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On February 28, Beijing Genecradle Therapeutics Co., Ltd. independently developed a gene therapy drug for Pompe disease.GC301 Adeno-Associated Virus Injection Receives Orphan Drug Designation from the U.S. Food and Drug Administration (FDA)（ODD）。Source of the image: Screenshot from the FDA Orphan Drug Designation Database websiteFDA Orphan Drug Designation (ODD) is an important policy introduced by the U.S. Food and Drug Administration to encourage the development of drugs for rare diseases. Since the introduction of the Orphan Drug Act in 1983, this policy has successfully channeled industry resources into the rare disease field through a "combination of incentives" such as tax credits, specific research grants, seven years of market exclusivity, and priority review pathways. It has cumulatively driven the approval of over 600 innovative drugs, allowing approximately 5% of rare diseases (out of more than 7,000 worldwide) to progress from "untreatable" to "treatable possibilities."GC301 Granted FDA Orphan Drug Designation: A Milestone for Genecradle's Innovative R&D Capability Recognized by International Authorities. This achievement not only demonstrates the strong R&D capabilities of China but also builds a bridge for over 20 million rare disease patients in China to access cutting-edge therapies aligned with global standards. It also provides valuable practical examples and references for China's advanced therapeutic drugs to enter the international stage and participate in global competition. As a pioneer in the gene therapy field, Genecradle will continue to advance the clinical and commercialization process of the GC301 gene therapy while actively exploring and expanding international collaborations to offer more accessible and effective treatment options for Pompe disease patients worldwide.About GC301 Adeno-Associated Virus Injection
GC301 AAV Injection is a Pompe disease gene therapy drug independently developed by Genecradle based on AAV delivery technology. It systemically compensates for the GAA enzyme gene defect through a one-time intravenous injection, covering key affected tissues such as the liver, myocardium, skeletal muscle, and central nervous system. As the world's first AAV gene therapy for infantile-onset Pompe disease (IOPD), GC301 Injection completed the first dosing in an IIT study in 2022. Its registrational clinical trial has been conducted as a multicenter study at several authoritative institutions in China, including Peking Union Medical College Hospital and PLA General Hospital. Currently, multiple IOPD subjects have completed a 52-week follow-up, with data showing consistently stable safety and efficacy. In the field of late-onset Pompe disease (LOPD) treatment, GC301 gene therapy has also made breakthrough progress. The first dosing of subjects in China’s first registered clinical trial for LOPD gene therapy was completed at the PLA General Hospital. Clinical data show that a single dose of GC301 can achieve long-term expression of the GAA enzyme, significantly improving patients' motor function and quality of life, providing a revolutionary treatment option for the Pompe disease population through root-cause intervention.Pompe disease, also known as glycogen storage disease type II, is a rare autosomal recessive genetic disorder caused by defects in the gene encoding lysosomal acid alpha-glucosidase (GAA). Variations in the GAA gene lead to a deficiency or reduction in GAA enzyme activity, preventing glycogen from being broken down and causing it to accumulate in the lysosomes of muscle cells. Based on the age of onset, affected organs, and disease progression rate, Pompe disease is divided into infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD). The infantile form primarily affects skeletal and cardiac muscles, with rapid disease progression; without effective treatment, patients often die within the first year of life from heart failure and respiratory failure. The late-onset form mainly affects trunk muscles, proximal limb muscles, and respiratory muscles, with significant individual variation in disease progression speed; respiratory failure is the leading cause of death. Pompe disease poses a serious threat to patients' health and has been included in China's first list of rare diseases.Beijing Genecradle Therapeutics Co., Ltd. is a national high-tech enterprise with core business in the development of gene therapy drugs mediated by AAV vector delivery technology. It aims to advance China's rare disease gene drugs from the laboratory to clinical and market stages, benefiting patients and their families. The company focuses on the development of gene therapy drugs for hereditary neuromuscular diseases, inherited metabolic disorders, lysosomal diseases, and ophthalmic conditions, promoting the research, development, and clinical application of gene-based treatments for rare diseases.
