BEIJING & BURLINGTON, Mass.--(BUSINESS WIRE)--CANbridge Pharmaceuticals, Inc. (1228.HK), a planetary biopharmaceutical company, with a instauration successful China, committed to the research, improvement and commercialization of transformative therapies to dainty uncommon diseases and oncology, announced that it has exercised its enactment to unafraid the exclusive planetary rights to develop, manufacture and commercialize a caller second-generation cistron therapy to dainty spinal muscular atrophy (SMA) from UMass Chan Medical School.
Animal information presented successful 2022 astatine the American Society of Gene and Cell Therapy (ASGCT), the European Society of Gene and Cell Therapy (ESGCT) and the World Muscle Congress, showed that the cistron therapy outperformed the benchmark therapy on aggregate cardinal endpoints successful a rodent exemplary of SMA and exhibited overmuch little liver toxicity erstwhile administered intravenously. The benchmark therapy uses a vector akin to that utilized successful the lone cistron therapy approved for SMA. CANbridge sponsored the probe and is evaluating the cistron therapy to find imaginable further advantages implicit the existent standard-of-care.
In addition, the institution announced that it has completed the afloat exertion transportation of cistron therapy products being developed for the attraction of Fabry and Pompe diseases from LogicBio® Therapeutics. Under an amended agreement, CANbridge retains an exclusive worldwide licence to the products successful the Fabry and Pompe cistron therapy programs that usage the AAV sL65 liver targeting capsid. CANbridge besides obtained non-exclusive worldwide rights to the LogicBio proprietary manufacturing process for Fabry and Pompe cistron therapies. In addition, the enactment rights to sL65-based therapies for 2 further indications, arsenic good arsenic to LB-001, an investigational attraction for methylmalonic acidemia, successful Greater China, which were granted nether the archetypal statement betwixt CANbridge and LogicBio, are removed from the amended agreement.
“The cistron therapy we developed with UMass Chan, and to which we present person planetary rights successful the field, holds committedness arsenic a imaginable best-in-class therapeutic for SMA,” said James Xue, Ph.D., founder, president and CEO of CANbridge Pharmaceuticals Inc. “As we besides adhd 2 further cistron therapy programs, for Fabry and Pompe diseases, to our pipeline astatine the precocious opened CANbridge Next-Generation Innovation and Process Development Facility, successful Burlington, Mass, we look guardant to processing imaginable best-in-class cistron therapies for these 3 uncommon diseases which presently person constricted attraction options.”
“The caller hSMN1 AAV cistron therapy vector, consisting of the endogenous SMN1 promoter and codon-optimized hSMN1, has a remarkably improved potency and information illustration arsenic compared to the benchmark vector, holding large committedness for further objective development,” said Guangping Gao, PhD, the Penelope Booth Rockwell Professor successful Biomedical Research, prof of microbiology & physiological systems, manager of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research. “We consciousness assured successful CANbridge’s quality to make this cistron therapy for spinal muscular atrophy, which could assistance much patients and families suffering from this devastating disease.”
About the Second-Generation Gene Therapy
The caller second-generation cistron therapy (scAAV9-SMN1p-co-hSMN1) is simply a self-complementary AAV9 cistron therapy expressing a codon-optimized quality SMN1 cistron nether the power of an endogenous promoter. When compared to a benchmark cistron therapy successful murine exemplary survey of spinal muscular atrophy (SMA) via intravenous administration, the second-generation therapy resulted successful a longer lifespan, amended restoration of centrifugal relation and much implicit neuromuscular junction innervation, without the liver toxicity seen with the benchmark vector. The benchmark vector, which is akin to that utilized successful the approved SMA cistron therapy, 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. Data were presented successful 2022 astatine the American Society of Gene and Cell Therapy (ASGCT), the European Society of Gene and Cell Therapy (ESGCT) and The World Muscle Society Congress.
The cistron therapy is being developed by CANbridge for the attraction of spinal muscular atrophy. CANbridge holds the exclusive planetary development, manufacture and commercialization rights.
About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is uncommon familial illness caused by the deficiency of a functional centrifugal endurance centrifugal neuron 1 (SMN1) gene, which codes for a macromolecule indispensable to centrifugal neuron survival. The effect is simply a accelerated and irreversible nonaccomplishment of centrifugal neurons, resulting successful debilitating centrifugal relation nonaccomplishment and, successful astir cases, death. The astir communal type, SMA1, onsets betwixt commencement and six months of property and accounts for 60 percent of the SMA cases, according to Cure SMA. If untreated, SMA1 leads to feeding and ventilation enactment oregon decease by property two. SMA2 onsets betwixt 6 and 24 months. Patients whitethorn beryllium capable to beryllium up by themselves but volition beryllium incapable to walk. 30 percent dice by the property of 25 (Cure SMA). SMA3 and SMA4 are rarer, aboriginal onset types of the illness that hap from puerility to aboriginal adulthood, and besides effect successful centrifugal relation nonaccomplishment and death. SMA affects astir 1 retired of 6,000 to 10,000 newborns worldwide, according to spinalmuscularatrophy.net.
Approved SMA attraction options are limited. Even with caller approvals, determination is inactive a ample unmet aesculapian need.
About CANbridge Pharmaceuticals Inc.
CANbridge Pharmaceuticals Inc. (HKEX:1228) 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 10 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. The CANbridge Next-Generation Innovation and Process Development Facility is processing novel, perchance curative, cistron therapies for uncommon familial diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA) and different neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. Animal information from the SMA cistron therapy was presented successful 2022 astatine the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. CANbridge planetary partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global.
For much connected CANbridge Pharmaceuticals Inc., delight spell to: www.canbridgepharma.com.
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