Foreword
Sometimes, we feel that everything is too slow. We hope that all the instruments, equipment, reagents and consumables we purchased can arrive quickly; we hope that the materials we consulted and applied for can be responded to quickly; we hope that the test time can be shortened and the results can be obtained quickly; we hope that the progression of the child's condition can be controlled quickly; we hope that the child can recover and grow healthily after taking the medicine; we hope that more rare disease drugs can be started quickly to benefit more people as soon as possible.
When we see that the gene drugs we developed have miraculous effects in treating rare diseases; when children reach the milestones of lifting their heads, turning over, sitting alone, standing with support, and walking alone; when we see the innocent eyes, flower-like smiles and babbling of children; when children who have lost the ability to walk stand up again and practice walking; when we receive sincere thanks and blessings from parents, we feel happiness, value and significance.
Although the road is long, we will reach our destination if we keep going; although the task is difficult, we will succeed if we do it. We are full of expectations for 2024.
2023
Derived from needs, beneficial to patients
- GC101 is an AAV gene therapy drug for spinal muscular atrophy. It improves motor function and enhances the quality of life of patients by expressing SMN1 protein in the central nervous system and systemic tissues and organs. At present, the registration clinical trial of GC101 injection is being carried out in multiple centers such as the Seventh Medical Center of the General Hospital of the Chinese People's Liberation Army, the First Hospital of Peking University, Shengjing Hospital Affiliated to China Medical University, and Wuhan Children's Hospital, and has taken the lead in entering the Phase II clinical trial stage. Clinical observations show that patients have shown good drug safety and significant improvement in various indicators after treatment.
- GC301 is an AAV gene therapy drug for Pompe disease. It corrects peripheral organ involvement and reduces the burden of the central nervous system of Pompe disease through a recombinant AAV viral vector preparation expressing GAA enzyme, and has therapeutic advantages that existing enzyme replacement therapy does not have. At present, GC301 is being carried out in multiple centers such as Peking Union Medical College Hospital, the First Medical Center of the General Hospital of the Chinese People's Liberation Army, and the Children's Hospital Affiliated to Zhejiang University School of Medicine. In the previous clinical trial initiated by researchers (IIT), the world's first infantile Pompe disease patient has completed 52 weeks of observation and clinical observations have shown definite efficacy and significant improvement.
- GC304 is an AAV gene therapy drug for hypertriglyceridemia. It expresses a recombinant AAV viral vector preparation that expresses lipoprotein lipase (LPL), a key enzyme in the human body that hydrolyzes plasma triglyceride-rich lipoproteins, to achieve long-term and sufficient degradation of triglycerides in circulating blood. It has a unique therapeutic advantage in changing the lifelong long-term medication situation for patients with lipid metabolism disorders, and is currently advancing Phase I clinical trials.
2023
Strategic layout, leapfrogging and upgrading
2023
Focus on design and be innovative
2023
Well-deserved, outstanding achievements
2023
System construction and continuous improvement
Since the company passed the ISO9001 quality management system certification for the first time in 2022, it has established a quality management system for the entire life cycle of gene therapy drugs (including drug research and development, clinical trials, drug vigilance, and drug manufacturing), and formulated a quality management policy of "originating from demand, daring to innovate, specializing in design, strict verification, sophisticated manufacturing, continuous improvement, and benefiting patients". In 2023, after the quality management system was fully operational for a year, it successfully passed the review. The company's entire staff has improved their understanding of quality and applied quality system thinking to drug research and development, clinical trials, and drug production management. Following the ISO9001 quality system specifications and principles, we creatively incorporated all aspects of research and development, production, clinical trials, and operations into the quality management system, established a quality and business management system suitable for new drug research and development companies, strengthened the system thinking of all employees in all positions, and strengthened the awareness and action norms of planning first, written records, risk-based thinking, and continuous improvement.
2023
Social responsibility, science and technology for public welfare
Letters from Children
In December, we received letters from children far away,
Dear children, thank you too
You made us deeply feel the sense of mission and value of making medicine;
Your courage and strength also inspired us,
The sweat on the road to recovery will pave a road, and you will walk thousands of miles with your own steps,
See the vast mountains and rivers, and the shining stars.
Outlook 2024
Rare diseases are both rare and numerous. The rare number of people with a single disease is small, but the number of diseases is large. The establishment and development of gene drugs for rare diseases is often a contradictory and lonely journey. When there is no light ahead, we carry lanterns and move forward, not only illuminating the team, but also bringing light to patients and families. We believe that a single spark can start a prairie fire, and a path is never lonely, and virtue will always have neighbors. We always adhere to the corporate values that are derived from needs and benefit patients, and make good drugs and gene drugs that ordinary people can afford.