Trace amine-associated receptor 1 (TAAR1) is a member of the G protein-coupled receptor (GPCR) TAAR family, which has been shown to be enriched in the central nervous system and periphery. TAAR1 can couple to diverse G protein subtypes, including Gs, Gq and Gi. Shandong University investigators have recently reported the discovery of a novel TAAR1 agonist as an antipsychotic drug candidate.
With the aim of overcoming drug resistance and reducing the toxicities associated with pan-fibroblast growth factor receptor (FGFR) inhibitors, scientists from 3H Pharmaceuticals Co. Ltd. developed a highly selective and potent small-molecule inhibitor of FGFR2, 3HP-2827, to be developed for the treatment of FGFR2‑driven solid tumors.
The FDA has granted orphan drug designation to the active ingredient in Soligenix Inc.’s Marvax, a heat stable subunit protein vaccine of recombinantly expressed Marburg marburgvirus (MARV) glycoprotein, for the prevention and post-exposure prophylaxis against MARV infection.
A research team at University of California, Los Angeles (UCLA), funded by Cancervax Inc., has created a promising new bispecific antibody vaccine for treating recurrent Ewing sarcoma.
Multiple myeloma (MM) stands as the second most common hematologic malignancy. Proteasome inhibitors are effective in MM, but many patients develop resistance, which is thought to be caused by mutations in the PSMB5 gene.
The autophagy process, a critical regulator of T-cell function, has been shown to control acute HIV-1 infection and play a crucial role also in HIV-1 disease pathogenesis.
Voyager Therapeutics Inc. has announced the selection of a lead development candidate in the GBA1 gene therapy program for the treatment of Parkinson’s disease and other GBA1-mediated diseases under its collaboration with Neurocrine Biosciences Inc.
Vaccination with infectious Plasmodium falciparum sporozoites (PfSPZ) administered with antimalarial drugs (PfSPZ-CVac) is more effective than vaccination with replication-deficient, radiation-attenuated PfSPZ. However, the requirement for drug administration is a significant limitation of the PfSPZ-CVac strategy.
An enzyme that activates cell death could be targeted to avoid the inflammation and lung lesions caused by influenza A virus (IAV). A collaborative study demonstrated that an inhibitor of receptor-interacting serine/threonine-protein kinase 3 (RIPK3) blocked necroptosis in infected alveolar epithelial cells and prevented the consequences in the lungs of severe disease.
Onkure Inc. has described phosphatidylinositol 3-kinase (PI3K) inhibitors reported to be useful for the treatment of cancer, congenital lipomatous overgrowth, vascular malformations, epidermal naevi and skeletal abnormalities and scoliosis (CLOVES syndrome), and PIK3CA-related overgrowth spectrum (PROS).