EdiGene Enters Global Licensing Agreement with Boston Children's Hospital to Advance Fetal Hemoglobin-Based Therapies for Hemoglobinopathies
Edigene
Genome Editing Technology Developer
Boston Children's Hospital
For over 140 years, Boston Children’s Hospital has been a world leader in pediatric care and the treatment of complex childhood diseases. As one of the largest pediatric medical centers globally, the hospital treats more than 500,000 patients annually and performs over 26,000 surgeries. Due to its favorable patient outcomes and exceptional quality of care, Boston Children’s Hospital has been repeatedly ranked as the number one children’s hospital in the nation by U.S. News & World Report.
On March 16, Boya jiyin (Beijing) Biotechnology Co., Ltd. announced a global non-exclusive licensing agreement with Boston Children's Hospital to apply the hospital’s proprietary methods and compositions for treating hemoglobin disorders by blocking BCL11A expression at the genomic level to increase fetal hemoglobin levels. Boya jiyin is a global clinical-stage biopharmaceutical company focused on the translation of gene-editing technologies, dedicated to developing innovative therapies for difficult-to-treat genetic diseases and cancers.
Scientific evidence shows that increasing fetal hemoglobin levels to compensate for the problems in adult hemoglobin production and function caused by genetic mutations is one of the methods for treating hemoglobinopathies. The expression of fetal hemoglobin naturally shuts down after birth in humans, but this shutdown mechanism can be reactivated during erythroid differentiation by blocking the expression of the BCL11A gene.
"For patients with beta-thalassemia who also suffer from hereditary persistence of fetal hemoglobin (HPFH), naturally occurring genetic variations allow them to have high fetal hemoglobin expression, significantly alleviating anemia symptoms, which highlights the potential of the aforementioned treatment," said Dr. Dong Wei, CEO of Boya jiyin (Beijing) Biotechnology Co., Ltd. "The achievement of this licensing agreement is an important strategic component in the development of our gene-editing therapy research product ET-01 for transfusion-dependent beta-thalassemia, and it marks a crucial step in accelerating the transformation of cutting-edge technology into innovative therapies to meet unmet patient needs."
The specific terms of this agreement were not disclosed by both parties.
Since its establishment in 2015, Boya jiyin (Beijing) Biotechnology Co., Ltd. has been building an increasingly comprehensive full-range industrialization capability and continuously expanding its product pipeline based on four gene-editing therapy platforms. In January 2021, the company's clinical trial application for ET-01, a gene-edited hematopoietic stem cell research product targeting transfusion-dependent β-thalassemia, was approved by the National Medical Products Administration. The multi-center Phase I clinical trial for this product is currently underway. ET-01 is an autologous CD34+ hematopoietic stem/progenitor cell injection with CRISPR/Cas9-modified BCL11A erythroid enhancer.
About Boston Children's Hospital
Boston Children's Hospital, ranked No. 1 in the U.S. by "U.S. News & World Report," is a major pediatric teaching institution of Harvard Medical School. Boston Children's Hospital is the largest research institute based on pediatric medicine globally, and since its establishment in 1869, its research achievements have benefited both children and adults. Currently, Boston Children's Hospital has 3,000 researchers, including a team comprised of 10 members of the National Academy of Sciences, 25 members of the National Academy of Medicine, and 12 Howard Hughes Medical Institute Investigators. Initially founded with only 20 beds, Boston Children's Hospital has now developed into a comprehensive healthcare center for children and adolescents with 415 beds.
About Boya jiyin
EdiGene, Inc. is a global biopharmaceutical company at the clinical stage, specializing in the translation of gene editing technologies. It is committed to developing innovative therapies for difficult-to-cure genetic diseases and cancers. EdiGene has established proprietary ex vivo cell gene editing treatment platforms targeting hematopoietic stem cells and T cells, an in vivo gene therapy platform based on RNA single-base editing technology, and a high-throughput genomic editing screening platform focused on targeted therapy development. Founded in 2015, EdiGene is headquartered in Beijing with offices in Guangzhou, Shanghai, and Cambridge, USA. For more information, please visit the company’s official website: www.EdiGene.com.