The EMA has awarded orphan drug designation to GC Biopharma Corp.’s intracerebroventricular enzyme replacement therapy (ERT) candidate, GC-1130A, for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A), developed in collaboration with Novel Pharma Inc.
Solid Biosciences Inc.’s SGT-003 has been granted orphan drug designation by the FDA. The company’s next-generation Duchenne muscular dystrophy gene therapy candidate was also granted fast track designation last month.
The FDA has awarded orphan drug designation to Ocelot Bio Inc.'s lead candidate OCE-205 for the treatment of ascites due to all etiologies except cancer. Ocelot Bio plans to initiate clinical studies of OCE-205 in refractory ascites next year.
Neuexcell Therapeutics Inc. has announced that NXL-004, an investigational AAV gene therapy product being developed for the treatment of malignant glioma, has been awarded orphan drug designation by the FDA.
Sernova Corp. has announced that its hemophilia A program, combining the Sernova Cell Pouch with a patient’s own cells corrected for the production of factor VIII (FVIII), has been awarded U.S. orphan drug and rare pediatric disease designations.
The FDA has awarded orphan drug designation to Neoimmunetech Inc.'s NT-I7 (efineptakin alfa, rhIL-7-hyFc) for the treatment of acute radiation syndrome (ARS). NT-I7, a novel long-acting recombinant human IL-7, has the potential to rapidly recover and improve the immune response following a high dose of radiation exposure.
Sirpant Immunotherapeutics Inc.’s lead product candidate, Sirpant-M (SI-101), has been awarded U.S. orphan drug designation for the treatment of T-cell lymphoma.
Epic Bio (Epicrispr Biotechnologies Inc.) has announced that EPI-321 has been awarded U.S. orphan drug designation for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
The FDA has awarded U.S. orphan drug designation and rare pediatric disease designation to Huidagene Therapeutics Co. Ltd.’s HG-204 (AAV-hfCas13Y-gMECP2), an RNA editing therapy based on CRISPR/Cas13Y, for the treatment of methyl-CpG binding protein 2 (MeCP2) duplication syndrome (MDS).
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