Aruvant is developing ARU-1801, a one-time, potentially curative gene therapy for sickle cell disease and β-thalassemia. In an ongoing clinical phase 1/2 study, ARU-1801, administered with only ...

Betibeglogene autotemcel gene therapy led to transfusion independence in 89% of patients with severe transfusion-dependent β-thalassemia. The trial involved 18 patients with specific genotypes ...

Patients with the rare blood disorders transfusion-dependent β-thalassemia (TDT) have shown spectacular results in clinical trials of bluebird bio's gene therapy, raising hopes the treatment ...

Earlier this year, the US Food and Drug Administration (FDA) approved the first potentially curative gene therapy to treat beta-thalassemia. While multiple companies have been working to bring ...

Yoltech Therapeutics Co. Ltd. has advanced YOLT-204 into the clinic for the treatment of transfusion-dependent β-thalassemia (TDT). If successful, YOLT-204 may provide an off-the-shelf curative ...

who is now able to hear thanks to gene therapy ... approval of a CRISPR therapy designed for patients suffering from sickle cell anemia and the blood disorder beta thalassemia.

SHANGHAI, Jan. 21, 2025 /PRNewswire/ -- YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204, ...

an investigational therapy for the treatment of transfusion-dependent beta-thalassemia (TDT). TDT is a severe genetic blood disorder where mutations in the beta-globin gene leads to reduced or ...

SHANGHAI, Jan. 20, 2025 /PRNewswire/ -- YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204, ...