Researchers from Johns Hopkins University, National Institutes of Health and University of Iowa have synthesized superstolide derivatives reported to be useful for the treatment of cancer and viral infections.

Writing in Molecular Therapy Nucleic Acids, researchers hypothesized that using poly(A) tail mimetics to enhance mRNA expression from haploinsufficiency-associated genes could be a disease-modifying treatment strategy.

Johns Hopkins University has discovered daunorubicin derivatives reported to be useful for the treatment of cancer, including glioblastoma, diffuse midline glioma and diffuse intrinsic pontine glioma.

Proteasome inhibitors such as bortezomib have shown promise in ovarian cancer treatment but limitations like route of administration or severe side effects restrict their use. Targeting proteasomal ubiquitin receptor ADRM1, also known as RPN13, has emerged as an alternative strategy and some RPN13 inhibitors are in early development.

Scientists at Johns Hopkins University and The Lieber Institute for Brain Development have identified peripherally restricted GABA(A) receptor subunits α3β2γ2S (GABRA3) positive allosteric modulators reported to be useful for the treatment of inflammatory bowel disease, lactose intolerance and abdominal pain.

Researchers from Johns Hopkins University and affiliated organizations have published details on the discovery and preclinical characterization of a purine antimetabolite, Pro-905, which is being developed for the treatment of malignant peripheral nerve sheath tumors (MPNST).

A new drug that inhibits the glutamate carboxypeptidase II (GCPII) enzyme could be used to treat inflammatory bowel disease (IBD), according to a new study in mice and human organoids. After decades of research trying to design GCPII inhibitors against neurological disorders, the new compound could be effective for another use.