RARE Daily

CANbridge and UMass Enter Gene Therapy Research Collaboration

June 10, 2020

Rare Daily Staff

CANbridge Pharmaceuticals has initiated a research program with the Horae Gene Therapy Center at the UMass Medical School for gene therapy research in rare genetic diseases, with a focus on neuromuscular conditions.

Guangping Gao, co-director of the Li Weibo Institute for Rare Diseases Research, and director, the Horae Gene Therapy Center and Viral Vector Core, will direct the research program. Gao is also a professor of microbiology and physiological systems in biomedical research at UMass Medical School. He has played a key role in the discovery and characterization of adeno-associated virus serotypes now widely used in the field.

CANbridge has named Gao, along with 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, founder, chairman and CEO of CANbridge Pharmaceuticals.

The Horae Gene Therapy Center is an interdisciplinary center with physicians and researchers working 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, ALS, TNNT1 nemaline myopathy, Rett syndrome, N-Gly 1 deficiency, Pitt-Hopkins syndrome, maple syrup urine disease, sialidosis, GM3 synthase deficiency, Huntington’s disease, 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.

Photo: Guangping Gao, co-director of the Li Weibo Institute for Rare Diseases Research

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