Biography
Yael Weiss is currently CEO of Mahzi Therapeutics, a company focused on the development of therapies for ultra-rare genetic neurodevelopmental disorders. Mahzi works closely with patient foundations to support their journey towards drug development and bring programs into Mahzi once pre-clinical proof of concept is established. Yael completed her MD at Hadassah Medical School at the Hebrew University in Jerusalem and her PhD at the Weizmann Institute of Science in Rehovot, Israel. She has over 20 years of industry experience in medical-clinical and business development roles at Genzyme, Merck and Ultragenyx. Yael is a member of the NIH driven Bespoke Gene Therapy (BCTG) consortium, ASGCT translational committee, N1 collaborative and is a 2022 Termeer Fellow Board member and advisor to ADNP and FOXG1 foundations.

Biography
Elizabeth Berry-Kravis established the Fragile X Clinic and Research Program at Rush University Medical Center in 1992 and has provided care to over 800 patients with fragile X syndrome (FXS). She conducts research on FXS, including genotype phenotype, molecular, biomarker, outcome measure, and natural history studies, and clinical trials. She has expanded this clinical and translational work to other neurogenetic diseases in the past 10 years, including work on PMS, NPC, Angelman syndrome, Rett syndrome, Battens disease, PKAN, and creatine transport deficiency. She has led the effort to develop new targeted treatments for FXS and other genetic neurological diseases, and has implemented novel trial designs, including Nof1 trials.

Biography
Aron spearheads Intellia's efforts in developing groundbreaking gene editing products targeting patients with rare diseases.Prior to joining Intellia, Aron was the Vice President of Global Regulatory Affairs at Sangamo Therapeutics, Inc., where he led regulatory strategies for 3 years and 5 months. His tenure at Bavarian Nordic further exemplifies his expertise, where he was responsible for global regulatory strategies for vaccine programs in oncology and infectious diseases.Aron's deep experience in the industry is marked by his tenure at various leading pharmaceutical companies, including Pfizer, where he managed a wide range of therapeutic areas and oversaw key regulatory programs for drugs like VIAGRA, TIKOSYN, and NORVASC. His earlier work at DuPont Pharmaceuticals honed his skills in toxicology, emphasizing his comprehensive understanding of drug development and safety evaluation.

Biography
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and HematologyMedical Oncology training at Brigham and Women's Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in 2016.

Biography
Jennifer Puck served on the faculties of U of Pennsylvania Medical School and the National Human Genome Research Institute before becoming a Professor of Pediatrics at UC San Francisco in 2006. While caring for patients with immune disorders, she has researched the genetic causes and mechanisms for rare human immune deficiencies. She developed the newborn screening test for severe combined immunodeficiency (SCID), now adopted throughout the USA and in many countries, enabling early diagnosis and optimal treatment. She has participated in multiple gene therapy trials for SCID and is currently CoPI of the successful first human Artemis SCID lentiviral gene addition trial at UCSF. While these advanced clinical studies offer health benefits to those with rare diseases, they also illuminate important basic immune pathways that were previously unknown.

Biography
Matthew Porteus is the Sutardja Clark Professor of Definitive and Curative Medicine in the Department of Pediatrics and Institute of Stem Cell Biology and Regenerative Medicine at Stanford. He is the coDirector for the Stanford Center for Definitive and Curative Medicine (CDCM). His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood and immune system (including sickle cell disease) and other organ systems. His goal is to combine his research and clinical interests to develop innovative curative therapies. His dream is to one day develop gene editing so that patients are cured of their disease before they or their parents even knew they had it.

Biography
Dr. Crombez joined Ultragenyx following the acquisition of Dimension Therapeutics in November 2017 and is responsible for strategic leadership of the company's clinical development and translational research programs. He also oversees global development functions including Clinical Development, Clinical Operations, BioMetrics, Endpoint Development and Strategy, Regulatory Affairs and Drug SafetyPharmacovigilance. Dr. Crombez is an appointed industry representative on the FDA Cellular, Tissue and Gene Therapies Advisory Committee. Before joining industry, he was assistant professor, Department of Pediatrics, Division of Medical Genetics at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA). Dr. Crombez is a board certified clinical geneticist and completed residencies in pediatrics and medical genetics and a fellowship in clinical biochemical genetics.

Biography
Sal Rico is a biotech executive and clinical pharmacologist with broad experience in patient centered clinical development, covering small molecules, biologics, and medical devices. His expertise is particularly focused on orphan diseases and gene therapy. Before joining Encoded Therapeutics, he was Senior Vice President of Clinical Development at Audentes Therapeutics (now Astellas Gene Therapies). There, he managed the clinical development of AT132, an AAVbased gene therapy for Xlinked myotubular myopathy. He has designed, conducted, and analyzed Phase 14 clinical trials across various therapeutic areas and has played a significant role in garnering regulatory approval for several drugs, biological products, and medical devices such as INTERCEPT Blood System, Intermezzo, Hibor, and others. Prior to Audentes, he led clinical development teams at Cerus Corporation and TransceptPharmaceuticals. Additionally, he was an investigator at the Centre for Drug Research, Hospital de la Santa Creu i Sant Pau in Barcelona, Spain.

Biography
Charlene Son Rigby has spent her career building organizations at the intersection of data, technology, and life sciences. Charlene was previously Chief Business Officer at Fabric Genomics and held executive roles at enterprise software and genomics companies, including Oracle and Doubletwist. She started her career in neuroscience research at Roche. When Charlenes daughter was diagnosed with a rare genetic disease, she cofounded the STXBP1 Foundation. Charlene is also CEO of Global Genes, a onprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. She is committed to finding a cure for her daughter's disorder. Charlene's unplanned connection between her personal life and profession has helped push forward the search for a cure for her daughter and kids like her, and given her work deeper meaning.

Biography
Adrian Veres is a scientist focused on applying next generation sequencing and machine learning approaches to create advanced therapeutics. Prior to founding Dyno to engineer AAVs that solve the in vivo delivery challenge, Adrian worked on generating human beta cells in vitro to enable cell replacement therapeutics in Type 1 Diabetes. Adrian leads Dyno’s RD team to create AAVs with transformative properties that can broaden the reach and efficacy of gene therapies.

Biography
Mike's son was diagnosed with SYNGAP1 in 2018. He founded the SynGAP Research Fund and continues to lead it as a volunteer. Mike comes from a career in public policy, international development and strategy. Previous roles have included establishing a new program at New America, a DC think tank, Budget Planning at both the Gates Foundation Emerson Collective, healthcare consulting at BCG, developing world university support for the World Bank Group, managing a refugee program for the International Catholic Migration Commission in Zimbabwe, and teaching math in Peace Corps Namibia. Graglia has an MBA from Columbia where he was a Bronfman Fellow, an MA from Johns Hopkins School of Advanced International Studies where he was a Soros Fellow, and a BS in mathematics from Gonzaga University, S.J.Graglia lives with his wife Ashley and two sons in Northern California.

Biography
Katherine A. High is a distinguished physician-scientist and currently a Visiting Professor at Rockefeller University. She is also Professor Emerita at the Perelman School of Medicine at the University of Pennsylvania and previously served as President of Therapeutics at AskBio. Dr. High is renowned for her contributions to the field of gene therapy, particularly in the development of therapies for rare diseases. She was the co-founder, president, and chief scientific officer of Spark Therapeutics, where she led the development of the first FDA-approved gene therapy for genetic disease, a rare form of inherited blindness, and played a pivotal role in the development of gene therapies for hemophilia B and hemophilia A. Dr. High's groundbreaking research has earned her numerous awards and honors, including the E.Donnall Thomas Prize from the American Society of Hematology, the Karl Landsteiner Award from the American Association of Blood Banks, the Outstanding Achievement Award from the American Society of Gene and Cell Therapy, and the Sanford Lorraine Cross $1 Million Medical Innovation Award, shared with Dr. Jean Bennett. She is an elected member of the National Academy of Medicine, the American Academy of Arts and Sciences, the Royal College of Physicians, and the National Academy of Sciences. Dr. High's ongoing commitment to advancing precision medicine and improving patient outcomes has had a transformative impact on the field of gene therapy and rare disease research. Her work has led to the development of innovative therapies that have the potential to cure or significantly improve the lives of patients with rare diseases.

Biography
Timothy Yu, MD, PhD, is recognized as a leader in the field of gene therapy, particularly in the development of personalized gene therapies for individuals with rare genetic disorders. A graduate of Harvard College, he completed MD-PhD training at UCSF, neurology residency at Massachusetts General Hospital and Brigham and Women’s Hospital, and a fellowship in neurogenetics at Boston Children’s Hospital. He joined the faculty of Boston Children’s Hospital in the Division of Genetics and Genomics in 2013. He is also an Associate Member at the Broad Institute Dr. Yu’s research group works at the intersection of genomics, informatics, and neurobiology with a focus on understanding and treating disorders of brain development. He was an early pioneer in methodology for genome sequencing and interpretation, which he applied to discover genes responsible for brain development and autism. He is also recognized for having developed the world’s first individualized genomic medicine: milasen, an antisense oligonucleotide targeting a unique genetic mutation causing a child’s fatal neurodegenerative condition – in under one year. Milasen was the first in a pilot series of individualized therapeutics for severe neurogenetic conditions that his team has since created. It also inspired efforts to advance individualized therapeutics for dozens of neglected genetic conditions worldwide, including the N-of-1 Collaborative, an international coalition founded by Dr. Yu to advance scientific, regulatory, and ethical best practices for this budding field. Dr. Yu has authored more than 80 scientific publications and has been recognized with numerous awards and honors, including the NORD Rare Impact Award, the Global Genes Rare Champion award, and the ANA Denny-Brown Young Neurologic Scholar award. He is dedicated to advancing precision medicine and improving patient outcomes through his research and clinical practice.

Biography
Timothy Yu, MD, PhD, is recognized as a leader in the field of gene therapy, particularly in the development of personalized gene therapies for individuals with rare genetic disorders. A graduate of Harvard College, he completed MD-PhD training at UCSF, neurology residency at Massachusetts General Hospital and Brigham and Women’s Hospital, and a fellowship in neurogenetics at Boston Children’s Hospital. He joined the faculty of Boston Children’s Hospital in the Division of Genetics and Genomics in 2013. He is also an Associate Member at the Broad Institute Dr. Yu’s research group works at the intersection of genomics, informatics, and neurobiology with a focus on understanding and treating disorders of brain development. He was an early pioneer in methodology for genome sequencing and interpretation, which he applied to discover genes responsible for brain development and autism. He is also recognized for having developed the world’s first individualized genomic medicine: milasen, an antisense oligonucleotide targeting a unique genetic mutation causing a child’s fatal neurodegenerative condition – in under one year. Milasen was the first in a pilot series of individualized therapeutics for severe neurogenetic conditions that his team has since created. It also inspired efforts to advance individualized therapeutics for dozens of neglected genetic conditions worldwide, including the N-of-1 Collaborative, an international coalition founded by Dr. Yu to advance scientific, regulatory, and ethical best practices for this budding field. Dr. Yu has authored more than 80 scientific publications and has been recognized with numerous awards and honors, including the NORD Rare Impact Award, the Global Genes Rare Champion award, and the ANA Denny-Brown Young Neurologic Scholar award. He is dedicated to advancing precision medicine and improving patient outcomes through his research and clinical practice.

Biography
Katherine A. High is a distinguished physician-scientist and currently a Visiting Professor at Rockefeller University. She is also Professor Emerita at the Perelman School of Medicine at the University of Pennsylvania and previously served as President of Therapeutics at AskBio. Dr. High is renowned for her contributions to the field of gene therapy, particularly in the development of therapies for rare diseases. She was the co-founder, president, and chief scientific officer of Spark Therapeutics, where she led the development of the first FDA-approved gene therapy for genetic disease, a rare form of inherited blindness, and played a pivotal role in the development of gene therapies for hemophilia B and hemophilia A. Dr. High's groundbreaking research has earned her numerous awards and honors, including the E.Donnall Thomas Prize from the American Society of Hematology, the Karl Landsteiner Award from the American Association of Blood Banks, the Outstanding Achievement Award from the American Society of Gene and Cell Therapy, and the Sanford Lorraine Cross $1 Million Medical Innovation Award, shared with Dr. Jean Bennett. She is an elected member of the National Academy of Medicine, the American Academy of Arts and Sciences, the Royal College of Physicians, and the National Academy of Sciences. Dr. High's ongoing commitment to advancing precision medicine and improving patient outcomes has had a transformative impact on the field of gene therapy and rare disease research. Her work has led to the development of innovative therapies that have the potential to cure or significantly improve the lives of patients with rare diseases.

Biography
Timothy Yu, MD, PhD, is recognized as a leader in the field of gene therapy, particularly in the development of personalized gene therapies for individuals with rare genetic disorders. A graduate of Harvard College, he completed MD-PhD training at UCSF, neurology residency at Massachusetts General Hospital and Brigham and Women’s Hospital, and a fellowship in neurogenetics at Boston Children’s Hospital. He joined the faculty of Boston Children’s Hospital in the Division of Genetics and Genomics in 2013. He is also an Associate Member at the Broad Institute Dr. Yu’s research group works at the intersection of genomics, informatics, and neurobiology with a focus on understanding and treating disorders of brain development. He was an early pioneer in methodology for genome sequencing and interpretation, which he applied to discover genes responsible for brain development and autism. He is also recognized for having developed the world’s first individualized genomic medicine: milasen, an antisense oligonucleotide targeting a unique genetic mutation causing a child’s fatal neurodegenerative condition – in under one year. Milasen was the first in a pilot series of individualized therapeutics for severe neurogenetic conditions that his team has since created. It also inspired efforts to advance individualized therapeutics for dozens of neglected genetic conditions worldwide, including the N-of-1 Collaborative, an international coalition founded by Dr. Yu to advance scientific, regulatory, and ethical best practices for this budding field. Dr. Yu has authored more than 80 scientific publications and has been recognized with numerous awards and honors, including the NORD Rare Impact Award, the Global Genes Rare Champion award, and the ANA Denny-Brown Young Neurologic Scholar award. He is dedicated to advancing precision medicine and improving patient outcomes through his research and clinical practice.

Talk
Maturing technologies for precision therapeutics offer hope to patients in the long tail of rare genetic diseases, but often require action at the individual mutation level, stressing an ecosystem organized around tackling one disease at a time. Through clinical examples I will discuss how scientific and regulatory pioneering will be required to enable this promise.

Biography
Swati Tole is the Chief Medical Officer at Capsida Biotherapeutics, a fully integrated gene therapy platform company focused on creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening disorders. Dr. Tole's drug development experience spans a wide range of disease areas in adults and children, including rheumatology, gastroenterology, hepatology, and infectious disease. Prior to joining Capsida, she spent 13 years at Genentech in roles of increasing responsibility, most recently as Vice President of Clinical Development. Prior to joining industry, Dr. Tole was a practicing internist at UCSF.

Biography
Adam Shaywitz leads the clinical organization for Gene Therapy at BridgeBio Pharma, focused on developing gene therapy treatments for a range of monogenic rare diseases including clinical stage programs for Canavan disease and congenital adrenal hyperplasia. Prior to joining BridgeBio Adam led clinical stage programs at BioMarin Pharmaceutical aimed at treating a broad range of rare diseases including Sanfilippo Syndrome (MPS III), achondroplasia, endocrinologic disorders, Morquio A syndrome and other lysosomal storage disorders. Before joining BioMarin Adam spent four years at Amgen in their early clinical development group, focused on advancing promising diabetes and metabolism agents into the clinic. He received his M.D. and Ph.D. degrees from Harvard Medical School, did his internal medicine training at MGH and subsequently went on to pursue a fellowship in endocrinology, diabetes and metabolism at Beth Israel Deaconess Medical Center and the Joslin Diabetes Center. Adam currently serves on the Board of Directors at Cure Sanfilippo Foundation.

Biography
Pooja Agarwal is responsible for driving gene therapy programs from concept to late stage clinical development at BioMarin. She leads a team that maximizes the latest technologies to develop new and potentially best in class gene therapies. Her team is also responsible for the evaluation of novel gene therapy modalities with the potential to overcome some of the current constraints in gene delivery. Prior to joining BioMarin, Pooja served as Senior Vice President, Research and Development at Krystal Biotech, where she led the development of Vyjuvek, which was approved earlier this year as the first topical redosable gene therapy for Recessive Dystrophic Epidermolysis Bullosa.

Biography
Thomas Wechsler is the head of Gene Therapy as part of the RNA, Gene Therapy and Delivery team in Therapeutics Discovery. In this role, he is responsible for leading the discovery and optimization of gene therapy clinical candidates for a range of therapeutic areas including retinal diseases and neuroscience. Prior to joining Janssen, Thomas was an Independent Consultant and the VP of Research at Grace Science, LLC developing an AAVbased gene therapy for the rare disease NGLY1 deficiency. Previously, at Sangamo Therapeutics he contributed to the first ever in vivo gene editing program to gain FDA approval to start clinical trials using a nucleasebased mechanism and to the ongoing STAAR gene therapy trial for the treatment of Fabry disease. Thomas has earned his Ph.D. from LMU, Germany and completed his Ph.D. Thesis work at UCSF. He conducted his Postdoctoral Fellowship working on DNA repair at Cancer Research UK.

Biography
James Chung, MD, PhD, is Chief Medical Officer at Kyverna Therapeutics. Dr. Chung brings extensive commitment to the autoimmune field within the biopharmaceutical industry working across the entire drug development process. Prior to Kyverna, Dr. Chung began in Early Development and served as the Inflammation Therapeutic Area Head. He also worked in latestage development as the Global Development Leader for Enbrel and as the Inflammation head for the US and Global Medical Organizations. Dr. Chung obtained his MD and PhD in immunology at the University of Pennsylvania where he also completed his residency in Internal Medicine and fellowship in Rheumatology.

Biography
Jennifer has worked in drug development for over 20 years, primarily focused on advancing global development programs for the treatment of rare neurological diseases. Her background and experience working with global regulatory agencies to support diverse drug and biological therapy development programs in both the development and postmarketing strategies will greatly benefit the FAST community. Jennifer has a niece, Samantha who lives with Angelman syndrome.

Biography
Morten Sogaard is President, Research TechOps at Astellas Gene Therapies. Morten oversees Research and nonGMP CMC in South SF and Japan with a remit to deliver 2 clinical gene therapy candidates p.a. Before Astellas Morten led human genetics, functional genomics, CompBio and diagnostics functions as Global Head of Genome Sciences and a member of Pfizer's RD LT, and later led Target Sciences. He also headed up Search Evaluation for Platform Technologies and Oncology. Morten started his industry career at Pharmacia (Sweden), where he oversaw NextGen immunooncology antibody therapeutics. Later at AZ he led Molecular Sciences providing biologics and genomics capabilities to TAs. 2004, Morten moved to BI (CT) as global head, enabling technology, building a companywide platform of global skill centers, research informatics and briefly served as Head of RD Informatics. Morten received his Ph.D. in Biochemistry from Univ. of Copenhagen and did postdoctoral studies at MSKCC.

Biography
Dr. Kirn is an Adjunct Professor of Bioengineering and Molecular Cellular Biology at Cal Berkeley. Over 30 years, he has co founded and been CEO of four viral vector based genetic medicines companies, all of which to date were either acquired or went public. Dr. Kirn was at Onyx Pharmaceuticals (VP) and Celgene (SVP), and was a senior advisor to Novartis, Bayer, Pfizer, Biogen and others. He received a BA in Physiology (Departmental Citation; Phi Beta Kappa) from Cal Berkeley, an MD (Alpha Omega Alpha) from UCSF and internal medicine residency training at Harvard Medical School, Brigham and Women's Hospital (including as Chief Medical Resident at affiliated VA hospital). He completed oncology and clinical research fellowships at UCSF and completed a certificate of business excellence from the Haas Business School at UC Berkeley. In 2013, he was awarded the Johnson Johnson Entrepreneur Innovator award from the JJ Innovation Center.

Biography
Dominic Borie is an accomplished immunologist and transplant surgeon with extensive experience in drug development. Before joining Kyverna, he held leadership positions at Horizon Therapeutics, Genentech, Amgen, and Roche. Dr. Borie has an extensive publication history with over 50 publications in peer reviewed journals, 10 book chapters, and four issued patents. He received his Ph.D. in transplantation immunology from the University of Paris V Descartes and his M.D., Masters degree in Immunology, and Certificate of Immunology and Immunopathology from the University of Paris XII.

Talk
Transformative Outcomes With CAR-T Cell Therapy for Autoimmune Diseases
Kyverna Therapeutics is working to transform outcomes for patients with autoimmune diseases with KYV101, a fully human antiCD19 CAR-T cell therapy with structural features that confer a safety profile well suited for use in autoimmune diseases. Kyverna has treated patients with multiple autoimmune diseases in sites around the world.

Biography
Lucas drives the development of next generation CRISPR products and leads the scientific research program for new discoveries. He focuses on harnessing microbial diversity to build novel CRISPR applications for DNA detection and genome editing. At UC Berkeley, Lucas worked in Jennifer Doudna's lab and authored multiple scientific papers, the latest of which features his discovery of the newest Cas system to date, Cas14, in Science. He received a BS in Chemistry from Washington University in St. Louis, where he researched bacterial photosynthesis.

Biography
Wendy Erler is the Vice President of Patient Experience, at Astra Zeneca, Alexion Rare Disease where she is leads the enterprise innovation model to incorporate patient insights throughout the drug development and commercialization processes. She is dedicated to working with the rare disease community as a true partner. Wendy serves on the board of Global GenesRARE-X and the advisory council for the n-Lorem Foundation. Previously, Ms. Erler was on the executive leadership team at Wave Life Sciences where she led Patient Advocacy and Commercial. There, Wendy contributed to the strategic selection of clinical candidates, operationalizing and opening 5 clinical trials, and building corporate culture patient centric initiatives. Previously, Wendy worked for Biogen in expanding leadership roles across many functions including commercial launch strategy, field team leadership roles, program management, business development and public affairs.Wendy graduated from Miami University and earned her MBA from St. Josephs University.

Biography
Collaborating with multiple academic and industry partners around the world. Current programs include drug repurposing, de novo small molecules, ASOs, RNAi, and gene editing. Building a clinical trial ready community through various initiatives including patient registry, digital natural history study, biobank, blood and EEG biomarker discovery and patient education.

Biography
Maria Grazia is the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine, founder of the Center for Definitive and Curative Medicine, and former co-director of the Institute for Stem Cell Biology and Regenerative Medicine at Stanford University. Dr. Roncarolo has spent her career translating discoveries in immune-mediated diseases and regenerative medicine into novel patient therapies. Dr. Roncarolo and her team were the first to discover T regulatory Type 1 cells in patients, and the first to develop curative treatments for ADA-SCID. A pediatric immunologist by training, she was instrumental in bringing novel cell and gene therapies to market including Strimvelis and Libmeldy. Dr Roncarolo is a co-founder of the gene-editing company Graphite Bio (NSDQ: GRPH), where she still serves as the Chair of the Scientific Advisory Board.