Genetic Disorders

FDA grants orphan drug status to Ionis’ olezarsen for FCS treatment

The US Food and Drug Administration (FDA) has granted orphan drug designation to Ionis Pharmaceuticals’ olezarsen, an investigational ligand conjugated…

Kyowa Kirin acquires Orchard Therapeutics for $477.6m

Kyowa Kirin, a Japan-based global speciality pharmaceutical company, has announced the successful completion of its acquisition of Orchard Therapeutics, a…

Palvella and Ligand partner for Phase III rare lymphatic tumour trial

Palvella Therapeutics has partnered with Ligand Pharmaceuticals for the Phase III clinical trial investigating topical Qtorin rapamycin for the treatment…


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