Although there are different methods of nuclear gene editing, there are still no effective treatments against mitochondrial disorders due to genetic alterations. Now, a group of researchers at Precision Biosciences Inc. and the University of Miami (UM) has developed a genetic edition platform that targets mitochondrial DNA (mtDNA) to delete its mutations.
“The ARCUS technology that we use is based on an enzyme found in nature called I-CreI. It is an enzyme that recognizes a 22 base pair DNA sequence within a species of green algae. And when it finds that DNA sequence, it will generate double-strand breaks,” first author Wendy Shoop, a scientist at Precision Biosciences, told BioWorld. Read More
Replay Holdings LLC and Miltenyi Biotec BV & Co. KG have announced a licensing and manufacturing agreement to support the development of a GMP-compliant T-cell receptor (TCR) natural killer (NK) cell therapy targeting the tumor-associated neoantigen, PRAME. Read More
Isozymes that are overexpressed in cancer and key in some metabolic processes are potential therapeutic targets. Previous studies found that phosphoenolpyruvate carboxykinase 2 (PCK2) is required by cancer cells for maintaining high metabolic activity and proliferation in some cancer types, but no small-molecule PCK2 inhibitors currently exist. Read More
Researchers from Zhejiang University and affiliated organizations have reported the discovery of novel orally bioavailable bromodomain-containing protein 9 (BRD9) proteolysis-targeting chimera (PROTAC) candidates for the treatment of acute myelocytic leukemia (AML). Synthesis and optimization led to the discovery of a novel series of BRD9 PROTACs, with compound [I] selected as the lead candidate with the best degradation and proliferation inhibition activities in vitro. Read More
Dice Alpha Inc. has disclosed IL-17A inhibitors reported to be useful for the treatment of psoriasis, radiographic axial spondyloarthritis (ankylosing spondylitis), hidradenitis suppurativa, spondyloarthritis, psoriatic and rheumatoid arthritis. Read More
A sustained antiplatelet effect plus target selectivity are the two major requirements for developing new antithrombotic therapies. Increasing the levels of cAMP in the platelets by the action of a prostacyclin receptor (PTGIR) agonist is a possible approach for this purpose. Researchers from the University of Michigan have presented preclinical data on their PTGIR agonist CS-585, which has shown higher blood stability, as a potential therapeutic for thrombosis. Read More
The Novo Nordisk Foundation is committing up to DKK1.8 billion (US$260 million) to establish a research and vaccine development initiative that aims to create new or improved vaccines for respiratory diseases, including tuberculosis (TB) and influenza. Read More
Shanghai Meiyue Biotech Development Co. Ltd. has identified serine/threonine-protein kinase SIK2 (QIK) inhibitors reported to be useful for the treatment of fibrosis, cancer, autoimmune diseases, transplant rejection, cardiovascular, dermatological, endocrine, and inflammatory disorders, among others. Read More
Neuexcell Therapeutics Inc. has announced that NXL-004, an investigational AAV gene therapy product being developed for the treatment of malignant glioma, has been awarded orphan drug designation by the FDA. Read More
The KCNC1 gene encodes the voltage-gated potassium channel subunit KV3.1, and variants in this gene have been previously linked to developmental and epileptic encephalopathy (DEE). Read More
Hangzhou Healthytide Biotechnology Co. Ltd. has synthesized peptoids acting as integrin α5β1 (ITGA5; VLA-5) antagonists reported to be useful for the treatment of cancer and macular degeneration. Read More
Biocytogen Pharmaceuticals (Beijing) Co. Ltd. has entered into an antibody evaluation, option and license agreement with Ona Therapeutics SL. Read More
Are you trying to stay on top of developments in CAR T therapies? Drug resistance? Emerging compounds? Updates from certain regulatory agencies? Want to be the first to learn about new biomarkers or business deals? You can still find these articles in the daily BioWorld e-newsletters or on the website, but if you’re laser-focused on a specific topic, we have a way to highlight the news you need as it happens, whether we’ve published a full-length feature story or a brief update. You asked and we listened. Now, in addition to the daily news lineup, you can create topic alerts to be delivered directly to your inbox or via an RSS reader. It’s easy to set up. Instructions can be found here: https://www.bioworld.com/featured-feeds. Happy holidays! Read More
