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.

After 45 years as a domestic Japanese biopharmaceutical company, JCR Pharmaceuticals is expanding into global markets. The company is betting that its manufacturing strength – which helped land a COVID-19 vaccine manufacturing deal with AstraZeneca – and blood-brain barrier crossing J-Brain Cargo technology will lead to success in Western markets.   

A new batch of marketing authorizations in Japan includes new therapies for high-need cancer indications and rare diseases, and the first nod worldwide for a novel lysosomal disorder treatment that can address serious CNS symptoms.