Dtx Pharma Inc.’s Dtx-1252, an investigational small interfering RNA (siRNA) therapeutic, has been awarded U.S. orphan drug designation for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A).
Vanda Pharmaceuticals Inc.'s antisense oligonucleotide (ASO) VCA-894A has been awarded orphan drug designation by the FDA for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within IGHMBP2.
Antengene Corp. Ltd.'s Claudin 18.2 antibody-drug conjugate (ADC) ATG-022 has been granted two orphan drug designations consecutively by the FDA for the treatment of gastric cancer and pancreatic cancer.
Ocugen Inc.’s OCU-410ST (AAV5-hRORA) has been awarded orphan drug designation by the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19) and cone-rod dystrophy 3 (CORD3) diseases.
Researchers in Australia have developed new cancer cell line models for pediatric cancers with the lowest survival rates, with a focus on high-risk central nervous system tumors such as glioblastoma.
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