Ningbo Kangjiale Medical Equipment Co., Ltd.has been firmly rooted in the domestic market since its establishment, while paying attention to the international market. Aiming at the strong domestic and foreign demand for medical equipment, the company strives to build a complete marketing network. At present, Kangjiale’s domestic sales network has covered 90% of the country (except Tibet), and its products have been exported to 2/3 of countries and regions around the world.
Recently, the 18th annual medical innovation summit led by the world's top medical center Cleveland Clinic was held online. One of the hot spots is the announcement of the top ten medical innovation products in 2021. Experts selected ten breakthrough medical devices and treatments that are expected to have an impact next year. These medical innovations will change the lives of countless patients.
The innovative technologies/therapies selected this time include CD20 targeted therapy ocrelizumab, a new drug for cystic fibrosis Trikafta, hepatitis C pan-genotype therapy, PARP inhibitors and many other innovative drugs, involving hemoglobinopathy and primary multiple Sexual sclerosis, cystic fibrosis, hepatitis C, prostate cancer, migraine and other diseases.
In addition to leading readers to uncover the reasons for the birth of the top ten medical innovations, Arterial Network has also carried out a detailed combing of the latest technological developments in the fields involved, and combined with related companies and markets, striving to explore the market development potential of the fields behind the readers.
Gene therapy for hemoglobinopathies
>>>>Current status of related diseases
Hemoglobinopathy is a group of hereditary blood diseases caused by abnormal hemoglobin molecular structure or abnormal synthesis rate of globin peptide chain. The most common diseases are thalassemia and sickle cell anemia, which affect more than 330,000 children worldwide each year.
Most of the children with thalassemia are of Mediterranean, African, and Asian descent. The "thalassaemia" gene carrying rate in our population is 1.92-14.95% (about 30 million people). It is more common in southern regions such as Guangdong, Guangxi, Hunan, and Hainan. There are about 300,000 patients with severe to severe thalassaemia and it is increasing at an annual rate of about 10%.
The usual treatment of thalassemia is expensive and difficult, and it brings great difficulties to millions of families. One of the current treatment methods is regular blood transfusion to maintain life, and the second method is the internationally recognized treatment method-allogeneic hematopoietic stem cell transplantation. However, this method is only suitable for some patients who can wait for a suitable match. Risks of entry failure, infection, and graft-versus-host disease.
Another common serious hereditary hemoglobin disease is sickle cell anemia. The disease is characterized by sickle-shaped red blood cells and a large number of abnormal red blood cells are destroyed, causing chronic anemia. Sickle cell anemia is especially common among people whose ancestors come from sub-Saharan Africa, India, Saudi Arabia, and Mediterranean countries. Immigration increased the frequency of the gene in the American continent. In some areas of sub-Saharan Africa, as many as 2% of all children have the disease at birth.
>>>>The latest progress of international research and development in recent years
In May 2019, the European Union approved Bluebird Bio's one-time gene therapy Zynteglo (LentiGlobin, autologous CD34+ cells containing the βA-T87Q globin-encoding gene) for the treatment of β-thalassemia of 12 years and older ( TDT) patients, Zynteglo became the world's first gene therapy for TDT, priced at $1.77 million. Subsequently, EMA granted Zynteglo the Orphan Drug Designation (ODD) for the treatment of sickle cell disease (SCD). In September of this year, Bluebird Bio announced that the European Medicines Agency (EMA) has granted Zynteglo the priority drug qualification (PRIME) for the treatment of sickle cell disease. In January this year, the FDA granted Aruvant Sciences company ARU-1801 an orphan drug designation for the treatment of sickle cell disease (SCD). ARU-1801 is a modified fetal hemoglobin gene therapy developed to treat sickle cell disease (SCD) and beta thalassemia.
The gene therapy trial CTX001, conducted by the Swiss gene editing company CRISPR Therapeutics in cooperation with the US pharmaceutical company Vertex Pharmaceuticals, also obtained positive interim data in the Phase 1/2 clinical trial, two patients with beta thalassemia and one severe sickle cell anemia After the patients received treatment, they all achieved the effect of stopping relying on blood transfusion.
Good news came from the country.
Recently, Xiangya Hospital of Central South University, East China Normal University and other institutions carried out "Clinical research on the safety and effectiveness of gamma globin-reactivated autologous hematopoietic stem cell transplantation for the treatment of β-thalassemia major". The clinical trial results showed two The patient who received treatment has got rid of blood transfusion dependence and has been cured and discharged. Domestic and foreign documents show that this is the first successful case of treating thalassemia through gene editing technology in the whole country and even in Asia, and it is also the first successful case of treating β0/β0 type severe thalassaemia through CRISPR gene editing technology in the world.
According to Professor Chen Fangping from the Department of Hematology, Xiangya Hospital of Central South University, "Overcoming the β0/β0 difficulty means that most transfusion-dependent β-thalassemias will be cured by this treatment strategy." "Activation" technology will become one of the treatment options for β-thalassemia with excellent effect, great feasibility and good safety.