Sofia Kakava is currently in her third year of a Ph.D. program at the University of Zurich. Her focus centers on the interactions between plasma-derived lipoproteins, particularly HDL, and the blood-brain barrier, with a keen interest in the health of the cerebrovasculature. Sofia's academic journey started at the University of Patras, where she completed her bachelor's degree, delving into the complexities of lipoprotein metabolism. Continuing her studies abroad, Sofia pursued her master's degree at Utrecht University in the Netherlands. Her work involved the development of a 3D in vitro model of dopaminergic neurons affected by Parkinson's disease, using iPSC-derived neurons. This innovative approach provided a unique perspective on neurodegenerative diseases and highlighted Sofia's commitment to pushing the boundaries of scientific exploration.During her master's program, Sofia got further experience through an internship at the University of Cambridge, UK. Here, she delved into the world of liver organoids, investigating lipid uptake, fatty liver, and liver regeneration. Sofia's research journey, marked by international experiences and a multidisciplinary approach, positions her as a promising scientist at the forefront of advancing our understanding of brain and lipid metabolism.

Dr Lorraine Kalia is an Associate Professor and Clinician-Scientist at the University of Toronto, Toronto, ON, Canada, whose clinical practice and research programme focus on Parkinson’s disease. She holds the Wolfond-Krembil Chair in Parkinson’s Disease Research. Dr Kalia is Co-Editor-in-Chief of the Journal of Parkinson’s Disease, Vice-Chair of the International Parkinson and Movement Disorder Society’s Scientific Issues Committee and Basic Science Interest Group, and Treasurer of the Canadian Movement Disorders Society. Her research programme, which spans the lab to the clinic, aims to understand the key molecular mechanisms responsible for neurodegeneration in Parkinson’s disease and to identify therapeutic agents that can modulate these molecular targets.

My occupation in the Alzheimercenter in Amsterdam is as a neuropsychologist and PhD student, meaning I work clinically in the memory clinic as well as in research. My research focusses on finding biomarkers for CTE (chronic traumatic encephelopathy), where I'm interested in imaging, fluids and clinical data.

SHORT BIOGRAPHY Professor and Director of Viral Vector Core, Department of Neurobiology, Duke University School of Medicine; Co-Founder at CLAIRIgene LLC INSTITUTION AND LOCATION DEGREE COMPLETION DATE FIELD OF STUDY Grodno State University, Belarus BSc 05/1992 Medical Studies Hebrew University, Israel MSc 05/1998 Immunology Hebrew University, Israel PhD 08/2004 Biochemistry and Human Molecular Genetics University of North Carolina at Chapel Hill Postdoc 01/2008 Gene Therapy, Virology Pertinent research contributions: (i) Development and optimization of Adeno-associated vectors and lentiviral vectors for delivery of CRISPR/Cas systems. Specifically, Kantor’s laboratory focused on the development of non-integrating delivery platforms, including adeno-associated vectors and integrase-deficient lentiviral vectors (IDLVs) carrying CRISPR/Cas9 components in the single virus (all-in-one systems). Kantor’s lab demonstrated that those all-in-one, transient vector systems are capable of efficient and safe gene-editing of the CNS targeted genes in vitro and in vivo [1], [2], [3], [4]. (ii) Development of all-in-one lentiviral vectors for epigenome-editing within SNCA gene. Epigenetic- based targeting of SNCA gene that encodes alpha-synuclein, highlighted a novel approach for the development of next-generation of treatment for Parkinson’s disease [5], [6], [7]. This work includes development of all-in-one lentiviral vector carrying deactivated dCas9 protein fused with de novo DNA methyltransferase targeting regulatory region within SNCA intron 1. The approach led to efficient and long-term downregulation of alpha-synuclein expression and rescuing Parkinson’s related phenotypes in neuroprogenitor cells derived from human IPSCs derived from the Parkinson’s patients. (iii) Development of and optimization of lentiviral and adeno-associated vectors for efficient delivery of epigenome-editing tools related to the Parkinson’s and Alzheimer’s diseases [8], [9]. In addition, Dr. Kantor serves as a Director of Viral Vector Core affiliated with the department of Neurobiology and School of Medicine at Duke University. The viral vector core is top-notch research facility focused on the pre-GMP viral services to broad research community inside and outside of Duke University. Dr. Kantor consults many internal and external faculties related to viral vector platforms and gene therapy approaches. Dr. Kantor is co-founder of CLARIgene LLC, a biotech company focused on the development of new therapeutics for the CNS diseases. SELECTED PUBLICATIONS 1. Ortinski, P.I., et al., Integrase-Deficient Lentiviral Vector as an All-in-One Platform for Highly Efficient CRISPR/Cas9-Mediated Gene Editing. Mol Ther Methods Clin Dev, 2017. 5: p. 153-164. 2. Tagliafierro, L., et al., Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models. J Vis Exp, 2019(145). 3. Brown, L.Y., W. Dong, and B. Kantor, An Improved Protocol for the Production of Lentiviral Vectors. STAR Protoc, 2020. 1(3): p. 100152. 4. Dong, W. and B. Kantor, Lentiviral Vectors for Delivery of Gene-Editing Systems Based on CRISPR/Cas: Current State and Perspectives. Viruses, 2021. 13(7). 5. Kantor, B., et al., Downregulation of SNCA Expression by Targeted Editing of DNA Methylation: A Potential Strategy for Precision Therapy in PD. Mol Ther, 2018. 26(11): p. 2638-2649. 6. Gu, J., et al., Cell-Type Specific Changes in DNA Methylation of SNCA Intron 1 in Synucleinopathy Brains. Front Neurosci, 2021. 15: p. 652226. 7. Chen, V., et al., The mechanistic role of alpha-synuclein in the nucleus: impaired nuclear function caused by familial Parkinson's disease SNCA mutations. Hum Mol Genet, 2020. 29(18): p. 3107-3121. 8. Rittiner, J., et al., Therapeutic modulation of gene expression in the disease state: Treatment strategies and approaches for the development of next-generation of the epigenetic drugs. Front Bioeng Biotechnol, 2022. 10: p. 1035543. 9. Yang, A., B. Kantor, and O. Chiba-Falek, APOE: The New Frontier in the Development of a Therapeutic Target towards Precision Medicine in Late-Onset Alzheimer's. Int J Mol Sci, 2021. 22(3).

Dimitrios Kapogiannis is a Senior Investigator and Deputy Chief of the Laboratory of Clinical Investigation at the National Institute on Aging (NIA/NIH), as well as adjunct Associate Professor of Neurology at Johns Hopkins Medicine. He earned a Medical Degree from the National and Kapodistrian University of Athens, Greece, in 1998 and completed a Neurology Residency training at Massachusetts General and Brigham and Women’s Hospitals of Harvard University, USA, in 2006. He completed a Fellowship in Cognitive and Behavioral Neurology at the National Institute of Neurological Disorders and Stroke, NIH, before joining the NIA in 2009. His research program includes basic, translational and clinical studies in Alzheimer’s disease and related disorders (AD/ADRD). He has pioneered the isolation of Extracellular Vesicles enriched for brain cell origin from blood for diagnosis and monitoring of AD and other neurological and psychiatric diseases and as outcomes in clinical trials. His clinical studies focus on metabolic and ketogenic interventions (anti-diabetic drugs, calorie restriction, oral ketone ester) to prevent and treat aging associated cognitive decline and AD/ADRD.