Give your opinion on a genetically modified medicine against Stargardt Disease

Give your opinion on a genetically modified medicine against Stargardt Disease

For each clinical trial application for the use of a genetically modified organism, the government organises a thirty-day public consultation, in accordance with the Royal Decree of 21 February 2005. As a citizen, you can give your opinion on this clinical trial application with a genetically modified medicine.

Stargardt disease (STGD1) is a hereditary autosomal recessive retinopathy. It is caused by mutations in the retina-specific ATP-binding cassette (ABC) transporter gene (ABCA4), located on chromosome 1. The age of onset and the rate of progression vary greatly: loss of central vision most commonly occurs in early childhood and young adulthood, but in some individuals it may occur later in adulthood.

Given the lack of available therapies, there is an unmet medical need in patients with STGD1.

In this clinical trial, the study drug AAVB-039 will be tested as a potential therapy for the treatment of patients with STGD1 through a single subretinal administration into the eye.

AAVB-039 is a gene replacement therapy that is being developed for the production of a functional human ABCA4 protein. Non-clinical pharmacology studies have shown that a single subretinal administration of AAVB-039 can reconstitute the full-length human ABCA4 protein in the retina of both small and large animals, including cynomolgus monkeys, whose eye is very similar to the human eye in terms of size, structure and presence of a macula.

The aim of this clinical trial is to assess the safety and tolerability and to determine the preferred dose of AAVB-039 in patients with STGD1.

The clinical trial will take place at the University Hospital in Gent.

How to give your opinion?

The public consultation runs from February 28^th to March 30^th 2026 inclusive. You can access the various data in the application file, as well as an online form to send your comments or ask your questions.

General information on GMO - public consultation.