MILAN, Oct. 04, 2026 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), the clinical-stage biotechnology company developing genetic medicines for inherited retinal diseases (IRDs) using its proprietary dual-AAV technology, has today announced the presentation of updated clinical data from the LUCE-1 study and preclinical and clinical development data for AAVB-039 for Stargardt disease at the 26th European Society of Retina Specialists (EURETINA) Annual Congress (EURetina 2026), taking place in Vienna 1-4 October 2026.
Prof. Francesca Simonelli presented preliminary findings from the LUCE-1 Phase 1/2 study (NCT06591793), an open-label study evaluating subretinal administration of AAVB-081, a dual AAV8.MYO7A gene therapy using a DNA-splicing approach, in adults with Usher type B retinitis pigmentosa. Enrollment in LUCE-1 was completed in January 2026, with 15 participants treated across three dose cohorts. The presentation included safety data across all treated participants and efficacy assessments from participants with at least six months of follow-up.
As of the August 3, 2026 data cut-off point, no serious adverse events or dose-limiting toxicities had been reported, and no participants had discontinued the study. Ocular inflammation was limited, in-line with expectations and responsive to corticosteroid treatment.
Among the 12 participants in the low- and mid-dose cohorts with at least six months of follow-up, early signals of visual function improvement were observed. Seven participants achieved at least a one-line improvement in best-corrected visual acuity (BCVA), including four participants with at least a two-line improvement. Six participants achieved at least a one-line improvement in low-luminance visual acuity (LLVA), including four participants with at least a three-line improvement. Exploratory assessments also showed supportive signals in fixation stability and microperimetry in several participants.
The data support continued clinical development of AAVB-081 in USH1B and further evaluation of visual function outcomes with longer follow-up.
Prof. Paulo Eduardo Stanga presented new data supporting the clinical translation of AAVB-039, AAVantgarde's dual AAV8.ABCA4 gene therapy for STGD1. AAVB-039 uses a dual AAV intein-mediated protein trans-splicing approach to deliver the full-length ABCA4 gene to photoreceptors.
Across mouse, pig and non-human primate models, the dual AAV8.ABCA4 approach demonstrated reconstitution of full-length ABCA4 proteins and a reduction of lipofuscin accumulation, together with a favourable ocular safety profile. In a STGD1 pig model, treatment resulted in full-length ABCA4 expression exceeding 100% of endogenous ABCA4 levels in the reported analysis, with reduced lipofuscin accumulation in treated areas compared with sham-injected knockout eyes. In non-human primates, 76–99% of photoreceptors were co-transduced with the two vector components across approximately 60% of the analysed retinal section. Ocular effects were described as mild and transient, with electroretinography changes that were temporary and dose-related and histological findings that were minimal, localized and improved over time.
The AAVB-039 clinical development program comprises the STELLA natural history study, which has completed enrolment of 150 patients, and the CELESTE first-in-human Phase 1/2 study. CELESTE is a phase 1/2 study evaluating the safety and preliminary efficacy of AAVB-039 in participants with STGD1 caused by biallelic pathogenic ABCA4 variants. The study is recruiting in the US, UK and Europe.
Dr. Jayashree Sahni, CEO of AAVantgarde commented: “The data presented at EURetina 2026 provide important updates across both of our lead programs. In LUCE-1, the continued absence of serious adverse events or dose-limiting toxicities, together with early signals of improved visual function in the low- and mid-dose cohorts, support the continued development of AAVB-081 in Usher syndrome type 1B. At the same time, the preclinical data for AAVB-039 demonstrate consistent ABCA4 expression, lipofuscin reduction and favourable ocular safety across relevant large animal models, supporting the ongoing clinical translation of our dual AAV intein platform in Stargardt disease.”
About the LUCE-1 Trial (NCT06591793)
LUCE-1 is a Phase 1/2 multicenter, open-label, dose escalation study investigating safety, tolerability and preliminary efficacy of 3 dose levels of dual AAV8.MYO7A (AAVB-081) administered subretinally in participants with retinitis pigmentosa associated with Usher Syndrome Type 1B. You can find further information on the LUCE-1 study in the link below:
LUCE-1 clinical study
About Usher syndrome type 1B
Usher syndrome type 1B (Usher1B) is an inherited disease that affects the retina and the inner ear. Usher1B is caused by mutations in the MYO7A gene. The therapeutic gene to treat Usher1B is 6.7 kb long and is therefore too large to fit inside a standard AAV vector. Approximately 20,000 patients in the U.S. and E.U. have Usher1B. These children are born deaf, have vestibular dysfunction, and begin to progressively lose vision in their first decade of life. Although there are surgical treatments available to treat deafness in these patients, there are no treatments available to treat progressive vision loss and blindness in these patients.
About the CELESTE trial (NCT07161544)
CELESTE is a Phase 1/2 multicenter, open-label, dose escalation study investigating safety, tolerability and preliminary efficacy of 3 dose levels of dual AAV8.ABCA4 (AAVB-039) administered subretinally in participants with Stargardt disease secondary to biallelic mutation of the ABCA4 gene.
About Stargardt disease
Stargardt disease is the most common form of inherited macular degeneration. Inherited in most cases as autosomal recessive, Stargardt disease is caused by mutations in the ABCA4 gene, leading to the accumulation of toxic retinoid byproducts in the retina and progressive vision loss. The ABCA4 gene is 6.8 kilobases in length, too large to be packaged within a standard, single AAV vector. Stargardt disease affects an estimated 60,000 to 75,000 individuals across the U.S. and E.U and currently there are no approved treatments.
About AAVantgarde
AAVantgarde is a clinical stage, biotechnology company advancing best-in-class therapies for patients with inherited retinal diseases. The company’s lead programs target Stargardt disease and retinitis pigmentosa due to Usher syndrome type 1B, two severe, inherited retinal diseases with no approved treatments. With a strong foundation in translational science and a commitment to clinical excellence, AAVantgarde is working to bring transformative therapies to patients. For more information, please visit: www.aavantgarde.com
Media Contact:
Barnaby Pickering – Director, 59 North Communications
Barnaby.Pickering@59north.bio
