Jinlan Gene and Buyang Gene Announce Strategic Collaboration to Develop Next-Generation Gene Therapy for Duchenne Muscular Dystrophy (DMD)

Feb 25, 2025 11:09 CST Updated 11:09

Genecradle

Gene Therapy Drug Developer

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Recently, Beijing Genecradle Therapeutics Co., Ltd. (Genecradle) and Beijing Buyang Gene Technology Co., Ltd. (Buyang Gene) officially signed a strategic cooperation agreement in Beijing. The two parties will jointly collaborate to develop the next generation of DMD (Duchenne Muscular Dystrophy) gene therapy to meet the clinical needs of DMD patients.

Genecradle officially launched the development of DMD gene therapy drugs in 2021, focusing on the optimization and modification of Dystrophin protein and regulatory elements. Compared with mini-Dystrophin or micro-Dystrophin, Genecradle has innovatively designed Smart-Dystrophin with more comprehensive biological activity, which has been tested in various DMD model animals. Its accurate biological positioning characteristics have been verified, improving the disease progression in model animals.

Meanwhile, Genecradle, relying on its self-developed DynEvoLib® capsid discovery platform, has successfully developed a series of novel AAV capsids with muscle-targeting properties. Compared with traditional vectors, the new AAV capsids demonstrate significant advantages in transgene expression and safety, providing a more efficient delivery tool for DMD gene therapy.

This strategic cooperation combines Genecradle's profound expertise in AAV gene drug development with Buyang Gene's unique advantages in novel AAV capsid development. It is expected to enhance therapeutic efficacy and safety while reducing the dosing level, bringing new hope for DMD treatment. In the future, both parties will continue to move forward hand in hand, jointly promoting the development and innovation of China's self-developed gene therapies, and bringing good news to more patients.

About Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is an X-linked recessive genetic disorder. Its cause lies in mutations of the Dystrophin gene, leading to the absence of dystrophin in muscle fibers, which in turn causes muscle fiber damage, muscle atrophy, and loss of function. DMD primarily affects boys, with one in every 3,500 newborn males globally being afflicted. Patients experience muscle weakness or atrophy due to progressive degeneration of skeletal muscles before school age, resulting in mobility difficulties. Between the ages of 7 and 12, patients typically lose the ability to walk entirely and often succumb to cardiac or respiratory muscle weakness by their 20s.

The clinical manifestations of DMD are diverse, including progressive symmetrical muscle weakness, with the proximal muscles more severely affected than the distal ones. Children often exhibit delayed walking, frequent falls, and slow walking. As the condition progresses, patients gradually develop difficulties climbing stairs, squatting, and standing up, and may even completely lose their ability to walk independently. Additionally, some patients may experience non-progressive cognitive abnormalities, such as intellectual disabilities, learning difficulties, and attention deficit hyperactivity disorder (ADHD).

Currently, there is no specific cure for DMD. The main treatment methods include regular rehabilitation training, nutritional enhancement, and other symptomatic supportive treatments. In terms of drug therapy, glucocorticoids such as prednisone are commonly used medications that can improve patients' strength and lung function, but long-term use has significant side effects. Although existing treatments can delay disease progression to a certain extent, there is currently no effective cure targeting the root cause of DMD—the mutation in the dystrophin gene—indicating a huge unmet clinical need for patients. Therefore, developing new treatment methods, such as gene therapy, is a major focus and challenge in current DMD clinical research both in China and internationally.

About Genecradle

Beijing Byongen Therapeutics Co., Ltd. was established in July 2021, focusing on the development of adeno-associated virus (AAV) capsids to provide precise and safe next-generation vectors for the gene therapy field. The company's proprietary capsid discovery platform, DynEvoLib®, utilizes computer-aided design and directed evolution technology to screen for novel AAV capsids with precise targeting, high transduction efficiency, and low immunogenicity, overcoming the bottleneck of in vivo gene delivery.

About Genecradle

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. Its mission is to advance China's rare disease gene therapies from basic research to clinical and market applications, 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. By promoting the research, development, and clinical application of rare disease gene drugs, the company seeks to deepen the understanding of life and health, transitioning gene therapy technologies and products from rare diseases to the treatment and rehabilitation of chronic and other major diseases.

Source of the article:BJGC Genecradle