Intraosseous delivery of gene therapy vectors for hemophilia A
Official title: Intraosseous delivery of lentiviral vectors for hemophilia A gene therapy
Funded at Seattle Children's Hospital · NIH-11389762 · Data updated 2026-11-12
A gene therapy approach using vectors delivered into bone to help people with hemophilia A reduce or eliminate the need for frequent FVIII treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Seattle Children's Hospital NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | August 15, 2016 |
| Project end | May 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $822,265 |
| NIH project number | 2R01HL134321-09 |
| Project ID | NIH-11389762 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates include people with hemophilia A who struggle with current FVIII therapies or have inhibitors, pending successful translation to human trials.
✕Less likely to help
Individuals without hemophilia A or those whose condition is not related to FVIII deficiencies are unlikely to benefit.
What this research studies
This project explores delivering gene therapy vectors directly into bone to produce factor VIII and help people with hemophilia A. It focuses on overcoming barriers to current AAV-based therapies and aims for a durable, potentially one-time or limited-treatment option. The work involves preclinical models to assess safety, efficacy, and long-term expression, with an eye toward translating promising results to patients who struggle with current therapies. If successful, the approach could reduce bleeding episodes and improve quality of life for individuals with hemophilia A, including those who have developed inhibitors. The study is led by Seattle Children’s Hospital and collaborates across biomedical engineering and hematology.
Why it matters
Potential benefit: If successful, this gene therapy could provide long-lasting or lifelong reduction in bleeding risk for hemophilia A patients, lowering the need for regular FVIII infusions.
How similar studies have gone: Past studies with AAV-based FVIII gene therapy have shown promise but face access and inhibitor challenges; this approach seeks to address those gaps with intraosseous delivery.
Where this research is happening
-
Seattle Children's Hospital
Seattle, United States Active
Researchers
- Principal investigator: Miao, Carol H — Seattle Children's Hospital
- Study coordinator: Miao, Carol H
Related research
Other NIH-funded projects on the same topics.
-
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.
-
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.
-
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.
-
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.
-
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.
-
Intergenerational Hemophilia A Program to Understand Factor VIII Immune Responses
This program follows mothers who carry hemophilia A and their babies with severe hemophilia to learn how immune reactions to factor VIII develop in early life.
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.