New breakthrough in enterprise-enterprise cooperation at Shanghai Institute of Biomedicine: The world’s first congenitally deaf child can have daily conversations after gene therapy
There has been a new breakthrough in the field of biomedicine in Shanghai: At the end of last year, the Eye, Ear, Nose and Throat Hospital of Fudan University carried out the world's first gene therapy for children with hereditary deafness. After nearly 10 months of follow-up, the child's hearing has been clearly improved and he can now... Have everyday conversations. Experts say that this gene therapy "drug" technology opens up a new direction in the treatment of congenital deafness and will bring good news to congenitally deaf children around the world.
Data shows that there are 26 million people with congenital deafness in the world. About 30,000 deaf children are born in my country every year, 60% of which are related to genetic factors, seriously damaging children's hearing, speech and intellectual development. However, there are no clinical treatments.
With the innovation and development of biomedical technology, gene therapy is considered to be one of the most promising and ideal strategies to cure hereditary deafness. This method can introduce normal genes or genes with therapeutic effects into target cells, compensate or correct defective genes or abnormalities, achieve disease treatment, fundamentally improve hearing, and restore hearing closer to the natural sound state. Defects in ototypic proteins are the most common genetic cause of auditory neuropathy, often causing severe, profound, or complete hearing loss and speech impairment in children.
Chief Physician Shu Yilai and the deafness gene therapy team, deputy director of the Otolaryngology Research Institute and director of the Hereditary Deafness Diagnosis and Treatment Center of Fudan University Eye, Ear, Nose and Throat Hospital, strongly support the hospital and the director of the Otolaryngology Research Institute and the Department of Otolaryngology Under the leadership of the director, Professor Li Huawei, and in collaboration with Professor Chen Zhengyi, a well-known hearing medicine expert at Harvard Medical School, after years of hard work, the company successfully developed my country's first gene therapy candidate drug for the treatment of deafness caused by otogene protein defects, and became the first in the world. Initiate clinical trials.
The treatment team uses gene compensation to inject gene therapy drugs into the inner ear of the child through minimally invasive localization to compensate for the defective ototypic protein, thereby restoring or improving the hearing and speech functions of children with OTOF mutation deafness. At present, the team has included many children in an orderly manner according to regulations. Clinical trial results show that inner ear gene therapy strategies are safe and effective in deaf patients. This study provides specific gene therapy candidates for patients with deafness caused by OTOF gene mutations, which is also an important breakthrough in the treatment of hereditary deafness. The team has also established a systematic and mature clinical treatment plan in terms of preoperative assessment, drug administration, intraoperative monitoring, and postoperative care, providing strong support and guarantee for subsequent research and clinical application.
Achievements from China's biomedical field have attracted the attention of international experts. The world's first multiple gene therapy clinical trials at the Eye, Ear, Nose and Throat Hospital, including this child, have been officially accepted as a conference report at the annual meeting of the European Society for Gene and Cell Therapy, and will be presented at the 30th ESGCT Conference in Brussels, Belgium. Share it with the world.
This major clinical breakthrough in gene therapy for congenital deafness is also an attempt to cooperate between institutions and enterprises and use complementary advantages to efficiently promote the transformation of industry, academia and research. Leveraging the company's experience in drug development, the team has opened up channels for gene therapy drug research and development, industrial design, process production, and safety assessment, established a new industry-university-research development model, and opened a new era of gene therapy for deafness. In the future, the Eye, Ear, Nose and Throat Hospital will further continue to promote the development and clinical transformation of a variety of safe and effective deafness treatment drugs, striving to bring more deaf patients back to the world of hearing.
It is also reported that Chief Physician Shu Yilai's deafness gene therapy achievement "Research and Development of Gene Therapy Drugs for Sensorineural Deafness" won the first prize in the "Chunsheng Cup" Medical Innovation Talent Competition. The "Chunsheng Cup" Medical Innovation Talent Competition is under the guidance of the Shanghai Municipal Party Committee Talent Work Leading Group Office and jointly organized by the Municipal Health Commission, the Municipal Science and Technology Commission, the Municipal Economic and Information Technology Commission, the Municipal Intellectual Property Office, and the Municipal Drug Administration. To further discover and cultivate innovative medical talents, stimulate the transformation of scientific and technological achievements in Shanghai's medical and health institutions, and create a medical highland with international influence.
