How Does a Gene Therapy Get Approved?
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At Curing Retinal Blindness Foundation (CRBF), we are helping to support the development of promising gene therapy treatments that are currently in the preclinical testing phase. This is a crucial step in the journey to finding a cure for CRB1 retinal disease. Thanks to advancements in gene therapy—especially the success of Luxturna (the first FDA-approved retinal gene therapy for RPE65-related disease)—we have a clearer path forward. But how does a gene therapy go from the lab to patients? Here’s what it takes:
The Approval Process
1. Discovery & Early Research (1-3 years)
Scientists first study the disease and find a gene therapy that might work. They test it in cells and animals to see if it’s safe and effective.
2. Preclinical Testing (2-4 years)
Before testing in people, researchers conduct more studies in the lab to check for safety. They determine the best way to deliver the therapy (such as using a virus to carry the gene). CRBF is actively supporting the development of a potential CRB1 gene therapy in this stage!
3. FDA Application (IND Submission) (~1 year)
If early results look promising, researchers submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA). This outlines all safety data and how they plan to test the therapy in humans. The FDA must approve this before human trials can begin.
4. Clinical Trials in Humans (5-7 years)
Gene therapy is tested in people through clinical trials, which happen in three phases:
- Phase 1 – Tests safety in a small group (dozens of people).
- Phase 2 – Tests effectiveness and side effects in more people (hundreds).
- Phase 3 – Confirms safety and effectiveness in a larger group (hundreds to thousands).
5. FDA Review (Biologics License Application – BLA) (~1-2 years)
If clinical trials show the therapy is safe and works well, the company submits a Biologics License Application (BLA) to the FDA. The FDA reviews all the data to decide if the therapy should be approved.
6. Approval & Availability
If approved, the gene therapy can be given to patients. The FDA continues to monitor safety, and researchers may conduct more studies (Phase 4) to track long-term effects.
Faster Timelines For Retinal Diseases
The approval of Luxturna, a gene therapy for RPE65-related retinal disease, has paved the way for other retinal gene therapies. Since Luxturna has already proven that gene therapy can work for retinal diseases, future treatments (like those for CRB1) may move faster through regulatory approval, potentially shortening the timeline.
While gene therapy development still takes 8-12 years, your support can help push CRB1 treatments through this process as fast as possible. With funding, researchers can accelerate preclinical work, launch clinical trials, and bring life-changing treatments to patients sooner.