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).
Bloomsbury Genetic Therapies Ltd. has announced it received orphan drug designations from the FDA and the European Commission for BGT-INAD, an investigational gene therapy for the treatment of infantile neuroaxonal dystrophy (INAD).
Bloomsbury Genetic Therapies Ltd. has announced U.S. and E.U. orphan drug designations for BGT-NPC, an investigational gene therapy for the treatment of Niemann-Pick disease type C (NPC). BGT-NPC is an investigational AAV9 gene therapy designed to provide a potentially curative solution to NPC patients following a one-time injection in the cerebrospinal fluid.
Baudax Bio Inc.’s lead clinical candidate, TI-168, has been awarded U.S. orphan drug designation by the FDA for the treatment of hemophilia A with inhibitors. TI-168 is a next-generation, factor VIII (FVIII)-specific regulatory T-cell (Treg) therapy designed to address hemophilia A in patients with FVIII inhibitors.
Pepgen Inc. has received a no objection letter from Health Canada for its clinical trial application (CTA) to initiate a phase I study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). Initial data from the study are expected next year.
The FDA has awarded orphan drug designation to Healx Ltd.’s HLX-1502 for the treatment of neurofibromatosis type 1 (NF1). HLX-1502, discovered through the company’s proprietary artificial intelligence (AI) drug discovery platform, has a first-in-class mechanism and supporting data that indicate the potential for a good safety profile.
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