Brightpath Biotherapeutics Co. Ltd.’s iPS cell-derived BCMA CAR-natural killer T cell therapy candidate has been awarded orphan drug designation by the FDA for the treatment of multiple myeloma.

GLIX-1 is a first-in-class small-molecule therapeutic targeting DNA repair vulnerabilities in cancer cells.

Diagonal Therapeutics Inc.’s DIAG-723 has been awarded orphan drug designation by the FDA for the treatment of hereditary hemorrhagic telangiectasia (HHT). Additionally, the EMA has provided a positive opinion for orphan drug designation, confirming that DIAG-723 meets the criteria for designation as an orphan drug in the E.U.