EdiGene Obtains Worldwide License from Boston Children’s Hospital to Methods and Compositions for Increasing Fetal Hemoglobin Levels to Treat Hemoglobinopathies

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, entered a non-exclusive, worldwide license agreement with Boston Children’s Hospital (BCH) for intellectual property related to methods and compositions for increasing fetal hemoglobin levels by disrupting BCL11A expression at the genomic level to treat hemoglobinopathies.

There is scientific evidence that suggests one way to address hemoglobinopathies is to upregulate the production of fetal hemoglobin to compensate for the genetic mutations that affect the proper production and function of hemoglobin. Fetal hemoglobin is naturally shut down after birth but can be reactivated by disrupting the BCL11A gene expression during erythropoiesis.

“In β-thalassemia patients with hereditary persistence of fetal hemoglobin (HPFH), naturally occurring genetic variants lead to higher fetal hemoglobin level and significantly alleviate symptoms, suggesting the potential of this therapeutic approach,” said Dong Wei, Ph.D., CEO of EdiGene. “The license is an important component of our strategy to develop ET-01, an innovative gene-editing therapy for patients with transfusion-dependent β-thalassemia, in an effort to advance rapidly evolving technology into therapeutics for patients with unmet needs.”

The terms of the agreement were not disclosed.

Founded in 2015, EdiGene has developed integrated capabilities and boosts a growing pipeline that extends across four gene-editing platforms. In January 2021, the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) approved the company’s Investigational New Drug (IND) application for ET-01, autologous CD34+ hematopoietic stem/progenitor cells with the elytroid-specific enhancer of the BCL11A gene modified by CRISPR/Cas9, for patients with transfusion-dependent β-thalassemia. A multi-center Phase I clinical trial is ongoing in China.

About Boston Children’s Hospital

Boston Children’s Hospital is ranked the #1 children’s hospital in the nation by U.S. News & World Report and is the primary pediatric teaching affiliate of Harvard Medical School. Home to the world’s largest research enterprise based at a pediatric medical center, its discoveries have benefited both children and adults since 1869. Today, 3,000 researchers and scientific staff, including 10 members of the National Academy of Sciences, 25 members of the National Academy of Medicine and 12 Howard Hughes Medical Investigators comprise Boston Children’s research community. Founded as a 20-bed hospital for children, Boston Children’s is now a 415-bed comprehensive center for pediatric and adolescent health care. For more, visit our Answers blog and follow us on social media @BostonChildrens, @BCH_Innovation, Facebook and YouTube.

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.