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

Nov 20, 2022

BEIJING and WALTHAM, Mass. – (November 18, 2022) – 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.

The Phase I clinical trial is a multicenter, open-label, single-arm study to assess the safety and efficacy of a single dose of ET-01 in TDT patients with eight subjects enrolled. EdiGene announced the activation of investigational sites and the first patient enrollment in September 2021.


About ET-01

ET-01 is an autologous CD34+ hematopoietic stem/progenitor cell with the erythroid-specific enhancer of the BCL11A gene modified by CRISPR/Cas9. It is the first gene-editing experimental therapy and the first hematopoietic stem cell experimental therapy with IND application approval by China National Medical Products Administration (NMPA).

About EdiGene, Inc.

EdiGene Inc. is a global, clinical-stage company focusing on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs. Anchored by its proprietary technologies of gene editing, bioinformatics, and high throughput genome-editing screening, EdiGene advances the pipeline on therapeutic platforms of LEAPERTM-based in vivo RNA base editing, ex vivo gene-editing hematopoietic stem cells, and ex vivo gene-editing T cells for allogeneic CAR-T. Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou and Shanghai, China, and Waltham, Massachusetts, USA. More information can be found at https://www.EdiGene.com.

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