CANbridge-UMass Chan Medical School Gene Therapy Research to be Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

First Study from the CANbridge UMass Chan Medical School Rare Disease Gene Therapy Research Collaboration 


Beijing, Cambridge, Mass., May 3, 2022- CANbridge Pharmaceuticals Inc., (“CANbridge,”  1228.HK) 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 from its gene therapy research agreement with the Horae Gene Therapy Center, at the UMass Chan Medical School, will be presented at the  ASGCT 25th Annual Meeting, being held in Washington D.C. from May 16 - 19th. Researchers will present findings from an animal study exploring a novel second-generation gene therapy for the potential treatment of spinal muscular atrophy (SMA). This is the first data to come out of the CANbridge rare disease gene therapy research collaboration with the UMass Chan Medical School, which is focused on developing gene therapy treatments for neuromuscular conditions with unmet medical needs.


Presentation Details:

Title: Endogenous Human SMN1 Promoter-driven Gene Replacement Improves the Efficacy and Safety of AAV9-mediated Gene Therapy for Spinal Muscular Atrophy in Mice

Poster #:M-144

Category: Neurologic Diseases I

Session Date and Time:   Monday, May 16, 5:30-6:30 PM

Authors: Qing Xie, Hong Ma, Xiupeng Chen, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Qin Su, Phillip Tai, Guangping Gao, Jun Xie

Abstracts are available on the ASGCT website:


About the Horae Gene Therapy Center at UMass Chan Medical School

The faculty of the Horae Gene Therapy Center is dedicated to developing therapeutic approaches for rare inherited disease for which there is no cure. We utilize state of the art technologies to either genetically modulate mutated genes that produce disease-causing proteins or introduce a healthy copy of a gene if the mutation results in a non-functional protein.

The Horae Gene Therapy Center faculty is interdisciplinary, including members from the departments of Pediatrics, Microbiology & Physiological Systems, Biochemistry & Molecular Pharmacology, Neurology, Medicine and Ophthalmology. Physicians and PhDs work together to address the medical needs of rare diseases, such as Alpha 1-Antitrypsin Deficiency, Canavan Disease, Tay-Sachs and Sandhoff diseases, Retinitis Pigmentosa, Cystic fibrosis, Lou Gehrig's disease, TNNT1 nemaline myopathy, Rett syndrome, N-Gly 1 deficiency, Pitt-Hopkins syndrome, Marple Syrup Urine Disease, Sialidosis, GM3 synthase deficiency, Huntington's disease, ALS and others. More common diseases such as cardiac arrhythmia and hypercholesterolemia are also investigated. The hope is to treat a wide spectrum of diseases by various gene therapeutic approaches. Additionally, the University of Massachusetts Chan Medical School conducts clinical trials on site and some of these trials are conducted by the investigators at the Gene Therapy center.


About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (“CANbridge,” 1228.HK) is a China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies.

CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with significant market potential, targeting some of the most prevalent rare diseases and rare oncology.

These include Hunter syndrome (MPS II) and other lysosomal storage disorders (LSDs), complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) as well as glioblastoma multiforme (GBM).

CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments. CANbridge global partners include, but are not limited to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Chan Medical School, the University of Washington School of Medicine, LogicBio and Scriptr.

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CANbridge Pharmaceuticals Inc.



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Planet Communications