Modern molecular techniques have progressed to the point where sequencing can seem almost quaint. At the Basic Science Symposium of the American Association for the Study of Liver Diseases 2022 meeting (AASLD 2022), new techniques were on full display, with sessions devoted to epigenetics, microbiome analysis and spatial transcriptomics. But the first session was still on genetic variants in all their forms – rare variants, common variants and nongermline mutations. Read More
Targeted therapies and immunotherapies have revolutionized cancer treatment in recent years. However, achieving durable responses and, ultimately, curing metastatic cancers driven by intracellular oncogenes remains a primary unmet medical need. Although fragments of intracellular oncoproteins can act as neoantigens presented by the major histocompatibility complex (MHC), recognizing the typically minimal differences between oncoproteins and their normal counterparts makes this approach quite challenging. Read More
Inhibition of emerging polyclonal on-target acquired resistance mutations remains a critical unmet need in the treatment of fibroblast growth factor receptor 2 (FGFR2)-driven tumors. In the current study, researchers from Tyra Biosciences Inc. presented the preclinical characterization of a novel FGFR1/2/3 inhibitor, TYRA-200, being developed for the treatment of cancer. Read More
The soluble form of human angiotensin-converting enzyme 2 (hsACE2) could prevent SARS-CoV-2 from binding to the host cell receptors through competitive inhibition, which may avoid viral infection. However, the relatively short half-life of the recombinant hsACE2 limits its clinical application. Read More
Navrogen Inc. has entered into a cooperative research and development agreement (CRADA) with researchers at the U.S. National Cancer Institute (NCI), part of the National Institutes of Health (NIH). Under this CRADA, Navrogen will sponsor the clinical investigation of the experimental antibody-drug conjugate (ADC) NAV-001, under supervision of the NCI. Read More
Selenophosphate synthetase 1 (SEPHS1) plays an essential role in the metabolism of selenium and has ATPase activity that synthesizes selenophosphate from ATP and selenide. Researchers have hypothesized the potential involvement of SEPHS1 in genetic disorders. They presented a series of case reports involving 9 individuals with heterozygous missense variants in the SEPHS1 gene; all these variants resided in the C-terminal domain or near the AIR synthase-related domain of the gene. Read More
Olix Pharmaceuticals Inc. has obtained promising data with OLX-702-A, an investigational GalNAc-asiRNA therapeutic for the treatment of nonalcoholic steatohepatitis (NASH), in a nonhuman primate NASH model. Administration of OLX-702-A resulted in a significant reduction in liver fat content, measured by magnetic resonance imaging proton density fat fraction (MRI-PDFF). Read More
The University of Texas System and Massachusetts Institute of Technology (MIT) have synthesized transcription factor PU.1 inhibitors reported to be useful for the treatment of Alzheimer's disease. Read More
Researchers from Bern University Hospital and University of Bern presented data from a case study that identified the presence of anti-neurochondrin antibodies as a potential biomarker of primary autoimmune cerebellar ataxia (PACA). Read More
The Cleveland Clinic Foundation has disclosed citron rho-interacting kinase (CRIK; CIT) inhibitors reported to be useful for the treatment of medulloblastoma and prostate cancer. Read More
Spanish researchers at Hospital Universitario La Paz have aimed to investigate the correlation between extracellular vesicles (EVs) from the immune and nervous system with the diagnosis and disease activity of multiple sclerosis (MS). EVs were isolated and collected from 86 patients with MS and 29 healthy controls from their neurons, oligodendrocytes and B and T lymphocytes. Read More
Dimethylfumarate (DMF) is largely used for treating multiple sclerosis (MS), but about 61% of DMF-treated patients develop lymphopenia, with its consequent associated risks. Biomarkers for monitoring treatment efficacy with DMF in patients with MS would be helpful in this field. Read More
Sunovion Pharmaceuticals Inc. has divulged new trace amine-associated receptor 1 (TAAR1; TAR1) agonists and/or 5-HT2A and/or 5-HT7 receptor antagonists reported to be useful for the treatment of bipolar and autism spectrum disorders, schizophrenia, substance abuse and dependence, Alzheimer's, Parkinson's and metabolic diseases. Read More
Mirati Therapeutics Inc. has described GTPase KRAS (G12C mutant) inhibitors reported to be useful for the treatment of cancer, in particular non-small-cell lung cancer (NSCLC). Read More
Cyagen Biomodels LLC has established a strategic collaboration with Neurophth Therapeutics Inc. to codevelop next-generation AAV gene therapy vectors for specific types of genetic ophthalmic disorders. Read More
Sunovion Pharmaceuticals Inc. has identified substituted sulfonamide-chroman compounds reported to be useful for the treatment of essential tremor, among others. Read More
New and updated preclinical data presented at the Society for Immunotherapy of Cancer's Annual Meeting
in Boston, by: Actym, Calidi, Cantargia, Cytovia, Enara Bio, Immunome, Leap, Lyell Immunopharma, TCR2. Read More
