YolTech Therapeutics, a Shanghai-based start-up focusing on in vivo gene editing, recently announced the initiation of an early Phase I trial with YOLT-204, a first-in-class therapy, in transfusion dependent β-thalassemia (TDT).
The dose-escalation study will provide a preliminary assessment of the safety, tolerability and efficacy of a single dose in three to nine Chinese patients, and if successful may pave the way for an off-the-shelf treatment for TDT
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