BEIJING & BURLINGTON, Mass.--(BUSINESS WIRE)--CANbridge Pharmaceuticals Inc. (HKEX:1228), a starring planetary biopharmaceutical company, with a instauration successful China, committed to the research, improvement and commercialization of transformative uncommon illness and uncommon oncology therapies, announced that information from its cistron therapy probe statement with the Horae Gene Therapy Center, astatine the UMass Chan Medical School, was presented astatine the 29th European Society of Gene and Cell Therapy Annual Congress successful Edinburgh, Scotland, today.
In an oral presentation, Guangping Gao, Ph.D., Co-Director, Li Weibo Institute for Rare Diseases Research, Director, the Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems and Penelope Booth Rockwell Professor successful Biomedical Research astatine UMass Chan Medical School, discussed the survey that was led by the researcher Jun Xie, Ph.D., and his squad from Dr. Gao’s lab, and titled “Endogenous quality SMN1 promoter-driven cistron replacement improves the efficacy and information of AAV9-mediated cistron therapy for spinal muscular atrophy (SMA) successful mice”.
The survey showed that a caller second-generation self-complementary AAV9 cistron therapy, expressing a codon-optimized quality SMN1 gene. nether the power of its endogenous promoter, (scAAV9-SMN1p-co-hSMN1), demonstrated superior safety, potency, and efficacy crossed respective endpoints successful an SMA rodent model, erstwhile compared to the benchmark vector, scAAV9-CMVen/CB-hSMN1, which is akin to the vector utilized successful the cistron therapy approved by the US Food and Drug Administration for the attraction of SMA. The benchmark vector expresses a quality SMN1 transgene nether a cytomegalovirus enhancer/chicken β-actin promoter for ubiquitous look successful each compartment types, whereas the second-generation vector utilizes the endogenous SMN1 promoter to power cistron look successful antithetic tissues. Compared to the benchmark vector, the second-generation vector resulted successful a longer lifespan, amended restoration of musculus function, and much implicit neuromuscular junction innervation, without the liver toxicity seen with the benchmark vector.
This, the archetypal information to beryllium presented from the cistron therapy probe collaboration betwixt CANbridge and the Gao Lab astatine the Horae Gene Therapy Center, was besides presented astatine the American Society for Cellular and Gene Therapy (ASGCT) Annual Meeting successful May 2022. Dr. Gao is simply a erstwhile ASCGT president.
Oral Presentation: Poster #: 0R57
Category: AAV adjacent procreation vectors
Presentation Date and Time: Thursday, October 13, 5:00 PM BST
Authors: Qing Xie, Hong Ma, Xiupeng Chen, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Qin Su, Phillip Tai, Guangping Gao, Jun Xie
Abstracts are disposable connected the ESGCT website: https://www.esgctcongress.com/
About the Horae Gene Therapy Center astatine UMass Chan Medical School
The module of the Horae Gene Therapy Center is dedicated to processing therapeutic approaches for uncommon inherited illness for which determination is nary cure. We utilize authorities of the creation technologies to either genetically modulate mutated genes that nutrient disease-causing proteins oregon present a steadfast transcript of a cistron if the mutation results successful a non-functional protein. The Horae Gene Therapy Center module is interdisciplinary, including members from the departments of Pediatrics, Microbiology & Physiological Systems, Biochemistry & Molecular Pharmacology, Neurology, Medicine and Ophthalmology. Physicians and PhDs enactment unneurotic to code the aesculapian needs of uncommon diseases, specified arsenic alpha 1-antitrypsin deficiency, Canavan disease, Tay-Sachs and Sandhoff diseases, retinitis pigmentosa, cystic fibrosis, amyotrophic lateral sclerosis, TNNT1 nemaline myopathy, Rett syndrome, NGLY1 deficiency, Pitt-Hopkins syndrome, maple syrup urine disease, sialidosis, GM3 synthase deficiency, Huntington disease, and others. More communal diseases specified arsenic cardiac arrhythmia and hypercholesterolemia are besides being investigated. The anticipation is to dainty a wide spectrum of diseases by assorted cistron therapeutic approaches. Additionally, the University of Massachusetts Chan Medical School conducts clinical trials connected site and immoderate of these trials are conducted by the investigators astatine The Horae Gene Therapy Center.
About CANbridge Pharmaceuticals Inc.
CANbridge Pharmaceuticals Inc. (HKEX:1228) is simply a planetary biopharmaceutical company, with a instauration successful China, committed to the research, improvement and commercialization of transformative therapies for uncommon illness and uncommon oncology. CANbridge has a differentiated cause portfolio, with 3 approved drugs and a pipeline of 11 assets, targeting prevalent uncommon illness and uncommon oncology indications that person unmet needs and important marketplace potential. These see Hunter syndrome and different lysosomal retention disorders, complement-mediated disorders, hemophilia A, metabolic disorders, uncommon cholestatic liver diseases and neuromuscular diseases, arsenic good arsenic glioblastoma multiforme. CANbridge is besides gathering next-generation cistron therapy improvement capableness done a operation of collaboration with world-leading researchers and biotech companies and interior capacity. CANbridge’s planetary partners see Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School and LogicBio.
For much connected CANbridge Pharmaceuticals Inc., delight spell to: www.canbridgepharma.com.
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