CANbridge Sets Its Sights on Gene Therapy Leadership

We are thrilled to announce that we have secured exclusive global rights to a potentially best-in-class gene therapy for Spinal Muscular Atrophy from UMass Chan Medical School. To top it off, we have also successfully completed the technology transfer of two gene therapies for Lysomal Storage Diseases. This is a major breakthrough in the fight against these devastating conditions.

CANbridge Pharmaceuticals, Inc. (1228.HK) is thrilled to announce that it has secured exclusive worldwide rights to develop, manufacture, and commercialize a breakthrough second-generation gene therapy for the treatment of Spinal Muscular Atrophy (SMA). This major milestone was made possible thanks to the groundbreaking research conducted by the UMass Chan Medical School. CANbridge is committed to bringing transformational therapies to those living with rare diseases and cancer.

At the American Society of Gene and Cell Therapy (ASGCT), the European Society of Gene and Cell Therapy (ESGCT) and the World Muscle Congress in 2022, groundbreaking research was presented on the potential for gene therapy to outperform the current standard-of-care for Spinal Muscular Atrophy (SMA). Sponsored by CANbridge, the data from mouse models revealed that this gene therapy not only outperformed the benchmark therapy across multiple key endpoints, but also showed much less liver toxicity when administered intravenously. With this exciting research, a new hope is emerging for those living with SMA.

CANbridge has completed the full technology transfer of gene therapy products from LogicBio® Therapeutics, granting it exclusive worldwide license to the products in the Fabry and Pompe gene therapy programs that use the AAV sL65 liver targeting capsid. The company also obtained non-exclusive worldwide rights to the LogicBio proprietary manufacturing process for Fabry and Pompe gene therapies, as well as option rights to sL65-based therapies for two additional indications, as well as to LB-001, an investigational treatment for methylmalonic acidemia, in Greater China. This agreement marks a major milestone in CANbridge’s mission to develop innovative treatments for rare diseases.

CANbridge Pharmaceuticals Inc. is excited to announce the development of a potential best-in-class gene therapy for Spinal Muscular Atrophy (SMA). Developed in collaboration with UMass Chan, the therapy holds great promise in treating this rare disease which currently has limited treatment options. Additionally, the company has recently opened their CANbridge Next-Generation Innovation and Process Development Facility in Burlington, Massachusetts, where they will be working on two additional gene therapy programs for Fabry and Pompe diseases. It is CANbridge’s hope that these therapies will bring relief to those suffering from these rare diseases.

CANbridge’s novel hSMN1 AAV gene therapy vector is an exciting breakthrough in the treatment of spinal muscular atrophy. With its improved potency and safety profile compared to benchmark treatments, this vector has the potential to make a tremendous difference in the lives of those affected by this devastating disease. According to Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research, we can have great confidence in CANbridge’s ability to develop this groundbreaking therapy.

About the Second-Generation Gene Therapy

The new, second-generation gene therapy (scAAV9-SMN1p-co-hSMN1) for spinal muscular atrophy has been found to be a major breakthrough in the fight against this devastating disease. Compared to the benchmark gene therapy that is used in the presently approved treatment, this therapy resulted in longer life spans, improved muscle function, and better nerve conduction, without any of the liver toxicity associated with the benchmark vector. The secret to its success lies in its self-complementary AAV9 gene therapy, which expresses a codon-optimized human SMN1 gene under the control of an endogenous promoter. The findings were presented in 2022 at various conferences, such as the American Society of Gene and Cell Therapy (ASGCT), the European Society of Gene and Cell Therapy (ESGCT) and The World Muscle Society Congress, and are set to revolutionize the treatment of SMA.

CANbridge is on the brink of revolutionizing the treatment of spinal muscular atrophy. Through pioneering gene therapy research and development, they are pushing the boundaries of what is possible, and have secured exclusive global rights to develop, manufacture and commercialize the treatment.

About Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an insidious genetic disorder that affects approximately 1 in 6,000 to 10,000 newborns worldwide. It is caused by a lack of a functioning motor survival motor neuron 1 (SMN1) gene, which codes for a critical protein essential for motor neuron survival. Without treatment, SMA1 can lead to feeding and ventilation support or death by age two, while SMA2 can result in an inability to walk by 24 months. SMA3 and SMA4, which occur from childhood to adulthood, both cause debilitating motor function loss and, in many cases, premature death. For those afflicted, SMA is a devastating and irreversible condition that can tragically cut their lives short.

Although recent approvals have opened up new treatment options for SMA, there remains an urgent need for more therapeutic options. Despite advancements in medical care, the vast majority of people living with SMA still lack an effective treatment.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) is an innovative biopharmaceutical company with a strong presence in China dedicated to the research, development and commercialization of life-changing therapies for rare diseases and rare oncology. With three approved drugs in its diverse drug portfolio and a pipeline of 10 assets targeting prevalent rare diseases, CANbridge is leading the way in addressing unmet medical needs. Its Next-Generation Innovation and Process Development Facility is developing groundbreaking gene therapies that could be curative for rare genetic diseases such as Pompe disease, Fabry disease, spinal muscular atrophy (SMA) and other neuromuscular conditions. CANbridge has presented animal data from its SMA gene therapy at the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. As a global leader, CANbridge is proud to partner with world-renowned organizations such as Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global.

Forward-Looking Statements

Looking to the future, it’s important to be aware that our actual results or performance may differ from our expectations. We may alter our intentions at any time based on future developments, so it’s important to stay up-to-date with the latest news. As of the date of this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made. We undertake no obligation to update or revise publicly any forward-looking statements, so be sure to read this article in its entirety.

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