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  • Ana Rita Batista, PhD, (left) instructor in neurology, pictured with Miguel Sena-Esteves, PhD, associate professor of neurology and director of the Translational Institute for Molecular Therapeutics..
    Media, Research News

    UMass Chan researchers achieve gene therapy milestone for potential Cockayne syndrome treatment

    Researchers working with UMass Chan Medical School’s Translational Institute for Molecular Therapeutics announced progress in developing a vector to deliver gene replacement therapy in mice models with Cockayne syndrome, a rare and fatal neurodegenerative disease that largely affects children and young adults.

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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.

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  • First gene therapy for Tay-Sachs disease successfully given to two children
    Research News

    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
    Media, Research News

    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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  • UMass Chan launches Translational Institute for Molecular Therapeutics
    Research News

    UMass Chan launches Translational Institute for Molecular Therapeutics

    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
  • Sio Gene Therapies reports positive interim data for gene therapy trial
    Research News

    Sio Gene Therapies reports positive interim data for gene therapy trial

    Sio Gene Therapies, a clinical-stage company focused on developing gene therapies for neurodegenerative diseases, presented positive interim data from its Phase I/II study of AXO-AAV-GM1, an adeno-associated viral vector-based gene therapy candidate for GM1 gangliosidosis that was licensed from UMass Chan.

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  • Riaan Research Initiative funds Cockayne syndrome gene replacement therapy research at UMass Chan Medical School
    Research News

    Riaan Research Initiative funds Cockayne syndrome gene replacement therapy research at UMass Chan Medical School

    Riaan Research Initiative, a non-profit organization, and UMass Chan Medical School are entering into an agreement to fund, research and develop a gene replacement therapy to combat Cockayne syndrome, a fatal autosomal recessive disorder.

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  • USA Today reports on first baby in gene therapy clinical trial for Sandhoff, Tay-Sachs diseases
    Research News

    USA Today reports on first baby in gene therapy clinical trial for Sandhoff, Tay-Sachs diseases

    Thomas Feldborg and Daria Rokina, of Denmark, chose to enroll their baby daughter, Alissa, in an investigational gene therapy for the treatment of GM2 gangliosidosis, which causes Tay-Sachs and Sandhoff diseases, to “let hope conquer the fears,” according to a report in USA Today chronicling their journey.

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  • Innovative neurosurgical technique used in Tay-Sachs gene therapy clinical trial
    Research News

    Innovative neurosurgical technique used in Tay-Sachs gene therapy clinical trial

    Oguz Cataltepe, MD, describes the innovative neurosurgical procedure used to treat patients in the new clinical trial for a gene therapy targeting infantile Tay-Sachs and Sandhoff diseases.

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  • First patient dosed in clinical trial of gene therapy for Tay-Sachs and Sandhoff diseases
    Research News

    First patient dosed in clinical trial of gene therapy for Tay-Sachs and Sandhoff diseases

    Sio Gene Therapies announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase I/II trial evaluating AXO-AAV-GM2, an investigational gene therapy for the treatment of GM2 gangliosidosis, which causes Tay-Sachs and Sandhoff diseases. Miguel Sena-Esteves, PhD, is a principal scientist of the research program at UMMS.

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  • FDA clears IND application for Tay-Sachs gene therapy
    Research News

    FDA clears IND application for Tay-Sachs gene therapy

    Axovant Gene Therapies, a clinical-stage company developing innovative gene therapies based on UMass Medical School research discoveries, has announced that the FDA has lifted its clinical hold and cleared Axovant’s Investigational New Drug application to initiate a registrational study of gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease.

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