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BRIDGE grants support research into rare Sialidosis disease and patch to prevent pressure ulcers

Heather Gray-Edwards, PhD, DVM; Miguel Sena-Esteves, PhD; and Raymond Dunn, MD; are the recipients of the Office of Business Development & Innovation 2021 BRIDGE Fund grants.

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

An experimental gene therapy was little Alissa's only hope. Now, instead of certain death, she faces an uncertain future.

An experimental gene therapy was little Alissa s only hope. Now, instead of certain death, she faces an uncertain future. Karen Weintraub, USA TODAY © Feldborg family photo Alissa Feldborg BOSTON Thomas Feldborg and Daria Rokina set off nearly every afternoon to explore this city. They leave from their hotel in the Charlestown neighborhood, pushing their baby carriage, some days heading deep into downtown, others choosing a path along the Charles River.  Every few minutes, Rokina stops to peek inside and check on 16-month-old Alissa. She gently rubs Alissa s cheek and coos a few soothing words, making sure the little girl is warm enough in her yellow snowsuit and adjusting her sparkly unicorn earmuffs. 

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

Innovative neurosurgical technique used in Tay-Sachs gene therapy clinical trial By Susan E.W. Spencer March 03, 2021 When the first patient in a new clinical trial was given a gene therapy targeting infantile Tay-Sachs and Sandhoff diseases in January, an innovative procedure that represents a “new frontier for neurosurgery” was used to deliver the therapeutic. Red arrows indicate the location in the thalamus where the infusion is targeted. The trial, by Sio Gene Therapies (formerly known as Axovant), is an open-label, two-stage, clinical study designed to evaluate safety and dose-escalation and safety and efficacy of surgical delivery of AXO-AAV-GM2 to the brain and spinal cord of trial participants with infantile or juvenile GM2 gangliosidosis. Sio licensed exclusive worldwide rights from UMass Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including

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