Creating a resource platform for curative therapies for sickle cell disease
CureSC Manufacturing Resource Platform
This study is working to create a helpful resource platform for making gene therapies that could cure sickle cell disease, starting by finding out what the sickle cell community needs to ensure these treatments can be produced effectively.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Beckman Research Institute/city of Hope NIH-funded |
| Lab location | 1 site (Duarte, United States) |
| Project ID | NIH-10934634 on NIH RePORTER |
What this research studies
This research aims to establish a manufacturing resource platform specifically for curative genetic therapies targeting sickle cell disease (SCD). The project will begin with a needs assessment to understand the requirements of the sickle cell community regarding cell and vector product manufacturing. Experts from City of Hope will evaluate current manufacturing capabilities and develop a strategy for a consortium approach to ensure that the necessary resources are available for effective treatment. This initiative will facilitate the production of essential components for gene therapies that could potentially cure SCD.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with sickle cell disease who may benefit from advanced genetic therapies.
Not a fit: Patients with other blood disorders or those not diagnosed with sickle cell disease may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved access to curative gene therapies for patients with sickle cell disease.
How similar studies have performed: Previous research has shown promise in the field of gene therapy for blood disorders, indicating that this approach could be effective.
Where this research is happening
Duarte, United States
- Beckman Research Institute/city of Hope — Duarte, United States (Active)
Researchers
- Principal investigator: Cardoso, Angelo a. — Beckman Research Institute/city of Hope
- Study coordinator: Cardoso, Angelo a.
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.