Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by the loss of function of the maternally inherited ubiquitin E3 ligase UBE3A gene. AS is characterized by severe symptoms, including lack of speech, epilepsy, developmental and motor skills delays, sleep disturbances and cognitive impairment. Currently, no treatments are available for this disease.

NS Pharma Inc.’s NS-051 (NCNP-04) has been awarded orphan drug designation by the FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping.

Experts agree that the earlier Alzheimer's disease is detected, the sooner action can be taken. And so, the key to preventing deterioration is identifying the most effective early biomarkers, those that can spot the disorder and help halt its progression. Recent advances in the field have pushed a new era of early detection through blood-based biomarkers and personalized medicine strategies based on each patient’s genetic, immunological and clinical profile.

Vectory Therapeutics BV and Shape Therapeutics Inc. have entered into an option and license agreement granting Vectory an exclusive option to evaluate Shape’s deep brain penetrating AAV capsid, SHP-DB1, for vectorized antibody payloads against three therapeutic targets.

At the 2025 World Sleep Congress, researchers from Bioprojet SA, Harmony Biosciences Holdings Inc. and Teijin Pharma Ltd. presented preclinical efficacy data on BP1.15205, a novel, orally available OX2R agonist under development for the treatment of narcolepsy.

Coya Therapeutics Inc. has released results of a study designed to evaluate the effects of COYA-303 (low dose IL-2 and a GLP-1 receptor agonist) in an established in vivo lipopolysaccharide mouse model of systemic and neuroinflammation. COYA-303 is an investigational proprietary combination for subcutaneous administration, under development for the treatment of diseases driven by chronic and sustained inflammation.

Neural stem cell-derived exosomes (Nexo) are small, membrane-bound extracellular vesicles secreted by neural stem cells. Due to their role in cell-to-cell communication within the central nervous system (CNS), they are considered a promising strategy for modulating neuronal function and promoting repair processes in conditions such as stroke, traumatic brain injury and neurodegenerative diseases. Researchers at the Chinese Academy of Sciences developed an advanced Nexo platform incorporating ultrasmall nanoselenium via lipid-mediated nucleation, termed Senexo.

A tangle of DNA can look like a knotted ball in the cell nucleus. However, the genetic machinery has a complex and regulated structure. Its long repetitive sequences also seemed to have no function. They were called junk DNA, although they were not. The same happened with proteins and low-complexity domains, disordered chains of amino acids that were poorly understood. Nevertheless, that protein noise has turned into music for the 2025 Lasker Awards. These prizes have recognized the work of scientists who were able to see order in chaos.