Monash University has identified cyclic peptides acting as melanocortin MC5 receptor (MC5R) agonists reported to be useful for the treatment of diabetes, obesity, cardiomyopathy, heart failure, renal disorders, Rabson Mendenhall syndrome, Donohue syndrome and lipodystrophy.
Linkcure Therapeutics has synthesized molecular glue degraders acting as zinc finger protein 803 (ZNF803; WIZ) degradation inducers reported to be useful for the treatment of sickle cell anemia and β-thalassemia.
Hanmi Pharmaceutical Co. Ltd. has disclosed polypeptides acting as triple agonists of glucagon like peptide 1 receptor (GLP-1R), glucagon receptor (GCGR) and glucose-dependent insulinotropic receptor (GDIR; GPR119) reported to be useful for the treatment of dyslipidemia.
Disruption of alternative splicing can generate abnormal mRNA variants, producing nonfunctional proteins or triggering nonsense-mediated decay, and is implicated in many splicing-related diseases. Therapeutic approaches that modulate splicing, therefore, hold promise to redirect aberrant transcripts toward normal isoforms and reestablish functional protein expression.
The treatment of Pseudomonas aeruginosa infections is significantly challenged by the pathogen’s diverse resistance mechanisms, with biofilm formation being a key driver of antibiotic tolerance. Furthermore, P. aeruginosa pathogenicity is amplified by virulence factors that both evade host defenses and facilitate biofilm development.
Adenosine deaminase acting on RNA (ADAR) is the enzyme responsible for adenosine-to-inosine (A-to-I) RNA editing, a process essential for regulating how cells respond to double-stranded RNA. ADAR1 generates two isoforms, p150 and p110, with distinct roles in cancer biology. Researchers from the Wistar Institute of Anatomy & Biology reported the preclinical characterization of ADAR1-i-124, an ADAR1 inhibitor designed for the treatment of cancer.
Enodia Therapeutics SAS has raised €20.7 million ($25 million) in seed financing to advance its work developing novel small-molecule therapies for targeted protein degradation at the point of synthesis.
The U.S. FDA has cleared Tessera Therapeutics Inc.’s IND application for TSRA-196, its lead in vivo gene editing therapy for α-1 antitrypsin deficiency (AATD). The Australian Human Research Ethics Committee (HREC) has also granted approval for the company to begin the phase I/II clinical study.
Keiferx LLC has entered into a research collaboration and option agreement with Amneal Pharmaceuticals Inc. to support the pre-IND development of KFRX-06, Keiferx’s brain-penetrant preclinical candidate designed to inhibit leucine-rich repeat kinase 2 (LRRK2), a gene strongly implicated in Parkinson’s disease biology.
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