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.
Researchers at Abbvie Inc. and Calico Life Sciences LLC have described protein tyrosine phosphatase inhibitors, particularly tyrosine-protein phosphatase non-receptor type 2 (PTPN2; TCPTP) and/or PTPN1 (PTP-1B), reported to be useful for the treatment of non-small-cell lung cancer (NSCLC).
Qilu Regor Therapeutics Inc. has divulged protein-arginine deiminase type-4 (PADI4) inhibitors reported to be useful for the treatment of sepsis, cancer, bacterial, viral infections, inflammatory disorders, autoimmune diseases and metabolic diseases.
Insilico Medicine IP Ltd. has identified small-molecule inhibitors of ubiquitin carboxyl-terminal hydrolase 1 (USP1) reported to be useful for the treatment of cancer.
Bayer AG and Cedilla Therapeutics Inc. have entered into an exclusive license agreement to develop and commercialize Cedilla Therapeutics' cyclinE1/cyclin dependent kinase 2 (CDK2) complex inhibitors, which selectively address oncogenic drivers.
Medigene AG has announced its pipeline expansion into neoantigens with KRAS as the first target in its MDG-10xx program, a T-cell receptor engineered T cell (TCR-T) therapy being developed in combination with the company's PD1-41BB switch receptor technology.