EdiGene Announces ET-01, China's First CRISPR/Cas9 Gene-Edited Therapy for Transfusion-Dependent Beta Thalassemia, Approved for Clinical Trial

Beijing, China and Cambridge, Massachusetts, USA, January 18, 2021 – Boya JiYin announced that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has approved its Investigational New Drug (IND) application for ET-01, a CRISPR/Cas9 gene-editing therapy product for transfusion-dependent β-thalassemia. This marks the first gene-editing therapy product and hematopoietic stem cell product in China to receive NMPA approval for clinical trials. Boya JiYin is a biopharmaceutical company leveraging genome editing technologies to accelerate drug research and develop innovative therapies for a wide range of diseases. Previously, on October 27, 2020, Boya JiYin announced that the NMPA had formally accepted its IND application for ET-01.

“We are delighted that the clinical trial application for ET-01 has been approved by the National Medical Products Administration (NMPA), and Phase I clinical trials will be initiated as soon as possible,” said Dr. Wei Dong, CEO of Boya jiyin(Beijing)Biotechnology Co., Ltd. “This also marks the company’s formal entry into the clinical development stage. We will continue to dedicate ourselves to the high-quality translation of gene-editing technologies, steadily advance the R&D progress of our other product pipelines, and bring more new therapeutic options to patients in China and around the world.”

ET-01 is an autologous, ex vivo gene-editing therapy product currently in the research and development stage. The clinical trial approved for initiation is a multicenter, open-label, single-arm study designed to evaluate the safety and efficacy of a single transplantation of ET-01 in patients with transfusion-dependent β-thalassemia. In China, there are 300,000 patients with moderate-to-severe thalassemia, and those with transfusion-dependent β-thalassemia continue to face significant unmet medical needs.

About ET-01

The generic name of ET-01 is Autologous CD34+ Hematopoietic Stem and Progenitor Cell Injection with CRISPR/Cas9-Mediated Editing of the BCL11A Erythroid Enhancer. It is an autologous, ex vivo gene-edited cell therapy product under development for the treatment of transfusion-dependent β-thalassemia. The drug substance of ET-01 is manufactured by collecting autologous mobilized peripheral blood mononuclear cells from patients, enriching the CD34+ cell population, and editing the erythroid enhancer of the BCL11A gene using the CRISPR/Cas9 system.

About Thalassemia

Thalassemia refers to a group of hereditary hemolytic anemias caused by deletions or point mutations in globin genes, leading to partial or complete suppression of globin chain synthesis. Based on the specific type of impaired globin chain synthesis, thalassemia is generally classified into α-thalassemia, β-thalassemia, γ-thalassemia, δ-thalassemia, δβ-thalassemia, and εγδβ-thalassemia. Clinically, the most common forms are α-thalassemia and β-thalassemia, which result from reduced synthesis of one of the two polypeptide chains (α or β) that constitute normal adult hemoglobin (HbA, α2β2).

Meanwhile, according to the classification and clinical features of the Thalassemia International Federation, thalassemia can be divided into transfusion-dependent thalassemia (TDT) and non-transfusion-dependent thalassemia (NTDT). Patients with TDT require lifelong regular blood transfusions to sustain life; without transfusions, they will develop severe complications and face premature death¹.

About Boya Jiyin

EdiGene, Inc. is a clinical-stage biopharmaceutical company dedicated to accelerating drug research and developing innovative therapies for various genetic diseases and cancers through cutting-edge international genome editing technologies. EdiGene has established an ex vivo cell gene editing therapy platform targeting hematopoietic stem cells and T cells with independent intellectual property rights, an in vivo gene therapy platform based on RNA single-base editing technology, and a high-throughput genome editing screening platform committed to targeted drug development. Founded in 2015, EdiGene is headquartered in Beijing, with branches in Guangzhou and Cambridge, USA.

References:

1.Guidelines for the Management of Transfusion Dependent Thalassaemia(TDT) 3rd Edition: Thalassaemia International Federation(TIF); 2014.