Orphan designation: single guide RNA containing a sequence complementary to human ALB locus gene, intron 1, target region, Ziclumeran Treatment of haemophilia B, 25/03/2025 Positive

Orphan designation: single guide RNA containing a sequence complementary to human ALB locus gene, intron 1, target region, Ziclumeran Treatment of haemophilia B, 25/03/2025 Positive

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