IRD Patient and Family Day 2024 | Gene therapy: a Luxturna implementation example

Описание: On 23 March 2024, the Inherited Retinal Disease Patient & Family Engagement Day was hosted in Sydney, Australia. This event was proudly co-hosted by UNSW Medicine & Health (Discipline of Paediatrics) and the Stem Cell Medicine Group at Children’s Medical Research Institute (CMRI). It was made possible by funding from the Medical Research Future Fund Stem Cell Mission (Chief Investigator: Associate Professor Anai Gonzalez-Cordero, CMRI), UNSW Medicine & Health, and Luminesce Alliance.

Presentation summary:
Robyn discusses the journey to commence the first gene therapy for RPE65-related retinal dystrophy. She shares the eligibility for trial enrolment, what is involved and trial outcomes to date.

Speaker bio:
Professor Robyn Jamieson is Head of the Eye Genetics Research Unit at CMRI, Sydney Children's Hospitals Network and Save Sight Institute, University of Sydney. Professor Jamieson's pioneering genomics research in Australia led to greater availability of genomic testing for IRDs. Her team's genomic and stem cell research is now leading to development and delivery of novel genetic therapies for IRDs.

For more information about the event, contact UNSW at [email protected]

0:00 Introduction
4:53 Gene therapy in Australia
7:05 Eligibility for RPE65 gene therapy
8:48 Multidisciplinary team
15:20 Steps to start gene therapy
20:20 Cost of inherited retinal diseases