Network leads gene therapy education program

Kids Research would like to congratulate Dr Michelle Lorentzos, Clinical Trials Lead at The Children’s Hospital at Westmead, for receiving a Pfizer Global Medical Grant.

The grant has been awarded for her project “Multidisciplinary Gene Therapy Education - Recombinant Adeno-Associated Virus (rAAV)” which is a multidisciplinary approach to education regarding AAV gene therapy in Australia.

“Genetic diseases affect over 50 million people worldwide. Many of these conditions have traditionally been considered untreatable, with medical care aimed at modifying symptoms, as opposed to significantly altering the outcome of the disease,” Dr Lorentzos said.

Adeno-Associated Virus (AAV) vectors use a non-enveloped virus as a vehicle to safely deliver vaccines and gene therapies into the cells or tissue of a patient with an otherwise difficult-to-treat genetic condition.

Gene therapy using AAV as a vector is emerging as a potential game-changer in the treatment of genetic diseases. 

“Take Spinal Muscular Atrophy (SMA) type 1 as an example. This is a devastatingly progressive neuromuscular diagnosis from which children typically die before their second birthday. Due to AAV-related gene therapy, there are patients with SMA type 1 that are not only surviving, but living symptom-free.”

Dr Lorentzos’ project is designed to build upon existing world-class infrastructure and expertise at Sydney Children’s Hospitals Network (SCHN) by facilitating a structured, multidisciplinary education focused on the safe, effective and ethical delivery of adenovirus-based gene replacement therapy.

The education program will be designed and implemented at SCHN, with the potential for adaption and delivery to other health services in the future. 

“SCHN is emerging as a leader in gene replacement therapy and we are increasing our capacity to undertake more work in this exciting area. However, the delivery of complex and novel treatments is only possible through effective workforce education." 

Pfizer Global Medical Grants support independent initiatives of global healthcare, including research, quality improvement or education, to improve patient outcomes in areas of medical need aligned with Pfizer’s medical and scientific strategies.

“I am honoured to receive the Pfizer Global Medical Grant and to contribute to the pivotal role that SCHN is playing in the changing landscape of medical therapies,” Dr Lorentzos said.