Lindsey A. George, MD
Areas of expertise: Hemostasis and thrombosis disorders, Hemophilia gene therapy, Hemophilia
About Lindsey A. George, MD
Dr. George is an Assistant Professor of Pediatrics at University of Pennsylvania and Director of Clinical In Vivo Gene Therapy at Children’s Hospital of Philadelphia. Her laboratory focuses on the development of gene-based therapies for coagulation disorders and on elucidating the molecular mechanisms underlying blood coagulation, with a particular emphasis on factor VIII biology and hemophilia A. Her studies on the regulation of factor VIII have led to the development of a next-generation gene therapy approach for hemophilia A that is currently being evaluated in a phase 2b clinical trial. Her laboratory also investigates the immunologic and molecular basis of unexplained clinical observations that have emerged from adeno-associated virus (AAV) gene therapy trials, with the goal of improving their safety and efficacy. In parallel with her laboratory research, Dr. George founded and directs the Clinical In Vivo Gene Therapy group at the Children’s Hospital of Philadelphia, which provides regulatory support for investigator-initiated studies, operational efforts for a large portfolio of interventional in vivo gene addition and editing trials and clinical infrastructure to safely and efficiently implement commercial in vivo gene therapies into clinical practice. She is a member of the National Academy of Medicine Emerging Leaders in Health and Medicine and past member of the Board of Directors of the American Society of Gene and Cell Therapy.
Titles
Director, Clinical In Vivo Gene Therapy
Tenure Assistant Professor of Pediatrics, The Perelman School of Medicine at the University of Pennsylvania
Certifications
Pediatrics – American Board of Pediatrics
Awards and Honors
2026-2029, Victor J. Dzau Emerging Leaders in Health and Medicine Scholar, National Academy of Medicine
2022, Outstanding New Investigator Award, American Society of Gene and Cell Therapy
2022, American Society of Clinical Investigation, Young Physician-Scientist Award
2022, Martin Villar Basic Science Award, Milan, Italy
2019, Scholar Award of the National Blood Foundation, Washington, D.C.
2019, National Top 10 Clinical/Translational Research Award, Clinical Research Forum, Washington, D.C.
2018, Professor Heimburger Award in Hemostasis, Marburg, Germany
2017, Eberhard Mammen Young Investigator Award for Excellence in Thrombosis and Hemostasis
2017, Next Gen Innovator, HemOnc Today
2015, Young Investigator Award, International Society of Thrombosis and Hemostasis, Toronto, CA
2015, Best Oral Abstract Presentation, Philadelphia Workshop in Thrombosis and Hemostasis, Philadelphia, PA
2015, Young Investigator Award, International Society of Thrombosis and Hemostasis, Toronto, CA
2014, Outstanding Abstract, Thrombosis and Hemostasis Society of North America, Chicago, IL
2014, Future Leaders in Hematology, Celgene Biopharmaceuticals, Anaheim, CA
2013, Abstract Achievement Award, American Society of Hematology, New Orleans, LA
2012, Abstract Travel Award, European Hematology Association, Amsterdam, NE
2011, Distinguished Clinician in Training, New York-Presbyterian Hospital, New York, NY
Leadership and Memberships
Memberships in Professional Organizations
International
2013-present, International Society of Thrombosis and Haemostasis
National
2022-present, American Society of Clinical Investigation
2015-present, American Society of Gene and Cell Therapy
2010-present, American Society of Hematology
Editorial and Academic Positions
Editorial Positions
Ad hoc reviewer
2021-present, The New England Journal of Medicine
2019-present, Science Translational Medicine
2019-present, Journal of Thrombosis and Hemostasis
2019-present, Molecular Therapy
2019-present, Hematology (American Society of Hematology Education Program)
2019-present, Blood
2018-present, Human Gene Therapy
2018-present, Nature Reviews Drug Discovery
2018-present, Human Gene Therapy
2017-present, British Journal of Haematology
2016-present, Pediatric Blood and Cancer
2015-present, Haematologica
2015-present, Thrombosis Research
2015-present, Haemophilia
Education & training
Graduate Degree
MS in Translational Research - The Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA
Medical Degree
MD - State University of New York School of Medicine at Buffalo, Buffalo, NY
Residency
Pediatrics - Weill Cornell Medical College New York, NY (Chief Resident)
Fellowship
Pediatric Hematology/Oncology - The Children’s Hospital of Philadelphia, Philadelphia, PA
Publications
Publications
2026
Keselman DJ, Small JC, Seneviratne T, McCague S, Kaschak TL, Yum SW, O'Brien A, Brandsema JF, Diamond T, Loomes KM, Lin KY, Wittlieb-Weber CA, Mayer OH, Daniel SG, Waldman AT, Samelson-Jones BJ, Matesanz SE, George LA: Real-World Outcomes of Delandistrogene Moxeparvovec Gene Therapy: Motor Outcomes and Emerging Safety Concerns. Mol Ther 34(1): 123-130, Jan 2026 Notes: published with an accompanying editorial.
Samelson-Jones BJ, Rasko JEJ, Ducore JM, McGuinn CE, George LA, von Mackensen S, Borgonuovo G, Agathon D, Smith L, Wilcox LJ, Biondo F, Plonski F: Safety, efficacy, and patient-reported outcomes 6 years after fidanacogene elaparvovec in adults with hemophilia B. Blood Adv 10(10): 3517-3526, May 2026.
Ahrens-Nicklas RC, Kotch C, Roche AM, Everett JK, Reddy S, Santi M, Madsen PJ, Martos-Rus C, Yrigollen CM, Small JC, McCague S, Davidson BL, Samelson-Jones BJ, Surrey LF, Li M, Ficicioglu C, Wang R, Bushman FD* and George LA*: Neuroepithelial Tumor Associated with AAV Integration After Intracisternal Magna Vector Delivery. New England Journal of Medicine 394(12): 2126-2133, Jun 2026.
Morris JJ, Davidson RJ, Watson CT, George LA: Factor VIII Aurora: A Naturally Occurring Gain of Function FVIII Variant with Enhanced FIXa Affinity. Blood (in press), 2026.
Samelson-Jones BJ, Martos-Rus C, Yrigollen CM, Tecedor L, Small JC, Matesanz SE, Watson CT, Vanden Heuvel AR, Carrig S, Yum SW, Brandsema JF, Lin KY, Wittlieb-Weber C, Viaene AN, Russo PP, Partington S, Huynh K, Seneviratne T, Juarez Rojas S, Doshi BS, Shieh PB, Flanigan KM, Frair EM, Nicolau S, Lawlor MW, Crudele JM, Davidson BL, George LA: Cardiac Toxicity and Molecular Efficacy after Delandistrogene Moxeparvovec Gene Therapy for Duchenne Muscular Dystrophy. Accepted (under embargo), 2026.
Sternberg AR, Watson CT, Davidson RJ, Uguen M, Kiialainen A and George LA: One-stage Assay Factor VIII Activity Reflects AAV-Derived Factor VIII Enhanced Thrombin Activation and Predicts Phenotype. Blood (in press), 2026.
2025
Doshi BS, Markmann CA, Novak N, Rojas SJ, Davidson R, Chau JQ, Wang W, Carrig S, Rus CM, Samelson-Jones BJ, Small JC, Bhoj VG, George LA: Use of CD19-targeted Immune Modulation to Eradicate AAV Neutralizing Antibodies. Mol Ther 8(1): 177-7, Mar 2025 Notes: published with an accompanying editorial.
Rasko JEH*, Samelson-Jones BJ*, George LA, Giermasz A, Ducore JM, Teitel JM, McGuinn CE, High KA, de Jong YP, Chhabra A, O'Brien A, Smith LM, Winburn I, Rupon J: Fidanacogene Elaparvovec for Hemophilia B: A Multi-Year Follow-Up Study. N Engl J Med 392(15): 1508-1517, Apr 2025.
Musunuru K, Grandinette SA, Wang X, Hudson TR, Briseno K, Berry AM, Hacker JL, Hsu A, Silverstein RA, Hille LT, Ogul AN, Robinson-Garvin NA, Small JC, McCague S, Burke SM, Wright CM, Bick S, Indurthi V, Sharma S, Jepperson M, Vakulskas CA, Collingwood M, Keogh K, Jacobi A, Sturgeon M, Brommel C, Schmaljohn E, Kurgan G, Osborne T, Zhang H, Kinney K, Rettig G, Barbosa CJ, Semple SC, Tam YK, Lutz C, George LA, Kleinstiver BP, Liu DR, Ng K, Kassim SH, Giannikopoulos P, Alameh MG, Urnov FD, Ahrens-Nicklas RC: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. N Engl J Med 392(22): 2235-2243, Jun 2025.
Morris JJ, Parsons NA, Wilhelm AR, Davidson RJ, Olenick LK, Watson CT, Vanden Heuvel A, George LA: Factor IXa and Factor X Influence Factor VIIIa Stability and Inactivation Mechanisms In Vitro and In Vivo. Blood 146(18): 2259-2271, Sep 2025 Notes: published with an accompanying editorial.
2024
Sternberg AR, Martos-Rus C, Davidson RJ, Liu X, George LA: Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice. Nat Commun 15(1): 7193, Aug 2024.
Lee K, Chau JQ, Suber YB, Sternberg AR, Pishko A, George LA, Bhoj V, Doshi BS, Samelson-Jones BJ: Enhanced Procoagulant Activity of Select Hemophilia B Causing Factor IX Variants with Emicizumab. Blood 144(11): 1230-1235, Sep 2024.
2023
Kaczmarek R, Piñeros AR, Patterson PE, Bertolini TB, Perrin GQ, Sherman A, Born J, Arisa S, Arvin MC, Kamocka MM, Martinez MM, Dunn KW, Quinn SM, Morris JJ, Wilhelm AR, Kaisho T, Munoz-Melero M, Biswas M, Kaplan MH, Linnemann AK, George LA, Camire RM, Herzog RW: Factor VIII trafficking to CD4+ T cells shapes its immunogenicity and requires several types of antigen-presenting cells. Blood 142(3): 290-305, Jul 2023.
Cao W, Trask AR, Bignotti AI, George LA, Doshi BS, Sabatino DE, Yada N, Zheng L, Camire RM, Zheng XL: Coagulation factor VIII regulates von Willebrand factor homeostasis in vivo. J Thromb Haemost 21(12): 3477-3489, Dec 2023.
2022
Xue F, Wang P, Yuan Z, Shi C, Fang Y, Liu W, Wang Y, Xiao X, Yang R, George LA, Zhang L: Total Knee Arthroplasty After Gene Therapy of Hemophilia B. N Engl J Med 387(12): 1622-1624, Oct 2022.
2021
Robinson MM, George LA, Carr ME, Samelson-Jones BJ, Arruda VR, Murphy JE, Rybin D, Jeremy Rupon J, High KA, Tiefenbacher S: Factor IX Assay Discrepancies in the Setting of Liver Gene Therapy Using a Hyperfunctional
Variant Factor IX-Padua. J Thromb Haemost 19(5): 1212-1218, May 2021.
Wilhelm AR, Parson NA, Samelson-Jones BJ, Davidson RJ, Esmon CT, Camire RM, George LA: Activated Protein C has a Regulatory Role in Factor VIII Function. Blood 137(18): doi: 10.1182/blood.2020007562, May 2021.
George LA: Hemophilia Gene Therapy: Ushering in a New Treatment Paradigm? American Society of Hematology Education Program Dec 2021.
George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA: Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med 385(21): 1961-1973, Nov 2021 Notes: Corresponding author.
2020
Doshi BS, Raffini LJ, George LA: Combined anti-CD20 and mTOR inhibition with factor VIII for immune tolerance induction in hemophilia A patients with refractory inhibitors. J Thromb Haemost 18(4): 848-852, Mar 2020.
Konkle BA, Coffin D, Pierce GF, Clark C, George LA, Iorio A, Mahlangu J, Naccache M, O'Mahony B, Peyvandi F, Pipe S, Quartel A, Sawyer EK, Skinner MW, Tortella B, Watson C, Winburn I, Members of the WFH Gene Therapy Registry Steering Committee. : World Federation of Hemophilia Gene Therapy Registry. Haemophilia 26: 563-564, May 2020.
George LA, Ragni MV, Rasko JEJ, Raffini LJ, Samelson-Jones BS, Hazbon M, Runowski AR, Wellman JA, Wachtel K, Chen Y, Anguela XM, Kuranda K, Mingozzi F, High KA: Long-Term Follow-Up of the First In Human Intravascular Delivery of AAV For Gene Transfer: AAV2 hFIX16 For Severe Hemophilia B. Mol Ther 28(9): 2073-2082, June 2020.
Konkle BA, Coffin D, Pierce GF, Clark C, George L, Iorio A, Mahlangu J, Naccache M, O'Mahony B, Peyvandi F, Pipe S, Quartel A, Sawyer EK, Skinner MW, Tortella B, Watson C, Winburn I: World Federation of Hemophilia Gene Therapy Registry. Haemophilia 26(4): 563-564, Jul 2020.
Konkle B, Pierce G, Coffin D, Naccache M, Clark RC, George L, Iorio A, O'Mahony
B, Pipe S, Skinner M, Watson C, Peyvandi F, Mahlangu J: Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH. J Thromb Haemost 18(11): 3074- 3077, Nov 2020.
2019
Samelson-Jones BJ, Finn JD, George LA, Camire RM, Arruda VR1: Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity. JCI Insight 4(14), June 2019.
Ragni MV, George LA: Scientific Priorities and Innnovative Implementation Strategies for FVIII Inhibitor Clinical Trials: Report from Working Group 1 of the NHLBI State of the Science Worskhop on FVIII Inhibitors. Haemophilia 25(4): 581-589, July 2019.
2018
Bergmeier W, Antoniak S, Conway EM, Denis CV, George LA, Isermann B, Key NS, Krishnaswamy S, Lam WA, Lillicrap D, Liu J, Looney MR, L pez JA, Maas C, Peyvandi F, Ruf W, Sood AK, Versteeg HH, Wolberg AS, Wong PC, Wood JP, Weiler H.: Advances in Clinical and Basic Science of Coagulation: Illustrated abstracts of the 9th Chapel Hill Symposium on Hemostasis. Res Pract Thromb Haemost 2(3): 407-428, Apr 2018.
2017
George LA, Sullivan SK, Giermasz G, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majuddar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hu Di,Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA: Hemophilia B Gene Therapy with a High Specific Activity Factor IX Variant N Engl J Med 377(2): 2215-2227, Dec 2017 Notes: Published with accompanying editorial. PMCID: 29211678
2016
Nguyen GN, George LA, Siner JI, Davidson RJ, Zander CB, Zheng XL, Arruda VR,Camire RM, Sabatino DE: Novel human factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia. J Thromb Haemost 14(10): 1-12, Oct 2016.
2015
George LA, Thalji NK, Raffini LJ, Gimotty PA, Camire RM: A new bypass strategy: zymogen-like Factor XaI16L corrects Hemophilia A whole blood thromboelastometry findings. J Thromb Haemost 13(9): 1694-8, Sep 2015.
2014
Greene LA, Law C, Jung M, Walton S, Ignjatovic V, Monagle P, Raffini LJ: Lack of anti-factor Xa assay standardization results in significant low molecular weight heparin (enoxaparin) dose variation in neonates and children. J Thromb Haemost 12(9): 1554-7, Sep 2014.
Greene LA, Chen S, Seery C, Imahiyerobo AM, Bussel JB: Beyond the platelet count: immature platelet fraction and thromboelastometry correlate with bleeding in patients with immune thrombocytopenia. Br J Haematol 166(4): 592-600, Aug 2014.
2013
Greene LA, Goldenberg NA, Simpson ML, Villalobos-Menuey E, Bombardier C, Acharya SS, Santiago-Borrero PJ, Cambara A, DiMichele DM: Use of global assays to understand clinical phenotype in congenital factor VII deficiency. Haemophilia 19(5): 765-72, Sep 2013.
2004
Cordeiro JM, Greene L, Heilmann C, Antzelevitch D, Antzelevitch C: Transmural heterogeneity of calcium activity and mechanical function in the canine left ventricle. Am J Physiol Heart Circ Physiol 286(4): H1471-9, Apr 2004.