RARE Daily

CANbridge and UMass Enter Second Gene Therapy Collaboration

September 9, 2020

Rare Daily Staff

Three months after initiating its collaboration focused on rare neuromuscular conditions, Chinese biopharma CANbridge Pharmaceuticals expanded the gene therapy collaboration with the Horae Gene Therapy Center at the UMass Medical School to include a second lab.

The second sponsored research program will be under the direction of Miguel Sena-Esteves, 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.

The first collaborative agreement is with Guangping Gao, 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, acting chief scientific officer and board member, CANbridge Pharmaceuticals. “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.”

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.

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.

“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 Sena-Esteves. “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.”

Photo: Miguel Sena-Esteves, associate professor of neurology at the University of Massachusetts Medical School

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