Brian Bigger was awarded a Bachelors degree from the University of Bath in Applied Biology. His PhD was conducted in the Gene Therapy Research Group, Imperial College, London, where he worked with Professor Charles Coutelle on developing a gene delivery vehicle for mitochondrial gene therapy. On completion of his PhD in 2000, Dr Bigger joined Dr Mike Themis, Imperial College, London to work on a Wellcome Trust collaborative project with Cancer Research UK, investigating gene delivery to stem cells for liver diseases. In 2004 he joined Dr Suzanne Watt’s group in Oxford University and the National Blood Service as a Senior Research Scientist to work on mechanisms of stem cell homing. In 2006 Dr Bigger set up the Stem Cell & Neurotherapies laboratory at the University of Manchester and the Royal Manchester Children’s hospital.

Dr. Carmine Settembre graduated in 2002 in pharmaceutical chemistry at the university of Naples “Federico II”. In 2002 he joined TIGEM institute as PhD student, then he moved in the United States as post-doctoral fellow first at Columbia University in New York and then at the Baylor College of Medicine, in Houston.  In 2011 he became assistant professor at the Baylor college of medicine. In 2013, thanks to the Dulbecco Telethon Institute (DTI) career award program he set up his own laboratory at TIGEM. In 2018 he became associate professor in Histology and Embryology at the University of Naples “Federico II”.

Christian Haass graduated in Molecular Biology at the University of Heidelberg, Germany. He was a postdoc and Assistant professor of Neurology at the Harvard Medical School in the Institute of Dr. Dennis Selkoe. Since 1999 he is the head of the division of Biochemistry at the Ludwig-Maximilians University. Haass is also the coordinator of the German Center for Neurodegenerative Disorders (DZNE) in Munich and the speaker of the DFG cluster of excellence “Systems Neurology (SyNergy)”. Haass received many awards, among them the Brain Prize in 2018.

Dr. Juan S. Bonifacino is a National Institutes of Health (NIH) Distinguished Investigator and Associate Scientific Director for the Neuroscience and Cellular and Structural Biology Division (NCSBD) of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), NIH. Bethesda, Maryland, USA. His laboratory studies the molecular mechanisms of intracellular protein and organelle transport, and the diseases that result from dysfunction of these mechanisms.

I graduated in 2005 at the University of Naples Federico II. Then I joined the TIGEM as an Open University PhD student, in Andrea Ballabio’s laboratory. My main PhD research project was focused to the study of the mechanisms leading to the neurodegeneration in lysosomal storage disorders (LSDs). I spent more than three years in the United States where I worked at Baylor College of Medicine (Houston) as visiting PhD student. During this period, I also contributed to the identification of the Transcription factor EB (TFEB) as a master regulator of lysosome biogenesis and autophagy. As postdoctoral researcher I discovered a new transcriptional regulation, mediated by TFEB, controlling mTORC1 signaling in response to nutrients. More recently, I demonstrated that this mechanism promotes cystogenesis and tumor growth in Birt-Hogg-Dube’ syndrome, a genetic condition due to loss of function mutations in the gene encoding FLCN, a key player of mTORC1 signaling. I’m an independent investigator at Tigem since January 2021. My research activity is currently supported by Kidney Cancer Association, Worldwide Cancer Research and Telethon foundation.

James (“Jim”) Hurley is a professor in the Department of Molecular and Cell Biology at the University of California, Berkeley. He graduated in Physics from San Francisco State University and obtained his Ph.D. in Biophysics in 1990 from the University of California, San Francisco. He was a senior investigator in the intramural program of the National Institutes of Health from 1992-2013, and joined the faculty of the University of California, Berkeley in 2013. He is a member of the National Academy of Sciences.

Dr. Hurley uses structural biology, biophysics, biochemistry, and cell biology approaches to understand the structure and function of cell membranes in health and disease. He is known for his work on the structure and mechanism of the ESCRT membrane scission machinery, coated vesicle and endosome biogenesis, lipid transporters and second messenger systems, and the autophagy core complexes.

Dr. Hurley’s lab currently has four major projects in the general area of mechanistic membrane biology. He has a long-standing interest in the core mechanisms of autophagy, and is currently focusing on the structural and biophysical basis of mitophagy and its role in Parkinson’s Disease. He is investigating the mechanism of membrane scission by ESCRT complexes in the contexts of HIV budding and release from infected cells, autophagy, and membrane repair. In a third project, the hijacking of coated vesicle trafficking by HIV is being worked out by structural, reconstitution, and live cell imaging approaches. Lastly, in the newest project, he is using structural and biochemical approaches to study the roles of the lysosomal GTPase regulatory complexes FLCN-FNIP and C9orf72-SMRC8 in neurodegenerative diseases.

PhD in Molecular Biology in 2002 at Faculty of Sciences of the University of Porto. Auxiliary Researcher in the Department of Human Genetics in the Instituto Nacional de Saúde Dr. Ricardo Jorge (INSA) and Head of the Lysosomal Storage Diseases Research Group.
Her scientific activity has been developed in the Human Genetics field with a major focus on LSDs. She has been conducting studies in order to expand the knowledge on the mutational spectrum of these diseases in Portugal. Mucolipidoses (ML) and mucopolysaccharidoses (MPS) are her major research focus. Presently, her group aims to develop RNA based therapeutic approaches to address these pathologies. The correction of LSDs causing mutations and the decrease of accumulated substrates are some of examples of aims that drive the development of such approaches.

Dr. Federico Mingozzi is the chief scientific officer at Spark Therapeutics, bringing two decades of experience in gene therapy, immunology, as well as biochemistry and molecular biology in both industry and academic settings.

Federico began his scientific career studying the genetic basis of bleeding disorders. At the Children’s Hospital Philadelphia (CHOP) he conducted pioneering studies on liver gene transfer with adeno-associated virus (AAV) vectors and immunology. Federico was involved in several first-in-human clinical studies of gene therapy based on the AAV vector platform while serving as the director of translational research the Center for Cellular and Molecular Therapeutics, at CHOP. He also led studies aimed at the characterization of human immune responses to AAV vectors and the development of strategies to modulate vector immunogenicity.

Eric Adler, MD, is a Professor of Medicine and Medical Director of the Heart Transplant Program at the University of California, San Diego (UCSD). He earned his medical degree from Boston University School of Medicine. He completed his internship and residency at the University of Washington and a cardiology fellowship at Mount Sinai School of Medicine. He is board certified in cardiovascular disease and advanced heart failure. He specializes in caring for patients with advanced heart failure, including those who need ventricular assist devices (VADs) or heart transplants.

His research is focsed on the study and treatment of cardiomyopathy. He has contributed to multipe papers demonstrating techniques for the isolation of enriched populations of cardiovascular progenitor cells from pluripotent stem cells and the utility of these cells for modeling cardiovascular disease. Recently, his work has led to the development of a novel gene therapy for Danon disease, a rare cardiovascular disorder associated with severe hypertrophic cardiomyopathy. The treatment is currently being evaluated in a Phase 1 trial. He also performs clinical research in heart failure and have high-impact publications on a variety of topics including cardiac transplant, mechanical circulatory support, palliative care and machine learning.