In cell and animal models of amyotrophic lateral sclerosis (ALS), the expression of toxic dipeptides in neurons led to changes in the extracellular matrix (ECM) as a protective response. The authors wrote that their findings, which appeared in Nature Neuroscience on Feb. 29, 2024, could suggest new strategies for how to approach ALS. Read More
In allergic diseases, STAT6 is a critical transcription factor in the IL-4 and IL-13 signaling pathways and the key driver of Th2 inflammation. Because STAT6 functions through protein-protein and protein-DNA interactions, selectively and potently inhibiting STAT6 with traditional small-molecule inhibitors has been a challenge. However, it is well suited for a targeted protein degradation approach, whereby a binding event is adequate to direct degradation. Read More
Fractyl Health Inc. has reported promising new preclinical findings supporting RJVA-001, the first clinical candidate in its Rejuva pancreatic gene therapy platform. The company’s GLP-1 gene therapy candidate has potential to treat metabolic diseases, including obesity and type 2 diabetes. Read More
The activation of the NLRP3 inflammasome exacerbates neuronal dysfunction in several neurological diseases such as Alzheimer’s and Parkinson’s diseases, as well as multiple sclerosis. Targeting NLRP3 is an approach to overcome brain inflammation, among others. Read More
Lamassu Bio Inc. has been awarded a $2.05 million grant from the NIH and National Cancer Institute (NCI) for the development of SA-53 as a treatment for p53 wild-type sarcomas. SA-53 is designed to trigger the body’s natural defense mechanism p53 by blocking MDM2, a protein that deactivates p53 and contributes to treatment resistance. Read More
Hidradenitis suppurativa (HS) is an inflammatory skin disease with significant diagnostic delay. Type XXII collagen is a fibrillar collagen located in the skin epidermis. Reliable biomarkers to aid in the diagnosis of HS and monitor the severity of disease are needed. Read More
Glenmark Pharmaceuticals Ltd. has disclosed E3 ubiquitin-protein ligase CBL-B (CBLB) inhibitors reported to be useful for the treatment of cancer. Read More
TET2 is a master epigenetic enzyme that converts 5-methylcytosine (5-mC) to 5-hydroxymethylcytosine (5-hmC), reprograming tumor cells and causing them to enter a dormant state. Read More
Beijing Neox Biotech Co. Ltd. has synthesized proteolysis targeting chimeras (PROTACs) comprising a cereblon (CRBN)-targeting moiety covalently bound to a Bruton tyrosine kinase (BTK)-targeting moiety through a linker reported to be useful for the treatment of cancer. Read More
Agenebio Inc. has identified benzodiazepine derivatives acting as GABA(A) receptor subunits α5β3γ2 (GABRA5) positive allosteric modulators reported to be useful for the treatment of mild cognitive impairment, amyotrophic lateral sclerosis, schizophrenia, post-traumatic stress, autism, substance abuse and dependence, Alzheimer’s disease and Parkinson’s disease, among others. Read More
Researchers from Henan University and affiliated organizations disclosed the discovery of new bis-substituted aromatic amides as potent antibiotic agents. Read More
Recursion Pharmaceuticals LLC has described molecular glue degraders comprising DDB1- and CUL4-associated factor 15 (DCAF15) and RNA-binding protein 39 (RBM39) protein acting as RBM39 degradation inducers and RBM39/DCAF15 interaction inducers reported to be useful for the treatment of renal cell carcinoma. Read More
In a new study, researchers from Harvard Medical School and Regulus Therapeutics Inc. further investigated the role of miR-155 in Alzheimer's disease (AD). Read More
