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luspatercept + thalidomide
- Development stage
- Unknown
- Lead developer
- Bristol Myers Squibb
- Modality
- Small Molecules, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Administration
- Subcutaneous, Oral
01
Overview
luspatercept + thalidomide is a combination therapy being investigated for the treatment of transfusion-dependent β-thalassemia. Luspatercept is a recombinant fusion protein that acts as an erythroid maturation agent by binding specific TGF-β superfamily ligands (such as GDF11 and activin B), thereby inhibiting SMAD2/3 signaling and promoting late-stage erythropoiesis. Thalidomide is an immunomodulatory small molecule that, in the context of hemoglobinopathies, is used at low doses to induce fetal hemoglobin (HbF) production, potentially through the modulation of the cereblon (CRBN) E3 ubiquitin ligase complex. This combination, currently being evaluated in a Phase 2 trial sponsored by Rongrong Liu, aims to provide a synergistic effect by both enhancing red blood cell maturation and increasing hemoglobin levels to reduce transfusion requirements.
- Other names
- luspatercept-aamt
02
Targets
BMP6 (Bone morphogenetic protein 6)GDF8 (Myostatin)CRBN (Cereblon)GDF11 (Growth differentiation factor 11)INHBB (Activin B)
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