Improved AAV3 gene delivery for hemophilia
Capsid- and genome-modified AAV3 vectors for hemophilia gene therapy.
['FUNDING_R01'] · UNIVERSITY OF FLORIDA · NIH-11248395
This work develops better AAV3-based gene delivery to help people with hemophilia make missing clotting factors using lower doses and fewer side effects.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF FLORIDA (nih funded) |
| Locations | 1 site (GAINESVILLE, UNITED STATES) |
| Trial ID | NIH-11248395 on ClinicalTrials.gov |
What this research studies
This project creates next-generation AAV3 viral vectors with both capsid (shell) and genome (ITR) changes to boost delivery into human liver cells. The team will test these engineered vectors in primary human hepatocytes and preclinical models to measure how much clotting-factor gene expression can be achieved. The aim is to reach therapeutic levels of factor VIII or IX using much lower vector doses so patients would need less immune-suppression. If the lab and preclinical results are promising, the optimized vectors would be prepared for eventual clinical testing in people with hemophilia A or B.
Who could benefit from this research
Good fit: Adults with moderate-to-severe hemophilia A or B who are considering gene therapy and meet clinical eligibility (for example, without prohibitive liver disease and with acceptable AAV antibody levels).
Not a fit: Children, people with advanced liver disease, or those with high pre-existing antibodies to AAV may not be eligible or may not benefit from this approach.
Why it matters
Potential benefit: Could enable lower-dose, longer-lasting gene therapy for hemophilia with fewer immune-related complications.
How similar studies have performed: AAV-based gene therapies for hemophilia have led to FDA approvals, showing the overall approach can work, while AAV3 capsid and ITR genome modifications are newer and less tested in people.
Where this research is happening
GAINESVILLE, UNITED STATES
- UNIVERSITY OF FLORIDA — GAINESVILLE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SRIVASTAVA, ARUN — UNIVERSITY OF FLORIDA
- Study coordinator: SRIVASTAVA, ARUN
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 below.
Conditions: Blood Coagulation Disorders, Christmas Disease, Coagulation Disorder