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Scientific Founders

Jose-Alain Sahel, MD, PhD

Jose-Alain Sahel, MD, PhD

Jose-Alain Sahel, MD, PhD

Jose-Alain Sahel, MD, PhD

Jose-Alain Sahel is Professor at Université Pierre et Marie Curie, and Director of Institut de la Vision and Chairman of the Department of Ophthalmology at the Quinze-Vingts National Eye Hospital and at the Rothschild Ophthalmology Foundation in Paris, France. He is also Professor at the Institute of Ophthalmology at the University College in London.

He has been conducting pioneering research into the understanding of the pathological mechanisms involved in retinal cell degeneration.

Prof. Sahel recently received awards from the US Foundation Fighting Blindness and the French “Académie des Sciences”, for his work on retinitis pigmentosa.

Botond Roska, MD, PhD

Botond Roska, MD, PhD

Botond Roska, MD, PhD

Botond Roska, MD, PhD

Botond Roskais a senior group leader at the Friedrich Miescher Institute in Basel Switzerland. He was educated at University of California Berkeley, Harvard University and Harvard Medical School as well as at Semmelweis Medical School.

His group studies the structure and function of the retina. His group pioneered retina cell type specific optogenetic vision restoration (Lagali et al., Nature Neuro. 2008, Busskamp et al, Science, 2010). He received several international prizes and awards: EMBO member (2011) Alcon Award (2011) VIVA Award (2010) ERC Starting Investigator (2010) EMBO Young Investigator (2009) Marie Curie Excellence Grant (2006) HFSP Young Investigator (2003) Harvard Society Fellow (2002) HFSP Short Term Fellow (2001) Bearden Memorial Award for biophysics (2001) Fulbright Fellow (1997).

Prof. Jean Bennett, MD, PhD

Prof. Jean Bennett, MD, PhD

Prof. Jean Bennett, MD, PhD

Prof. Jean Bennett, MD, PhD

Jean Bennett, M.D., Ph.D., tenured professor of Ophthalmology and Cell and Developmental Biology, and vice chairman for research in Ophthalmology at the University of Pennsylvania (Penn) School of Medicine, is a pioneer in retinal gene therapy and internationally recognized for her work in this field.

She has developed gene transfer approaches to test treatment strategies for retinal degenerative and ocular neovascular diseases, to elucidate retinal differentiation pathways and to identify pathogenetic mechanisms that lead to blindness. In addition, her laboratory has developed gene transfer approaches to treat syndromic diseases resulting in vision loss, studies the immune response of the eye to viral vectors, and characterizes and utilizes cell specific promoters for the delivery of therapeutic nucleic acids.

Recently she was named the F.M. Kirby Professor of Molecular Ophthalmology. Her research, conducted at Penn over the past 16 years, has established the scientific underpinnings which made it possible to test the first potential definitive retinal gene therapy treatment for patients with blinding retinal degenerations.

Dr. Bennett is the principal investigator of the Foundation Fighting Blindness-funded The Children’s Hospital of Philadelphia (CHOP)-Penn Pediatric Center for Retinal Degenerations and the Scientific Advisor of a clinical trial that evaluates the safety and efficacy of gene augmentation for a Leber Congenital Amaurosis. This study was initiated in the fall of 2007 at The Children’s Hospital of Philadelphia.

Luk H. Vandenberghe, PhD

Luk H. Vandenberghe, PhD

Luk H. Vandenberghe, PhD

Luk H. Vandenberghe, PhD

Luk Vandenberghe is Assistant Professor at Harvard Medical School and runs an active research laboratory at the Massachusetts Eye and Ear Infirmary and the Schepens Eye Research Institute.

He directs the Grousbeck Gene Therapy Center which is focused on the biology of somatic gene transfer, the development of enabling technologies in gene therapy, and the translation of clinical programs with a particular emphasis on vision and hearing restoration.

He is an Associate Director of the Ocular Genomics Institute, a collaborative venture within the Harvard Medical School Department of Ophthalmology, with the ambition to provide diagnosis, treatment, and advancement of the science of the genetic basis of blinding disorders.

Dr. Vandenberghe trained at the Katholieke Universiteit Leuven, Belgium in biological engineering and biomedical sciences. At the University of Pennsylvania, PA, USA he was a scholar and researcher in several premier translational gene therapy laboratories there. He discovered, developed and characterized some of the most widely used gene therapy carriers for in vivo and ex vivo applications including AAV, adeno- and lentiviral-based vectors.

Dr. Vandenberghe made groundbreaking contributions to better understand immune responses to AAV vectors and the targeting of cell types in the macula and retina important for genetic therapies for blindness. In his active career, he published over 40 peer reviewed articles and reviews. He is co-inventor on over a dozen published or pending patents including novel serotypes and improved manufacturing technologies of AAV.

Dr. Serge Picaud, PhD

Dr. Serge Picaud, PhD

Dr. Serge Picaud, PhD

Dr. Serge Picaud, PhD

Serge Picaud is director of research at the Vision Institute in Paris.

Over the last 15 years, he has developed many cellular and animal models of different retinal diseases for assessing the efficacy of neuroprotection or other therapeutic strategies. He thus developed the culture of post-mortem human retinal tissue, which provides the means to test AAV vectors efficacy on human retinal neurones. He has also characterized many animal models using a phenotyping platform inspired from the clinical trial center.

He was a founder and advisor of the biotech company Fovea, which was bought by Sanofi-Aventis to become its ophthalmological division.

Connie Cepko, PhD

Connie Cepko, PhD

Connie Cepko, PhD

Connie Cepko, PhD

Connie Cepko is Professor at Harvard Medical School. She is working on the mechanisms that direct development of the central nervous system (CNS) of vertebrates, and particularly on the vertebrate retina. Connie has produced seminal works in the mechanisms that lead to the death of photoreceptors in the many inherited forms of human blindness.