In a study published in the journal Molecular Therapy Methods and Clinical Development, researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with researchers at the School of Veterinary Medicine at the University of Pennsylvania, found that adeno-associated viral (AAV) gene therapy has the potential to be a life-changing treatment for companion dogs –pets – with severe hemophilia, an inherited bleeding disorder caused by deficiency of clotting factor.
While relatively rare, hemophilia is the most common inherited bleeding disorder in dogs, often treated with blood transfusions. Because hemophilia is caused by spontaneous genetic mutations, researchers hope AAV gene therapy’s success in humans for a variety of conditions, including hemophilia A and B, will make it more accessible for companion dogs.
“Our findings show that AAV-liver-directed gene therapy could be a promising treatment for dogs with hemophilia,” said Benjamin J. Samelson-Jones, MD, PhD, an attending physician in the Division of Hematology at CHOP. “While more research is needed before AAV gene therapy can enter veterinary clinics, our research suggests that companion dogs could reside in their homes with their owners and receive regular blood clotting tests and immune system checks.”
In the study, researchers followed 12 dogs with severe hemophilia that were treated with AAV gene therapy in a real-world setting. Following the treatment, the companion dogs demonstrated a 94% decrease in bleeding rates and 61% improvement in quality of life over an average of 4.1 years.
This first study to measure quality of life in hemophilic dogs found that gene therapy led to noticeable improvements. Owners reported their dogs were more active, had fewer health issues, and showed fewer negative behaviors—occurring less than once or twice a week. Positive physical behaviors, like daily activity and play, also increased.
The study was funded by National Heart, Lung and Blood Institute (HL158781, HL156073 and N01_75N92019D00041), the National Center for Advancing Translational Sciences (UL1TR000003) and the Institute for Translational Medicine and Therapeutics of the Perelman School of Medicine and the School of Veterinary Medicine at the University of Pennsylvania.
Doshi, Benjamin J. Samelson-Jones et al. “AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life.” Molecular Therapy Methods and Clinical Development. Online March 14, 2024. DOI: 10.1016/j.omtm.2024.101205.
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In a study published in the journal Molecular Therapy Methods and Clinical Development, researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with researchers at the School of Veterinary Medicine at the University of Pennsylvania, found that adeno-associated viral (AAV) gene therapy has the potential to be a life-changing treatment for companion dogs –pets – with severe hemophilia, an inherited bleeding disorder caused by deficiency of clotting factor.
While relatively rare, hemophilia is the most common inherited bleeding disorder in dogs, often treated with blood transfusions. Because hemophilia is caused by spontaneous genetic mutations, researchers hope AAV gene therapy’s success in humans for a variety of conditions, including hemophilia A and B, will make it more accessible for companion dogs.
“Our findings show that AAV-liver-directed gene therapy could be a promising treatment for dogs with hemophilia,” said Benjamin J. Samelson-Jones, MD, PhD, an attending physician in the Division of Hematology at CHOP. “While more research is needed before AAV gene therapy can enter veterinary clinics, our research suggests that companion dogs could reside in their homes with their owners and receive regular blood clotting tests and immune system checks.”
In the study, researchers followed 12 dogs with severe hemophilia that were treated with AAV gene therapy in a real-world setting. Following the treatment, the companion dogs demonstrated a 94% decrease in bleeding rates and 61% improvement in quality of life over an average of 4.1 years.
This first study to measure quality of life in hemophilic dogs found that gene therapy led to noticeable improvements. Owners reported their dogs were more active, had fewer health issues, and showed fewer negative behaviors—occurring less than once or twice a week. Positive physical behaviors, like daily activity and play, also increased.
The study was funded by National Heart, Lung and Blood Institute (HL158781, HL156073 and N01_75N92019D00041), the National Center for Advancing Translational Sciences (UL1TR000003) and the Institute for Translational Medicine and Therapeutics of the Perelman School of Medicine and the School of Veterinary Medicine at the University of Pennsylvania.
Doshi, Benjamin J. Samelson-Jones et al. “AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life.” Molecular Therapy Methods and Clinical Development. Online March 14, 2024. DOI: 10.1016/j.omtm.2024.101205.
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