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
Pavan is a Principal in the Boston office with a focus on Oncology and Precision Medicine. He co-leads ZS Biomarker Center of Excellence and works extensively with clients in the area of Precision Medicine. Over the last 12+ years at ZS, Pavan has focused on supporting Oncology companies across a full spectrum of medical, marketing and sales challenges. With experience working in most tumor types in Oncology, he brings deep therapeutic area expertise in Oncology. He has helped companies assess organization structures, develop commercialization strategy, segmentations, design forward looking local and global commercial models to successfully navigate the rapidly evolving Oncology market dynamics. With expertise in Precision Medicine and Oncology, Pavan has helped clients with market landscape and opportunity assessments, disease area strategy, and brand strategy including positioning. In addition, he has partnered with Oncology diagnostic companies across a range of engagements including portfolio strategy development, sales planning, customer segmentation, and competitive assessment. He holds an M.B.A from Yale School of Management, and B.S. in Mechanical Engineering from Indian Institute of Technology Madras. Prior to joining ZS, Pavan worked as a Design Engineer at Caterpillar Inc.

Biography
Carolyn R. Bertozzi, a distinguished figure in the field of bioorthogonal chemistry, has made significant contributions to gene and cell therapies across various domains. Her groundbreaking work has earned her the prestigious Nobel Prize, recognizing her innovative techniques that allow precise modification of biomolecules within living cells. Dr. Bertozzi’s research not only advances our understanding of cellular processes but also provides novel strategies for cell targeting and engineering. Her academic journey began with a focus on chemistry at Harvard University, followed by biochemistry studies at the University of California, Berkeley. Since receiving the Nobel Prize, Dr. Bertozzi has continued to push the boundaries of her field, developing new cancer immunotherapies and point-of-care tests for tuberculosis, and unraveling connections between rare diseases and cancer. Her remarkable contributions exemplify the intersection of chemistry and biology, revolutionizing therapeutic approaches and paving the way for precision medicine.

Biography
Mathieu Nonnenmacher is currently leading the Novel Capsid Discovery group at Voyager Therapeutics, a Massachusetts gene therapy company using AAV for treatment of severe neurological diseases including Huntingtons disease, Friedreichs Ataxia and amyotrophic lateral sclerosis. During his postdoctoral training at Mount Sinai School of Medicine, Mathieu identified a natural mechanism of capsid-genome correlation that had important implications for the development of AAV libraries for Directed Evolution. Mathieu Joined Voyager Therapeutics in 2014 to develop capsid engineering for brain delivery. Since then, he established the TRACER biopanning platform which led to the discovery of numerous neurotrophic capsid variants in mouse and non-human primates. The Voyager team has recently discovered the mechanism of action of certain capsids, leading to the developments of novel viral and non-viral shuttles capable of crossing the blood-brain barrier.

Biography
James M. Wilson, MD, PhD, is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he has led an effort to develop the field of gene therapy. His research career spanning over 40 years has focused on rare diseases and ways to treat them by gene therapy. Dr. Wilson has published over 600 papers and is named on more than 1200 patents worldwide. The Wilson lab identified a new type of vector based on novel isolates of adeno-associated viruses which have become best in class for gene therapy. More recently Dr. Wilsons laboratory has focused on improved vectors for gene therapy and clinical applications of genome editing and mRNA therapy.

Biography
Dr. Kunwoo Lee is the Co-Founder and CEO of GenEdit. He is an innovator and believer of gene therapy. He is the inventor of several delivery technologies for gene therapy including CRISPR gene editing. Prior to GenEdit, he completed graduate research at UC Berkeley in the Department of Bioengineering. He was named as a Forbes 30 under 30 in 2017 and was a Siebel Scholar in 2016.

Biography
Carolyn R. Bertozzi, a distinguished figure in the fields of glycoscience and chemical biology, has made significant contributions to gene and cell therapies across various domains. Her groundbreaking work in the area of bioorthogonal chemistry has earned her the prestigious Nobel Prize, recognizing her innovative techniques that allow precise modification of biomolecules in living cells and organisms. Dr. Bertozzis research not only advances our understanding of cellular processes but also provides novel strategies for cell targeting and engineering. Her academic journey began with a focus on chemistry at Harvard University, followed by biochemistry studies at the University of California, Berkeley. Since receiving the Nobel Prize, Dr. Bertozzi has continued to push the boundaries of her field, developing new cancer immunotherapies and point-of-care tests for tuberculosis, and unraveling connections between rare diseases and cancer.Her remarkable contributions exemplify the intersection of chemistry and biology, revolutionizing therapeutic approaches and paving the way for precision medicine.

Talk
This presentation will highlight new therapeutic modalities developed in my lab that are rooted in glycobiology.

Biography
Haig Aghajanian, Ph.D., is Co-Founder and Vice President of Research at Capstan, as well as Adjunct Assistant Professor of Medicine at the Perelman School of Medicine at the University of Pennsylvania. His research has focused on the etiology of cardiac disease and cell therapy for the treatment of fibrosis culminating in his recent work published in Nature and Science that served as the inception of Capstan.

Biography
Nadav Ahituv is a Professor in the Department of Bioengineering and Therapeutic Sciences and the Interim Director of the Institute for Human Genetics at the University of California, San Francisco. He received his PhD in human genetics from Tel-Aviv University working on hereditary hearing loss. He then did his postdoc, specializing in functional genomics, in the Lawrence Berkeley National Laboratory and the DOE Joint Genome Institute. His current work is focused on identifying gene regulatory elements and linking nucleotide variation within them to various phenotypes including morphological differences between species, drug response and human disease. His lab was one of the co-developers of massively parallel reporter assays (MPRAs) that allow for high-throughput functional characterization of gene regulatory elements and pioneered the use of gene regulatory elements as therapeutic targets. Finally, the lab also utilizes short DNA sequences for diagnostic purposes.

Biography
Carolyn R. Bertozzi, a distinguished figure in the field of bioorthogonal chemistry, has made significant contributions to gene and cell therapies across various domains. Her groundbreaking work has earned her the prestigious Nobel Prize, recognizing her innovative techniques that allow precise modification of biomolecules within living cells. Dr. Bertozzi’s research not only advances our understanding of cellular processes but also provides novel strategies for cell targeting and engineering. Her academic journey began with a focus on chemistry at Harvard University, followed by biochemistry studies at the University of California, Berkeley. Since receiving the Nobel Prize, Dr. Bertozzi has continued to push the boundaries of her field, developing new cancer immunotherapies and point-of-care tests for tuberculosis, and unraveling connections between rare diseases and cancer. Her remarkable contributions exemplify the intersection of chemistry and biology, revolutionizing therapeutic approaches and paving the way for precision medicine.

Biography
Dr. Courtney Silverthorn is a distinguished professional with a robust background in science policy and technology transfer. Currently, she oversees business development opportunities for the Foundation for the NIH. Dr. Silverthorn has also played a pivotal role in the Bespoke Gene Therapy Consortium, a collaborative project focused on accelerating the development of gene therapies for diseases that currently have no commercial interest. Her contributions to this consortium underscore her commitment to advancing treatments for rare diseases.In addition to her work at FNIH and prior roles at several federal agencies, Dr. Silverthorn has shared her insights on platforms such as the Rare Disease Advisor Podcast. In an interview with Larry Luxner, she discussed the consortium's impact on rare diseases, highlighting her expertise in scientific partnerships and innovation. Dr. Silverthorns dedication to enhancing healthcare through scientific research and collaboration continues to make her a significant figure in the field.

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
James M. Wilson, MD, PhD, is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he has led an effort to develop the field of gene therapy. His research career spanning over 40 years has focused on rare diseases and ways to treat them by gene therapy. Dr. Wilson has published over 600 papers and is named on more than 1200 patents worldwide. The Wilson lab identified a new type of vector based on novel isolates of adeno-associated viruses which have become best in class for gene therapy. More recently Dr. Wilsons laboratory has focused on improved vectors for gene therapy and clinical applications of genome editing and mRNA therapy.

Biography
James M. Wilson, MD, PhD, is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he has led an effort to develop the field of gene therapy. His research career spanning over 40 years has focused on rare diseases and ways to treat them by gene therapy. Dr. Wilson has published over 600 papers and is named on more than 1200 patents worldwide. The Wilson lab identified a new type of vector based on novel isolates of adeno-associated viruses which have become best in class for gene therapy. More recently Dr. Wilsons laboratory has focused on improved vectors for gene therapy and clinical applications of genome editing and mRNA therapy.

Biography
Charles A. Gersbach, PhD, is the John W. Strohbehn Distinguished Professor of Biomedical Engineering at Duke University and Director of the Center for Advanced Genomic Technologies. His research focuses on developing and applying genome engineering technologies, particularly CRISPR-based methods, for therapeutic genome editing, synthetic biology, and functional genomics. Dr. Gersbach's work aims to bridge the gap between fundamental research and clinical applications, significantly advancing gene and cell therapies for genetic diseases and regenerative medicine. His contributions have been recognized with awards such as the NIH Directors Pioneer Award, and he has been inducted into the AIMBE College of Fellows. Dr. Gersbach earned his PhD in Biomedical Engineering from the Georgia Institute of Technology and completed postdoctoral training at the Salk Institute for Biological Studies.

Biography
Prashant Mali, PhD, is a Professor in the Shu Chien-Gene Lay Department of Bioengineering at the University of California San Diego. His research spans the exciting fields of synthetic biology and regenerative medicine. Dr. Malis lab is at the forefront of developing toolsets for systematic genome interpretation and gene therapy applications, focusing on genome and transcriptome engineering. Dr. Mali has been instrumental in pioneering CRISPRs and ADARs as powerful tools for DNA and RNA editing, significantly impacting basic biology and human therapeutics. He holds degrees in Electrical Engineering from the Indian Institute of Technology Bombay and a doctorate in Biomedical Engineering from Johns Hopkins University. He has been named a Highly Cited Researcher by Clarivate and was elected to the American Institute for Medical and Biological Engineering in 2022 and the National Academy of Inventors in 2023.

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
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
Dr. Blythe Sather, a leader at Tune Therapeutics, has focused her career on genetic and epigenetic modifications for disease treatment. She earned her Ph.D. in Immunology from the University of Washington, studying regulatory T cells, and conducted postdoctoral research on lentiviral gene therapies at Seattle Childrens Research Institute. At Lyell Immunopharma, Dr. Sather developed CAR and TCR products for solid tumors as Senior Director. At Juno Therapeutics, she helped build the CAR T cell platform and led the multiple myeloma CAR T cell program, collaborating with Editas Medicine on CRISPR-mediated gene editing. Now at Tune, she develops technologies to regulate gene expression, aiming to create therapies for viruses, cancer, and rare genetic disorders.

Biography
Fyodor Urnov co-developed the toolbox of human genome and epigenome editing, co-named genome editing, and was on the team that advanced all of its first-in-human applications to the clinic. He also led the effort that identified the genome editing target for an approved medicine to treat sickle cell disease and beta-thalassemia. A major goal for the field of genome editing and a key focus of Fyodor's work is expanding access to CRISPR therapies for genetic disease. As part of that effort Fyodor directs the Danaher-IGI Beacon for CRISPR Cures - a first-in-class academia-industry partnership developing and advancing to the clinic scalable CRISPR-based approaches to treat diseases of the immune system.

Biography
François Vigneault is a serial biotechnologist, co-founder of Pi.Bio, ShapeTX, and AbVitro. Pi.Bio develops next-generation human-derived small proteomic-ligand therapies for immuno-inflammatory diseases, oncology, and gene delivery. ShapeTX is an AI-driven RNA editing gene therapy company leveraging ADAR technology. AbVitro, acquired by Juno Therapeutics (later Celgene and Bristol Myers Squibb), pioneered high-throughput immune single-cell sequencing for antibody and TCR discovery. François completed his postdoctoral work with George Church at Harvard Medical School and served as a Maritime Warfare Officer in the Canadian Navy.

Biography
Ben Dewees is a leader at Kyverna Therapeutics, specializing in the development of cell therapies for autoimmune diseases. Ben is a dedicated Regulatory professional with over 25 years of industry experience and over 20 of those years in Regulatory Affairs. He brings extensive experience in developing novel drug development pathways for patients with unmet medical needs. Ben is dedicated to enhancing patient care through innovative therapeutic solutions.

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
Prior to MacroGenics, Dr. Eck served as Chief Medical Officer of Immatics US, and as CEO of Aravive Biologics. Prior to these roles, Dr. Eck was Vice President and Global Head of Oncology Medical Sciences at Astellas Pharma, managing a portfolio of assets which included enzalutamide (Xtandi), erlotinib (Tarceva) and gilteritinib (Xospata). Dr. Eck has also held leadership positions in drug development as Vice President of Translational Medicine and Pharmacogenomics at Eli Lilly and as V.P Translational Medicine. . Dr. Eck currently serves on the Board of Directors for Circulogene, 1cBio , the Personalized Medicine Coalition and the Central Pennsylvania Clinic.

Biography
Tim joined ARM as CEO in 2022 with more than 20 years of experience in the biotechnology industry. A company-builder with a focus on corporate culture, he has helped early-stage organizations create internal cohesion and advance strategic initiatives. As chief executive, Tim divides his time between managing ARM and its global staff of nearly 30 employees and serving as an external advocate on behalf of ARMs membership and the broader cell and gene therapy sector. Tim received a B.A. in history and philosophy from Boston College and a J.D. from the Columbus School of Law at the Catholic University of America.

Biography
Vaishnav is the Founder CEO of Sequome, a company that decodes designs sequences to develop Sequence Medicines, working with Fortune 500 Pharmaceutical Technology companies. Prior to Sequome, Eeshit received his PhD from MIT as a Presidential Fellow, where he was advised by Prof. Aviv Regev (currently Head of Roche gRED), and collaborated with leading institutions in academia (Broad Institute) and industry (Google). Eeshit led the development of Gene Genie, the sequence-function AI for decoding and designing sequences, published on the cover of Nature.

Biography
Paul Kruszka is a board-certified clinical geneticist who uses genomic and precision medicine to enhance the delivery of health care to individuals with rare diseases. Prior to working at GeneDx, Dr. Kruszka spent a decade at the National Institutes of Health conducting genomic research and taking care of individuals with rare genetic diseases. Dr. Kruszka is credited with the clinical and molecular delineation of multiple novel genetic conditions. Dr. Kruszka attended medical school at the University of Michigan and completed a family medicine residency at the University of Virginia and a clinical genetics residency at the National Human Genome Research Institute at the National Institutes of Health.

Biography
Tay Salimullah is a Venture Partner at 4BIO Capital and Managing General Partner of Renovamen Advisory LLC, offering strategic support to early-stage biotech, venture capital, and private equity firms. With over 20 years of leadership in rare diseases and MedTech, Tay has a proven record of scaling high-value technologies, including cell and gene therapies. At Novartis Gene Therapies, he transformed AveXis into a multibillion-dollar global business, pioneering Zolgensma's commercial model, securing approvals in 55+ countries, treating 4,000+ patients, and generating $5B+ in revenue. Earlier roles include a decade at Pfizer and strategic positions in investment and healthcare. Tays expertise spans drug development, market access, pricing, and operational excellence. He serves on the board of Trojenix and is a Fellow at the Alliance for Regenerative Medicine, advancing Cell and Gene Therapy Ethics and Society. Tay is passionate about driving innovation in regenerative medicine.

Biography
James M. Wilson, MD, PhD, is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he has led an effort to develop the field of gene therapy. His research career spanning over 40 years has focused on rare diseases and ways to treat them by gene therapy. Dr. Wilson has published over 600 papers and is named on more than 1200 patents worldwide. The Wilson lab identified a new type of vector based on novel isolates of adeno-associated viruses which have become best in class for gene therapy. More recently Dr. Wilsons laboratory has focused on improved vectors for gene therapy and clinical applications of genome editing and mRNA therapy.

Biography
Dr. Courtney Silverthorn is a distinguished professional with a robust background in science policy and technology transfer. Currently, she oversees business development opportunities for the Foundation for the NIH. Dr. Silverthorn has also played a pivotal role in the Bespoke Gene Therapy Consortium, a collaborative project focused on accelerating the development of gene therapies for diseases that currently have no commercial interest. Her contributions to this consortium underscore her commitment to advancing treatments for rare diseases.In addition to her work at FNIH and prior roles at several federal agencies, Dr. Silverthorn has shared her insights on platforms such as the Rare Disease Advisor Podcast. In an interview with Larry Luxner, she discussed the consortium's impact on rare diseases, highlighting her expertise in scientific partnerships and innovation. Dr. Silverthorns dedication to enhancing healthcare through scientific research and collaboration continues to make her a significant figure in the field.

Biography
Dr. Kira Radinsky is the CEO and CTO of Diagnostic Robotics, leveraging advanced AI to revolutionize healthcare by making it more accessible and affordable. She previously founded SalesPredict, acquired by eBay in 2016, where she served as Chief Scientist (IL). Dr. Radinsky gained global recognition for her predictive algorithms identifying early warning signs of epidemics and political unrest, earning her a spot in MIT Technology Reviews 35 Innovators Under 35 and Forbes 30 Under 30 in Enterprise Tech. A frequent speaker at global conferences like TEDx and Wired, she also publishes in HBR. In 2022, she co-founded Mana.bio and serves on the boards of Esh Digital Bank and Nebius (NASDAQ: NBIS). She previously held board roles with the Israel Securities Authority and HSBCs tech board. Dr. Radinsky is also a visiting professor at the Technion, focusing on predictive data mining in medicine.

Biography
Dr. Robert Califf, a distinguished cardiologist and clinical trial expert, has significantly contributed to the field of precision medicine. Serving twice as the Commissioner of the U.S. Food and Drug Administration (FDA), Dr. Califf has played a pivotal role in advancing regulatory science and fostering the development of innovative medical products. His tenures at the FDA were marked by efforts to modernize clinical trials, enhance drug safety, and accelerate the approval of life-saving therapies. Additionally, Dr. Califf's leadership in the NIH's Precision Medicine Initiative has helped lay the groundwork for personalized treatment strategies that are transforming patient care.

Biography
Broad experience working in Pharma and Biotech both as a team lead and in Strategy and BD. Amir previously lead discovery teams at two Flagship Pioneering companies Repertoire Immune Medicine and Sail Therapeutics. He has also held positions at Ceptur Therapeutics leading their corporate development efforts as well as working to build Ginkgo Bioworks first product in Antibodies.