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EdiGene approved for clinical trial of China's first gene-editing therapy

chinadaily.com.cn | Updated: Jan 22, 2021 L M S

The State Food and Drug Administration recently approved the clinical trial application of Zhongguancun Biopharmaceutical Enterprise EdiGene's gene-editing therapy product ET-01 for transfusion-dependent β-thalassemia.

As an innovative treatment with no precedent in China, this is the country's first approved clinical trial application of gene-editing therapy and the first approved clinical trial application of a hematopoietic stem cell product. 

"The clinical trial of ET-01 will be launched as soon as possible," said EdiGene CEO Wei Dong. "We will strive to bring more updated treatment options to patients in China and around the world, and even create the possibility of a one-time cure."

EdiGene is a biomedical company headquartered in Beijing Zhongguancun Life Science Park with branches in Guangzhou and the United States. It has also established an in-vitro cell gene-editing therapy platform for hematopoietic stem cells and T cells with independent intellectual property rights.

Data from the Blue Book of Thalassemia in China shows that more than 30 million people carry the thalassemia gene in the country, and patients still have vast and unmet medical needs.

The condition of children with β-thalassemia will gradually worsen after birth. In addition to anemia symptoms, they are prone to splenomegaly, delayed development and impaired organ functions caused by weakened immunity. 

Therefore, 50 percent of patients with severe thalassemia will die before the age of 5. It is rare for them to live longer than 20 years without adequate treatment.

For patients with transfusion-dependent β-thalassemia, the current standard treatment is a blood transfusion every two to five weeks and regular use of iron removers to reduce the body's iron level. The iron overload caused by long-term blood transfusion can damage the heart and liver function, leading to death from heart failure and growth and development disorders.

The medical treatment expenses are around 100,000 yuan ($ 15,480) per year. 

Although the disease may be cured through one-time hematopoietic stem cell transplantation, the transplant risks and side effects of immune rejection are great.

Last September, the State Food and Drug Administration proposed guidelines for developing gene-editing therapy drugs, updating the regulatory policy along with the new treatment.

From a global perspective, gene therapy is an innovative remedy. Novartis's ground-breaking drug Zolgensma is used for the treatment of spinal muscular atrophy. The drug is a gene therapy treatment, and it has achieved positive results.

For the treatment of thalassemia, gene-editing therapy has become a new treatment option around the world. 

At this stage, Swiss biopharmaceutical company CRISPR Therapeutics, American pharmaceutical company Forte Pharmaceuticals, and Bioverativ, a subsidiary of French pharmaceutical company Sanof, are all conducting related research and development.

Francis Collins, the chief scientist of the International Human Genome Project and president of the National Institutes of Health, once said that gene-editing technology has great potential in disease treatment.