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Dr. Jin Lei: Jinsai Pharmaceutical will increase R&D investment in the field of rare diseases
Release time:
2021-09-15
Rare diseases refer to diseases with low prevalence and rare, usually chronic, serious diseases, often life-threatening. The World Health Organization defines rare diseases as diseases or lesions with the number of affected people between 0.65‰ and 1‰ of the total population. According to statistics, there are more than 7,000 rare diseases in the world, accounting for about 10% of human diseases, and the number of patients has exceeded 300 million. In my country, there are at least 20 million rare disease patients. Therefore, rare diseases are not actually "rare".
On the occasion of the 15th "International Rare Disease Day", Dr. Jin Lei, General Manager and Chief Scientist of Jinsai Pharmaceutical, was interviewed by the People's Daily Health Client, Xinhuanet and other media, and introduced the company's efforts to address rare diseases over the years. The efforts of the Ministry of Public Security have called on more people to pay attention to rare diseases, so that the rare disease group is no longer alone.
Innovative research and development, so that children with rare diseases have drugs to treat
Over the years, Kinsey has done solid work in the research and development of rare disease drugs and clinical trials. Taking chubby-Willi syndrome as an example, the current incidence rate is 0.33-1/10,000, the awareness rate is low, and a large number of patients have experienced misdiagnosis. If not intervened, the mortality rate is high. Research over the past 10 years has found that growth hormone therapy not only helps to improve the height and weight of obese patients, but also affects the metabolism, exercise capacity, behavioral and cognitive development of obese patients, continuous treatment in adulthood and the entire life cycle. will bring about revolutionary changes. "We have carried out a series of clinical studies on growth hormone in the treatment of chubby-Willi syndrome, and also on growth hormone in the treatment of achondroplasia, Turner syndrome, Sliver-Russell syndrome, etc. Although Turner syndrome is not currently In the list of rare diseases, but the diagnosis rate of this disease is similar to that of rare diseases, so we feel that this disease also needs more attention from the society, so that the families of these patients can get help." Dr. Jin Lei said.
Not limited to growth hormone, many of Kinsey's R&D pipelines involve the development of rare disease drugs. Rheumatic immune diseases are a major cause of affecting the growth and development of children, among which systemic juvenile idiopathic arthritis (sJIA) is an autoinflammatory disease that seriously affects the growth and development of children, but there are few specific drugs for the treatment of the disease. . At present, the products developed by Kinsey are in the phase II clinical trial stage, hoping to help more children with sJIA grow up healthily as soon as possible. "In the future, we expect to continue to invest more than 15% of the company's annual revenue in research and development, and with the comprehensive construction of Shanghai Zhangjiang R&D Center, not only will there be a larger investment in rare disease research, I believe Kinsey's overall There will also be very great progress in drug research and development.”
Industry support, children with rare diseases can be cured if they are sick
In addition to clinical research, Kinsey Pharmaceuticals also actively supports the work of domestic rare disease academic organizations. After supporting the Endocrinology, Genetics and Metabolism Group of the Pediatric Branch of the Chinese Medical Association to establish the China Prader-Willi Syndrome (PWS) Clinical Standardized Multidisciplinary Diagnosis and Treatment Collaborative Alliance, it also supported the experts of the group to establish the Silver-Russell Syndrome Diagnosis and Treatment Collaborative Group. "We are working with relevant clinical experts to support scientific research institutions and clinicians to actively explore, hoping to effectively improve the life and health of children with rare diseases."
At present, there are many other rare diseases affecting the growth and development of children, such as hypophosphatemic rickets, methylalaninemia, etc. Kinsey is assisting experts in developing growth hormone to improve the height and basal metabolism of patients with these rare diseases Research has also given great support. "Although the population affected by rare diseases is limited, as the diagnosis and treatment methods become more and more advanced, the types of rare diseases will increase significantly, and the total population will increase. As a high-tech pharmaceutical company, we shoulder social responsibilities and hope to Together with the government, society and regulatory authorities, we will promote the development of rare disease research and development, and ultimately enable children to receive comprehensive treatment."
Public welfare assistance to help children with rare diseases grow up healthily
In order to help patients with rare diseases improve their quality of life and reduce the financial pressure on their families, Kinsey is also constantly using public welfare to practice corporate social responsibility. On December 15, 2018, the Chinese Red Cross Foundation launched the "Little Fat Willy Patient Care Project", and Jinsai Pharmaceutical donated 10 million yuan of medicines and funds to fund the project to carry out medical assistance, physician training, popular science education, academic research, etc. Up to now, a total of 200 people have received love funding through this project. On December 3, 2021, the Growth Angel Fund and the National Children's Turner Syndrome Diagnosis and Treatment Collaborative Group jointly launched the "Turner Syndrome Love Relief Project". Kinsey Pharmaceutical Co., Ltd. helped 100 families with Turner Syndrome in financial difficulties nationwide. Patients, provide free one-year subsidy for growth hormone drugs, and send health care and growth hope to girls.
Dr. Jin Lei said: "In the future, we will continue to increase the promotion of public welfare projects, which will also benefit more children. During this period, we will not only simply subsidize medicines, but also support the Chinese Red Cross Foundation to hold a large number of popular science activities to promote public awareness. We hope that more people and families can increase the diagnosis and treatment rate by paying attention to the group, so that more children can get medical treatment in time.”
Rare diseases are not far away from us. Understanding rare diseases and caring for rare patients should start with you and me, and start from this moment.
Today, the 15th "International Rare Disease Day", Kinsey Pharma invites you to pay attention and hope that more "rare diseases" will be seen.
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