Phosphatidylinositol 4,5-bisphosphate (PI(4,5)P2), also known as PIP2, is a cellular membrane phospholipid generated by phosphatidylinositol phosphorylation that regulates the functions of several proteins.

Bristol Myers Squibb Co. has exercised its option for an exclusive global license for PRX-019, a potential treatment of neurodegenerative diseases with an undisclosed target, under an agreement with Prothena Corp. plc.

Since utrophin compensates for lack of dystrophin in mdx mice, which results in a milder phenotype of muscular dystrophy compared to humans, the mdx/utrn-/- mouse has been developed to mimic early onset of muscle dystrophy, severe muscle weakness and premature death.

Scientists from the PsychENCODE Consortium have analyzed the brain transcriptome in a coordinated series of studies to map all the cell types, genes, epigenetic factors, and molecular pathways involved in different psychiatric disorders. After a first set of projects based on bulk analysis, the second phase of this project included 14 simultaneous publications that revealed the cellular atlas of post-traumatic stress disorder (PTSD) and major depressive disorder (MDD), among others.

Proof-of-concept murine data demonstrated that delivery of anticodon engineered tRNAs to muscle cells restored production of full-length, native dystrophin.

Understanding psychiatric disorders at a cellular and molecular level could provide a different perspective to design diagnostic and therapeutic tools searching for the origin of these disorders and the alterations they cause. Fourteen simultaneous studies from the PsychENCODE Consortium have delved into the cellular atlases of human neurodevelopment, reporting the broadest view of neuropsychiatry to date.

Degron Therapeutics Inc. has entered into a collaboration and exclusive license agreement with Takeda Pharmaceutical Co. Ltd. to discover and develop novel molecular glue degraders for multiple targets in oncology, neuroscience and inflammation.