Creating a therapy using stem cells to help patients without a thymus
Development of a stem-cell derived thymic cell therapy to treat patients with athymia
['FUNDING_SBIR_2'] · THYMMUNE THERAPEUTICS, INC · NIH-11042805
This study is exploring a new way to help people born without a thymus, which makes it hard for their immune system to work properly, by using special stem cells to create thymus cells that can be implanted to boost their immune function.
Quick facts
| Phase | ['FUNDING_SBIR_2'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | THYMMUNE THERAPEUTICS, INC (nih funded) |
| Locations | 1 site (CAMBRIDGE, UNITED STATES) |
| Trial ID | NIH-11042805 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a new therapy for patients born without a thymus, a condition known as athymia, which leads to a lack of functional T cells and severe immunodeficiency. The approach involves using induced pluripotent stem cells (iPSCs) to create thymic cells that can be implanted to restore T cell function in these patients. Current treatments are limited by the availability of donor tissues, but this innovative method aims to overcome those challenges by generating thymic cells from stem cells. The research is being conducted by a team with extensive experience in drug development, ensuring a robust approach to this critical health issue.
Who could benefit from this research
Good fit: Ideal candidates for this research are infants and young children diagnosed with athymia, particularly those with conditions like 22q11.2 deletion syndrome.
Not a fit: Patients who do not have athymia or those with other unrelated immunodeficiencies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a scalable and effective treatment option for patients with athymia, significantly improving their immune function and survival rates.
How similar studies have performed: While the approach of using stem cells for thymic cell therapy is innovative, similar strategies in regenerative medicine have shown promise, indicating potential for success.
Where this research is happening
CAMBRIDGE, UNITED STATES
- THYMMUNE THERAPEUTICS, INC — CAMBRIDGE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LIM, BING — THYMMUNE THERAPEUTICS, INC
- Study coordinator: LIM, BING
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: 22q11 Chromosomal Microdeletion Syndrome, 22q11 Deletion Syndrome, 22q11.2 deletion syndrome