New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria. The advance opens the door to the development of programmable methods for rearranging DNA, using recombinase enzymes guided by RNA. The two different approaches to using insertion sequences (IS) – some of the simplest and most compact mobile genetic elements – are described in two papers published in Nature and Nature Communications. Read More
Transmembrane serine protease 6 (TMPRSS6) is a negative regulator of hepcidin, which is the main iron homeostasis-regulating hormone. Regeneron Pharmaceuticals Inc. has recently presented preclinical data for the monoclonal antibody targeting TMPRSS6, REGN-7999, which is being developed for the treatment of iron overload. Read More
CDR-Life Inc. has expanded its pipeline of novel T-cell engagers (TCE) with the addition of CDR-813 and CDR-505. CDR-813 is a highly potent and selective TCE candidate targeting tumors expressing PRAME (preferentially expressed antigen in melanoma) in HLA-A*02:01 patients. Read More
Defence Therapeutics Inc. has demonstrated that its second-generation ARM-X anticancer vaccine is therapeutically effective against pre-established ovarian cancer (ID8 model) when combined with an anti-PD-1 immune checkpoint inhibitor. Read More
The Centre National de la Recherche Scientifique (CNRS) and the University of Strasbourg have patented nonpeptidergic oxytocin receptor agonists potentially useful for the treatment of aging, alcoholism, pain and more. Read More
Nervgen Pharma Corp. has outlined its plans to develop its new drug candidate, NVG-300, with a focus on three initial indications: ischemic stroke, amyotrophic lateral sclerosis (ALS) and spinal cord injury (SCI). Read More
The generation of anti-citrullinated protein autoantibodies (ACPA) are known key drivers in the pathogenesis of rheumatoid arthritis (RA) and are generated by peptidyl arginine deiminases (PADs). Read More
A Leo Pharma A/S patent describes new interleukin-17 (IL-17) modulators reported to be useful for the treatment of psoriasis, psoriatic arthritis, and radiographic axial spondyloarthritis (ankylosing spondylitis), among other diseases. Read More
CSPC Pharmaceutical Group Ltd. has obtained clearance from China’s National Medical Products Administration (NMPA) to conduct clinical trials in China with the first mRNA-lipid nanoparticles (LNP)-based CAR T-cell injection, SYS-6020. Read More
Research at Idorsia Pharmaceuticals Ltd. has led to the discovery of aryl- and heteroaryl-sulfonamide derivatives acting as C-C chemokine receptor type 8 (CCR8) antagonists. Read More
CAR T-cell therapy that targets BCMA is an effective option for treating relapsed/refractory multiple myeloma, but there is a lack of persistence due to the inability to develop a memory phenotype. Read More
Fibrobiologics Inc. has announced the development of a novel artificial thymus organoid that can restore immune function in a severe combined immunodeficiency (SCID) mouse model. Read More
Gen1e Lifesciences Inc. has prepared new mitogen-activated protein kinase 14 (MAPK14; MAPK p38α) inhibitors reported to be useful for the treatment of cancer, inflammatory disorders, autoimmune diseases, viral infections, acute respiratory distress syndrome, amyotrophic lateral sclerosis, chronic obstructive pulmonary disease and cystic fibrosis. Read More
Janssen Pharmaceutica NV and Johnson & Johnson (China) Investment Ltd. patents disclose new menin (MEN1)/MLL interaction inhibitors reported to be useful for the treatment of cancer. Read More
