European Medicines Agency (EMA) | What's new Follow Orphan designation: peptide-oligonucleotide conjugate against CNOT3 mRNA Treatment of inherited retinal dystrophies due to mutation in the PRPF31 gene, 20/05/2026 Positive https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-26-3230 ema.europa.eu
