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Recent popular reports from Yimaike★Heavy! New type of CAR-NK cell therapy is effective against mutated strains of new coronary pneumoniaYimaihei Technology ★ "spot type" red blood cells treat a variety of cancers, preclinical data reveals its characteristicsYimaihei broke the news, click on the picture, and sign up immediately July 2021 20th/MedClub News/--Recently, the 923th Hospital of the Joint Logistics Support Force of the People’s Liberation Army and Shanghai Bangyao Biotechnology Co.
, Ltd.
jointly carried out the "gamma globin reactivated autologous hematopoietic stem cell transplantation for the treatment of severe β-Mediterranean "Anemia (thalassaemia) safety and effectiveness" clinical research has sent good news again, and another child bid farewell to thalassaemia through gene editing
.

This is the first project in Guangxi to cure severe β-thalassemia through gene therapy.
So far, three children have got rid of blood transfusion with the help of gene editing technology
.

Next, the target age of this research project will be further expanded to 35 years old, which is expected to benefit the "old and difficult groups" in the field of thalassaemia transplantation-older and hopeless patients
.

The clinical trial is a multi-center clinical trial, which was successively carried out in Xiangya Hospital and 923 Hospital of Central South University
.

On July 22, 2020, Bangyao Biotechnology reported that the project carried out in cooperation with Xiangya Hospital of Central South University has achieved initial results.
The two patients treated have already got rid of blood transfusion after treatment.
The follow-up period has been more than one year, and the total hemoglobin is still Maintain it within the normal range
.

Through multi-center clinical trials, the gene therapy provided by Bangyao Biotechnology has cured 5 children with severe β-thalassemia, which further confirmed the feasibility and good efficacy of the technology
.

At present, Bangyao Biotechnology is making every effort to promote the process of new drug declaration, and strive to realize the market of new drugs as soon as possible to benefit a wider population of patients with severe thalassaemia
.

The clinical trial results of the 923 Hospital brought more surprises to the research team
.

At present, the three children from the 923 Hospital have been cured and discharged.
Similar to the two patients treated by Xiangya Hospital, they got rid of blood transfusion dependence as soon as one and a half months
.

Because of the close cooperation of the pediatric team of the hospital, no infection occurred in the three children after transplantation
.

According to Wang Li, the director of the pediatrics department of the hospital, children's medication is different from that of adults, and the dose is calculated based on body weight.
The complications are also different from those of adults
.

Only by making every detail accurate, can children smoothly overcome the difficulties of infection, drug toxicity, hematopoiesis and immune reconstruction
.

▲During Lele's treatment, just in time for the first two children (first from right, second from right) to return to the hospital for follow-up.
Two "coming people" cheered Lele on and after continuous observation, Lele's hemoglobin, platelet and other indicators were maintained It means that he got rid of the blood transfusion after more than a month of gene therapy
.

By July 15th, Lele's hemoglobin had risen to 92 grams per liter, and his white blood cells and platelets had become normal, which meant he had bid farewell to thalassaemia
.

▲Lele’s mother, Chen Mei (pseudonym), who accompanies Lele’s treatment, told reporters that Lele usually has a blood transfusion every 25 days, but due to the tight blood source, hemoglobin often drops to 60 grams per liter before the blood transfusion
.

Chen Mei remembers that Lele’s last blood transfusion was on June 22, when hemoglobin dropped to 68 grams per liter, and rose to 86 grams per liter after the blood transfusion
.

The success of the treatment of thalassaemia depends on whether the patient can get rid of the blood transfusion
.

According to reports, if gene therapy is not accepted, Lele’s hemoglobin will theoretically drop to 76 g/l after one week of blood transfusion, and to 66 g/l after two weeks
.

But this time is different.
After two weeks, the test results show that the value has not fallen.

.

Chen Mei said: "The matching is hopeless, and long-term blood transfusion is not a solution
.

Because blood resources and family financial conditions are very tight, relatives and friends are afraid of receiving calls from his home, because we call either to borrow money or ask them to help each other donate blood.
We believe that science will bring miracles
.

"Gene therapy is expected to completely cure hereditary blood diseases.
Hematopoietic stem cell transplantation is currently the only cure for thalassemia major
.

For patients, the path to healing is to find someone with the same type as you, but not everyone has good luck
.

Now they have another way in front of them-to change their bad genes
.

A 7-year-old Lele (a pseudonym) and his mother lived in a ward of the Department of Pediatrics of the 923th Hospital of the Joint Logistics Service of the People's Liberation Army
.

They are from Hezhou.
As a severe beta-thalassemia patient, Lele has been relying on blood transfusions to maintain his life since birth
.

Since Lele was one and a half years old, his family has been planning to give him a matching transplant, but many attempts have failed
.

Lele didn't match his parents or brother, and he couldn't find an identical donor in the Chinese bone marrow bank and Taiwan bone marrow bank
.

However, gene therapy provides a way to "don't ask for help" for patients with thalassaemia
.

Professor Zhang Xinhua from the Department of Hematology of the No.
923 Hospital introduced that the main globin chain that composes human hemoglobin has a magical change before and after birth-alpha globin chain and gamma globin chain are transformed into alpha beads shortly after birth.
Protein chain and beta globin chain
.

Therefore, for β-thalassaemia caused by β-globin gene defects, the goal of treatment can be achieved by awakening the "silent" γ-globin gene after birth to restore the hematopoietic characteristics of the fetus
.

▲Based on the strategy of gene editing technology to treat thalassaemia.
This operation is similar to the "switch" principle.
The gamma gene that is turned off after birth is reopened to replace the inactive beta gene
.

This requires the use of gene editing technology
.

The CRISPR/Cas9 "gene scissors" technology currently used in this project was invented by two female scientists in France and the United States, which can achieve targeted cutting of DNA
.

At the cutting place, scientists can precisely change the DNA of organisms and rewrite the code of life
.

These two scientists won the 2020 Nobel Prize in Chemistry
.

The Nobel Committee stated in the official award speech that "CRISPR/cas9 gene scissors have completely changed the molecular life sciences, and may make the human dream of curing hereditary diseases come true
.
"
Gene therapy is expected to allow older patients to say goodbye to thalassaemia.
The recently released "Blue Book of Thalassemia in China (2020)" shows that as of April 1, 2021, there have been fewer than 4,000 thalassaemia stem cell transplants nationwide, most of which are siblings.
Compatible transplantation
.

Zhang Xinhua introduced that all-in-one transplantation is very mature and has a high success rate
.

However, the success rate is closely related to the patient's age-the younger the age, the higher the success rate; conversely, the success rate is reduced due to factors such as the number of blood transfusions, iron overload, and organ function impairment
.

Therefore, most hospitals that carry out transplants prefer to accept children under 10 years of age
.

For haploidentical transplantation, due to the risk of rejection and other issues, only transplant teams with long-term thalassemia hematopoietic stem cell transplantation and rich clinical experience can perform it.
Therefore, the progress is slow and the number of completed cases is relatively small
.

Guangxi started the "Guangxi Thalassemia Prevention and Control Plan" in 2010 and implemented the "Zero Birth" plan for severe thalassaemia
.

After ten years of hard work, the birth rate of thalassaemia fetuses has been greatly reduced
.

In the past ten years, the government has drastically reduced the births of severe thalassaemia children while vigorously promoting the transplantation technology of thalassaemia, and has achieved remarkable results
.

However, thalassaemia patients born before 2010 are over 10 years old, and as they grow older, it is more and more difficult for them to cure
.

"In other words, the patient is almost better to do has been done, the rest of the patient as well as older children who can not find a suitable donor, they need to find a way out
.
"
Professor Zhang Xinhua said 923 hospitals
.

Gene editing technology has opened a door for these patients.
The current stage of this project is for patients aged 5 to 15, and the age will be expanded to 35 years old in the next stage
.

"Gene therapy is a brand new thing after all
.

" Professor Zhang Xinhua said frankly, "The unpredictable consequences of gene editing are really unknown at present
.

Therefore, the observation period for these cases will last for several years.

.

"focus areas genes, state Yiu continue to make breakthroughs in upgrading a previous study, a team of scientists in the biological state Yiu editing tools to develop thalassemia gene and the field of gene therapy, has been going on has made a series of breakthroughs, the project uses gene technology to re-edit Turning on the expression of gamma globin in the fetal period, instead of defective beta globin, is likely to become one of the treatment options for patients with thalassaemia
.

The medical field represented by gene therapy and cell therapy is facing unprecedented opportunities and is a major breakthrough in the medical field in the next ten years
.

Bangyao Biotech has always been committed to the development of innovative drugs for gene therapy.
Since its establishment, it has established an international strategy of "based on China, global deployment, and benefiting the world" to meet the rapidly growing medical market in China and the world, and provide for those who urgently need revolutionary genes.
The patient of the therapy brings good news
.

Bangyao has differentiated competitive advantages in terms of product line selection and technology platform.
For the core pipeline product-gene therapy beta thalassaemia, Bangyao Biotechnology overcomes industry barriers, not only has a comprehensive grasp of gene editing technology, but also has a deep understanding of hematopoietic stem cell editing.
Strategies, and then continuously optimize the process, so that the key problems of gene-edited hematopoietic stem cells in the treatment process can be solved
.

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