On March 29, 2023, the new drug clinical trial (IND) application for the AAV gene therapy drug "GC301 adeno-associated virus injection" for the treatment of Pompe disease submitted by Beijing GeneCradle Technology Co., Ltd. (hereinafter referred to as "GeneCradle") was officially approved by the National Medical Products Administration for clinical implicit approval (acceptance number: CXSL2300013), and the indication is late-onset Pompe disease (LOPD). This is another new indication for GC301 injection after the approval of the clinical trial (IND) for infantile Pompe disease.

Pompe disease can be divided into infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD) according to the age of onset and severity. Children with IOPD have complete or almost complete loss of GAA enzyme activity, and progressive myocardial hypertrophy and muscle weakness occur within a few months after birth. Untreated children often die of cardiopulmonary failure within 1 year old. Compared with infantile-onset Pompe disease, patients with late-onset Pompe disease retain some GAA activity, with an onset age of >12 months. The heart involvement is milder than that of IOPD, and is often manifested as progressive muscle weakness and respiratory failure. At the same time, there are multiple system injuries such as the central nervous system, leading to symptoms such as dysarthria, dysphagia, osteoporosis, scoliosis, sleep apnea and hearing loss, which seriously affect the quality of life of patients. GC301 injection delivers normal GAA genes to patients through a one-time intravenous injection and expresses normal GAA enzymes, in order to treat Pompe disease from the root.

While the treatment research and clinical practice of infantile Pompe disease continue to make progress, it is equally urgent for patients with late-onset Pompe disease to enter the clinic as soon as possible for gene therapy drugs. The approval of the new IND brings hope for the accessibility of gene therapy drugs for patients with late-onset Pompe disease, and marks that the applicable population for GC301 injection has expanded from infants to children and adolescents. At present, no gene therapy drugs for Pompe disease have been approved for marketing at home and abroad. The continuous expansion of the applicable population of GC301 injection will greatly meet the urgent clinical needs of Pompe disease patients.

About Pompe Disease
Pompe disease, also known as Pompe disease or Glycogen Storage Disease Type II (GSD II), is a rare lysosomal storage disease. Due to the GAA gene mutation, the activity of acid-α-1,4-glucosidase (α-GAA) in the body is reduced or absent, which makes it impossible to decompose and metabolize intracellular glycogen. A large amount of glycogen accumulates in the cells, causing tissue damage and organ dysfunction. Pompe disease is a multi-system damaging disease, and the impact on tissues with high energy metabolism requirements such as myocardium and skeletal muscle is more significant.

About GC301 Gland-Related Injection
GC301 injection is an AAV gene therapy drug designed and developed by GeneCradle for the treatment of Pompe disease. It adopts a one-time intravenous injection strategy for widespread systemic expression in order to directly compensate for the GAA enzyme gene defects in tissues such as the liver, myocardium, skeletal muscle, and central nervous system. Preclinical experimental results show that GC301 injection can widely transduce various tissues in the animal body and express active GAA protein after a single intravenous injection, improve skeletal muscle and myocardial damage, significantly increase mouse muscle strength, and significantly prolong the survival of model animals, achieving long-term and effective treatment of Pompe disease.

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 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 technologies and products from rare diseases to the treatment and rehabilitation of chronic diseases and other major diseases.