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).
Sironax Ltd. has divulged nicotinamide phosphoribosyltransferase (NAmPRTase; Nampt) activators reported to be useful for the treatment of cancer, neurodegeneration, inflammation, and mitochondrial, metabolic, cardiovascular, muscle and renal disorders, among others.
Ildong Pharmaceutical Co. Ltd. has identified novel tetraheterocycle compounds acting as GTPase KRAS inhibitors reported to be useful for the treatment of cancer.