Gene therapy for sickle cell disease using a new viral approach
Official title: In vivo Sickle Cell Disease Gene Therapy Using a Novel Virus Like Particle
Funded at Gigamune, INC. · NIH-11004909 · Data updated 2026-11-12
This study is testing a new gene therapy that uses a special delivery system to help people with sickle cell disease and β-thalassemia make healthy red blood cells, aiming to offer a long-lasting cure without the need for complicated hospital treatments.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Gigamune, INC. NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | September 10, 2024 |
| Project end | September 9, 2025 |
| Funding year | 2024 |
| Award (this year) | $300,000 |
| NIH project number | 1R43HL174204-01A1 |
| Project ID | NIH-11004909 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals aged 21 and older who have been diagnosed with sickle cell disease or β-thalassemia.
✕Less likely to help
Patients with other forms of hemoglobinopathies or those who are not diagnosed with sickle cell disease or β-thalassemia may not benefit from this research.
What this research studies
This research focuses on developing a gene therapy for sickle cell disease (SCD) and β-thalassemia, which are genetic disorders affecting hemoglobin production. The approach involves using a novel virus-like particle to deliver genetic material directly into patients' cells, potentially allowing for the production of healthy red blood cells without the need for extensive hospital procedures. Unlike traditional methods that require the removal and editing of stem cells, this in vivo method can be administered in a more accessible manner at various hospitals. The goal is to provide a long-term cure for patients suffering from these conditions.
Why it matters
Potential benefit: If successful, this research could lead to a life-long cure for patients with sickle cell disease and β-thalassemia.
How similar studies have gone: Other gene therapies for β-thalassemia have shown success, indicating that this novel approach may also be promising.
Where this research is happening
-
Gigamune, INC.
San Francisco, United States Active
Researchers
- Principal investigator: Johnson, David Scott — Gigamune, INC.
- Study coordinator: Johnson, David Scott
Related research
Other NIH-funded projects on the same topics.
-
Digital twins of the spleen to help design better therapies for sickle cell disease
A computer-driven replica of the spleen is being developed to speed up creating safer and more effective treatments for people with sickle cell disease.
-
Accelerating hydroxyurea use for sickle cell disease in Nigeria
A pragmatic study in Nigeria aims to help doctors use hydroxyurea more consistently for people with sickle cell disease.
-
Immune and nerve signals behind acupuncture relief for sickle cell pain
This NIH grant explores how acupuncture helps reduce pain and opioid use for people with sickle cell disease.
-
Mindfulness-based Support for Pain and Sleep in Young People with Sickle Cell Disease
A digital mindfulness program is being tailored for adolescents and young adults with sickle cell disease to help with pain and sleep and is being tested for practicality and early effectiveness.
-
How diet affects white blood cells and sickle cell disease
A study looks at how nutrition, from early life to adulthood, influences sickle cell disease by examining neutrophils and disease severity in people with SCD and in a mouse model.
-
Nutrition-based plan to ease pain in sickle cell disease
A nutrition-based approach is being tested to reduce pain for people living with sickle cell disease.
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.