(Yicai Global) Jan. 19 -- China has green lit the country’s first clinical trials of a gene editing therapy, or a form of treatment for genetic diseases that revises, removes or replaces mutated DNA, for the inherited blood disorder thalassemia by biotech start-up EdiGene.
The study of EdiGene’s gene editing therapy ET-01 will assess its safety and efficacy in the treatment of transfusion-dependent thalassemia, the Beijing-based firm said yesterday.
Thalassemia patients are born with less hemoglobin than normal, leading to less oxygen in the blood, which causes anemia, depressed immunity, stunted growth and fatigue.
Around 30 million people in China carry the gene mutation. Some 300,000 people suffer from moderate to severe thalassemia, according to a 2015 report. Half of those with the severe form die before the age of five. Patients need life-long blood transfusions in order to prolong their life, costing around CNY100,000 (USD15,440) a year.
ET-01 is an injection of genetically modified hematopoietic stem and progenitor cells, which are present in the blood and bone marrow and help to form red blood cells, taken from the patient.
Set up in 2015, EdiGene raised CNY450 million (USD69.4 million) last October, the largest single amount for a Chinese gene editing firm so far. The company has branches in Guangzhou, southern China and Cambridge, Massachusetts in the US.
Editor: Kim Taylor