UMass Chan Medical School in Worcester announced on Thursday it is launching the Translational Institute for Molecular Therapeutics, a center which will focus on gene therapy clinical trials for rare genetic diseases.
At the same time, the school also announced a collaboration with the Raiden Science Foundation of Oregon, which will raise $3 million for a research project related to an extremely rare genetic disorder called UBA5 deficiency. The research was jump-started by an $85,000 gift ahead of the funding campaign.
The Translational Institute will be led by neuroscientist Miguel Sena-Esteves. It is designed to support researchers with manufacturing, regulatory matters, and investigative new drug studies, which are needed to conduct first-in-human clinical trials, UMass Chan said in its announcement.
Clinical trials are currently underway for therapeutics related to Tay-Sachs disease and ALS.
“Having all the infrastructure, together, in the Translational Institute for Molecular Therapeutics, will make it easier for UMass Chan investigators to bring new therapeutics to clinic for these forgotten diseases that have difficulty attracting investment from outside entities,” Sena-Esteves said in a statement.
UMass Chan plans to seek additional philanthropic support the institute’s activities.
