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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.

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About Miguel Sena-Esteves, PhD

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

 

Meet Dr. Miguel Sena-Esteves

 

Our research

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.

 

Learn more about our research

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Click the link below to get in contact with us

 

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  • Top story: UMass Chan launches Translational Institute for Molecular Therapeutics
    Research News

    Top Story: UMass Chan launches Translational Institute for Molecular Therapeutics

    Top story: The Translational Institute for Molecular Therapeutics leverages UMass Chan Medical School’s extensive experience in researching and developing gene therapies for early-stage clinical trials. Miguel Sena-Esteves, PhD, is director of the new institute.

    Read more
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    First gene therapy for Tay-Sachs disease successfully given to two children

    In a piece written for The Conversation, Miguel Sena-Esteves, PhD, talks about research leading to the first ever gene therapy for Tay-Sachs disease and the launch of the Translational Institute for Molecular Therapeutics at UMass Chan.

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  • Raiden Science Foundation makes gift to UMass Chan for UBA5 gene therapy research
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    Raiden Science Foundation makes gift to UMass Chan for UBA5 gene therapy research

    An Oregon couple whose baby was diagnosed with an ultra-rare genetic disorder has launched a foundation to raise money to accelerate development of therapies for the condition and recently signed a gift agreement with UMass Chan Medical School to jump start the research.

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