Roche has synthesized pyruvate dehydrogenase kinase 1 (PDK1) inhibitors reported to be useful for the treatment of systemic lupus erythematosus, psoriasis, inflammatory bowel diseases, idiopathic pulmonary fibrosis, chronic obstructive pulmonary disease and cancer.
Blossomhill Therapeutics Inc. has identified macrocyclic compounds acting as GTPase KRAS (G12D mutant) inhibitors reported to be useful for the treatment of cancer.
Tuberous sclerosis complex (TSC) is a multisystemic disorder caused by loss-of-function mutations in the TSC1 or TSC2 genes. Its clinical phenotype is heterogenous, but most patients with TSC have epilepsy.
Qrons Inc. has established a collaboration with scientists at a public research university in Israel, by which Tellurium-based compounds in combination with Qrons' QS-200 product candidate and other configurations will be explored as treatment for diffused axonal injuries (concussions), which accounts for approximately 89% of traumatic brain injuries (TBIs).
Everest Medicines Ltd. has achieved a preclinical proof-of-concept milestone for its mRNA rabies vaccine program. This new vaccine candidate for rabies post-exposure prophylaxis was developed in partnership with Providence Therapeutics Holdings Inc. utilizing a clinically validated mRNA technology platform.
The HIV regulatory protein transactivator of transcription (Tat) is a viral protein believed to play a key role in the neurotoxicity and cognitive impairment seen in HIV-associated neurocognitive disorders. Tat allosterically modulates dopamine (DA) reuptake through the human DA transporter (hDAT). In the current study, researchers from University of South Carolina and affiliated organizations aimed to assess the effects of the novel allosteric modulator of DAT, SRI-32743, on the Tat-DAT interaction.
Abata Therapeutics Inc. has selected its first development candidate, ABA-101, an autologous regulatory T cell (Treg) therapy for the treatment of progressive multiple sclerosis (MS). ABA-101 targets MS patients with nonrelapsing progressive disease who have a DRB1*15:01 genetic haplotype and for whom imaging evidence of ongoing inflammatory tissue injury has been observed.
The hexanucleotide repeat expansion (HRE) GGGGCC in the noncoding region of the chromosome 9 open reading frame 72 (C9ORF72) gene is the most common cause of hereditary (40%) and apparently sporadic (5%-6%) amyotrophic lateral sclerosis (ALS).