Gene editing for hemophilia A using targeted lipid nanoparticles
Official title: Gene Editing for Hemophilia A Treatment Using Lipid Nanoparticles
Funded at Seattle Children's Hospital · NIH-11332298 · Data updated 2026-11-12
A patient-friendly approach uses tiny lipid bubbles to deliver gene editing tools that fix the FVIII gene in the liver, aiming to reduce or eliminate the need for frequent FVIII infusions.
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, 2024 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $719,642 |
| NIH project number | 5R01HL169793-03 |
| Project ID | NIH-11332298 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals diagnosed with Hemophilia A who could benefit from gene-editing-based therapies and meet safety and eligibility criteria for future trials.
✕Less likely to help
Patients with conditions outside hemophilia A or with contraindications to gene editing or liver-targeted therapies may not benefit.
What this research studies
This project investigates a nonviral, targeted gene editing strategy to correct the FVIII gene responsible for hemophilia A using lipid nanoparticles that home to liver sinusoidal endothelial cells. By combining LNP technology with CRISPR/Cas9, researchers aim to permanently fix the faulty gene and restore normal clotting function. The work focuses on optimizing LNP formulations and endothelial-targeting ligands to improve delivery and minimize side effects, with the goal of a safer, longer-lasting treatment option for patients. If successful, this could lessen treatment burden and improve quality of life for people living with HemA.
Why it matters
Potential benefit: If successful, the approach could provide a durable treatment for hemophilia A with fewer infusions and better blood clotting control.
How similar studies have gone: Previous viral gene therapies show promise but have durability and safety concerns; nonviral, liver-targeted gene editing is a newer, evolving approach with early supportive data.
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.
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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.