Gene therapy to induce immune tolerance in dogs with hemophilia A
Official title: Immune tolerance induction by AAV-FVIII gene therapy for canine hemophilia A with inhibitors
Funded at Children's Hosp of Philadelphia · NIH-10851822 · Data updated 2026-11-12
This study is testing a new gene therapy for dogs with hemophilia A who have developed issues with their treatment, aiming to help their bodies accept a key protein they need for blood clotting, which could make their care easier and less costly.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 1, 2021 |
| Project end | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $723,852 |
| NIH project number | 5R01HL158781-04 |
| Project ID | NIH-10851822 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are dogs diagnosed with hemophilia A who have developed inhibitors to factor VIII.
✕Less likely to help
Dogs without hemophilia A or those who do not have inhibitors to factor VIII may not benefit from this research.
What this research studies
This research investigates a new gene therapy approach to treat hemophilia A in dogs, specifically targeting those that have developed inhibitors to factor VIII. The therapy uses adeno-associated virus (AAV) vectors to deliver the canine F8 gene, which is responsible for producing factor VIII, directly to the liver. By doing so, the aim is to induce immune tolerance, allowing the dog's body to accept factor VIII without producing harmful antibodies. This could potentially reduce the need for expensive and prolonged treatment regimens currently in use.
Why it matters
Potential benefit: If successful, this research could provide a more effective and less costly treatment option for dogs suffering from hemophilia A with inhibitors.
How similar studies have gone: Previous studies using AAV gene therapy for hemophilia A without inhibitors have shown promising results, indicating potential for success in this novel approach.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Samelson-Jones, Ben J — Children's Hosp of Philadelphia
- Study coordinator: Samelson-Jones, Ben J
Related research
Other NIH-funded projects on the same topics.
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cGMP production of FVIII-making placental cells to help people with Hemophilia A
A new, patient-friendly therapy aims to make FVIII-producing placental cells in the lab to help adults with Hemophilia A, potentially offering a longer-lasting and more affordable treatment.
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A safer, longer-lasting gene delivery approach for Hemophilia A using improved nonviral vectors
Researchers are testing a new type of nonviral gene delivery that could provide longer expression of a therapeutic gene for adults with Hemophilia A.
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Hemophilia A: studying why some people develop inhibitors to FVIII across families
This NIH grant funds a multi-year program to follow families affected by severe hemophilia A to understand why some individuals develop antibodies against factor VIII.
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Intraosseous delivery of gene therapy vectors for hemophilia A
A gene therapy approach using vectors delivered into bone to help people with hemophilia A reduce or eliminate the need for frequent FVIII treatments.
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Targeting FVIII production to eye-opening blood vessels in hemophilia A
Aims to improve treatment for people with hemophilia A by directing how the body makes clotting factor VIII, using a targeted gene-delivery approach.
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Platelet-targeted FVIII Gene Therapy for Hemophilia A
A gene therapy approach aims to make platelets carry FVIII to help people with hemophilia A, even if they have inhibitors against FVIII.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.