On February 25, 2023, the Phase I/IIa clinical trial project launch meeting of GC101 adeno-associated virus injection, a gene therapy drug for the treatment of type I spinal muscular atrophy (SMA) patients, developed by Beijing GeneCradle Technology Co., Ltd. (hereinafter referred to as "GeneCradle"), was successfully held at the Seventh Medical Center of the General Hospital of the Chinese People's Liberation Army, marking the official launch of the clinical trial of GC101 for the treatment of type I SMA in the research center.

Deputy Director Dong Cheng of the Seventh Medical Center of the General Hospital of the People's Liberation Army, Deputy Director Cai Yun of the Drug Clinical Research Laboratory of the General Hospital of the People's Liberation Army, Professor Feng Zhichun, the principal investigator of the Seventh Medical Center of the General Hospital of the People's Liberation Army, Teacher Xu Juan of the Drug Clinical Trial Institutional Office of the Seventh Medical Center of the General Hospital of the People's Liberation Army, Teacher Liang Hong, Secretary of the Ethics Office of the Seventh Medical Center of the General Hospital of the People's Liberation Army, Professor Zhu Zhiming, Deputy General Manager of Clinical Operations of Beijing GeneCradle Technology Co., Ltd., and the person in charge of the clinical trial collaboration were invited to attend the project launch meeting. The experts and representatives of all parties at the meeting expressed warm congratulations on the launch of this project and made speeches.
Teacher Xu Juan of the Drug Clinical Trial Institutional Office of the Seventh Medical Center of the General Hospital of the People's Liberation Army extended sincere congratulations on the launch of this project and said that the drug clinical trial institution will fully support this project. At the same time, it is also hoped that all participants will cooperate sincerely, play their respective responsibilities, and complete this project with quality and quantity in accordance with GCP regulations and program requirements, and wish this project a complete success.
Deputy Director Dong Cheng of the Seventh Medical Center of the General Hospital of the Chinese People's Liberation Army expressed his sincere congratulations on the successful launch of the project, and said that the successful implementation of this project is of great significance to the discipline construction of the center and the treatment of children with type 1 SMA. He wished the project a complete success with the joint efforts and support of all parties.
At the launch meeting, GeneCradle representatives and relevant persons in charge of the clinical contract research organization reported on the clinical trial of the GC101 type I SMA project, and made detailed introductions to the key contents of clinical trial process management such as clinical trial plan, clinical operation, subject management, drug management, safety management, etc. The participants had a full discussion on the details of the implementation of the clinical trial. Professor Zhu Zhiming, deputy general manager of clinical operations of Jinlan, expressed his sincere gratitude to all parties for their support for the project, and said that he would actively coordinate with all parties to control the progress of the trial and ensure timely and smooth communication. He also hoped that all parties would make persistent efforts in the next work, overcome difficulties, and jointly promote the progress of the project according to the established goals and plans to achieve the expected target results.

As the principal investigator of this clinical trial project, Professor Feng Zhichun of the Seventh Medical Center of the General Hospital of the Chinese People's Liberation Army highly praised the technical strength of the GeneCradle project team and expressed his confidence in the clinical trials of this project. He pointed out that this project is the world's first clinical study of gene therapy drugs for the treatment of type I spinal muscular atrophy via intrathecal administration, which is of milestone significance for expanding the existing treatment methods for type I spinal muscular atrophy.

In the previous clinical study initiated by researchers at the Seventh Medical Center of the PLA General Hospital, GeneCradleGC101 adeno-associated injection has been successfully used in 18 SMA patients, fully demonstrating the team's rich experience in gene therapy and providing a practical basis for the smooth implementation of IND research.

The launch meeting was successfully concluded after full discussion and confirmation by all parties, marking the official launch of the Phase I/IIa clinical trial of the GeneCradleGC101 project. As the first gene drug for intrathecal administration to treat type I SMA in China, GC101 injection is expected to provide clinicians with a new long-term treatment method for the benefit of SMA patients.

About GC101 injection

GC101 adeno-associated virus injection is a recombinant adeno-associated virus type 9 vector (rAAV9) carrying a normal SMN1 gene expression unit. It is administered once by intrathecal injection to express the SMN1 gene in motor neuron cells, thereby improving the function of affected cells such as motor neurons. Assisted by respiratory and motor rehabilitation training, it can improve the respiratory and motor abilities of SMA patients.

About GeneCradle

Beijing GeneCradle Technology Co., Ltd. is a national high-tech enterprise with AAV vector delivery technology-mediated gene therapy drug development as its core business. Its mission is to promote China's rare disease gene drugs from basic to clinical and market, benefiting patients and families. The company focuses on the development of gene therapy drugs in the fields of hereditary neuromuscular diseases, genetic metabolic diseases, lysosomal diseases and ophthalmic diseases. By promoting the development and clinical application of rare disease gene drugs, it has a deeper understanding of life and health, and has transitioned gene therapy technology and products from rare diseases to the treatment and rehabilitation of chronic diseases and other major diseases.