Orphan designation: mRNA encoding Cas12HF endonuclease, Single guide RNA against the human HAO1 gene Treatment of primary hyperoxaluria, 22/08/2025 Positive | European Federation of Internal Medicine

Submitted by Anonymous (not verified) on 10 April 2026 - 10:26

Orphan designation: mRNA encoding Cas12HF endonuclease, Single guide RNA against the human HAO1 gene Treatment of primary hyperoxaluria, 22/08/2025 Positive

Link:

https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3118

Source:

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