On April 6, 2023, the new drug clinical trial (IND) application submitted by Beijing GeneCradle Technology Co., Ltd. (hereinafter referred to as "GeneCradle") for the AAV gene therapy drug "GC101 adeno-associated virus injection" for the treatment of spinal muscular atrophy was granted clinical implicit approval by the National Medical Products Administration (acceptance number: CXSL2300035). The approved indication is type 3 spinal muscular atrophy (type 3 SMA). This is the third new indication for GC101 injection after the approval of clinical trials for type 1 SMA and type 2 SMA.
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular genetic disease. According to the severity of the disease and the age of onset, SMA is mainly divided into five phenotypes. Type 3 SMA patients develop the disease 18 months after birth, with normal early motor development, can walk independently, and some have delayed independent walking. With age, proximal muscle weakness appears, with the lower limbs being more severe than the upper limbs, and eventually the ability to walk independently is partially lost, and gradually becomes dependent on a wheelchair. As the disease progresses, limb fasciculations and foot deformities may occur, and some patients' daily lives are affected by scoliosis and respiratory insufficiency. Compared with type 1 and type 2 SMA patients, the life expectancy of type 3 patients is not shortened or slightly reduced, but their normal life will also be affected by factors such as long-term medication and gradual loss of motor ability.
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 neurons, 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. The newly added clinical trial indications include type 3 SMA patients aged 2 to 25 years.
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.