Regulation of advanced therapies has come under scrutiny at a conference in London, as activity in the sector heats up amid Europe’s root and branch review of pharmaceutical legislation.

Circadian rhythm disorders are associated with psychiatric disorders including depression, bipolar disease and schizophrenia. To target casein kinase I isoform delta (CKI-delta), Janssen Pharmaceutica NV scientists sought potent, selective, brain-penetrant CKI-delta inhibitors which can be dosed daily and orally.

Mas-related G-protein coupled receptor X2 (MRGPRX2) is expressed particularly on mast cells and its activation leads to the mast cell degranulation with release of inflammatory mediators.

Atrial fibrillation (AF) is a very common diagnosed arrhythmia and catheter ablation is an effective treatment, but recurrence occurs after catheter ablation and remains a medical challenge. MicroRNAs can be easily detected in the circulation, and they are epigenetic regulators in many biological processes; their usefulness as biomarkers of risk stratification in patients with AF was investigated.

The crosstalk between immune cells and multiple myeloma (MM) cells is an important determinant of MM progression, but the mechanisms behind this have not been fully defined.

There is a lack of biomarkers for disease activity in aortic stenosis (AS). On the other hand, citrullination is involved in many chronic inflammatory processes and citrullinated proteins have been found within atherosclerotic plaque.

Researchers from Kira Pharmaceuticals LLC and University of Pennsylvania have developed a novel mouse model of rapidly progressing lethal C3 glomerulopathy (C3G), with the aim of assessing whether inhibition of proximal alternative pathway (AP) complement components such as factor D (FD) may be more efficacious than C5 inhibition for the treatment of C3G.