1st Study from the CANbridge UMass Chan Health-related Faculty Uncommon Condition Gene Therapy Investigation Collaboration
BEIJING & CAMBRIDGE, Mass., May perhaps 04, 2022–(Company WIRE)–CANbridge Pharmaceuticals Inc., (“CANbridge,”1228.HK) a foremost China-centered international biopharmaceutical organization fully commited to the research, growth and commercialization of transformative rare disorder and unusual oncology therapies, introduced that info from its gene treatment research settlement with the Horae Gene Therapy Centre, at the UMass Chan Health-related School, will be presented at the ASGCT 25th Annual Assembly, being held in Washington D.C. from May perhaps 16 – 19th. Scientists will existing results from an animal analyze discovering a novel 2nd-technology gene remedy for the probable treatment method of spinal muscular atrophy (SMA). This is the initially data to appear out of the CANbridge uncommon disease gene remedy investigation collaboration with the UMass Chan Health care School, which is targeted on building gene treatment treatment options for neuromuscular disorders with unmet health care demands.
Presentation Aspects:
Title: Endogenous Human SMN1 Promoter-driven Gene Substitute Enhances the Efficacy and Security of AAV9-mediated Gene Therapy for Spinal Muscular Atrophy in Mice
Poster #:M-144
Category: Neurologic Diseases I
Session Day and Time: Monday, May perhaps 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 offered on the ASGCT web page: https://annualmeeting.asgct.org
About the Horae Gene Treatment Center at UMass Chan Professional medical University
The school of the Horae Gene Remedy Middle is committed to building therapeutic strategies for uncommon inherited ailment for which there is no treatment. We employ point out of the art systems to both genetically modulate mutated genes that generate ailment-leading to proteins or introduce a healthy copy of a gene if the mutation outcomes in a non-purposeful protein.
The Horae Gene Treatment Centre faculty is interdisciplinary, which includes members from the departments of Pediatrics, Microbiology & Physiological Units, Biochemistry & Molecular Pharmacology, Neurology, Medicine and Ophthalmology. Medical professionals and PhDs work alongside one another to deal with the healthcare demands of unusual ailments, this sort of as Alpha 1-Antitrypsin Deficiency, Canavan Sickness, Tay-Sachs and Sandhoff ailments, Retinitis Pigmentosa, Cystic fibrosis, Lou Gehrig’s disorder, TNNT1 nemaline myopathy, Rett syndrome, N-Gly 1 deficiency, Pitt-Hopkins syndrome, Marple Syrup Urine Sickness, Sialidosis, GM3 synthase deficiency, Huntington’s sickness, ALS and others. Far more typical diseases this kind of as cardiac arrhythmia and hypercholesterolemia are also investigated. The hope is to deal with a wide spectrum of conditions by a variety of gene therapeutic techniques. In addition, the University of Massachusetts Chan Health-related Faculty conducts medical trials on web page and some of these trials are carried out by the investigators at the Gene Treatment middle.
About CANbridge Prescription drugs Inc.
CANbridge Pharmaceuticals Inc. (“CANbridge,” 1228.HK) is a China-based mostly world wide rare illness-centered biopharmaceutical corporation committed to the study, development and commercialization of transformative scarce condition and rare oncology therapies.
CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with major industry likely, concentrating on some of the most common rare conditions and scarce oncology.
These consist of Hunter syndrome (MPS II) and other lysosomal storage conditions (LSDs), enhance mediated ailments, hemophilia A, metabolic problems, uncommon cholestatic liver illnesses including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) as perfectly as glioblastoma multiforme (GBM).
CANbridge strategically combines worldwide collaborations and interior research to create and diversify its drug portfolio and invest in up coming-generation gene therapy technologies for exceptional ailment remedies. CANbridge world-wide partners contain, but are not restricted to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Chan Medical Faculty, the College of Washington University of Drugs, LogicBio and Scriptr.
For far more on CANbridge Prescribed drugs Inc., you should go to: www.canbridgepharma.com.
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View source variation on businesswire.com: https://www.businesswire.com/news/residence/20220503005199/en/
Contacts
U.S. Trader Relations:
Chris Brinzey
ICR Westwicke
[email protected]
China Trader Relations
CANbridge Prescribed drugs Inc.
[email protected]
Media:
Deanne Eagle
World Communications
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917.837.5866
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