On February 19, 2025, Beijing Genecradle Therapeutics Technology Co., Ltd. (Genecradle Therapeutics) and Beijing Byongen Therapeutics Co., Ltd. (Byongen Therapeutics) formally signed a strategic cooperation agreement in Beijing to jointly develop the next-generation gene therapy for Duchenne Muscular Dystrophy (DMD), aiming to meet the clinical needs of DMD patients.
Genecradle Therapeutics initiated the development of DMD gene therapy in 2021, focusing on the optimization and modification of the Dystrophin protein and regulatory elements. Compared to mini-Dystrophin or micro-Dystrophin, Genecradle Therapeutics has originally designed Smart-Dystrophin, which has more comprehensive biological activity. This design has been tested in multiple DMD animal models, where it has demonstrated accurate biological localization features and improved disease progression in the models.
Meanwhile, Byongen Therapeutics has successfully developed a series of novel AAV capsids with muscle-targeting properties using its proprietary DynEvoLib® capsid discovery platform. Compared to traditional vectors, the new AAV capsids show significant advantages in transgene expression and safety, providing a more efficient delivery tool for DMD gene therapy.
This strategic cooperation combines Genecradle Therapeutics' profound experience in AAV gene drug development with Byongen Therapeutics' unique strengths in novel AAV capsid development. It is expected to enhance therapeutic efficacy and safety while reducing the dosage required for treatment, bringing new hope to DMD therapy. In the future, the two parties will continue to work together to promote the development and innovation of domestically developed gene therapies in China, bringing good news to more patients.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is an X-linked recessive genetic disorder. The disease is caused by mutations in the Dystrophin gene, leading to the absence of Dystrophin protein in muscle fibers, which in turn causes muscle fiber damage, muscle atrophy, and loss of function. DMD primarily affects boys, with an incidence rate of approximately 1 in 3,500 male newborns globally. Patients begin to experience muscle weakness or atrophy due to the continuous degeneration of skeletal muscles in early childhood, leading to difficulties in walking. Around the ages of 7 to 12, patients typically lose their ability to walk and often succumb to cardiomyopathy or respiratory muscle weakness in their twenties.
The clinical manifestations of DMD are diverse, including progressive symmetrical muscle weakness that is more pronounced in the proximal muscles than in the distal ones. Affected children often exhibit delayed walking, frequent falls, and slow gait. As the disease progresses, patients gradually experience difficulty in climbing stairs and rising from a squat, eventually losing their ability to walk independently. In addition, some patients may also present non-progressive cognitive abnormalities, such as intellectual disability, learning difficulties, and attention deficit hyperactivity disorder (ADHD).
Currently, there is no specific cure for DMD. The main treatment methods include regular rehabilitation training and symptomatic supportive therapies such as enhanced nutrition. In terms of pharmacological treatment, corticosteroids like prednisone are commonly used to improve patients' strength and pulmonary function, although long-term use is associated with significant side effects. While existing treatments can delay disease progression to some extent, there is currently no effective cure targeting the root cause of DMD—the mutation in the Dystrophin gene. The unmet clinical needs of patients are substantial. Therefore, the development of new therapeutic approaches, such as gene therapy, is a current hot topic and challenge in DMD clinical research both domestically and internationally.
About Byongen Therapeutics
Beijing Byongen Therapeutics Co., Ltd. (Byongen Therapeutics) was established in July 2021, focusing on the development of AAV capsids to provide next-generation vectors that are precise and safe for gene therapy. The company's proprietary capsid discovery platform, DynEvoLib®, utilizes computer-aided design and directed evolution techniques to screen for novel AAV capsids with precise targeting, high transduction efficiency, and low immunogenicity, breaking through the bottleneck of in vivo gene delivery.
About Genecradle Therapeutics
Beijing Genecradle Therapeutics Technology Co., Ltd. is a national high-tech enterprise with core business in the development of gene therapy drugs mediated by AAV vector delivery technology. The company is committed to advancing rare disease gene drugs from the laboratory to clinical and market applications, benefiting patients and their families. It specializes in the development of gene therapy drugs for genetic neuromuscular diseases, inherited metabolic diseases, lysosomal diseases, and ophthalmic diseases. By promoting the research and clinical application of rare disease gene drugs, the company aims to deepen the understanding of health and life, transitioning gene therapy technology and products from rare diseases to the treatment and rehabilitation of chronic and other major diseases.