AAVantgarde Bio, a clinical-stage biotechnology company, announced on March 10, 2025, that it will present data from two of its leading gene therapy programs at the upcoming Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, scheduled for May 4-8, 2025, in Salt Lake City, Utah.
The company will showcase results from its innovative dual AAV vector platform technology, which addresses a critical limitation in gene therapy—the cargo capacity of adeno-associated viral (AAV) vectors—enabling the delivery of larger therapeutic genes for inherited retinal diseases.
LUCE-1 Clinical Trial for Usher Syndrome Type 1B
AAVantgarde's oral presentation will feature updated data from the LUCE-1 clinical trial, a first-in-human Phase I/II study evaluating dual AAV8.MYO7A gene therapy for patients with Usher syndrome Type 1B (USH1B). This rare genetic disorder causes combined hearing loss and retinitis pigmentosa, a progressive vision loss condition.
The presentation builds upon preliminary results shared at the FLORetina medical conference in December 2024, now including data from additional treated patients. Professor Francesca Simonelli, Head of the Ophthalmology Unit at the University Hospital of Campania "Luigi Vanvitelli" in Naples and Principal Investigator for AAVantgarde, will deliver the presentation.
"The LUCE-1 trial represents a significant advancement in addressing the underlying genetic cause of Usher syndrome Type 1B, for which there are currently no approved treatments," said an ophthalmology expert familiar with the study. "The ability to deliver the large MYO7A gene using AAVantgarde's dual vector approach could potentially preserve vision in these patients who typically experience progressive blindness."
The presentation is scheduled for May 4, 2025, from 2:30 to 2:45 PM MST, as part of Session 125: "First-in-human and preclinical gene therapies."
Stargardt Disease Program Advances
In a poster presentation, AAVantgarde will share preliminary data from its non-human primate (NHP) Good Laboratory Practice (GLP) study for Stargardt disease. The study evaluated the safety and pharmacokinetics of subretinal administration of dual AAV8.ABCA4, the company's gene therapy candidate for this condition.
Stargardt disease is the most common form of inherited juvenile macular degeneration, typically causing vision loss in childhood or adolescence. The disease results from mutations in the ABCA4 gene, which is too large to fit within a standard AAV vector—making it an ideal candidate for AAVantgarde's dual vector technology.
According to the company, preliminary results confirm a positive safety profile that supports the initiation of first-in-human clinical trials. This represents a significant milestone for patients with Stargardt disease, who currently have no FDA-approved treatment options.
The poster presentation (Number 5956 - A0021) titled "GLP safety study of intein-based Dual AAV8.ABCA4 for Stargardt Disease" will be available on May 8, 2025, from 11:45 AM to 1:30 PM MST during Session 534: "Gene therapy."
Addressing Unmet Needs in Inherited Retinal Diseases
Dr. Natalia Misciattelli, CEO of AAVantgarde, emphasized the significance of these developments: "We are excited to present very promising data from our two lead programs that clearly show the therapeutic potential of these programs and our platform technology and provide hope to the underserved Usher 1B and Stargardt patients."
Both Usher syndrome Type 1B and Stargardt disease represent significant unmet medical needs. Current management approaches focus primarily on supportive care rather than addressing the underlying genetic causes of vision loss.
Innovative Platform Technology
AAVantgarde's proprietary technology overcomes a fundamental limitation in gene therapy. Standard AAV vectors, while effective for gene delivery, can only accommodate genetic material up to approximately 4.7 kilobases in size. Both the MYO7A gene (implicated in Usher syndrome) and the ABCA4 gene (involved in Stargardt disease) exceed this capacity.
The company's dual vector approach effectively splits larger genes into two complementary AAV vectors that, once inside target cells, reconstitute the full-length therapeutic gene. This innovation potentially expands the range of genetic conditions amenable to gene therapy.
Industry analysts note that success in these programs could validate AAVantgarde's platform technology for application beyond ophthalmology, potentially addressing other conditions requiring delivery of large genes to various tissues.
Future Directions
With positive preliminary data from both programs, AAVantgarde appears positioned to advance its gene therapy candidates through clinical development. The company has indicated that the safety profile observed in the Stargardt program supports progression to human trials, while the Usher 1B program continues to generate clinical data.
The upcoming presentations at ARVO 2025 will provide the scientific community with more detailed insights into the efficacy and safety of these innovative approaches, potentially marking a significant step forward in the treatment of inherited retinal diseases.
