New paper out!

ESR10, Melita Kaltak is happy to share the latest breakthrough in our mission to combat Stargardt disease (STGD1). Our recent scientific article presents the remarkable versatility of QR-1011, an antisense oligonucleotide (AON), initially designed to correct splicing in the most common severe variant associated with STGD1. Here, QR-1011 showed significant splice correction abilities for two additional severe STGD1 variants.
This discovery offers the possibility for a broader therapeutic impact, potentially benefiting a larger group of STGD1 patients.