Hemophilia and Rare Bleeding Disorders » Hemophilia Gene Therapy

Abstract Number: PB0211

A translational analysis of immune components in peripheral blood from severe hemophilia A patients treated with TAK-754, an AAV8 vector with a codon-optimized B-domain–deleted factor VIII transgene

J. Chapin, M. Ayash-Rashkovsky, J. Kenniston, M. Wagoner, Q. Wang

Abstract Number: OC 12.2

AAV mediated CRISPR/Cas9 based therapeutic gene-editing with a bypass coagulation factor in a murine model of hemophilia.

P. Sarangi¹, S. Bammidi¹, R. Sambasivan², G. Jayandharan¹

Abstract Number: OC 12.4

An Enhanced Hemostatic Factor VIII Variant for Hemophilia A Gene Therapy: Prothrombotic and Immunological Risk Assessment

A. Sternberg, R. Davidson, C. Martos-Rus, A. Wilhelm, L. George

Abstract Number: OC 01.1

Base and Prime editing of DNA as a new therapeutic option for Hemophilia A

E. Tonetto¹, S. Pignani², G. Roman², A. Follenzi², F. Bernardi³, D. Liu⁴, M. Pinotti¹, D. Balestra¹

Abstract Number: PB0210

Blood Biodistribution and Vector Shedding of Valoctocogene Roxaparvovec in People with Severe Hemophilia A: Results from the Phase 3 GENEr8-1 Trial

S. Agarwal¹, K. Obrochta¹, M. Vora¹, A. Bowen¹, B. Bunch¹, J. Holcomb¹, D. Guo¹, T. Robinson², K. Jayaram¹, C. Russell¹, C. Vettermann¹, J. Henshaw¹

Abstract Number: PB0212

Collaboration between the World Federation of Hemophilia and National Registries for the Long-Term Follow-up of People with Hemophilia Treated with Gene Therapy

M. Naccache¹, B. Konkle², D. Coffin¹, C. Clark³, L. George⁴, A. Iorio⁵, W. Miesbach⁶, D. Noone⁷, F. Peyvandi⁸, S. Pipe⁹, M. Recht¹⁰, M. Skinner¹¹, L. Valentino¹², J. Mahlangu¹³, G. Pierce¹, F. Leebeek¹⁴

Abstract Number: OC 21.5

Comparative effectiveness of valoctocogene roxaparvovec and prophylactic factor VIII replacement estimated through propensity scoring

H. Liu¹, C. Hawes², C. Hsu¹, P. You¹, X. Yang¹, V. Newman², T. Robinson¹, A. Hatswell³, D. Hinds¹

Abstract Number: OC 21.4

Effect of Growth on AAV-mediated Expression of FIX-R338L in Juvenile Hemophilia B Dogs

T. Nichols¹, S. Rakhe², V. Arruda³, B. Samelson-Jones⁴, M. Caughey⁵, E. Merricks⁶, J. Murphy², D. Pittman⁷

Abstract Number: OC 01.3

Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy

A. Sternberg, B. Samelson-Jones, L. George

Abstract Number: OC 21.2

Hemostatic results for up to 6 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A

M. Laffan¹, S. Rangarajan², W. Lester³, E. Symington⁴, B. Madan⁵, D. Hart⁶, M. Li⁷, T. Robinson⁸, G. Pierce⁹, W. Wong⁷

Abstract Number: OC 01.2

Improvements in Health-Related Quality of Life in Adults with Severe or Moderately Severe Hemophilia B After Receiving Etranacogene Dezaparvovec Gene Therapy

R. Itzler¹, J. Miller², R. Robson², P. Monahan³, S. Pipe⁴

Abstract Number: OC 01.5

Innate and Adaptive Immune Responses to Adeno-Associated Viral Gene Therapy in the Severe Hemophilia A Dog Model

P. Batty¹, A. Mo², M. Ashrafali³, B. Yates³, A. Gonzalez³, B. Handyside³, L. Harpell², D. Hurlbut², A. Menard⁴, A. Pender², L. Razon³, C. Sihn³, A. Winterborn², S. Fong³, D. Lillicrap⁵

Abstract Number: OC 12.5

Liver Sinusoidal Endothelial Cells Targeted with Ultrasound Mediated Gene Delivery Shows Long Term FVIII Expression in Hemophilia A Mice

S. Lawton¹, M. Manson², M. Fan³, T. Chao³, P. Kim³, C. Campbell³, C. Chen³, X. Cai⁴, A. Vander Kooi³, C. Miao³

Abstract Number: LB 01.3

Relationship between transgene-produced FVIII and bleeding rates 2 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

J. Mahlangu¹, H. Chambost², S. Chou³, A. Dunn⁴, A. von Drygalski⁵, R. Kaczmarek⁶, G. Kenet⁷, M. Laffan⁸, A. Leavitt⁹, B. Madan¹⁰, J. Mason¹¹, J. Oldenburg¹², M. Ozelo¹³, F. Peyvandi¹⁴, D. Quon¹⁵, M. Reding¹⁶, S. Shapiro¹⁷, H. Yu¹⁸, T. Robinson¹⁸, S. Pipe¹⁹

Abstract Number: OC 12.3

Rescue of the endogenous FVIII expression in hemophilia A mice using CRISPR/Cas9 mRNA LNPs

C. Chen, X. Cai, C. Miao

Abstract Number: PB0213

Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients with Hemophilia B

G. Young¹, P. Chowdary², S. Barton³, D. Yee³, F. Ferrante³

Abstract Number: OC 12.1

Transgene expression patterns after AAV8 delivery using hepatocyte promoter elements reveal expression in both hepatocytes and liver sinusoidal endothelial cells (LSECs)

J. Lindgren¹, D. Sabatino¹, G. Nguyen¹, A. Follenzi², A. Henriksen¹, R. Fama¹

Abstract Number: OC 21.3

Treatment of Canine Hemophilia A via Intraosseous Delivery of a Platelet-Specific Factor VIII-Lentiviral Vector

C. Rementer¹, C. Li¹, T. Nichols², X. Cai¹, J. Joo¹, X. Wang¹, E. Merricks³, H. Ochs¹, D. Rawlings¹, C. Miao¹