Chinese gene therapy ‘clinically cured’ patient with blood disorder thalassaemia, researchers say
- One-time treatment offers hope that people with one of the world’s most common inherited blood disorders could avoid transfusions
- Innovative gene-editing technique CS-101 has distinct advantages over existing CRISPR-based therapies, according to researchers
Shanghai-based CorrectSequence Therapeutics, along with collaborators from Guangxi Medical University, announced on Monday that they had cured an adolescent patient with transfusion-dependent beta thalassaemia.
“To the best of our knowledge, this is the world’s first report of a successful clinical cure of haemoglobinopathy with base-editing technology,” CorrectSequence said in its news release.
The patient, who had been undergoing blood transfusions every two weeks to manage the condition, has not required a transfusion for more than two months since receiving the treatment, the company said.
The patient “has resumed normal life” after receiving the one-time treatment, known as CS-101, CorrectSequence said.
While studies on CS-101 are still pending long term data, Androulla Eleftheriou, executive director of the Thalassaemia International Federation (TIF), said the design of CorrectSequence’s editor “greatly enhances the safety of the approach compared to other advanced therapy application”.
Beta thalassaemia is an inherited recessive blood disorder that results in a person’s blood cells lacking enough haemoglobin, a blood protein that carries oxygen throughout the body.