Luspatercept ameliorates disease phenotype and complications in the Townes mouse model of sickle cell disease

J Clin Invest. 2026 Feb 2;136(3):e197706. doi: 10.1172/JCI197706.

Abstract

Preclinical data demonstrating that luspatercept mitigates vaso-occlusive and anemia-related complications in the Townes sickle cell model provide a strong rationale for its evaluation in SCD patients.

Keywords: Hematology; Hematopoietic stem cells; Immunology.

MeSH terms

  • Activin Receptors, Type II
  • Anemia, Sickle Cell* / complications
  • Anemia, Sickle Cell* / drug therapy
  • Anemia, Sickle Cell* / genetics
  • Anemia, Sickle Cell* / pathology
  • Animals
  • Disease Models, Animal
  • Humans
  • Immunoglobulin Fc Fragments* / pharmacology
  • Mice
  • Phenotype
  • Recombinant Fusion Proteins* / pharmacology
  • Vaso-Occlusive Crises

Substances

  • Recombinant Fusion Proteins
  • luspatercept
  • Immunoglobulin Fc Fragments
  • Activin Receptors, Type II