SUZHOU – November 15, 2024— CANbridge Pharmaceuticals Inc. (1228.HK), a global biopharmaceutical company committed to the research, development and commercialization of transformative therapies to treat rare diseases, today announced the publication of a research article in the journal Science https://www.science.org/doi/10.1126/science.adp8179 that describes the discovery of the StitchR™ RNA assembly technology and its application to treat muscle diseases caused by mutations in large genes, including Duchenne muscular dystrophy (DMD). The title of the publication is “Ribozyme-activated mRNA Trans-ligation Enables Large Gene Delivery to Treat Muscular Dystrophies.”

The StitchR technology was developed by Douglas Anderson, Ph.D. and colleagues at the University of Rochester and Scriptr Global Inc. to address the limited payload capacity of adeno-associated viruses (AAV).  vectors, the most commonly used gene therapy vector. This technology enables the efficient delivery of larger gene payloads via two independent AAV. The dual AAV vectors express the left and right halves of a gene sequence that are seamlessly stitched together end-to-end by ribozymes at the messenger RNA level, thus enabling the production of large proteins.

In 2021, CANbridge entered into a research collaboration and exclusive world-wide license agreement with Scriptr Global for the development of StitchR-enabled gene therapies targeting dystrophinopathies, including DMD, Becker muscular dystrophy (BMD) and X-linked dilated cardiomyopathy (DCM) driven by DMD mutations. The new study demonstrates that StitchR can reconstitute high levels of a large midi-dystrophin protein encoded by StitchR-enabled dual AAV vectors in the muscles and heart of mdx mice, a key preclinical model for studying DMD. The midi-dystrophin is approximately twice the size of the micro-dystrophins that are currently approved or in clinical trials, and more than half the size of native dystrophin. The authors further show that the midi-dystrophin is functional and leads to significant improvements in muscle health in treated mice. StitchR is the basis of CANbridge’s CAN204 DMD gene therapy program, which is currently in the preclinical research stage.  

 “The publication in Science demonstrates that this technology for creating large proteins from dual AAV vectors is recognized externally across the broader scientific community as both novel and extremely powerful,” said James Xue, Ph.D., founder, chairman and CEO of CANbridge Pharmaceuticals Inc. “DMD is an X-linked genetic muscle disease that is caused by mutations in the dystrophin gene, which is too large to fit into a single or even dual AAV vectors. The StitchR RNA Assembly Technology enables a doubling of the size of a gene that AAV can deliver, and in the case of DMD, we believe that that by providing a larger recombinant dystrophin than the current micro-dystrophins, CANbridge’s next generation AAV approach may lead to a more potent treatment to address the significant unmet needs of DMD patients.”

Jason West, Ph.D., Vice President of Research of CANbridge, and co-author of the publication added “CAN204 may represent a best-in-class therapy compared to other dual and single vector AAV approaches because of the high efficiency and limited byproducts demonstrated by the StitchR technology when used to deliver large dual vector-encoded DMD genes to mice and human skeletal muscle cells during our collaboration with Scriptr and in our preclinical research studies.  We are excited to continue to advance this program as rapidly and safely as possible for DMD patients.”

About dystrophinopathies

Dystrophinopathies are X-linked genetic muscle diseases, which include Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and X-linked dilated cardiomyopathy (DCM) driven by mutations in the DMD gene.  DMD presents in early childhood and is characterized by rapidly progressive muscle degeneration and weakness, leading to loss of ambulation by about 12 years of age. BMD is characterized by later-onset skeletal muscle weakness and loss of ambulation in adulthood. X-linked DCM  is characterized by a large and poorly contracting heart without significant skeletal muscle involvement.  DCM, which also occurs in DMD and BMD patients, often progresses to congestive heart failure and is a common cause of morbidity and death in patients with dystrophinopathies.

About CAN204

CAN204 is a dual AAV vector gene therapy currently in the discovery research stage of development. CAN204 utilizes the StitchR™ RNA Assembly Technology that is exclusively licensed to CANbridge from Scriptr Global Inc. for the worldwide treatment of dystrophinopathies.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare diseases. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 9 assets, targeting prevalent rare disease indications that have unmet needs and significant market potential. These include Hunter syndrome (Mucopolysaccharidosis type II) and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. CANbridge global partners include: Apogenix, GC Biopharma, Mirum Pharma, WuXi Biologics, Privus Biologics, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global.

For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.

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