Abdera Therapeutics Inc.’s ABD-147 has been granted U.S. orphan drug designation for the treatment of neuroendocrine carcinoma. ABD-147 is a next-generation precision radiopharmaceutical biologic therapy designed to deliver actinium-225 (225Ac) to solid tumors expressing DLL3.

South Korean artificial intelligence-based drug developer Pharos Ibio Co. Ltd. said that the Ministry of Food and Drug Safety (MFDS) granted an orphan drug designation for PHI-101, a second-line therapy for acute myeloid leukemia (AML).

Vivet Therapeutics SAS has announced its gene therapy VTX-806 has been awarded European orphan drug designation for the treatment of cerebrotendinous xanthomatosis (CTX), a rare disorder of bile acid metabolism.

Skyline Therapeutics (Shanghai) Co. Ltd.’s SKG-1108, a novel one-time intravitreally delivered gene therapy, has been awarded U.S. orphan drug designation for the treatment of retinitis pigmentosa.

Invenra Inc.’s bispecific antibody, INV-724, developed for the treatment of neuroblastoma, has been awarded orphan drug and rare pediatric disease designations by the FDA.

Ractigen Therapeutics Co. Ltd.’s small activating RNA (saRNA) therapeutic, RAG-18, has been awarded U.S. orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy.

Actio Biosciences Inc.’s ABS-0871 has received both orphan drug designation and rare pediatric disease designation from the FDA. ABS-0871 is in preclinical development for the treatment of Charcot-Marie-Tooth disease subtype 2C (CMT2C).

Innorna Co. Ltd.’s IN-016 has received orphan drug status from the U.S. FDA for the treatment of progressive familial intrahepatic cholestasis (PFIC). Earlier in July, IN-016 had been granted Rare Pediatric Disease Designation. PFIC is a group of rare genetic disorders associated with defects in bile acid secretion or transport, resulting in unwanted bile accumulation within the liver.

Parvus Therapeutics Inc.'s PVT-201 has received orphan drug status from the U.S. FDA for the treatment of primary biliary cholangitis (PBC), an autoimmune disease of the liver.