BangYao biological to overcome the heavy "land poverty", China's gene therapy development in the future.

Read: This is the first successful case in Asia to treat poverty through gene editing.
recently, Shanghai Bangyao Biotech Co., Ltd. announced the preliminary results of the clinical trial conducted by Bangyao Bio and Xiangya Hospital of Central South University in cooperation with the clinical trial of "clinical study on the safety and effectiveness of autologous hematopoietic stem cell transplantation for heavy beta thalassemia in the treatment of heavy beta thalassemia".
note, this is the first time in Asia that gene-editing technology has been used to treat poverty in the world, and the first successful case of severe poverty in beta-0/beta-0 in the world through CRISPR gene editing technology.
Dr. Wu Yuxuan, vice president of biological research and development of Bang Yao, a scientist involved in the project, and a researcher at East China University, once again shared more stories about Bang Yao Bio in the field of thalassemia.
thalassemia ("terrestrial poverty") is the world's most widely distributed, most affected population of a single genetic disease, divided into alpha, beta, beta and pyrethroaia, 4 types, beta and alpha thalassemia is more common.
China is also a large country of poverty, at present, China's "land poverty" gene carriers about 30 million people, involving 100 million family population, moderate to severe poverty patients about 300,000 people and is increasing at a rate of about 10% per year, in the south of China's Yangtze River, especially in Guangxi, Guangdong and Hainan provinces, but the treatment method is extremely limited. Dr Wu Yuxuan,
, said: "At present, the routine treatment of poverty is regular blood transfusion and full iron removal treatment, almost every half a month."
and if the frequency of blood transfusion can not meet the standard, first of all, the survival of patients is very poor, many TDT patients due to potential disease and iron overload and experience serious complications and organ damage, such as many patients in the country will appear liver swelling, spleen swelling and other symptoms, the quality of life is very low.
however, the traditional life-long blood transfusion iron treatment life cost about 4.8 million yuan, is the vast majority of families can not bear the heavy burden, but also because of the huge cost and limited blood sources, China's patients rarely receive standardized treatment, and without high-cost frequent blood transfusion and iron treatment, the majority of heavy children died within 5 years of age.
", the "ground-poor gene therapy strategy developed by the scientific team of BangYao Bio, isolated HSC from the patient, used CRISPR/Cas9 gene editing technology to perform gene editing at specific gene sites in HSC, and then implanted the gene-edited HSC for self-transplant.
this gene edit ingitkines the patient's own HBG (the gene that encodes the mitle protein) expression, thereby greatly increasing the content of niobium glocy protein in red blood cells, so that the patient's own hemoglobin level reaches the normal range, completely get rid of blood transfusion dependence. Dr Wu Yuxuan,
, said: "The results of this treatment are very significant in both children, this is arguably the first clinical trial in China for the gene therapy for thalassemia through gene editing technology, and the first gene therapy trial in the world for the most severe beta-globulin gene deficiency."
", in fact, gene therapy strategies based on different technologies have made encouraging progress in the development of thalassemia diseases, and several of the fastest-moving gene therapies are CRISPR Therapeutics and Vertex, which have developed CTX001 based on CRISPR gene editing technology, and Bluebird Biobird, inc. developed LentiGlobin, a slow-virus vector." Zynteglo, Sangamo and Sanofi, a joint stat-to-the-statin(ZFN) genome editing technology, are genetically modified for HSCs isolated from the patient's own, with Zynteglo having been tested in clinical phase III and listed in Europe, and officially launched in Germany in January for the first time.
but contrasted, BangYao's strategy has a unique competitive advantage.
similar to the CRISPR gene editing technology CTX001, the current clinical trial sidons two patients are beta-0/beta-type, not the most serious type of land poverty, and Bangyao and Central South University Xiangya Hospital this collaboration treatment is beta-pearl completely missing beta-0 type of poverty, treatment is more difficult, overcome the difficulties of beta 0/beta 0 type poverty, also all blood transfusion dependence will be able to be treated through this treatment.
for Zynteglo, which uses a slow virus vector, is also targeted at non-beta 0/beta 0 genotype TDT patients, while the slow virus has a risk of cancer in the genome, and the preparation cost of the virus vector is very high, Zynteglo is priced at $1.77 million (1.575 million euros), is currently the world's second most expensive drug, which also makes many patients look away.
, Dr. Wu Yuxuan also said: "We are in the reconstruction of neutrophils, the speed of reconstruction is also faster than they, so it can be said that in our country has developed a world-wide leader in gene-editing gene therapy to treat thalassemia disease, hope that in the future can benefit a wider range of patients in southern China."
" and it is well known that the current gene editing technology is complex, relative to the cost of general treatment strategies are much higher, for the future price trend of gene therapy products, Dr. Wu also said: "the cost of gene editing is still relatively high, but than the previous slow virus therapy has actually been a great improvement."
Bonyo Bio after years of exploration and development in the process, the final exploration of this set of CRISPR/Cas9 gene editing technology based on the treatment strategy will greatly reduce costs, but the cost of the general treatment strategy may still be more expensive, our future pricing may be estimated to be in the millions of yuan.
",""In fact, the annual cost of blood transfusions and iron-free treatment for poor patients is about 100,000 to 200,000 yuan, and the cost of this lifetime treatment is very expensive.
, it consumes a lot of blood resources, because every patient in a poor area would consume 250 to 300 blood donations a year if they were treated with a standardized blood transfusion.
so we have developed this gene therapy strategy, both economically and from social blood resources.
"China's gene therapy industry prospects optimistic Shanghai Bangyao Biotech Co., Ltd. is committed to become the world's leading gene cell pharmaceutical company in the new commercial civilization era, with the mission of "leading innovation with gene editing, developing breakthrough therapy for the benefit of all mankind", relying on the independent research center and the "Shanghai Gene Editing and Cell Therapy Research Center" jointly established with East China Normal University, has produced 105 patent achievements in the past five years, 5 projects have been in 8 well-known hospitals to carry out IIT trials Twelve high-level academic papers have been published in internationally renowned journals such as Nature Medicine.
for the growth and changeof since the founding of Bangyao Bio and the future expectations, Dr. Wu Yuxuan said: "Bangyao has completed a number of gorgeous transformation satios since its founding year."
from cooperation with Huashi University, sharing laboratory base, to independent research and development, production, completed the transition from small test to pilot, set up a strict and perfect quality control system, while equipped with more than 60 CMC and clinical research professional team, efficient and smooth realization of the results of transformation.
in terms of the team of scientists, we now have a strong research and development team, mainly with Professor Liu Mingyao of the School of Life Sciences of East China Normal University.
we are currently doing a lot of international leadership in gene editing and gene therapy, both in basic research and clinical transformation.
", BangYao Bio has built three major technology platforms for gene editing, cell therapy and gene therapy, and has a 6000 square meter GMP pilot base and an operation team of nearly 100 people, effectively ensuring that innovative research results can be rapidly transformed and applied.
Bonyo Bio continuously drives rapid update iterations of research and development products through patient needs and clinical feedback.
and uphold an open, shared and win-win attitude, together with the global innovation biopharmaceutical ecological chain enterprises, accelerate the transformation and landing of innovative drugs, for the benefit of the global patients with genetic diseases and malignant tumors! With the company's multiple gene therapy and cell therapy projects into scientific clinical trials or IND declaration stage.
for the follow-up capacity follow-up, Dr. Wu Yuxuan also said: "Our future plan is to further expand the clinical production site, and so on in the future after our IIT experiment and IND trial large-scale, I believe we will have better hardware and software conditions to make both social and commercial value of more promising products."
and in my opinion, the world, especially China's gene therapy industry is currently very optimistic "first, the domestic strong support for new drug research and development in recent years, the environment to promote previous overseas studies, and even some high-end talent with experience in international large pharmaceutical companies are scrambling to return home to start a business, which also makes China has gradually formed a very good and strong talent pool.
and then on the basic research, after years of development, China in the world bioscience basic research level has been at a relatively high level.
finally, for indications, in our country, such as thalassemia, there is a very wide range of patients, with a certain market size.
, the combination of these points will promote the development of gene therapy and gene editing industry in China.
", in general, for a new area such as gene therapy, the global industry as a whole has not been developed for a long time, but also in the last 5-10 years to achieve some more successful commercial transformation results.
the current gene therapy industry in research and development technology, production processes and other aspects are still facing many challenges, but we believe that a group of leading research and development capabilities such as BangYao Bio outstanding enterprises will continue to break through innovation, accelerate the development of Gene Therapy in China, but also benefit more patients.
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