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.

The FDA has awarded orphan drug designation to Krystal Biotech Inc.’s KB-408 for the treatment of α1-antitrypsin deficiency (AATD).

Canariabio Inc.'s MAb-AR20.5 has been awarded orphan drug designation by the FDA for pancreatic cancer. MAb-AR20.5 is an IgG1k type murine monoclonal antibody that binds specifically to the circulating and tumor-associated antigen (MUC1) expressed on pancreatic cancer cells.

The FDA has granted orphan drug designation to Nuvectis Pharma Inc.'s NXP-800 for the treatment of cholangiocarcinoma.

Bloomsbury Genetic Therapies Ltd. announced that the FDA granted orphan drug designation for its investigational liver-targeted gene therapy, BGT-OTCD, for the treatment of ornithine transcarbamylase deficiency (OTCD).

Genprex Inc. announced that the FDA has granted orphan drug designation to the company's lead drug candidate, Reqorsa immunogene therapy (quaratusugene ozeplasmid, GPX-001), for the treatment of small cell lung cancer (SCLC). Reqorsa contains a plasmid that expresses the tumor suppressor gene TUSC2, the expression of which is reduced or not present in SCLC.

Satellos Bioscience Inc. has received orphan drug designation and rare pediatric disease designation from the FDA for SAT-3153 for the potential treatment of Duchenne muscular dystrophy (DMD). The first-in-class oral small-molecule therapeutic is designed to restore the innate muscle regeneration process independent of dystrophin and regardless of exon mutation status.