The first treatment base in China using CRISPR/Cas9 gene editing technology was launched.

Thalassemia, also known as marine anemia, is a group of hereditary hemolytic anemia.
people who usually suffer from severe thalassemia need to receive reinfusion of red blood cells and deiron therapy on a regular basis throughout life. the emergence and development of
gene editing technology has made it possible for poor patients to be exempt edited by blood transfusions and allogeneic cell transplants. On November 18,
, the first treatment base using CRISPR/Cas9 gene editing technology was launched in Nansha, Guangzhou, by Boya Zhing, and is expected to conduct clinical trials on beta-thalassemia in 2019.
1. Thalassemia thalassemia is one of the world's largest monogene icing diseases, is a genetic hemoglobin disease, due to the regulation of gloprotein synthesis of gene deficiency or mutation, resulting in the formation of hemoglobin alpha chain and beta-chain globulin synthesis imbalance, red blood cell life shortened a hemolytic anemia.
thalassemia (Thalassemia), first described by Cooley and Lee in 1925, was first found in the Mediterranean, so it is known as "thalassemia", also known as marine anemia or gloatin-producing anemia.
According to the WHO's Hemoglobin Epidemiology Report, more than 70% of the world's population is at risk of haemoglobin disease, with newborns accounting for nearly 90%.
thalassemia is mainly distributed in tropical and subtropical regions with a high global malaria rate, including southern China.
in China, thalassemia is mainly distributed in Guangdong, Guangxi, Guizhou, Sichuan, Hunan and other places, the prevalence rate is about 2%, of which the minority areas of Guizhou Province beta thalassemia gene carrying rate is as high as 5.4%.
the disease has been found in 160 countries around the world, with about 150 million people carrying the gene for beta-thalassemia.
, normative long-term blood transfusions and de-iron treatments are still the most important treatments for most heavy-duty beta thalassemia.
, traditional treatments are extremely expensive and require frequent life-long treatment.
According to the Beijing Angel Mom Charity Foundation, the China Siyuan Engineering Poverty Reduction Foundation, beijing Normal University China Public Welfare Research Institute in 2016 jointly released the "China Thalassemia Blue Book" shows that the traditional life-long blood transfusion iron treatment will cost about 4.8 million yuan.
the treatment of local poor hematopoietic stem cell transplantation (HSCT) is also a widely developed clinical treatment for poverty, but the premise of the treatment of hSCT in the ground is the success of bone marrow matching.
only a tiny fraction of the lucky patients who were able to successfully match.
2. Treatment of beta thalassemia from gene level therapy for beta thalassemia from gene levels is expected to free patients with beta thalassemia from the limitations of the treatment of the configuration and to access treatment to a wider range of patients.
2006, the world's first clinical trial of genetically modified therapies for thalassemia was conducted in France, led by international research teams such as Dr. Philippe Leboulch and Dr. Marina Cavazzana.
a male patient with thalassemia who received gene therapy was 18 years old and had beta thalassemia.
he started blood transfusions at the age of 3 and had blood transfusions every month when he was serious.
in the trial, the scientists first isolated hematopoietic stem cells from the patient's body and then used the virus as a vector to introduce the defect-free genes into the cells, chemically remove the excess cells and leave the cells behind, and eventually transplant them back into the patient.
results showed that the patient's normal red blood cells improved, and after one year of treatment, although there were still mild anemia symptoms, blood transfusion treatment was eliminated.
results were published in the journal Nature in 2010 under the title "Thesarapy of the country and HMGA2 activation after generapy of human beta-thalassaemia". The bottleneck of
gm therapy is mainly in the virus process.
many companies invest a lot of time and effort in virus processes.
12 years later, the results from Bluebird were published in the New England Journal of Medicine on April 19, 2018 under the title "Gene Therapy in Patients with The Se-Dependent beta-Thalassemia".
opens a new chapter in GM therapy.
study included 22 patients with blood transfusion-dependent thalassemia (12-35 years) in the United States, Thailand and France.
researchers first obtained the self-contained CD34 plus cells from the patient and transduced the cells in vitro with the LentiGlobin BB305 vector that encodes the adult hemoglobin (HbA).
then the cells are fed back after the patient's pretreatment of myelin.
found that the long-term blood transfusion needs of all 22 patients after treatment were significantly reduced or no longer needed blood transfusions after infusion of gene-modified cells.
the average hemoglobin synthesis level peaked in patients after 9-12 months after the retransmission of genetically modified cells.
, there are no serious adverse events associated with pharmaceutical products. After 26 months of follow-up
, 12 of the 13 non-beta 0/beta 0 genotype patients had stopped blood transfusions, and the hbAT87Q hemoglobin levels for gene expression were restored to the range of 3.4 to 10.0 g, the total hemoglobin levels were restored to 8.2 to 13.7 g;
these studies suggest that GM therapies are likely to save patients from blood transfusions and allogeneic cell transplants, but the complexity of the virus process adds to the difficulty.
. Boya Is a precise modification of the gene with the "God's wrist" gene editing.
in the treatment of poor patients, gene-edited therapy can be relatively simple compared to the gm therapy process.
it wasn't until 2012 that the CRISPR system was first used in gene editing of eukaryotic cells.
only six years later, the technology was approved by regulators in Europe and the United States and entered clinical trials. What
Is Now Working for Ao-Yabey is pushing gene-editing treatments into the clinic in China.
looking at the development pipeline of Boja,ogen, we found that eT-01, the gene-editing therapy used in beta-thalassemia, is using CRISPR/Cas9 gene editing technology, which is also the fastest-growing product for Breain and is expected to enter clinical research in 2019. On November 18,
, The Guangzhou Gene Editing Treatment Base was officially launched, which is the first treatment base in China using CRISPR/Cas9 gene editing technology. eT-01, the product of
's first gene-editing technique for the treatment of beta thalassemia, will be put on clinical terms here. Wei Wensheng, founder of
Boa, said that because it is a gene-editing technology for single-gene genetic diseases and the patient population is relatively accurate, it is expected to be approved for sale more quickly three years after clinical trials, and could lead to corner overtaking in the field of gene-editing and disease treatment around the world.
long-term concern about poor patients angel mother charity foundation founder Qiu Lili introduced, because of the difficulty of matching, in the traditional technical means, thalassemia patients face a lot of hardships, Boya Zhan because of the gene editing therapy being developed to solve the above problems to provide a new possibility for the vast number of unmatched poor patients to bring new hope.
Professor Li Chunfu, director of the Southern Chunfu (Children's) Blood Disease Research Institute and known as the "Father of China's Poverty", said that gene editing treatment will become a non-rejection response, the best cure effect, no need to seek transplant bone marrow sources, the most cost-effective program. Dr. Wei Dong, CEO of
, said that Boya Zingbey, as the first gene-editing company in China, will be the world's leading gene-editing company by keeping a close eye on the two main lines of gene editing to treat diseases and new drug research and development.
and Boya series because of the Guangzhou company - the establishment of the medical treatment and the establishment of the Guangzhou gene editing treatment base is one of the very important steps, marking the Boya in the new stage from scientific research to clinical.
the launch of the country's first treatment base using CRISPR/Cas9 gene editing technology, means that Boya is one step closer to finally turning genome editing technology into a clinical treatment.
we look forward to the development of more poor people benefiting from gene editing technology.
Source: Biological Exploration.