CANbridge Pharmaceuticals Enters Into Rare Disease Gene Therapy Research Agreement With UMass Medical School

Adds Gene Therapy Leaders to its Advisory Board

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- CANbridge Pharmaceuticals Inc., a biopharmaceutical company developing innovative drug candidates to treat underserved medical conditions, announced that it has initiated a research program with the Horae Gene Therapy Center at the UMass Medical School, in Worcester, MA, for gene therapy research in rare genetic diseases, with a focus on neuromuscular conditions. The research program will be under the direction of Guangping Gao, PhD, 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 in Biomedical Research at UMass Medical School. Dr. Gao is a groundbreaking gene therapy researcher who played a key role in the discovery and characterization of adeno-associated virus (AAV) serotypes now widely used in the field.

CANbridge also announced the addition of Guangping Gao, PhD. and Mark Bamforth, an industry veteran and founder and former CEO of Brammer Bio, to its recently-expanded Strategic Advisory Board.

“This collaboration with the Horae Gene Therapy Center at UMass, a pioneer in gene therapy research, affords CANbridge the potential to extend our rare disease pipeline by integrating next-generation technology as we advance as a leader in the treatment of rare diseases,” said James Xue, PhD, Founder, Chairman and CEO, CANbridge Pharmaceuticals Inc. “We are thrilled to be partnering with such a world-class institution.”

“As a longtime leader in the development of gene therapies for rare disease, with expertise in both capsid and genome engineering, Dr. Gao is the ideal partner for CANbridge as we expand our therapeutic capacity in rare genetic diseases,” said Richard J. Gregory, PhD, Acting Chief Scientific Officer, CANbridge Pharmaceuticals Inc.

“On behalf of everyone at CANbridge, I’m very pleased to welcome Guangping Gao and Mark Bamforth, each widely recognized as true global leaders in the broad areas of gene therapy science and manufacturing, to our Strategic Advisory Board,” said Jim Geraghty, CANbridge Pharmaceuticals Advisory Board Chair. “Together with Mark Goldberg and Rich Gregory, also recognized biotechnology leaders, they give CANbridge an unparalleled foundation for developing breakthrough therapies for patients in China and around the world.”

About the Horae Gene Therapy Center at UMass Medical School

The faculty of the Horae Gene Therapy Center is dedicated to developing therapeutic approaches for rare inherited disease for which there is no cure. We utilize 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 Horae Gene Therapy Center faculty is interdisciplinary, including members from the departments of Pediatrics, Microbiology & Physiological Systems, Biochemistry & Molecular Pharmacology, Neurology, Medicine and Ophthalmology. Physicians and PhDs work together to address 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 syndrome, N-Gly 1 deficiency, Pitt-Hopkins syndrome, Marple Syrup Urine Disease, Sialidosis, GM3 synthase deficiency, Huntington’s disease, ALS and others. More common diseases such as cardiac arrhythmia and hypercholesterolemia are also investigated. The hope is to treat a wide spectrum of diseases by various gene therapeutic approaches. Additionally, the University of Massachusetts Medical School conducts clinical trials on site and some of these trials are conducted by the investigators at the Gene Therapy center.

About the University of Massachusetts Medical School

The mission of the University of Massachusetts Medical School is to advance the health and well-being of the people of the commonwealth and the world through pioneering education, research, public service and health care delivery.

For more information, visit www.umassmed.edu.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. is a biopharmaceutical company accelerating development and commercialization of specialty healthcare products for orphan diseases and targeted cancers, focusing on products that are unavailable or address unmet medical needs.

CANbridge has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases. In greater China, where it is a recognized leader in orphan diseases, CANbridge has an exclusive licensing agreement to commercialize Hunterase^®, an enzyme replacement therapy for the treatment of Hunter syndrome, developed by GC Pharma and marketed in more than ten countries worldwide. The CANbridge oncology portfolio includes exclusive rights to develop and commercialize Puma Biotechnology’s NERLYNX^® (neratinib), which was recently approved in greater China, as well as rights to other novel candidates.

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

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