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 ?-thalassemia (TDT).

�The dosing of the 8^th 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.�

?-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 LEAPER^TM-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.

Contacts

Xiaomeng Zhang

EdiGene, Inc.

+86 10-80733899

[email protected]