UMass Chan researchers achieve gene therapy milestone for potential Cockayne syndrome treatment
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UMass Chan Medical School has established the Translational Institute for Molecular Therapeutics, ushering in the next phase of gene therapy development to accelerate new treatments for rare genetic diseases.
The institute, led by Miguel Sena-Esteves, PhD, leverages UMass Chan Medical School’s extensive experience in researching and developing gene therapies for early-stage clinical trials and includes infrastructure to assist investigators with manufacturing, regulatory matters and investigational new drug-enabling studies needed to conduct first-in-human clinical trials. Importantly, UMass Chan has GMP and GMP-like manufacturing facilities to produce the necessary biologicals needed for proof-of-concept and pilot clinical trials.
Miguel Sena-Esteves, PhD, is associate professor of neurology and a member of the Horae Gene Therapy Center. He received his PhD in biomedical sciences from the University of Porto, Portugal, where he started his work in gene therapy.
His laboratory at UMass Chan focuses on the development of adeno-associated virus (AAV) based gene therapies for lysosomal storage diseases, Huntington’s disease, ALS, FSHD and brain tumo
The Translational Institute for Molecular Therapeutics was formed to evaluate therapies for rare diseases, where there are few options for seeking treatment. The institute leverages UMass Chan Medical School’s extensive experience in researching and developing gene therapies for early-stage clinical trials. It is specifically focused on increasing the number of and reducing the costs associated with human gene therapy clinical trials for rare genetic diseases by establishing a pipeline that moves research seamlessly from discovery to clinical trials and regulatory support.
