CANbridge Pharmaceuticals Enters into Second Rare Disease Gene Therapy Research Agreement with UMass Medical School

Sept. 8, 2020 12:02 UTC

 CANbridge Pharmaceuticals Enters into Second Rare Disease Gene Therapy Research Agreement with UMass Medical School


BEIJING & CAMBRIDGE, Mass--(BUSINESS WIRE)-- CANbridge Pharmaceuticals Inc., a biopharmaceutical company developing innovative drug candidates to treat underserved medical conditions, announced that it has expanded its gene therapy collaboration with the Horae Gene Therapy Center at the University of Massachusetts Medical School. This second sponsored research program will be under the direction of Miguel Sena-Esteves, PhD, Associate Professor of Neurology at the University of Massachusetts Medical School, and will focus on the development of novel customized adeno-associated virus (AAV) vectors expected to have broad applications for the treatment of neuromuscular diseases. Earlier this year, CANbridge announced its first collaborative agreement with Guangping Gao, PhD, Director of the Horae Gene Therapy Center Gao Lab for the study of gene therapy-based rare disease treatments.

“Working with the Sena-Esteves lab will afford CANbridge access to next-generation gene therapy platform technology to treat neuromuscular diseases, which are severely underserved conditions in China and the rest of the world,” said Rich Gregory, PhD, Acting Chief Scientific Officer and Board Member, CANbridge Pharmaceuticals Inc. “This, our second research collaboration with the Horae Gene Therapy Center, places CANbridge at the cutting edge of the gene therapies that could offer new ways to treat rare diseases, which we hope to bring to patients worldwide.”

“Developing new AAV capsids to address dosing and immunological limitations that have emerged from clinical trials is critical for the ultimate success of in vivo gene therapy to deliver safe transformative therapies,” said Miguel Sena-Esteves PhD. “The high throughput techniques we have perfected over the last several years allows us to take an ‘a la carte’ approach to AAV capsid engineering in order to meet multiple performance characteristics.”

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. It utilizes 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.

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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. CANbridge also has a collaborative agreement with the Horae Gene Therapy Center at UMass Medical School for the research and development of gene therapies to treat rare genetic diseases. 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


CANbridge Pharmaceuticals Inc.
James Xue

Deanne Eagle
Planet Communications


Source: CANbridge Pharmaceuticals Inc.

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