Terence R. Flotte, MD

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.

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.

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.

UMass Medical School has received approval from the University of Massachusetts Board of Trustees to build a nine-story biomedical research and education facility on the Worcester campus to support laboratory research growth and the educational enterprise.

In a memo to the UMass Medical School community, Chancellor Michael F. Collins and Dean Terence R. Flotte say racism is a public health crisis and outline changes underway at UMMS.

Two young children with Tay-Sachs disease were treated safely at UMass Memorial Medical Center with a gene therapy developed at UMass Medical School and, in one case, the child’s condition has stabilized, according to an interview published by WBUR with Terence R. Flotte, MD.

Preliminary data from an expanded access study of an investigational gene therapy in two patients with infantile Tay-Sachs disease indicates the potential to modify the rate of disease progression, according to a recent report presented in Barcelona by Terence R. Flotte, MD.

Reforms are needed to ensure the integrity of the medical residency program selection process, according to commentary in the journal Academic Medicine by Chancellor Michael F. Collins and colleagues at UMass Medical School.

The Toronto-based Blu Genes Foundation, which is dedicated to developing gene therapies for rare disease, has given $1.4 million to UMass Medical School to advance a Phase I/II clinical trial for Tay-Sachs. Miguel Sena-Esteves, PhD, Terence Flotte, MD, Heather Gray-Edwards, PhD, DVM, and colleagues at  Auburn University are leading the research.

Dean Terence R. Flotte served as senior author on a call to action published in Academic Medicine, titled, “It is time for zero tolerance for sexual harassment in academic medicine.”