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
