A safer, longer-lasting gene delivery approach for Hemophilia A using improved nonviral vectors
Official title: Development of AAV Episome-Based Nonviral Vectors for Prolonged Gene Expression in Hemophilia A
Funded at M34 INC · NIH-11408326 · Data updated 2026-11-12
Researchers are testing a new type of nonviral gene delivery that could provide longer expression of a therapeutic gene for adults with Hemophilia A.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | M34 INC NIH-funded |
| Lab location | 1 site (Hillsborough, United States) |
| Project start | September 15, 2026 |
| Project end | September 14, 2027 |
| Funding year | 2026 |
| Award (this year) | $306,782 |
| NIH project number | 1R43HL182565-01A1 |
| Project ID | NIH-11408326 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with Hemophilia A who may benefit from future gene therapy approaches and participate in translational studies.
✕Less likely to help
Individuals seeking immediate, approved standard therapies may not benefit from this early-stage development.
What this research studies
This project aims to combine the benefits of AAV and nonviral delivery to achieve durable gene expression for Hemophilia A while potentially allowing re-dosing. It explores episome-based nonviral vectors that can carry large gene payloads and reduce issues seen with traditional AAV therapies, such as pre-existing antibodies and re-administration limitations. The work involves preclinical development and optimization to maximize safety and durability in adult samples, with careful attention to how the vector behaves in humans. If successful, this approach could pave the way for new treatment options that sustain clotting factor production with fewer restrictions on repeat dosing.
Why it matters
Potential benefit: If successful, this research could lead to a more durable and adaptable gene therapy for Hemophilia A with fewer dosing limits.
How similar studies have gone: Earlier efforts with nonviral approaches have shown promise but durability remains a key challenge; this work builds on those foundations with a novel episome-based strategy.
Where this research is happening
-
M34 INC
Hillsborough, United States Active
Researchers
- Principal investigator: Grieger, Joshua — M34 INC
- Study coordinator: Grieger, Joshua
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.
-
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.
-
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.
-
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.