AAVantgarde Bio presents positive data for retinitis pigmentosa and Stargardt disease therapies

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Clinical-stage biotechnology company AAVantgarde Bio announced safety and efficacy data from trials of two therapeutic candidates, both adeno-associated virus (AAV)-based treatments. The company, based in Milan, Italy, presented abstracts at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting in Salt Lake City, Utah. In a press release, AAVantgarde Bio shared information on the two studies, for therapeutic candidates AAVB-081 and AAVB-039.

AAVB-081 is designed for patients with retinitis pigmentosa (RP) related to Usher syndrome type 1 (USH1B). In the first-in-human phase 1/2 clinical trial, 8 patients were dosed in either a low-dose or medium-dose cohort. Efficacy data of the first subject dosed was provided at day 180. According to AAVantgarde, no serious adverse events or dose-limiting toxicities have been reported. Ocular inflammation was not severe and was controlled with steroids. The first patient treated with AAVB-081 showed >1 line improvement in best corrected visual acuity, and >3 lines improvement in low-luminance visual acuity. The patient also demonstrated positive results on additional functional endpoints, which were not specified in the press release.

In the preclinical AAVB-039 program, the candidate was assessed in a GLP-NHP study for its safety and pharmacokinetic profile. AAVB-039 is intended to treat patients with Stargardt disease via subretinal administration of dual AAV8.ABCA4. According to Jayashree Sahni, MD, EMBA, FRCOPHTH, chief medical officer of AAVantgarde Bio, the data from the preclinical programme demonstrated a positive safety profile. The promising outcome "supports the initiation of a first-in-human clinical study, providing hope for underserved Stargardt patients that don’t have any therapeutic options to prevent them from losing their sight," she said.

Francesca Simonelli, MD, head of the ophthalmology unit at the University Hospital of Campania “Luigi Vanvitelli” in Naples, Italy, also commented on AAVantgarde Bio’s announcement. Prof Simonelli serves as principal investigator of the LUCE-1 clinical trial for AAVB-081.

Ahead of the ARVO meeting, she said, “I am very excited to present updated positive safety results in 8 patients and day 180 results for our first subject dosed, showing preliminary efficacy signal. Through this innovative program, we aim to revolutionize our approach to understanding and treating these underserved patients.”

“We are poised to generate robust evidence that will not only advance scientific knowledge, but also directly impact patient care,” Prof Simonelli added.

Reference

1. AAVantgarde presents positive clinical data from its AAVB-081 program for Usher 1B and preclinical data from its AAVB-039 program for Stargardt at the ARVO 2025 annual meeting. Press release. AAVantgarde Bio. Published May 12, 2025. Accessed May 12, 2025. https://www.aavantgarde.com/en/news/aavantgarde-presents-updated-data-of-usher-1b-and-stgd-programs-at-arvo-2025/