CANbridge Starts Neuromuscular Gene Therapy Program with UMass

CANbridge Pharmaceuticals Inc., a biopharmaceutical company developing innovative drug candidates to treat underserved medical conditions, announced on September 09th 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. 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.

For further information, see CANbrige Pharma (http://www.canbridgepharma.com/index/newsdetail/79?lang=en)

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