Watch: Advanced Therapeutics webinar - Ocular Gene & Cell Therapies

Watch the video of this webinar to hear from two experts in the fields of genetic retinal diseases and stem cell biology discuss Ocular Gene and Cell Therapies and the tremendous new hope they bring to the treatment of these retinal diseases.

Ocular Gene Therapy is the first in vivo gene therapy approved by the Therapeutic Goods Administration (TGA) here in Australia for clinical use in inherited retinal diseases. Inherited retinal diseases are a group of conditions that disproportionately affect children and young adults and lead to blindness. Excitingly, there are now new genetic therapies and advances in retinal stem cell biology towards cellular therapies for even the most severe forms of retinal degeneration.

Watch the video of this webinar to learn about:

• Ocular gene therapy – precision therapy for genetic eye diseases
• Retinal organoids enabling access to genetic therapies
• Cellular therapies for retinal diseases, a new strategy

Professor Robyn Jamieson Professor Robyn Jamieson is Professor of Genomic Medicine, University of Sydney, Australia. She leads the Eye Genetics Research Unit at Children’s Medical Research Institute (CMRI), Sydney Children’s Hospitals Network (SCHN) and Save Sight Institute (SSI), University of Sydney, and clinical implementation of the Ocular Gene and Cell Therapy program (SCHN and OHMR, NSW Health). Professor Jamieson’s research is focussed on a comprehensive strategy for development and implementation of new therapies for genetic retinal diseases. A multidisciplinary team approach is taken including clinical genetic, ophthalmic and genomic laboratory science expertise for accurate genomic diagnoses and retinal phenotypic staging. For patients with inherited retinal diseases, Professor Jamieson’s laboratory undertakes functional genomic, stem cell and gene editing approaches to determine underlying disease mechanisms and develop new genetic therapies using patient-derived retinal organoids and mouse models. Professor Jamieson’s work contributed to the recent Australian Therapeutic Goods Administration (TGA) approval of the first clinical ocular gene therapy in Australia, a breakthrough for previously untreatable genetic retinal diseases.

Dr Anai Gonzalez Cordero Dr Anai Gonzalez Cordero is Group Leader of the Stem Cell Medicine team and also the manager of the Stem Cell & Organoid Facility at the Children’s Medical Research Institute (CMRI), with a joint appointment with the University of Sydney. Anai gained her degree in Developmental Biology in 2008 from University College London and was subsequently awarded a PhD in eye regeneration from the Wellcome Trust Developmental and Stem Cell Biology programme. Her work established proof of concept for stem cell therapy by transplantation of photoreceptor cells as a treatment for blindness due to retinal degeneration. She continued her work at UCL in the Institute of Ophthalmology as a Research Fellow. In collaboration with Moorfields Eye hospital in London, Anai led the expansion of the stem cell research programme investigating and modelling a number of inherited retinal and auditory degenerative conditions, using 3D retinal organoids. During her career Anai has received a number of awards for her research, including the ERA-NET NEURON Excellent Paper in Neuroscience (Gonzalez-Cordero et al., 2013) and the Ruskell Medal by the Worshipful Company of Spectacle Markers (UK). She was also invited to present her work in the House of Commons in the UK. In March 2019, Anai joined CMRI. At CMRI, her group is interested in developing novel technologies to improve and test cell and gene therapies for the retina and the modelling of retinal, auditory and brain diseases using organoids derived from pluripotent stem cells. She also leads a new Stem Cell and Organoids Facility that provides a number of human stem cell derived cells, tissues and organoids to researchers at CMRI and partners in NSW. The Facility provides a number of human stem cell derived cells, tissues and organoids to all groups and researchers at CMRI and affiliated Institutes. This is now expanding considerably the scope of Stem Cell Research in NSW and Australia.

Host & panelists (speakers joined by):

• Professor Ian Alexander

Head of the Gene Therapy Research Unit at Kids Research (a joint initiative with the Children's Medical Research Institute)

• Professor Craig Munns

Endocrinologist and Advanced Therapeutics Lead at Children’s Hospital at Westmead

• Dr Laura Fawcett

Respiratory Consultant and Advanced Therapeutics Lead at Sydney Children’s Hospital, Randwick