BEIJING AND CAMBRIDGE, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals Inc. (“CANbridge”, 1228.HK), a leading global biopharmaceutical company based in China and engaged in the research, development and commercialization of transformative therapies for rare diseases and rare oncology , announced that data from its gene therapy research agreement with the Horae Gene Therapy Center, at UMass Chan Medical School, will be presented at ASGCT 25and Annual Meeting, to be held in Washington DC May 16-19and. The researchers will present the results of an animal study exploring a novel second-generation gene therapy for the potential treatment of spinal muscular atrophy (SMA). This is the first data from the CANbridge Rare Disease Gene Therapy Research Collaboration with UMass Chan Medical School, which focuses on developing gene therapy treatments for neuromuscular diseases with unmet medical needs.
Title: Endogenous Human SMN1 Promoter-Induced Gene Replacement Improves Efficacy and Safety of AAV9-Mediated Gene Therapy for Spinal Muscular Atrophy in Mice
Poster #: M-144
Category: Neurological diseases I
Date and time of the session: Monday, May 16, 5:30-6:30 p.m.
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: https://annualmeeting.asgct.org
About Horae Gene Therapy Center at UMass Chan School of Medicine
The faculty of the Horae Gene Therapy Center is dedicated to developing therapeutic approaches for rare inherited diseases for which there is no cure. We use 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 faculty at the Horae Gene Therapy Center is interdisciplinary and includes members from the departments of Pediatrics, Microbiology and Physiological Systems, Biochemistry and Molecular Pharmacology, Neurology, Medicine, and Ophthalmology. Physicians and doctors work together to meet 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’s syndrome, N-Gly 1 deficiency, Pitt-Hopkins syndrome, Marple syrup urinary disease, sialidosis, GM3 synthase deficiency, Huntington’s disease, ALS and others . More common diseases such as cardiac arrhythmia and hypercholesterolemia are also studied. The hope is to treat a wide range of diseases by various gene therapy approaches. Additionally, the University of Massachusetts Chan Medical School conducts on-site clinical trials and some of these trials are conducted by investigators from the gene therapy center.
About CANbridge Pharmaceuticals Inc.
CANbridge Pharmaceuticals Inc. (“CANbridge”, 1228.HK) is a China-based global biopharmaceutical company focused on rare diseases and engaged in the research, development and commercialization of transformative therapies for rare diseases and rare oncology.
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 (LSD), complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases including syndrome d’Alagille (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 grow and diversify its drug portfolio and invest in next-generation gene therapy technologies for the treatment of rare diseases. CANbridge Global Partners include, but are not limited to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, University of Massachusetts Chan School of Medicine, University of Washington School of Medicine, LogicBio and Scriptr.
To learn more about CANbridge Pharmaceuticals Inc., visit: www.canbridgepharma.com.
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