Alexion, Astrazeneca Rare Disease, part of Astrazeneca plc, has entered a definitive purchase and license agreement for a portfolio of preclinical gene therapy programs and enabling technologies from Pfizer Inc.
The non-receptor tyrosine kinase (TK) Src is the first oncogene ever identified. In tumors, Src hyperactivation is usually linked to the aberrant activation of upstream receptor tyrosine kinases (RTKs). In previous work, researchers from IRCCS-Fondazione Santa Lucia and collaborators developed a library of pyrazolo[3,4-d]pyrimidines inhibiting Src and other cytoplasmic TKs. Several compounds showed in vitro and in vivo activity in tumor models characterized by a deregulation of those kinases, such as osteosarcoma, neuroblastoma and glioblastoma.
Protein quality control research is “almost exclusively focused on heat shock proteins, which are ubiquitously present” up and down the evolutionary chain, Xiaolu Yang told BioWorld. But “for more sophisticated organisms, which we humans like to think we are, it’s a little odd that we still use the system that bacteria started with…. It seems like we should have something more. The TRIM system,” he added, “fills that gap.”
TRIMs or tripartite motif proteins are a group of quality control proteins that are found only in animals. One of their functions is to add ubiquitin tags to proteins, marking them for transport to the proteasome system. TRIMs are part of the innate antiviral defense system. But in the July 27, 2023, issue of Science, Yang, who is a professor of cancer biology in the Perelman School of Medicine at the University of Pennsylvania, and his colleagues reported that TRIM11 interacts with tau protein in multiple ways that were beneficial in preventing tauopathies.
Atai Life Sciences AG has described 5-HT2A receptor agonists reported to be useful for the treatment of substance abuse and dependence, treatment resistant depression, major depression, anxiety, eating, obsessive-compulsive and stress disorder.
Beigene Co. Ltd. has divulged antibody-drug conjugates (ADC) comprising HER3 (erbB3) targeting antibody linked to an effector molecule through a linker reported to be useful for the treatment of cancer, metabolic diseases, neurodegeneration and inflammation.
Hangzhou Adamerck Pharmlabs Inc. has disclosed uracil derivatives reported to be useful for the treatment of cardiovascular and cerebrovascular disorders.
Juvenile neuronal ceroid lipofuscinosis (JNCL) is an inherited neurodegenerative disease caused by mutations in the gene encoding CLN3 lysosomal protein. Contrary to late-infantile neuronal ceroid lipofuscinosis (LINCL), caused by mutations in the lysosomal protein tripeptidyl peptidase 1 (TPP1), no animal models or effective treatment exist for JNCL. Previous research characterized mouse models with either mutated Tpp1 or Cln3, but the phenotype of a double Cln3/Tpp1 mutant, as well as the function of CLN3 protein, remain unclear.
Insilicotrials Technologies SpA has entered into a collaborative partnership with Axoltis Pharma SA in the field of central nervous system (CNS) diseases, with the aim of optimizing the clinical development plan for Axoltis’ drug candidate, NX-210c.
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