Trial reportThe New England journal of medicine2017
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
Registry-linked trialOpen access · bronzeAbstract readClinical Trial, Phase IClinical Trial, Phase IIMulticenter Study
In one paragraph
Trial report in The New England journal of medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02484092 (Gene Therapy, Open-label, Dose-escalation Study of PF-06838435), which is not on this map. Cited by 441 papers, 4 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
441citing papers in PubMed, 4 pooled it
54.4field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Gene Therapy, Open-label, Dose-escalation Study of PF-06838435 (SPK-9001) [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B
Who cites it
441 citing papers in PubMed, 4 syntheses or guidelines pooled it, 738 citations in OpenAlex.
- Incidence, timing, and clinical significance of adverse immune events after gene replacement therapy: A systematic review and meta-analysis.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Pooled it
- Adeno-associated virus-based gene therapy for hemophilia A and B: a systematic review and meta-analysis.Blood advances · 2024Pooled it
- A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Pooled it
- Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies.Gene therapy · 2023Pooled it
- Prospective collection of bleeding rate and factor usage in participants with hemophilia A and B in a noninvestigational product study.Research and practice in thrombosis and haemostasis · 2026Trial
- Safety, efficacy, and patient-reported outcomes 6 years after fidanacogene elaparvovec in adults with hemophilia B.Blood advances · 2026Trial
- Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials.Nature medicine · 2026Trial
- Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.Blood advances · 2025Trial
- Field Study and Correlative Studies of Factor IX Variant FIX-R338L in Participants Treated with Fidanacogene Elaparvovec.Thrombosis and haemostasis · 2024Trial
- Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results.Nature medicine · 2024Trial
- Trial
- Clinical immunogenicity in rAAV gene therapy: Insights and implications.Molecular therapy. Advances · 2026Review
- Sequence-based artificial intelligence-guided inhibitory peptides to counteract AAV neutralizing antibodies in gene therapy.Molecular therapy. Advances · 2026Article
- Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial.Nature medicine · 2026Article
- Operationalizing Precision Medicine in Drug Development: Predictive Biomarkers, Companion Diagnostics, and Regulatory Pathways.Clinical pharmacology and therapeutics · 2026Review
- Rapamycin nanoparticles mitigate anti-AAV antibody formation in a mouse model of ornithine transcarbamylase deficiency.Molecular therapy. Advances · 2026Article
- Increased gene therapy efficacy through the use of extended half-life clotting factors.Blood advances · 2026Article
- A humanized mouse model of APOB deficiency recapitulates hypobetalipoproteinemia.Journal of lipid research · 2026Article
- CAR T cell therapy beyond cancer: current status, challenges and future prospects.Signal transduction and targeted therapy · 2026Review
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
381 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
28 authors at 8 institutions in 3 countries.
Lindsey A GeorgeFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Spencer K SullivanFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Adam GiermaszFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
John E J RaskoFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Benjamin J Samelson-JonesFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Jonathan DucoreFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Adam CukerFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Lisa M SullivanFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Suvankar MajumdarFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Jerome TeitelFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Catherine E McGuinnFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Margaret V RagniFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Alvin Y LukFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Daniel HuiFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
J Fraser WrightFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Yifeng ChenFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Yun LiuFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Katie WachtelFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Angela WintersFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Stefan TiefenbacherFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Valder R ArrudaFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Johannes C M van der LooFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Olga ZelenaiaFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Daniel TakefmanFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Marcus E CarrFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Linda B CoutoFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Xavier M AnguelaFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Katherine A HighFrom the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).
Anna Needs Neuroblastoma Answers · USUniversity of Pennsylvania · USSpark Therapeutics (United States) · USChildren's Hospital of Philadelphia · USCornell University · USUniversity of California Davis Medical Center · USUniversity of Pittsburgh · USUniversity of Toronto · CA
Funding
Safety &Efficacy of Intravas. Del. of AAV-F.IX to Skeletal MuscleP01HL064190 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI PONCZ, MORTIMER · 2000 to 2015
$27.8MHEMATOLOGY CLINICAL RESEARCH TRAINING PROGRAMT32HL007439 · NHLBI · UNIVERSITY OF PENNSYLVANIA · PI LAWRENCE F BRASS, PETER S KLEIN · 1985 to 2026
$17.1MRED CELL HEMOGLOBIN RESEARCH TRAINING PROGRAMT32HL007150 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI STELLA T CHOU, Mortimer Poncz · 1985 to 2026
$7.4MNHLBI NIH HHS P01 HL064190NHLBI NIH HHS T32 HL007150NHLBI NIH HHS T32 HL007439
Abstract
backgroundThe prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in the treatment of hemophilia.
methodsWe infused a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-specific promoter and factor IX Padua (factor IX-R338L) transgene at a dose of 5×10
resultsNo serious adverse events occurred during or after vector infusion. Vector-derived factor IX coagulant activity was sustained in all the participants, with a mean (±SD) steady-state factor IX coagulant activity of 33.7±18.5% (range, 14 to 81). On cumulative follow-up of 492 weeks among all the participants (range of follow-up in individual participants, 28 to 78 weeks), the annualized bleeding rate was significantly reduced (mean rate, 11.1 events per year [range, 0 to 48] before vector administration vs. 0.4 events per year [range, 0 to 4] after administration; P=0.02), as was factor use (mean dose, 2908 IU per kilogram [range, 0 to 8090] before vector administration vs. 49.3 IU per kilogram [range, 0 to 376] after administration; P=0.004). A total of 8 of 10 participants did not use factor, and 9 of 10 did not have bleeds after vector administration. An asymptomatic increase in liver-enzyme levels developed in 2 participants and resolved with short-term prednisone treatment. One participant, who had substantial, advanced arthropathy at baseline, administered factor for bleeding but overall used 91% less factor than before vector infusion.
conclusionsWe found sustained therapeutic expression of factor IX coagulant activity after gene transfer in 10 participants with hemophilia who received the same vector dose. Transgene-derived factor IX coagulant activity enabled the termination of baseline prophylaxis and the near elimination of bleeding and factor use. (Funded by Spark Therapeutics and Pfizer; ClinicalTrials.gov number, NCT02484092 .).
Indexed as
Genetic VectorsTransgenesAdolescentAdultDependovirusFactor IXGenetic TherapyHemophilia BHemorrhageHumansMaleMiddle AgedYoung AdultFactor IX
Identifiers
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Registered trials
NCT02484092ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.