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EdiGene Announces Completion of Last Patient Dosing in Phase I Clinical Trial of ET-01, its Investigational Gene-editing Hematopoietic Stem Cell Therapy for Transfusion Dependent β-thalassemia

Excerpt from the Press Release:

BEIJING & WALTHAM, Mass.–(BUSINESS WIRE)–EdiGene, Inc., a global, clinical-stage company focused on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs, announced it had completed the last patient dosing in Phase I clinical trial of ET-01, its investigational gene-editing hematopoietic stem cell therapy for transfusion dependent β-thalassemia (TDT).

“The dosing of the 8th and last patient in our multi-center Phase I study is another important milestone in our efforts to bring a potentially one-time cure for patients with TDT,” said Dong Wei, Ph.D., CEO of EdiGene. “We are deeply grateful for the patients and their families, as well as investigators whose participation and dedication have played a critical role in advancing the Phase I study. The study outcome will shed important insight on the safety and efficacy profile of ET-01 in this particular patient population, and when at an appropriate time in 2023, we plan to discuss with regulators on Phase II clinical study strategy and design, bringing ET-01 one step closer to patients with TDT in China.”

β-thalassemia is a hereditary hemolytic anemia disease. The current standard treatment for TDT requires lifelong packed red blood cell transfusions and iron chelation therapy. ET-01 is designed to achieve a functional cure with a single administration of CRISPR/Cas9 gene-modified autologous hematopoietic stem and progenitor cells.

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