Today is the annual "International Rare Disease Day". Ltd. has entered the countdown to market for the first domestic teriflunomide. The drug is approved for relapsing multiple sclerosis (MS).

Multiple sclerosis (MS) is a lifelong, chronic, progressive disease that affects more than 2.3 million people worldwide, with a prevalence of approximately 0.03% and more than 30,000 patients in China.In May 2018, MS was included in the First Catalogue of Rare Diseases in China.Its cause stems from autoimmune system pathology, which causes the breakdown and denudation of nerve myelin, resulting in damage to the spinal cord, brain, and optic nerve function The cause of the disease is an autoimmune disorder that causes the breakdown and denudation of the myelin sheath, resulting in damage to the spinal cord, brain and optic nerve function. The neurological disability of the patient gradually increases, resulting in loss of self-care, blindness and even loss of life. According to the research of authoritative institutions, MS is incurable and patients have to live with the disease. Therefore, the earlier treatment can better control the patient's disease activity and reduce the occurrence of irreversible neurological damage.

Founded in 2010 in the park, with the philosophy of "every small group should not be abandoned", CGeneTech continues to focus on rare disease drug development and has built an integrated drug development technology platform covering the entire R&D chain of small molecule innovative drugs. Earlier this year, Teriflunomide was approved by the State Drug Administration for the treatment of relapsing forms of multiple sclerosis. "In the consistency evaluation with Sanofi's original drug, CGeneTech's teriflunomide reached a level consistent with the original drug in terms of efficacy, side effects and other dimensions." said Yu Qiang, founder and CEO of Centra Tech.

At present, Suzhou is accelerating to build an innovation cluster with international competitiveness and global influence. With an integrated drug R&D technology platform of the whole R&D chain and continuous layout in various fields such as rare diseases, hypoglycemic drugs and anti-tumor drugs, CGeneTech also has its own ideas for the creation of Suzhou innovation cluster.

In addition to focusing on rare disease drug R&D, CGeneTech is also committed to hypoglycemic and anti-tumor drug R&D. Shengliptin phosphate tablets, a novel and highly active and safe DPP-4 inhibitor, have been successfully selected for the National 12th Five-Year Plan and 13th Five-Year Plan in 2012 and 2018 respectively. In the second half of 2019, Shengliptin phosphate was formally approved by the National Drug Review Center, exempting Phase II clinical trials and entering Phase III trials directly, which is expected to be completed in the second half of this year and is expected to fill the gap of domestic DPP-4 inhibitors. In the field of tumor therapy, the company avoided the popular areas of antibody therapy such as PD-1/L1 and focused on the types of mutations that are currently difficult for tumor immunotherapy to be effective.

For a long time, the new target, route of administration, dosage form, strength of action and therapeutic effect often determine whether a domestic innovative drug will "fail" or not. The reason why Shengliptin Phosphate Tablets of Centene were able to "enter the clinical stage" is the breakthrough in molecular design and formulation process of the company.

"There are various proteins, or enzymes, in the human body that regulate physiological activities. Very often, people get sick because these enzymes are inactive or too hyperactive. Small-molecule drugs enter the body and achieve therapeutic purposes by regulating the relevant enzymes." Yu Qiang said that the efficacy and safety of small molecule drugs are closely related to the molecular design. In layman's terms, the molecule to be designed for small molecule drugs is the earliest form of the drug - the appearance of the lead compound, its fat and thin determines the size of the activity, whether it will "bite" determines whether there is toxicity. When everything is "broken" successfully, this small molecule drug can be registered, that is, registered patent, and then slowly grow into an adult.

It is reported that the core technical team of CGeneTech is composed of four people, all from the chemistry department of Peking University, which is good at molecular design. "We hope that high-quality small-molecule drugs can be combined with large-molecule antibody drugs across borders to bring more choices for patients." Yu Qiang said.