Scientists at the Center for Genomic Regulation (CRG) have developed an AI-based tool to design thousands of sequences that regulate DNA. They have also synthesized these molecules, called enhancers, to control gene activation in mouse hematopoietic stem cells, which they have tested in vitro. Read More

Approximately 90% of all kidney cancers involve renal cell carcinoma, against which researchers are racing to find more effective therapies. One of the major challenges in treating this and other cancers is ensuring that the immune cells that infiltrate the tumor remain activated and mount effective responses. Read More

Ahead Therapeutics SL has received positive feedback from the EMA on its way toward initiating regulatory toxicology studies for its lead program in myasthenia gravis. The feedback supports the company’s scientific approach. Read More

UTR Therapeutics Inc. has submitted an IND application to the FDA for UTRxM1-18, a novel therapeutic approach for targeting c-MYC-driven cancers. Pending approval, a first-in-human phase I trial is slated to begin next year. Read More

The KRAS G12D mutation is the most common oncogenic KRAS variant, identified in approximately 34% of pancreatic ductal adenocarcinoma cases, 12% of colorectal cancers and 4% of lung adenocarcinomas. Read More

The progression of neurodegeneration during experimental glaucoma is tied to the early degradation of anterograde transport along retinal ganglion cells to central brain targets, which is followed by frank optic nerve degeneration. Read More

Aptevo Therapeutics Inc. is advancing APVO-711, its bispecific antibody targeting PD-L1 x CD40 that combines checkpoint inhibition with immune activation in a single molecule. Read More

Neuboron Medtech Research Institute Ltd. has disclosed boron-containing lipoic acid derivatives and vesicle forms acting as boron neutron capture therapy (BNCT) agents reported to be useful for the treatment of cancer. Read More

Claudin-6 (CLDN6) is a protein found in the tight junctions of epithelial cells to modulate their permeability and barrier function, among other actions. Read More

Individuals with autoimmune diseases affecting the thyroid can suffer eye complications in the form of Graves’ orbitopathy, in which inflammation in and around the orbit leads to eyelid retraction, eye bulging and other symptoms. Read More

Suven Life Sciences Ltd. has synthesized muscarinic M4 receptor positive allosteric modulators (PAMs) reported to be useful for the treatment of pain, psychiatric disorders and neurological disorders. Read More

Teijin Pharma Ltd. has identified crystalline forms of oxazepine derivatives acting as orexin OX2 receptor agonists reported to be useful for the treatment of narcolepsy, hypersomnia and sleep apnea. Read More

Drug resistance to epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors, occurring via secondary mutations or bypass pathways, is frequent among non-small-cell lung cancer patients. Read More

Northridge Health Group (Hong Kong) Co. Ltd. has divulged proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to a tyrosine-protein phosphatase non-receptor type 2 (PTPN2; TCPTP)/PTP1B-targeting moiety via a linker. Read More

Folate receptor α (FOLR1) is highly expressed in the surface of tumoral cells in several cancer types, while it shows limited expression in normal tissues. CSPC Pharmaceutical Group Ltd. has developed a next-generation antibody-drug conjugate (ADC) targeting FOLR1 – SYS-6041 – for the treatment of mid-to-low FOLR1-expressing tumors. Read More

Individuals with cystic fibrosis show a notably higher rate of attention deficit hyperactivity disorder (ADHD) symptoms than the general population. This association points to a possible role of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, whose mutations cause cystic fibrosis, in the development of ADHD. Read More

Shenzhen Hujia Technology Co. Ltd. has described retinol derivatives acting as nitric oxide (NO) production inhibitors reported to be useful for the treatment of acne, aging, skin inflammation and skin hyperpigmentation. Read More

Additional early-stage research and drug discovery news in brief, from: Avista Therapeutics, E-therapeutics, Kamari Pharma, Telomir Pharmaceuticals. Read More