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Journal Articles

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

Blood (2018) 132 (Supplement 1): 487.

https://doi.org/10.1182/blood-2018-99-115495

Published: 2018

Journal Articles

Activity of a FIX-Padua Transgene Product in Commonly Used FIX:C One-Stage and Chromogenic Assay Systems Following PF-06838435 (SPK-9001) Gene Delivery

Mary Robinson, PhD, Lindsey A. George, MD, Benjamin J. Samelson-Jones, MD PhD, Valder R Arruda, MD PhD, Katherine A. High, MD, Marcus E Carr, MD PhD, Stefan Tiefenbacher, PhD

Blood (2018) 132 (Supplement 1): 2198.

https://doi.org/10.1182/blood-2018-99-119616

Published: 2018

Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence

Blood (2017) 130 (Supplement 1): 601.

https://doi.org/10.1182/blood.V130.Suppl_1.601.601

Published: 2017

Journal Articles

Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

Lindsey A. George, MD, Margaret V. Ragni, MD MPH, Ben J. Samelson-Jones, MD PhD, Adam Cuker, MD MS, Alexa R. Runoski, MS, Grace Cole, Fraser Wright, PhD, Yifeng Chen, MD, Daniel J Hui, PhD BS, Katie Wachtel, MS, Daniel Takefman, Linda B. Couto, PhD, Kathleen Z. Reape, Marcus E. Carr, MD PhD, Xavier M. Anguela, PhD, Katherine A. High, MD

Blood (2017) 130 (Supplement 1): 604.

https://doi.org/10.1182/blood.V130.Suppl_1.604.604

Published: 2017

Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

Daniel J Hui, PhD BS, Yun Liu, PhD, Ruchi Patel, Yifeng Chen, MD, Lindsey A. George, MD, Spencer K. Sullivan, MD, Adam Giermasz, MD, John Rasko, MBBS, PhD, Ben J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD, Sue Dasen, MS, Marcus E. Carr, MD PhD, Xavier M. Anguela, PhD, Katherine A. High, MD

Blood (2017) 130 (Supplement 1): 2056.

https://doi.org/10.1182/blood.V130.Suppl_1.2056.2056

Published: 2017

Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression

Blood (2016) 128 (22): 3.

https://doi.org/10.1182/blood.V128.22.3.3

Published: 2016

Journal Articles

Lack of Seasonal Variation in Bleeding and Patient-Assessed Pain Patterns in Patients with Hemophilia B Receiving On-Demand Therapy

Frank E. Shafer, MD, Lynne Smith, MBA, Nicholas Vendetti, MS, Pablo Rendo, MD, Marcus E. Carr, MD

Blood (2012) 120 (21): 1136.

https://doi.org/10.1182/blood.V120.21.1136.1136

Published: 2012

Journal Articles

Differential Dose Dependent Effects of Recombinant FVIIa and Enhanced Activity Analogue (NN1731) on Thrombin Generation, Platelet Function and Clot Structure in Blood from Normal Volunteers and Patients with Factor VIII Deficiency with or without Factor VIII Inhibitor Antibodies.

Donald F. Brophy, PharmD, Erika J. Martin, MT(ASCP), Melinda Nolte, R.N., Jan G. Kuhn, R.N., MPH, Sashidhar N. Reddy, MD, Mirella Ezban, MD, Marcus E. Carr, MD, PhD

Blood (2005) 106 (11): 4033.

https://doi.org/10.1182/blood.V106.11.4033.4033

Published: 2005

Journal Articles

Correlation between Antifactor Xa Activity and Thrombin Generation Time, Platelet Contractile Force and Clot Elastic Modulus Following Enoxaparin Exposure in Patients with and without Renal Dysfunction.

Donald F. Brophy, PharmD, Erika J. Martin, M.T.(ASCP), Todd W.B. Gehr, MD, Al M. Best, PhD, Marcus E. Carr, MD, PhD

Blood (2005) 106 (11): 4144.

https://doi.org/10.1182/blood.V106.11.4144.4144

Published: 2005

Journal Articles

At 90 Micrograms Per Kilogram, Recombinant Factor VIIa Partially Corrects Abnormal Hemostatic Function in Blood from Factor IX Deficient Patients.

Marcus E. Carr, MD, PhD, Erika J. Martin, MT, Janice G. Kuhn, MPH, Melinda Nolte, RN

Blood (2004) 104 (11): 1044.

https://doi.org/10.1182/blood.V104.11.1044.1044

Published: 2004

Journal Articles

Muscle Cells in Fibrin Matrix Actively Collapse the Network Leading to Rapid Allignment and Formation of Muscle Bundles.

Marcus E. Carr, MD, PhD, Phillipe Lam, PhD, David G. Simpson, PhD, Gary E. Wnek, PhD, Gary L. Bowlin, PhD

Blood (2004) 104 (11): 3992.

https://doi.org/10.1182/blood.V104.11.3992.3992

Published: 2004

NARROW

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

Activity of a FIX-Padua Transgene Product in Commonly Used FIX:C One-Stage and Chromogenic Assay Systems Following PF-06838435 (SPK-9001) Gene Delivery

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence

Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression

Lack of Seasonal Variation in Bleeding and Patient-Assessed Pain Patterns in Patients with Hemophilia B Receiving On-Demand Therapy

Differential Dose Dependent Effects of Recombinant FVIIa and Enhanced Activity Analogue (NN1731) on Thrombin Generation, Platelet Function and Clot Structure in Blood from Normal Volunteers and Patients with Factor VIII Deficiency with or without Factor VIII Inhibitor Antibodies.

Correlation between Antifactor Xa Activity and Thrombin Generation Time, Platelet Contractile Force and Clot Elastic Modulus Following Enoxaparin Exposure in Patients with and without Renal Dysfunction.

At 90 Micrograms Per Kilogram, Recombinant Factor VIIa Partially Corrects Abnormal Hemostatic Function in Blood from Factor IX Deficient Patients.

Muscle Cells in Fibrin Matrix Actively Collapse the Network Leading to Rapid Allignment and Formation of Muscle Bundles.

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American Society of Hematology

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American Society of Hematology

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