Fresh off of receiving Fast Track designation from the FDA, AAVantegarde Bio announced the federal agency has granted Orphan Drug designation (ODD) to the biotech’s investigational gene therapy for Stargardt disease: AAVB-039.
First, a quick rundown on this designation.
As part of the FDA’s special regulatory status for investigational drugs, ODD is granted to certain candidates showing promise for the treatment, prevention, or diagnosis of “orphan” diseases.
- To note: These are classified as rare, serious, or life-threatening diseases or conditions (such as Stargardt) that affect <200,000 patients in the United States.
More about these “orphan drugs”: Drugs in this category undergo the same scientific review process as any other drug seeking regulatory approval for commercial licensing in the United States—but are also qualified to receive:
- Tax credits for qualified clinical trials
- User fees exemption
- Potential 7 years of market exclusivity following approval
And how does this differ from Fast Track designation?
Both designations serve separate purposes for an investigational candidate intended to treat serious conditions with unmet needs.
Case in point: FTD focuses more on providing more FDA interaction and ensuring a faster review process for a drug.
- And speaking of this, see here for details on AAVB-039’s FTD, which was granted in August 2025.
Let’s talk about this Stargardt candidate.
AAVB-039 is an intra-retinal adeno-associated virus serotype 8 (AAV8)-intein-mediated product targeting the ABCA4 gene—which, when mutated, leads to the development of Stargardt.
- See here for a refresh on AAV8.
How this ABCA4 gene targeting works: AAVB-039 utilizes AAVantgarde’s proprietary dual AAV intein platform to deliver the full-length ABCA4-protein (which has historically proven difficult).
Back up a moment. What do we know about this proprietary platform?
The dual AA intein platform is based on protein trans-splicing, and Stargardt is its sole disease target.
- Check out its subretinal injection-administered process for treatment.
Got it. And the intended result?
Ideally, to address the root cause of Stargardt and enable treatment for all Stargardt patients (regardless of the specific mutation).
So … with these designations in place, what’s next for the candidate?
At the moment, AAVB-039 is currently undergoing clinical evaluation in the U.S.-based, first-in-human phase 1/2 CELESTE trial (for which the company received FDA clearance in July 2025).
See here for what we know about it (as well as a second global study also underway).