Beijing GeneCradle Technology Co., Ltd. (hereinafter referred to as "GeneCradle") independently developed the first AAV intrathecal gene drug for the treatment of type 1 SMA in China, GC101 adeno-associated virus injection, which has passed the Phase I safety assessment and is currently recruiting patients for the Phase II registration clinical trial.
The lead unit of this Phase II registration clinical trial is the Seventh Medical Center of the Chinese People's Liberation Army General Hospital, and will recruit 24-30 eligible children with type 1 SMA in multiple centers such as Peking University First Hospital, Shengjing Hospital Affiliated to China Medical University, and Children's Hospital Affiliated to Shandong University. See the table below for details:
GC101Clinical trial of treating SMA shows good safety
GC101 injection IND registration clinical trial has been launched in multiple research centers, including 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, Children's Hospital Affiliated to Shandong University, and Wuhan Children's Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology. Currently, 11 patients with type 1 SMA and 29 patients with type 2 SMA have received one-time gene therapy with GC101 injection. Existing data show the good safety and preliminary effectiveness of GC101 injection, and the young patients who have received treatment have also shown varying degrees of improvement in motor ability.
Please pay attention to the recruitment information of SMA type 3 registration clinical trial
On May 6, 2024, the registration clinical trial of GC101 injection for the treatment of type 3 SMA (acceptance number CXSL2300035) has been approved by the ethics committee of Beijing Tiantan Hospital affiliated to Capital Medical University, the lead unit. The lead unit will take the lead in recruiting subjects in the near future.Please pay attention to the subsequent information release。
About Spinal Muscular Atrophy
Spinal muscular atrophy is a hereditary neuromuscular disease caused by a defect in the function of the SMN protein due to a mutation in the survival motor neuron gene 1 (SMN1). It is one of the autosomal recessive genetic diseases that causes infant mortality. The carrier frequency of the pathogenic gene in the Chinese population is about 1/42, and the incidence rate in newborns is about 1/10,000. Type 1 SMA presents with obvious muscle weakness at birth or within a few days after birth. The symptoms are quite obvious by 6 months of age. Children with weakened muscle tone and reflexes have difficulty sucking, swallowing and breathing. Most patients with type 2 SMA lose the ability to sit alone and become paralyzed during adolescence, and patients often die from complications such as respiratory failure; patients with type 3 SMA are affected by long-term medication, gradual loss of motor ability and other factors that affect their normal life. In the classification of SMA diseases, children with type 1 SMA are the most serious. If they do not receive timely and effective treatment intervention, the median survival period is 13.6 months, and most do not exceed 24 months. It is the number one lethal genetic disease for infants and young children.
About GC101 Adeno-Associated Virus 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 via 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 the development of gene therapy drugs mediated by AAV vector delivery technology as its core business. Its mission is to promote China's rare disease gene drugs from basic to clinical and market, benefiting patients and their 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.