CANbridge-UMass Chan Medical School Spinal Muscular Atrophy Gene Therapy Animal Data to be Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

-Novel Second-Generation SMA Gene Therapy Rescues Motor Function in Mice,  Outperforms Benchmark Therapy


Burlington, Mass.; Beijing, China, May 5, 2023- CANbridge Pharmaceuticals Inc. (HKEX:1228), a leading China-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that data on the novel second-generation gene therapy, developed under the CANbridge and UMass Chan Medical School research agreement, will be presented at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, May 16-20, Los Angeles, CA.


The study reports that a novel second-generation hSMN1-AAV gene therapy vector, consisting of an endogenous SMN1 promoter and codon-optimized human SMN1 transgene in two different AAV serotypes, outperformed the benchmark gene therapy across several endpoints, including lifespan, weight gain and motor functions, in a mouse model of spinal muscular atrophy (SMA) when administered via intracerebroventricular (ICV) delivery. Furthermore, when delivered via ICV at one-tenth the dose used in both high- and low-dose cohorts in an earlier IV study, the second-generation gene therapy produced similar lifespan and body weight gains and improved motor functions. The benchmark therapy uses a vector similar to that used in the only gene therapy approved for SMA.


“ICV delivery of our second-generation SMA gene therapy at one-tenth the dose used in our IV study matches or improves upon the IV results we reported last year at ASGCT,” said Gerry Cox, MD, PhD, chief development strategist and interim chief medical officer, CANbridge Pharmaceuticals. “We believe that the transgene expression in our second-generation SMA gene therapy better mimics the natural physiological levels of SMN1 in transduced tissues. This gene therapy could offer a safer, more effective and more efficient clinical approach for treating SMA, which aligns with the CANbridge mission to create innovative therapies with improved market access to patients.”


Presentation Details:

Title: Low-dose intracerebroventricular delivery of a second-generation AAV gene therapy for spinal muscular atrophy achieves efficient and toxicity-free motor function rescue in mice


Poster: #698


Session Date and Time: “Wednesday Poster Session”

                                     May 17, 12PM PDT


Authors: Xiupeng Chen, Qing Xie, Hong Ma, Veena Kumanan, Jun Yang, Yijie Ma, Leila Jalinous, Qin Su, Phillip WL Tai, Guangping Gao, Jun Xie


Abstracts are available on the ASGCT website:


About UMass Chan Medical School

UMass Chan Medical School, one of five campuses of the University of Massachusetts system, comprises the T.H. Chan School of Medicine; the Morningside Graduate School of Biomedical Sciences; the Tan Chingfen Graduate School of Nursing; ForHealth Consulting of UMass Chan Medical School, , a public service consulting division; and MassBiologics, the only nonprofit, FDA-licensed manufacturer of vaccines, biologics and viral vector gene therapies in the United States. UMass Chan’s mission is to advance the health and wellness of our diverse communities throughout Massachusetts and across the world by leading and innovating in education, research, health care delivery and public service. In doing so, it has built a reputation as a world-class research institution and as a leader in primary care education, perennially ranked in the top 10 percent of medical schools for primary care by U.S. News and World Report. UMass Chan attracts more than $300 million annually in research funding, placing it among the top 50 medical schools in the nation. In 2021, the Medical School received a $175 million donation from The Morningside Foundation and was renamed UMass Chan Medical School.  


About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. Animal data from the SMA gene therapy was presented in 2022 at the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global.

For more on CANbridge Pharmaceuticals Inc., please go to:


Forward-Looking Statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the data on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.





U.S. Investor Relations:

Chris Brinzey

ICR Westwicke


China Investor Relations

CANbridge Pharmaceuticals Inc.



Deanne Eagle

Planet Communications