BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- EdiGene., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, will present its latest research on a novel surface marker of human hematopoietic stem cells (HSCs) which could support long-term HSCs repopulation, and on microtubule polymerization inhibition that could enhance HSC homing and engraftment at the 63^rd American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta on December 11-14, 2021.

The research to be presented during the ASH meeting shows that CD66e is a robust functional HSC bio-marker that can support efficient long-term HSCs identification and repopulation. In a separate presentation, the company demonstrates that short-term microtubule polymerization inhibition could enhance human HSC homing and engraftment. These findings could potentially be used to further optimize HSC gene therapy development and clinical HSC transplantation.

EdiGene’s ET-01, an investigational gene-editing HSC therapy for patients with transfusion-dependent β-thalassemia, is in Phase I clinical study in China. ET-01 is an autologous CD34+ hematopoietic stem/progenitor cells with the erythroid-specific enhancer of the BCL11A gene-modified by CRISPR. HSCs are first removed from a patient and gene-modified, afterward, these cells are reinfused into the patient to treat the disease.

“The current research strengthens our efforts to unlock the potential of gene-editing HSC Transplantation,” said Dong Wei, Ph.D., CEO of EdiGene. “By combining our in-depth studies of hematopoietic stem cells and knowledge of gene-editing technologies translation, we hope to engineer novel enhancement to this transformative platform, and help more patients with serious medical conditions.”

“As a platform technology, gene-editing can be organically integrated with life-saving technologies such as HSC Transplantation to develop novel therapies,” said Wensheng Wei, Ph.D., Scientific Founder of EdiGene. “Being able to specifically modify a gene in cell therapies has the potential to free patients from chronic therapies and potentially curing them of their serious diseases.”

Details of the presentations:

Title: CD66e Enrichment Enhances Repopulation of Human Long-Term Hematopoietic Stem Cells
Publication number: 2156
Session Name: 501. Hematopoietic Stem and Progenitor Cells and Hematopoiesis: Basic and Translation: Poster II
Date/Time: Sunday, December 12, 2021 6:00PM – 8:00 PM (ET)
Location: Georgia World Congress Center, Hall B5
Abstract: https://ash.confex.com/ash/2021/webprogram/Paper148235.html

Title: Microtubule Polymerization Inhibition Enhances Human Hematopoietic Stem Cell Homing and Engraftment
Publication Number: 3820
Session Name: 701. Experimental Transplantation Basic and Translational: Poster III
Date/Time: Monday, December 13, 2021 6:00PM – 8:00PM (ET)
Location: Georgia World Congress Center, Hall B5
Abstract: https://ash.confex.com/ash/2021/webprogram/Paper149294.html

About EdiGene, Inc

EdiGene is a global, clinical-stage biotechnology company focused on translating gene editing technologies into transformative therapies for patients with serious genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou and Shanghai, China and Cambridge, Massachusetts, USA. More information can be found at www.EdiGene.com.

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