CHA Advanced Research Institute and CHA Biotech signed an agreement with GenKOre to develop gene therapeutics for sickle cell anemia and beta-thalassemia (β-thalassemia).

Under the agreement, joint research combining CHA Biotech’s viral vector technology and GenKOre’s gene transfer technology will be utilized to develop a gene-based therapy for sickle cell anemia and β-thalassemia, which are blood diseases caused by hemoglobin gene mutations.

Sickle cell anemia and β-thalassemia are the most representative blood diseases caused by a single gene mutation, resulting in hemoglobin deficiency or abnormal production.

Annually, more than 300,000 babies worldwide are born with a gene mutation associated with sickle cell anemia or β-thalassemia, and severe cases require blood transfusions every two to five weeks for life. 

In August 2022, the FDA approved Zynteglo (ingredient: betibeglogene autotemcel), a gene therapy developed by Bluebird Bio for thalassemia patients. Additionally, another drug called "exa-cel" which was co-developed by Vertex, and CRISPR Therapeutics, is currently under FDA and EMA review.

However, CHA Biotech drew attention to the fact that Zynteglo is not easily accessible to patients due to the risk of developing blood cancer and the high cost of the treatment, which is about 3.7 billion won (approximately $2.8 million) per dose.

"By utilizing GenKOre's ultra-small gene locator technology, we will lay the foundation for the development of gene therapy for rare blood diseases," said Yoon Ho-sup, president of CHA Advanced Research Institute.

"Drug delivery vehicles such as adeno-associated viruses are essential for the development of safer therapies for these diseases," said GenKOre co-CEO Kim Yong-sam. "We will utilize each company’s strengths to accelerate the development of a thalassemia gene-based therapy."