel, BEAM-101), an investigational cell therapy using base editing enabling re-expression of γ-globin and production of anti-sickling fetal hemoglobin (HbF).

Data presented at the EHA Annual Congress (11 – 14 June 2026) from the BEACON phase 1/2 clinical trial for patients with SCD and frequent VOCs showed that:
• 43 patients with follow-up ranging from 1 – 24 months had been infused (cut-off: Jan 2026).

• The rate of sickling and max induced sickling were reduced post-infusion, demonstrating restoration of RBC health and function, thus correcting the SCD pathophysiology.

Source: Ristoglogene Autogetemcel Treatment Restored Red Blood Cell Health And Function in Patients with Sickle Cell Disease, with Sickling and Rheology Parameters Comparable to Sickle Cell Trait

 

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