On March 16, 2022 EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, reported that it 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 (Press release, EdiGene, MAR 16, 2022, View Source [SID1234610138]).
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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.