Multiple team members from the Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Program at Children's Hospital of Philadelphia (CHOP) were part of the program at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, held Dec. 10–13, 2022, in New Orleans, La.
The NoT Bleeding Program is a world-class center for the therapeutic development and advanced clinical care of inherited bleeding disorders. The program combines longstanding clinical expertise in hemophilia gene therapy and management of bleeding disorders with cutting-edge research. ASH is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment and prevention of blood disorders.
NoT Bleeding physicians and scientists at ASH were the following:
Lindsey A. George, MD — who leads the Not Bleeding Program and is the Director of Clinical In Vivo Gene Therapy — gave the oral abstract “Inhibiting Activated Protein C Cleavage of Factor VIII for Hemophilia A Gene Therapy.”
Rodney M. Camire, PhD, gave the oral presentation “An Antibody Targeting Human FV Promotes Thrombin Generation and Reduces Bleeding in a Hemophilic Mouse Model.”
George and Camire were co-authors of the oral presentation “Antigen Trafficking and Possibly Intrinsic Immunostimulatory Properties Shape High Immunogenicity of Factor VIII” and co-authors of the poster “Coagulation Factor VIII Enhances the Cleavage of Von Willebrand Factor By ADAMTS13 In Vivo.”
Benjamin J. Samelson-Jones, MD, PhD, presented the poster “Factor VIII Mimetic Rescue of Hemophilia B Causing Factor IX Variants.”
George and Samelson-Jones were co-authors of the oral presentation “Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011.”
Samelson-Jones was co-author of the poster “Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A.”
Samelson-Jones and Bhavya S. Doshi, MD, were co-authors of the poster “Long-Term Real-World Safety and Efficacy of Liver-Directed AAV Gene Therapy for Severe Hemophilia with and without Inhibitors in Privately Owned Dogs.”
Doshi was co-author of the poster “Vector Analysis of Multicentric Lymphoma in a Severe Hemophilia Α Dog after AAV Gene Therapy.”
Leslie J. Raffini, MD, Medical Director of the Hemostasis and Thrombosis Center, and Hilary Whitworth, MD, were co-authors of the abstract “Clinical Characteristics and Acute Management of Pediatric Pulmonary Embolism.”
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Multiple team members from the Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Program at Children's Hospital of Philadelphia (CHOP) were part of the program at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, held Dec. 10–13, 2022, in New Orleans, La.
The NoT Bleeding Program is a world-class center for the therapeutic development and advanced clinical care of inherited bleeding disorders. The program combines longstanding clinical expertise in hemophilia gene therapy and management of bleeding disorders with cutting-edge research. ASH is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment and prevention of blood disorders.
NoT Bleeding physicians and scientists at ASH were the following:
Lindsey A. George, MD — who leads the Not Bleeding Program and is the Director of Clinical In Vivo Gene Therapy — gave the oral abstract “Inhibiting Activated Protein C Cleavage of Factor VIII for Hemophilia A Gene Therapy.”
Rodney M. Camire, PhD, gave the oral presentation “An Antibody Targeting Human FV Promotes Thrombin Generation and Reduces Bleeding in a Hemophilic Mouse Model.”
George and Camire were co-authors of the oral presentation “Antigen Trafficking and Possibly Intrinsic Immunostimulatory Properties Shape High Immunogenicity of Factor VIII” and co-authors of the poster “Coagulation Factor VIII Enhances the Cleavage of Von Willebrand Factor By ADAMTS13 In Vivo.”
Benjamin J. Samelson-Jones, MD, PhD, presented the poster “Factor VIII Mimetic Rescue of Hemophilia B Causing Factor IX Variants.”
George and Samelson-Jones were co-authors of the oral presentation “Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011.”
Samelson-Jones was co-author of the poster “Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia A.”
Samelson-Jones and Bhavya S. Doshi, MD, were co-authors of the poster “Long-Term Real-World Safety and Efficacy of Liver-Directed AAV Gene Therapy for Severe Hemophilia with and without Inhibitors in Privately Owned Dogs.”
Doshi was co-author of the poster “Vector Analysis of Multicentric Lymphoma in a Severe Hemophilia Α Dog after AAV Gene Therapy.”
Leslie J. Raffini, MD, Medical Director of the Hemostasis and Thrombosis Center, and Hilary Whitworth, MD, were co-authors of the abstract “Clinical Characteristics and Acute Management of Pediatric Pulmonary Embolism.”
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