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The State of Gene Therapy


The State of Gene Therapy
May 4, 2021
Time: 11:00 am PT, 2:00 pm ET
Special Guests:
James Wilson, MD, PhD (Perelman School of Medicine, University of Pennsylvania)
Artur Padzik, PhD (AAV Production Manager, Biovian)
Gene therapy has made tremendous strides over the past decade or so. The first approved drugs are on the market, and literally hundreds of trials are underway offering unprecedented hope to patients with rare, debilitating genetic disorders. Nevertheless, safety issues continue to swirl around the field as adverse events are reported in trials involving both lentivirus and AAV vectors. Pricing and manufacturing pose additional hurdles that could significantly challenge the field. ....

Artur Padzik , James Wilson , Heather Gray Edwards , Phd Umass Medical School , University Of Pennsylvania , Phd Perelman School Of Medicine , Broadcast Date , Medical School , Perelman School , ஜேம்ஸ் வில்சன் , ஹீத்தர் சாம்பல் எட்வர்ட்ஸ் , பல்கலைக்கழகம் ஆஃப் பென்சில்வேனியா , ஒளிபரப்பு தேதி , மருத்துவ பள்ளி , பெரல்மேன் பள்ளி ,

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 g ....

Terencer Flotte , Douglas Martin , Heather Gray Edwards , Oguz Cataltepe , Miguel Sena Esteves , School Of Medicine , Umass Medical School , Auburn University College Of Veterinary Medicine , Sio Gene Therapies , Medical School , Isaac Haidak Professor , Auburn University College , Veterinary Medicine , டக்ளஸ் மார்டின் , ஹீத்தர் சாம்பல் எட்வர்ட்ஸ் , மிகுவல் சேனா ெஸ்டேவேச் , பள்ளி ஆஃப் மருந்து , உமஸ்ஸ் மருத்துவ பள்ளி , ஆபரன் பல்கலைக்கழகம் கல்லூரி ஆஃப் கால்நடை மருந்து , சிோ கீந் சிகிச்சைகள் , மருத்துவ பள்ளி , ஆபரன் பல்கலைக்கழகம் கல்லூரி , கால்நடை மருந்து ,