FDA Grants Fast Track Designation to AAVantgarde Bio for Stargardt Disease Gene Therapy AAVB-039

The US Food and Drug Administration (FDA) has granted Fast Track Designation to AAVantgarde Bio for AAVB-039, the company’s investigational gene therapy for Stargardt disease caused by biallelic mutations in the ABCA4 gene.

This decision follows recent clearance by the FDA of the Investigational New Drug (IND) application for AAVB-039, enabling the program to move forward into clinical testing.

Targeting the Underlying Genetic Cause

AAVB-039 is designed to address the root cause of Stargardt disease by delivering the full-length ABCA4 protein, with potential applicability to all patients carrying ABCA4 mutations.

“We are thrilled that the FDA has granted Fast Track Designation for AAVB-039, as it underscores the urgent unmet need and important potential of our program due to the serious nature of challenges faced by patients living with Stargardt disease,” said Natalia Misciattelli, PhD, CEO of AAVantgarde Bio. “This designation represents an important milestone for our program and enables us to accelerate development efforts as we work to bring a potentially transformative therapy to patients as quickly as possible.”

Clinical Development: CELESTE and STELLA Studies

AAVB-039 is currently being evaluated in the Phase 1/2 CELESTE clinical trial, which assesses safety, tolerability, and preliminary efficacy in patients with Stargardt disease.

In parallel, AAVantgarde is running STELLA, a prospective natural history study conducted across the US, Europe, and the UK. Data from STELLA has helped inform the design of the CELESTE trial, ensuring alignment with the disease’s progression and patient needs.

Additional Pipeline Programs

AAVantgarde Bio is also advancing AAVB-081, its first clinical-stage program, for the treatment of retinitis pigmentosa. Currently in Phase 1/2 development, AAVB-081 is the first dual AAV gene therapy to enter clinical testing for an ocular indication.