Stopping corneal transplant rejection with BDRK-401 gene therapy

Prevention of corneal transplant rejection using AAV-BDRK-401 therapy

NIH-funded research Bedrock Therapeutics, INC. · NIH-11180103

A one-time gene treatment is applied to donor corneas to help people at high risk keep their corneal transplants from being rejected.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionBedrock Therapeutics, INC. NIH-funded
Lab location1 site (Chapel Hill, United States)
Project IDNIH-11180103 on NIH RePORTER

What this research studies

If you need a cornea transplant, doctors would treat the donor cornea once with a modified virus (AAV) carrying BDRK-401 before surgery to protect it from the recipient's immune system. The treatment is designed to reduce blood vessel growth into the graft and locally suppress immune responses that drive rejection. The team completed Phase I milestones and showed treated corneas were fully protected from acute rejection in rabbit models, and they refined a process that could be used in hospitals. This Phase II effort moves the approach toward use in people and prepares for clinical testing in corneal transplant patients.

Who could benefit from this research

Good fit: Ideal candidates are people undergoing high-risk allogeneic corneal transplants, for example those with inflamed or vascularized corneas or prior graft failures.

Not a fit: Patients who do not need a corneal transplant, who are having low‑risk grafts, or who cannot receive AAV-based treatments are unlikely to benefit directly.

Why it matters

Potential benefit: If successful, this could reduce corneal graft rejection and lower the need for prolonged systemic immunosuppression after high-risk transplants.

How similar studies have performed: AAV-based gene therapies have worked for other eye conditions and this approach completely prevented acute rejection in rabbit cornea models, but using AAV-treated donor corneas in humans is novel.

Where this research is happening

Chapel Hill, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.