The human genome has yielded another round of secrets with the publication of two back-to-back papers in Nature on July 23, 2025. Both studies re-sequenced probands from the open-access 1000 Genomes Project, which was one of the first projects to sequence individuals from diverse populations. While one paper “goes very deep and tries to reconstruct a few genomes to basically near completion,” the other specifically looked at structural variants in a larger number of genomes. Together, they give new insights into genome variation. Read More
Alterations in the GABAergic pathway in the brain contribute to the pathophysiology of neurodevelopmental disorders, including autism spectrum disorder and Angelman syndrome. Targeting the GABA-A receptor α5 subunit (GABRA5) with positive allosteric modulators represents a new therapeutic strategy for the treatment of these conditions. Read More
Orbital Therapeutics Inc. has presented preclinical results supporting the development of OTX-201, a potential best-in-class in vivo CAR T therapy that comprises an optimized circular RNA encoding a CD19-targeted CAR delivered via targeted lipid nanoparticles. Read More
Rakovina Therapeutics Inc. has reported progress in its AI-driven KT-5000AI program, advancing the development of precision ATR (ataxia telangiectasia and Rad3-related) inhibitors designed to disrupt the DNA damage response (DDR) pathway in cancer cells. Through its collaboration with Variational AI Inc., Rakovina has evaluated a vast chemical space of potential molecular structures using Variational’s AI Enki platform to identify novel compounds Read More
Foresight Therapeutics (Hefei) Co. Ltd. has prepared and tested new cardiac myosin inhibitors reported to be useful for the treatment of hypertrophic cardiomyopathy. Read More
The U.S. FDA has granted Precision Biosciences Inc.’s PBGENE-DMD orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). PBGENE-DMD uses two complementary Arcus nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene in order to restore near full-length dystrophin protein within the body to improve functional outcomes. Read More
Superluminal Medicines Inc. has commenced IND-enabling studies for its lead program, an orally administered, highly selective, biased melanocortin MC4 receptor (MC4R) agonist initially targeting rare genetic forms of obesity and hypothalamic obesity, with potential as a treatment for general obesity in combination with GLP-1 drugs. IND submission is expected in the second half of 2026. Read More
CSPC Baike (Shandong) Biopharmaceutical Co. Ltd. has patented new polycyclic compounds acting as glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of Alzheimer’s disease, obesity, type 2 diabetes and nonalcoholic fatty liver disease (NAFLD; MASLD). Read More
The sodium-dependent phosphate transport protein 2b (NaPi2b), encoded by the SLC34A2 gene, is highly overexpressed in high-grade epithelial ovarian cancer and non-small-cell lung cancer while exhibiting minimal expression in normal adult tissues, making it a relevant tumor-associated antigen and a promising target for antibody-drug conjugates (ADCs). Read More
Shanghai Yuyao Biotech Ltd. has identified new signal transducer and activator of transcription 3 (STAT3) inhibitors reported to be useful for the treatment of cancer. Read More
E3 ubiquitin-protein ligase RNF31 inhibitors are disclosed in a Flindr Therapeutics BV patent as potentially useful for the treatment of cancer. Read More
Brain-derived neurotrophic factor (BDNF) is a neurotrophin that supports neuronal growth and survival and plays a critical role in regulating synaptic plasticity and cognitive function. Reduced BDNF levels have been observed in Alzheimer’s disease, Parkinson’s disease and mood disorders, suggesting that agents capable of enhancing BDNF expression may represent promising therapeutic strategies for both neurodegenerative diseases and depression. Read More
