Shots:
- Yoltech reported the initiation of a dose-escalation study for YOLT-204 (QD) to evaluate its safety & efficacy as an off-the-shelf treatment of transfusion-dependent beta-thalassemia (TDT) without CT & HSCT
- In preclinical studies, YOLT-204 showed consistent expression of fetal hemoglobin, associated with reduced polymerization of sickle hemoglobin, fewer complications, & lower mortality. These findings highlight its potential as a therapy for TDT & SCD
- YOLT-204 boosts RBC count by targeting the regulatory region of hemoglobin to activate fetal hemoglobin expression via targeted delivery to hematopoietic stem cells using YolTech’s lipid nanoparticles
Ref: Prnewswire | Image: Yoltech
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