Rob Collin
Dr. Rob Collin is a Full Professor and a Principal Investigator at the Radboud university medical center (RadboudUMC) in Nijmegen, The Netherlands, and affiliated with the Donders Institute for Brain, Cognition and Behaviour. His research group within the Department of Human Genetics focuses on the development and implementation of novel molecular therapies for inherited retinal diseases (IRDs). Main research topics include the development of splice modulation therapy and (micro)gene augmentation therapy for selected subtypes of IRD, by employing various cellular (e.g. fibroblasts, induced pluripotent stem cell-derived retinal cells) and animal (mice, zebrafish) model systems. Clinical trials based on his pioneer work to utilize splice correction therapy for CEP290-associated LCA is ongoing, with positive results reported so far. Currently, his pre-clinical research focuses on implementing similar strategies for a number of other subtypes of IRD, including Stargardt disease.
Bence Gyorgy
Bence Gyorgy, M.D., Ph.D. is currently Head of Clinical Translation at the Institute of Molecular and Clinical Ophthalmology Basel (Basel, Switzerland). He is leading three main preclinical research programs for vision loss: 1) cone-based optogenetics for vision restoration, 2) base editing for Stargardt disease and 3) precision gene correction for Usher Syndrome Type 2A. Besides these core translational activities he leads an academical group working on developing novel gene therapy solutions for neurosensory diseases. He is also active as a clinician and sees patients with inherited retinal dystrophies 10% of his time.
By training, Dr. Gyorgy is a medical doctor and he received his PhD in molecular genetics in 2013. During his PhD he studied how cells exchange information via extracellular vesicles in the lab of Prof. Edit I. Buzas. He spent five years as a post-doctoral fellow at Harvard Medical School, Department of Neurobiology and the Massachusetts General Hospital, Department of Neurology. Working in three different labs (Prof. David Corey, Prof. Xandra Breakefield and Prof. Casey Maguire), he developed adeno-associated vectors (AAVs) and precision gene editing tools for gene therapy.
His main area of expertise includes gene therapy, gene editing and translational research in the field of sensory diseases. Among others he received the RetinAward from the Swiss Vitreoretinal Group in 2020, the Excellence in Research Award from the American Society of Gene and Cell Therapy. He was a Edward R. and Anne G. Lefler Center Fellow between 2017 and 2018. During his research he published 37 research papers, including in Nature Medicine, Nature Communications and Neuron.
Knut Stieger
Knut Stieger is currently Professor of Experimental Ophthalmology and acting director Research and Teaching at the Department of Ophthalmology at Justus-Liebig-University in Giessen, Germany. Being a Veterinarian by training, he received his PhD from Nantes University in France in 2007. At the time he was developing (i) AAV based gene addition therapy for RPE65 deficiency in the canine model of disease and (ii) methods to control transgene expression in the retina of primates. The same year, he joined the group in Giessen and started working on experimental approaches to treat retinal degeneration. He was supported by an ERC starting grant in 2012 to develop genome editing based strategies for X linked retinitis pigmentosa. Since 2018, he is coordinator and speaker of the research priority program SPP 2127 “gene and cell based therapies to counteract neuroretinal degeneration” funded by the German Research Council (DFG). Currently, his research focuses on improving DNA double strand break repair mechanisms in photoreceptor cells for optimized use in genome editing based therapeutic applications.
Virginijus Siksnys
Virginijus Siksnys studied chemistry at Vilnius University and obtained his Ph.D from Moscow State University before returning to Vilnius where he moved through different research ranks at the Institute of Applied Enzymology/Institute of Biotechnology. Since 2002 he holds the position of Professor of Vilnius University and serves as Chief scientist at the Institute of Biotechnology. Dr. Siksnys has made a major and sustained contribution to the understanding of the structure and function of restriction enzymes. His research on the CRISPR-Cas has had a major impact on the field. His studies of the Cas9 protein paved the way for development of novel tools for genome editing applications. He is a member of Academia Europaea, EMBO, Lithuania Academy of Sciences and Norwegian Academy of Sciences and Letters. His work has been recently recognized with several awards and prizes.
Katrien Remaut
Prof. Katrien Remaut was born in Ghent, in 1978, where she studied Pharmacy and obtained her diploma with summa cum laude in 2001. She has been active in the field of nanomedicines as PhD student (2001), postdoc (2007) and professor (2014) were she focuses on the development of nanoparticles for ocular applications and on fundamental aspects of extracellular and intracellular delivery barriers. She has been the promotor or co-promotor of 8 defended PhD theses and has over 80 published or accepted papers in international scientific journals such as Nano Today, ASC Nano, Advanced Drug Delivery Reviews, Journal of Controlled Release, Bioconjugate Chemistry, Molecular Pharmaceutics, Acta Biomaterialia, ACS Applied Materials and Interfaces, etc… she has received several national and international awards such as the Price of the Royal Academy of Medicine for Scientific Research in Pharmacy, period 2008 – 2011. She is a member of the Editorial board of European Journal of Pharmaceutics and Biopharmaceutics and has been a member of the young academy in Flanders, where she helped in the development of Moendoes, a scientific board game for high school students. She teaches Biochemistry to the 2nd year bachelor students pharmaceutical sciences.
Ivana Trapani
Dr. Ivana Trapani is Principal Investigator at the Telethon Institute of Genetics and Medicine (TIGEM) in Pozzuoli and Research Associate at the University of Naples “Federico II”. She got a Master degree in Pharmaceutical Biotechnology in 2009. In 2010 she entered the PhD program in Molecular Medicine of the European School of Molecular Medicine (SEMM), at the Telethon Institute of Genetics and Medicine (TIGEM), where she after continued her studies as a postdoctoral researcher. Dr. Ivana Trapani main research interest over the last 10 years has been the development of gene therapy approaches for the treatment of inherited forms of blindness. In particular, her studies led to the identification of different strategies to overcome one of the main limitations of the virus that is most commonly used as a vehicle for retinal gene therapy, the adeno-associated virus (AAV), unable to carry genes longer than 5 kb.
Vasiliki Kalatzis
Vasiliki Kalatzis has a long-standing career dedicated to human genetic disorders and gene therapy. After receiving her Bachelor of Science degree, majoring in Genetics, from the University of Adelaide in Australia, she moved to Paris, France in 1993 to perform her Ph.D. training at the Pasteur Institute where she identified the gene responsible for Branchio-Oto-Renal syndrome. In 1998, she moved to the Necker Hospital in Paris as a post-doctoral fellow to study the pathophysiology of the lysosomal storage disease Cystinosis and, in 2001, as a tenured Inserm research scientist, she moved to the Institute of Molecular Genetics in Montpellier, to develop a gene therapy approach to treat this disorder. During this time she became interested in retinal diseases, and thus, in 2010, she joined the laboratory of Pr. Christian Hamel at the INM, to start a group working on the pathophysiology and treatment of inherited retinal disorders (IRDs) using human induced pluripotent stem cell (iPSC)-derived retinal models. Vasiliki Kalatzis is currently an Inserm research director and head of the Vision team at the INM. In addition, she is scientific cofounder of the start-up Horama, which serves as a bridge between preclinical proof-of-concept therapeutic studies and clinical trials for IRDs.