Speakers-2026

Lindsey George, M.D.

Dr. George is an Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania and Director of Clinical In Vivo Gene Therapy at the Children’s Hospital of Philadelphia. Her laboratory studies the molecular basis of coagulation with a focus on mechanisms that regulate factor VIII/VIIIa cofactor function and developing gene-based therapies for hemophilia A. Most recently her laboratory has described mechanisms of factor VIIIa and exploited this work to develop next generation approaches for hemophilia A gene therapy. This work is now in a phase 2b gene therapy trial for hemophilia A, sponsored by Roche and Genentech. Dr. George previously led early-phase, adeno-associated virus (AAV) mediated gene transfer trials for hemophilia A and B. Four years ago, she founded the Clinical In Vivo Gene Therapy group at the Children’s Hospital of Philadelphia that supports >20 ongoing interventional clinical AAV trials across the institution as well as investigator initiated personalized gene editing strategies. This group additionally implements commercial in vivo gene therapies. Dr. George has been member of the Board of Directors of the American Society of Gene and Cell Therapy and participates in multiple national and international professional societies for hemostasis and gene therapy research.

Terence Flotte, M.D.

Dr. Flotte is the Provost/Dean and Elisabeth Chair for the School of Medicine at UMass Chan Medical School, with faculty appointments in the Department of Genetic and Cellular Medicine and Pediatrics. Dr. Flotte is a pediatric pulmonologist and gene therapy scientist. He has been continuously NIH funded since 1993 for basic and early phase translational research in gene therapy for genetic diseases, including cystic fibrosis, alpha-1 antitrypsin deficiency, and disorders of fatty acid oxidation. Flotte has been the principal investigator of multiple clinical gene therapy trials, including the first-ever human use of AAV vectors, a trial in cystic fibrosis patients that began in 1995, and recent trials in alpha-1 antitrypsin deficiency and Tay-Sachs disease. He serves as the Editor in Chief of Human Gene Therapy, longest running journal in the field of gene therapy. He is currently the President of the American Society of Gene & Cell Therapy (ASGCT), the leading gene and cell therapy academic society in the world.

Laura Sepp-Lorenzino, Ph.D.

Laura Sepp-Lorenzino, Ph.D. is a biotech executive with expertise in drug development, particularly in nucleic acid therapies. Laura is Principal of GNMmeds LLC, consulting on genomic medicine research and development. She currently serves on the board of directors of Taysha Gene Therapies, AskBio Biopharmaceutical, URSA Medicines, the Alliance for Regenerative Medicine, and the American Association of Cell and Gene Therapy. She is the President Elect of the Oligonucleotide Therapeutics Society (2028-2029 term) and serves in their board. Additionally, she chairs the scientific advisory board of Travin Bio and is an SAB member for Thermo Fisher Scientific, Inverna Therapeutics, U.K. Nucleic Acid Therapy Accelerator, and Arsenal Capital Partners. Previously, Laura was Chief Scientific Officer at Intellia Therapeutics (2019–2025), where she played a pivotal role in shaping the company’s strategic direction, fostering a robust and diversified CRISPR platform, and driving groundbreaking advances in in vivo genome editing. Under her leadership, Intellia demonstrated the first successful in vivo genome editing in humans and advanced two candidates into pivotal trials. Before Intellia, she served as VP, Head of Nucleic Acid Therapies at Vertex Pharmaceuticals, as well as VP and entrepreneur-in-residence at Alnylam Pharmaceuticals, and Executive Director of RNA Therapeutics at Merck & Co. Laura’s expertise extends beyond nucleic acid therapies, encompassing oncology drug discovery and development, honed through leadership roles at Merck West Point and Memorial Sloan-Kettering Cancer Center. She holds a professional degree in Biochemistry from the University of Buenos Aires, Argentina, and earned her M.S. and Ph.D. in Biochemistry from New York University. She also contributes to the scientific community as an editorial board member of Nucleic Acid Therapeutics and Molecular Therapy Nucleic Acids.

Peter Marks, Ph.D., M.D.

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 Hematology/Medical 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. In 2012, he joined the Food and Drug Administration and served as Director of the Center for Biologics Evaluation and Research from 2016 to 2025. He is currently Senior Vice President for Molecule Development and Head of Infectious Disease at Eli Lilly and Company, has published extensively, and is a member of the National Academy of Medicine.

Sheila Mikhail, JD, M.B.A

Sheila Mikhail has almost 25 years of biopharmaceutical leadership experience. She is a serial entrepreneur, an advocate for equitable access to healthcare, and a philanthropist. Sheila co-founded and ran several companies, including AskBio (sold to Bayer in 2020 in a $4B transaction) and Bamboo Therapeutics, Inc. (sold to Pfizer in 2016 in a deal valued at $827M). She was also a founder and part of the management team of Chatham Therapeutics, LLC (sold to Baxter in 2014) and Viralgen VectorCore S.A., a gene therapy CDMO business (sold to Bayer in 2021). In further pursuit of expanded global access to healthcare, Ms. Mikhail recently co-founded Jurata Thin Film, Inc., to eliminate the need for cold chain storage requirements. Sheila is currently the CEO and Co-Founder of M34, which develops molecular therapy for neurodegenerative diseases. Committed to ensuring that gene therapy treatments are developed for patients suffering from ultra-rare diseases, in 2017, Ms. Mikhail co-founded Columbus Children’s Foundation (CCF). To date, CCF has supported the treatment of over 50 children suffering from amino acid decarboxylase deficiency (AADC), a devastating neurological disease with no viable treatment options. Following her breast cancer diagnosis in 2022, Ms. Mikhail formed BC-Ruckus to advocate for expanded access to supplemental screening for women with dense breasts so everyone’s breast cancer can be found early. Ms. Mikhail was recognized as the Ernst & Young 2021 Overall National US Entrepreneur of the Year.