AAVantgarde’s AAVB-039 gene therapy to treat Stargardt disease receives orphan drug status from FDA

The US Food and Drug Administration (FDA) has granted AAvantgarde’s AAVB-039 Orphan Drug Designation (ODD) and has also received Clinical Trial Authorisation (CTA) approval from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA).

AAVB-039 is an intra-retinal AAV8-intein-mediated investigational gene therapy targeting the gene associated with Stargardt disease, ABCA4.¹ AAVB-039 addresses the underlying genetic cause of Stargardt by providing the “full-length ABCA4 protein” and has the potential to “benefit all patients with ABCA4 mutations,” according to the company.¹

Natalia Misciattelli, PhD, CEO of AAVantgarde, commented on the news in a press release from the company,¹ saying, “The Orphan Drug Designation and UK CTA approval represent two important regulatory milestones for AAVB-039 and reflect the FDA’s and MHRA’s acknowledgement of the urgent need for treatments for patients living with Stargardt disease. With Fast Track Designation already in place, we now have a suite of regulatory incentives that will help accelerate development and bring this potentially transformative therapy to patients and families as efficiently as possible.”

As Misciattelli noted, AAVantgarde was recently granted fast track designation to AAVB-039 by the FDA.² In addition to the fast track designation, the investigational new drug (IND) application for AAVB-039 was also approved by the FDA this year.³

At the time of the IND application approval, Misciattelli commented,³ “With AAVB-039 now in clinical development, we are advancing our second therapeutic candidate toward addressing the urgent needs of patients with inherited retinal diseases. Moreover, the news reinforces our evolution from pioneering dual AAV-based, retina-targeting therapies into a clinical-stage company with a growing pipeline of candidates. Looking ahead, we remain focused on generating meaningful clinical data and building a portfolio of transformative treatments that can redefine what’s possible for patients living with severe genetic disorders.”

AAVB-039 is currently being evaluated in the phase 1/2 CELESTE clinical trial, which is assessing safety, tolerability, and preliminary efficacy of AAVB-039 in patients with Stargardt disease across 3 dose levels. The company has not released details on a projected timeline for trial results or updates.

In addition, AAVantgarde is conducting STELLA, a prospective natural history study across the US, Europe, and the UK, which has informed the clinical trial design of CELESTE, according to the company. The company is still recruiting for the STELLA study.

References:

1. AAVantgarde Announces FDA Orphan Drug Designation and UK CTA approval for AAVB-039 for the Treatment of Stargardt Disease. Published October 2, 2025. Accessed October 3, 2025. https://www.aavantgarde.com/en/news/aavantgarde-announces-fda-orphan-drug-designation-and-uk-cta-approval-for-aavb-039-for-the-treatment-of-stargardt-disease/

2. Harp MD. FDA grants fast track designation to AAVantgarde Bio for AAVB-039. Published August 12, 2025. Accessed October 3, 2025. https://www.ophthalmologytimes.com/view/fda-grants-fast-track-designation-to-aavantgarde-bio-for-aavb-039

3. Filkins K. AAVantgarde Bio receives IND clearance for AAVB-039. Published July 17, 2025. Accessed October 3, 2025. https://www.ophthalmologytimes.com/view/aavantgarde-bio-receives-ind-clearance-for-aavb-039