The past few months have seen the European Medicines Agency award its much sought-after priority medicines designation to treatments for Hunter syndrome, bubble boy disease and narcolepsy.

Among other Asia deals, Takeda adds to pair of recent gene therapy tie-ups with a CNS-focused cell therapy partnership with Immusoft; earlier it licensed ex-US rights to JCR’s Hunter syndrome drug.

Denali Therapeutics has reported promising early clinical data in Hunter syndrome for its fusion protein, DNL310, an investigational agent that crosses the blood-brain barrier and is expected to address disease-associated CNS effects; however, some biomarker data disappointed, and there are potential competitors also under development.

South Korean firm teams with Speragen to develop enzyme-replacement therapy for the genetic disorder and with Mirum for maralixibat. Plus deals involving Takeda/PeptiDream, Hanmi/LegoChem, Sun/Cassiopea, LegoChem/Cellectar, ChubLab/CJ CheilJedang Corp., Alebund/Chugai, Kintor/Fosun, Astellas/ExCellThera and I-Mab/neoX/Immorna.