Non-immunogenic gene therapy for DMD patients excluded from dystrophin trials
IND-enabling studies of non-immunogenic gene therapy for ultra-rare DMD patients excluded from dystrophin trials
['FUNDING_U01'] · UNIVERSITY OF PENNSYLVANIA · NIH-11146702
This project develops a gene therapy designed to avoid immune reactions for people with rare forms of Duchenne muscular dystrophy who were left out of prior dystrophin trials.
Quick facts
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PENNSYLVANIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11146702 on ClinicalTrials.gov |
What this research studies
The team is designing a shortened dystrophin gene delivered by an AAV vector that is altered to reduce the chance of T cell–mediated immune reactions that caused serious side effects in a small group of patients. They are using animal models that closely mimic human DMD, including the German Shorthaired Pointer muscular dystrophy model, to test safety and immune responses. The work is focused on IND-enabling studies—building the data package needed to seek permission to start human testing. If results support safety, this could lead to clinical trials aimed specifically at patients previously excluded for immune-risk reasons.
Who could benefit from this research
Good fit: Ideal candidates would be people with Duchenne muscular dystrophy who have rare dystrophin mutations and were excluded from earlier AAV-dystrophin trials because of immune-risk concerns.
Not a fit: Patients already eligible for effective, approved DMD therapies or those with contraindications to AAV-based gene delivery are unlikely to benefit directly from this preclinical project.
Why it matters
Potential benefit: If successful, this could make systemic AAV gene therapy safer and open treatment options to DMD patients who were previously excluded due to immune-related risks.
How similar studies have performed: AAV-based mini-dystrophin therapies have shown promise in restoring dystrophin but have also produced immune-related serious adverse events in some cases, and efforts to design non-immunogenic constructs are relatively novel and currently at the preclinical stage.
Where this research is happening
PHILADELPHIA, UNITED STATES
- UNIVERSITY OF PENNSYLVANIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: STEDMAN, HANSELL H — UNIVERSITY OF PENNSYLVANIA
- Study coordinator: STEDMAN, HANSELL H
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.