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ENWorld's First! Leadingpharm CXO · Deep Blue Ocean Bio-Pharmaceutical Assists Clinical Research of Non-Viral Vector Gene Therapy for DMD, Opening a New Paradigm in Gene Therapy!
This is also the world's first investigator-initiated trial (IIT) using extracellular vesicle delivery platform to introduce gene therapy drugs into the human body.
The project, entitled "A Novel mRNA Platform Based on Engineered Extracellular Vesicles (EV) for the Treatment of Duchenne Muscular Dystrophy", aims to evaluate the safety, tolerability and preliminary efficacy of FL-dystrophin mRNA delivered by engineered extracellular vesicles in DMD patients, providing a safer and more efficient gene therapy option for patients.
With its profound experience in the field of gene therapy clinical trials, Leadingpharm CXO · Deep Blue Ocean Bio-Pharmaceutical provides full-process empowerment from the application to the Ethics Committee of Human Genetic Resources Administration, ethical review support, clinical operation management to data quality control, helping this breakthrough technology smoothly transition from preclinical research to clinical trial stage.
01 Research Background and Scientific Basis
Duchenne muscular dystrophy is a fatal genetic disease caused by mutations in the dystrophin gene on the X chromosome, affecting 1 in 3,500 male infants worldwide¹. Due to the loss of muscle cell membrane stability, patients suffer from progressive muscle weakness, usually lose the ability to walk in adolescence, and die early from cardiopulmonary failure.
Currently, there is no cure for the disease. Although current therapies such as ASO or AAV vector gene therapy can partially restore dystrophin expression, they still have limitations:
Lack of universality to cure all mutation types;
Single treatment with AAV may activate innate and adaptive immune responses, leading to multiple organ failure, especially liver, heart and lung damage, or even death from liver failure²;
Clinical cases of AAV treatment show that its toxicity is dose-dependent and closely related to the patient's baseline status (such as liver disease)³. In addition, on June 15, 2025, Elevidys, the only gene therapy drug approved by the US FDA for DMD, was withdrawn from the market due to multiple cases of AAV-induced hepatotoxicity leading to patient deaths.
These events highlight the need to develop new and safe non-viral treatment strategies for muscular dystrophy.
02 Advantages of mRNA Therapy
The success of mRNA vaccines in combating the COVID-19 pandemic has drawn attention to mRNA therapy. mRNA therapy refers to the direct induction of target protein synthesis in cells by mRNA. Compared with AAV therapy, it has the advantages of low toxicity and low immunogenicity. In addition, mRNA therapy has good plasticity and can be used to encode any protein. Therefore, mRNA therapy can directly supplement the missing dystrophin in DMD patients, thereby restoring muscle fiber integrity and solving the problems of loss of muscle regeneration ability and mitochondrial dysfunction.
Many clinical and preclinical studies have found that AAV, as a vector for gene drugs, has many defects, such as causing hepatotoxicity, nerve and blood damage, renal failure caused by thrombotic microangiopathy, and death from acute liver failure. These adverse reactions limit the research and development of AAV therapy in the treatment of DMD, and there is an urgent need to innovate and develop safer and more effective treatment methods.
mRNA therapy can overcome the limitations of ASO and AAV therapies in the treatment of DMD, but its practical application in clinical practice requires safe and effective delivery vectors.
03 Innovative EV mRNA Platform
To break through this bottleneck, the research team took a unique approach and turned to engineered extracellular vesicles (EVs), a natural nanoscale delivery vector.
Engineered EVs, as tiny vesicles secreted by cells, have excellent biocompatibility and low immunogenicity, and can easily cross multiple biological barriers to accurately deliver the carried genetic material to target cells.
The research team has long been committed to research in the field of drug delivery. Based on the solid foundation of their previous preclinical and translational work on the therapeutic application of EVs, they have rich research experience in optimizing EV-mediated mRNA delivery strategies⁴˒⁵.
The EV mRNA platform breaks through technical bottlenecks through the following innovative designs:
Full-length protein supplementation: Delivering FL-dystrophin mRNA through EVs directly repairs gene defects, breaking the limitations of traditional therapies due to incomplete function of truncated proteins;
Non-viral delivery system: Unlike AAV vectors, which can cause immunogenicity and cardiotoxicity, there is no risk of viral toxicity. It is the first clinical trial using non-viral vector EVs for the treatment of rare diseases including DMD;
Broad applicability: Suitable for all mutation types, including patients with nonsense mutations and frameshift mutations;
Repeatable administration: Breaking the limitation of single treatment of AAV therapy due to immunogenic reactions, realizing a repeated administration strategy that can adjust the dose of delivered mRNA according to the patient's recovery.
Based on favorable preclinical data, the team decided to officially launch the IIT study, hoping that the success of this trial can provide more data on safety and efficacy for gene therapy of DMD.
References:
[1] https://www.frontiersin.org/journals/physiology/articles/10.3389/fphys.2023.1183101/full
[2] https://www.nejm.org/doi/full/10.1056/NEJMoa2307798
[3] https://www.nature.com/articles/s41587-020-0642-9
[4] https://www.nature.com/articles/s41551-022-00989-w
[5] https://academic.oup.com/eurheartj/article/46/17/1662/7959994
About Us
Deep Blue Ocean Bio-Pharmaceutical, a sub-brand of Leadingpharm (stock code: 600222) in the field of clinical CRO, was established in 2007. Adhering to the service concept of "starting from the end, top-level design, and being a full-process partner in new drug creation", it provides global pharmaceutical companies with services covering Phase I-III clinical research, bioequivalence research, post-marketing research, third-party audits, IND and NDA applications, and medical affairs for biological drugs, chemical drugs, traditional Chinese medicines, medical devices, cell and gene therapy, etc. It can meet the customized clinical research needs of different R&D-oriented enterprises and help domestic and foreign customers break through key bottlenecks before new drug launch.
At present, the company has carried out more than 2,000 clinical research projects, including dozens of Class I innovative drug projects and 50-100 BE studies every year. In addition to carrying out related businesses in the fields of rheumatology and immunology, oncology, cardiovascular and cerebrovascular diseases, and endocrinology, it has taken the lead in providing clinical research services in the fields of medical aesthetics, medical food for special medical purposes, cell and gene therapy drugs, filling multiple gaps at home and abroad.
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