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).
Tscan Therapeutics Inc. has received IND clearance by the FDA for TSC-200-A0201, a T-cell receptor (TCR) T-cell therapy targeting human papillomavirus 16 (HPV16), an oncogenic virus responsible for some cervical cancers and head and neck squamous cell carcinomas.
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.
Indapta Therapeutics Inc. has gained IND clearance from the FDA to commence a first-in-human phase I trial of IDP-023, an allogeneic natural killer (NK) cell therapy, in patients with relapsed or refractory multiple myeloma and lymphoma, anticipated to begin in the second half of this year. The study will explore three different dose levels of Indapta’s G-NK cells alone and in combination with IL-2 and the monoclonal antibodies rituximab and daratumumab.
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