The oncofetal antigen 5T4 is a cell surface glycoprotein overexpressed in many solid tumors, while its expression in normal tissues is limited, making it an attractive target for cancer therapies. Its association with tumor progression and poor prognosis further supports its therapeutic potential.
The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic mutations in the FKRP gene.
KAYO-1732 is a novel, orally available, small-molecule inhibitor of the aldehyde dehydrogenase 1A3 (ALDH1A3) enzyme, being developed for the treatment of type 2 diabetes and cardiovascular disorders.
Researchers from Opko Health Inc. and Entera Bio Ltd. recently presented preclinical pharmacokinetic data on OPK-8801003, an oral GLP-2 analogue developed for the treatment of short bowel syndrome.
Resvita Bio Inc. has held a pre-IND meeting with the FDA for RVB-003, its lead investigational therapy for Netherton syndrome. The FDA’s feedback gives the company a pathway to submit an IND for RVB-003 in the first half of next year, with a clinical efficacy read-out anticipated by early 2027.
Aera Therapeutics Inc. has nominated AERA-109, a targeted in vivo CAR T therapy designed to treat multiple B cell-mediated autoimmune diseases, as the company’s first development candidate. AERA-109 leverages Aera’s proprietary targeted lipid nanoparticle (tLNP) delivery platform and CAR T technology.
Clarivate plc has unveiled the 2025 Citation Laureates. Widely considered a predictor of the Nobel Prizes, this recognition has highlighted the discovery of biomolecular condensates in chemistry and the innate immunity signaling pathway in physiology or medicine, as well as the identification of leukemia stem cells and ghrelin, the so-called hunger hormone.
The Royal College of Psychiatrists in the U.K. has published its first-ever guidance to support research into psychedelic drugs as therapies for conditions including treatment-resistant depression, substance abuse disorders and posttraumatic stress disorder, saying that in a fast-moving field there is a risk of jumping ahead of the evidence.
China Pharmaceutical University has patented non-ubiquitin proteolysis targeting chimera (NuTACs) comprising proteasomal ubiquitin receptor ADRM1 (ARM1; hRpn13) linked to programmed cell death 1 ligand 1 (CD274; PD-L1) and/or bromodomain-containing protein 4 (BRD4; HUNK1)-targeting moiety. They are reported to be useful for the treatment of cancer, eye disorders, viral infections, autoimmune diseases, inflammatory disorders and systemic inflammatory response syndrome.
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