Similarities among three pediatric brain tumors that arise in different structures of the CNS – pineoblastoma, retinoblastoma and Group 3 medulloblastoma – have been linked to their shared origin during pineal gland development. Scientists at St. Jude Children’s Research Hospital have identified the molecular signatures that drive these tumors from pinealocyte progenitor cells that conserve a common differentiation program, providing a shared therapeutic target for these three cancer types. Read More
The exact genetic and epigenetic cause of the sporadic form of amyotrophic lateral sclerosis (ALS), which affects approximately 90% of patients, are largely unknown. Previous work found that mitochondrial dysfunction and metabolic dysregulation are crucial to ALS pathophysiology. Read More
C-Further, an international consortium supporting new therapeutics for pediatric cancers, has unveiled the first early-stage therapeutic programs in its pipeline. The company said it is advancing CF-012 and CF-033 through its collaborative model. Read More
In Duchenne muscular dystrophy (DMD), deficiency of dystrophin leads to cardiomyocyte membrane instability, abnormal calcium influx, and progressive fibrotic remodeling of cardiac tissue. Histone deacetylase 6 (HDAC6) contributes to disease progression by regulating cytoskeletal dynamics and proteostasis in dystrophic muscle cells. Consequently, inhibition of HDAC6 represents a potential therapeutic strategy for addressing both the skeletal and cardiac manifestations of DMD. Read More
Pfizer Inc. has identified 4-{4-[(1-{[4-(propan-2-yl)phenyl]carbamoyl)-D-prolyl)amino]cyclohexyl}benzoic acid derivatives acting as gastric inhibitory polypeptide receptor (GIPR) antagonists. As such, they are described as potentially useful for the treatment of type 2 diabetes and obesity. Read More
The signaling axis orchestrated by OX40L-OX40 is a T-cell costimulatory pathway implicated in multiple autoimmune diseases, such as atopic dermatitis (AD). Antibodies targeting this pathway have proven useful at treating AD in the clinical setting. Earendil Labs recently presented data regarding HXN-1021, an anti-OX40L antibody with potent activity and extended half-life, in vivo. Read More
CSN5, a key COP9 signalosome subunit, regulates protein stability in the cell cycle, apoptosis and DNA repair. Its overexpression in cancer promotes tumor growth, metastasis and therapy resistance, making it a potential therapeutic target. Read More
Shionogi & Co. Ltd. has disclosed heterocyclic derivatives characterized as reverse transcriptase/ribonuclease H (HIV-1) inhibitors for potential use in the treatment of HIV infection. Read More
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized pyridone carboxamide-derived compounds acting as soluble epoxide hydrolase inhibitors. They are reported to be useful for the treatment of diabetic retinopathy, neuropathic pain and neurodegenerative diseases. Read More
Aiming to develop a vaccine candidate against human adenovirus-55 (HAdV-55) infections, researchers from the First Affiliated Hospital of Guangzhou Medical University (China) and collaborators have generated a replication-incompetent rAd55-5E4. Read More
Work at Shanghai 3D Medicines Laboratory Co. Ltd. has led to the discovery of new sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers designed for use in the treatment of pain. Read More
Nanjing Synnocare Pharmaceutical Technology Co. Ltd. and Shanghai Synnocare Biopharmaceuticals Co. Ltd. have patented ATP-dependent RNA helicase A (DHX9) inhibitors with potential for use in the treatment of cancer, autoimmune and viral infections. Read More
Atea Pharmaceuticals Inc. recently presented preclinical data on two of its compounds, AT-2490 and AT-587, which have shown promising antiviral potential for treating hepatitis E virus infection. Read More
