Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease

Q: What does this research mean for the field?

Treatment with risto-cel, a base editing therapy targeting HBG1 and HBG2 promoters, leads to rapid engraftment, durable expression of fetal hemoglobin (HbF), and a reduction in sickle hemoglobin (HbS) in patients with sickle cell disease. Novelty: ClaimNovelty.NOVEL_FINDING. Consensus alignment: ConsensusAlignment.NEUTRAL.

Ashish O. Gupta; Akshay Sharma; Haydar Frangoul; J Kanter; Markus Y. Mapara; Jignesh Dalal; Asif Alavi; Jennifer Jaroscak; Ernesto Ayala; John F. DiPersio; Edward D. Ziga; Mary Eapen; Stacey Rifkin-Zenenberg; Alex Minella; Yinzhong Chen; Sarah Chesler; Srikanth Ambati; Thomas S. Bowman; Bahru Habtemariam; Marcelyne Joseney-Antoine; Priya S. Chockalingam; Ling Lin; Sunita Goyal; Amy Simon; Alexis A. Thompson; Matthew M. Heeney

doi:10.1056/nejmoa2504835

Key Points

To evaluate the efficacy of risto-cel in enhancing hemoglobin levels and reducing sickle hemoglobin in sickle cell disease.
Administered risto-cel treatment to evaluate its effects on hemoglobin expression.
Monitored engagement of patients to assess engraftment and expression of hemoglobin types.
Assessed endpoints such as HbF and HbS levels post-treatment.
Rapid engraftment of the treatment was observed.
Durable expression of fetal hemoglobin (HbF) was confirmed.
Significant reduction in sickle hemoglobin (HbS) levels associated with treatment.

Implication

Clinical investigation demonstrates improved hemoglobin expression in patients with sickle cell disease, suggesting a promising treatment avenue.

Abstract

Treatment with risto-cel was followed by rapid engraftment and durable expression of HbF and reduction in HbS. These data support further investigation of risto-cel to treat sickle cell disease. (Funded by Beam Therapeutics; BEACON ClinicalTrials.gov number, NCT05456880.).

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Cite This Study

Gupta et al. (2026) studied this question.

synapsesocial.com/papers/69d1fb20a79560c99a0a17cd https://doi.org/10.1056/nejmoa2504835

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Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease

Key Points

Implication

Abstract

Discussion

Cite This Study

Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease

Key Points

Implication

Abstract

Discussion

Cite This Study

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