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Shi Huang

Thalassaemia is the world’s most common genetic blood disorder. China currently has 30 million thalassaemia gene carriers, while the global total reaches 350 million.
Patient with severe thalassaemia could confront a stark reality: regular blood transfusions, potential organ transplants, and even the risk of premature death.
If two carriers of the gene marry, their children have a 25 per cent chance of developing the disease.
China’s latest original therapy, which integrates gene editing and stem cell technology, has given patients new hope.
The therapy used CS-101, a base-editing drug developed for thalassaemia patients by scientists at ShanghaiTech University using transformer Base Editor (tBE) technology.
Five Chinese patients with beta-thalassaemia who received CS-101 treatment rapidly restored haematopoietic function – the ability to produce healthy new blood cells. Total haemoglobin and fetal haemoglobin (HbF) levels rose quickly and stayed high, freeing them from blood transfusions.
The first patient treated with CS-101 therapy received it in October 2023, and has gone over 28 months without needing a blood transfusion. In the two-year follow-up, no patients experienced serious adverse reactions.
This also marks the world’s first successful cure of a haemoglobin disorder using in vitro base-editing therapy.


The results of the study, jointly conducted by ShanghaiTech, Fudan University and Guangxi Medical University, were published in Nature on April 8.
“Traditional gene editing technologies are like ‘molecular scissors’, achieving gene modification by cutting the DNA double helix,” Chen Jia, the paper’s corresponding author and developer of the CS-101 therapy, told China Science Daily on April 15.
“However, this method has its shortcomings: DNA cleavage within the body can easily lead to cell death, chromosomal rearrangements or translocations, and even pose a potential risk of inducing leukaemia, which limits its safe clinical application.”
First identified among Mediterranean residents in the 1920s, thalassaemia was once commonly referred to as “Mediterranean anaemia”.
This anaemia stems from a genetic mutation that originally evolved as a defence against malaria, as the haemoglobin changes it causes help to protect against the parasitic infection.
However, in today’s world, where malaria is no longer prevalent, this genetic mutation has brought about new problems.
In patients with this condition, red blood cells undergo abnormal self-destruction, leading to severe anaemia.
Patients with severe thalassaemia may require blood transfusions 10 or more times a year. Without this, they might face irreversible complications and premature death.
The traditional effective treatment is still bone marrow stem cell transplant, which requires perfect donor matching and carries the risk of “graft-versus-host disease” – where the new cells attack the recipient’s body.
CS-101 is a next-generation gene-editing technology that enables precise editing of a single base in patients ex vivo, or outside the body.

It first collects CD34-positive haematopoietic stem cells from peripheral blood, performs base editing after extraction and then reinfuses them into the bone marrow to rebuild the patient’s haematopoietic system and restore normal function.
Although yet to be commercialised, China’s CS-101 injection is projected to cost far less than Western alternatives that can cost millions of US dollars – potentially drawing thalassaemia patients from abroad for affordable treatment.
According to the official website of CorrectSequence, the company producing the CS-101 therapy, it has cured nearly 20 patients with beta-thalassaemia and sickle cell anaemia, including a four-year-old girl from Pakistan.
These patients have successfully eliminated their dependence on blood transfusions and maintained a high level of haemoglobin expression over the long term.
The research team is accelerating CS-101’s subsequent clinical trials and drug approval process. At the same time, they are advancing further upgrades to the technology.
“Our next focus will be on developing base-editing treatments for liver-related metabolic diseases,” Chen told China Science Daily.
The South China Morning Post has contacted Professor Chen for comment.


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Original therapy integrating stem cell tech and gene editing could treat a blood disorder with 350 million carriers worldwide


Original therapy integrating stem cell tech and gene editing could treat a blood disorder with 350 million carriers worldwide.


China’s thalassaemia jab raises hope for common inherited blood disorder

.css-1c6uqr6{color:inherit;font-weight:inherit;font-size:inherit;font-family:inherit;line-height:inherit;overflow-wrap:break-word;}Original therapy integrating stem cell tech and gene editing could treat a blood disorder with 350 million carriers worldwide

Original therapy integrating stem cell tech and gene editing could treat a blood disorder with 350 million carriers worldwide