Shape Therapeutics Inc. could bring in as much as $1.2 billion in a new option and license deal with Vectory Therapeutics BV. It’s another collaboration for both companies that are known for working with large and small pharmas. Vectory is getting the exclusive option to evaluate Shape’s brain-penetrating adeno-associated virus capsid, SHP-DB1, against three targets, including mHTT, TDP-43 for Huntington’s disease and phosphorylated tau for Alzheimer’s disease.

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.

Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4.

Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4.

Remynd NV has turned in positive phase IIa data for a new target in Alzheimer’s disease, showing that restoring calcium homeostasis in neurons reduces levels of pathological tau proteins, increases dopamine levels and has a positive effect on cognition in symptomatic patients.

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.

Arrowhead Pharmaceuticals Inc. has filed a request for regulatory clearance in New Zealand to initiate a phase I/IIa trial of ARO-MAPT, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies, including Alzheimer’s disease.