Kedrion SpA’s investigational plasma-derived treatment for congenital aceruloplasminemia has been awarded European orphan drug designation by the EMA. The company is working to advance this treatment toward clinical development in Europe.
Researchers from the Medical School of Nanjing University hypothesized that in ulcerative colitis, the gut-resident macrophages may be compromised, leading to impaired integrity of the epithelial barrier. “From a basic science standpoint, our work uncovers a novel etiology of ulcerative colitis. From a translational perspective, it identifies a promising therapeutic target, potentially paving the way for developing effective drugs to treat or even cure the disease,” senior author Minsheng Zhu told BioWorld.
Researchers from Stanford University have reported that inhibiting the enzyme 15-hydroxyprostaglandin dehydrogenase (15-PGDH) promoted cartilage regeneration in mouse models of osteoarthritis due to either aging or tissue injury. An oral version of the inhibitor that the team used is in a clinical trial for sarcopenia; it improved muscle mass and strength in preclinical studies. However, the mechanism by which 15-PDGH inhibition works appears to differ in the two conditions.
Brenig Therapeutics Inc. has described leucine-rich repeat kinase 2 (LRRK2; dardarin) and (LRRK2; dardarin) (G2019S mutant) inhibitors reported to be useful for the treatment of Parkinson’s disease, among others.
Hansoh Bio LLC, Jiangsu Hansoh Pharmaceutical Group Co. Ltd. and Shanghai Hansoh Biomedical Co. Ltd. have divulged transcriptional coactivator YAP1/transcriptional enhancer factor (TEAD) interaction inhibitors reported to be useful for the treatment of cancer.
Gluetacs Therapeutics (Shanghai) Co. Ltd. has synthesized molecular glue degraders comprising cereblon (CRBN) binding agents and protein degradation moieties reported to be useful for the treatment of cancer, infections, autoimmune diseases, anemia, transplant rejection, diabetes, cardiovascular disorders and inflammatory disorders, among others.
Blueprint Medicines Corp. has disclosed PROTAC compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to a cyclin-dependent kinase 4 (CDK4)-targeting moiety potentially useful for the treatment of cancer.
In an effort to identify stronger and safer α-glucosidase inhibitors, researchers at the Chinese Academy of Sciences prepared a series of diarylpentane derivatives, the most promising of which, [I], reversibly inhibited α-glucosidase with an IC50 of 18 µM.
In an effort to develop more effective estrogen receptor α (ERα) inhibitors, researchers at Nanjing University of Chinese Medicine and collaborators aimed to develop a proteolysis targeting chimera (PROTAC) against the receptor.
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