Uniqure NV is the latest firm to get caught between the FDA’s shifting demands for “gold standard” science and regulatory flexibility for rare disease therapies. The company disclosed in its latest earnings report that U.S. regulators are calling for a sham-controlled study before they will consider approval of gene therapy AMT-130 in Huntington’s disease, a requirement that could set the program back by two to three years and raises potential ethical issues.
Several neurodegenerative diseases, including Alzheimer’s disease, frontotemporal dementia and progressive supranuclear palsy, are classified as tauopathies due to the pathological accumulation of tau protein in specific brain nuclei. Researchers in Argentina have proposed the use of RNA interference (RNAi)-mediated therapies using viral vectors to target tau.
MicroRNAs (miRNAs) are small noncoding RNAs gaining increasing attention due to their crucial role in gene expression regulation and influence in various cellular processes and diseases. miRNAs can be encapsulated in extracellular vesicles (EVs), which are released by most cell types to modulate gene expression in recipient cells.
Regulus Therapeutics Inc. CEO Jay Hagan told investors in a January call the company had no interest in “simply out-licensing” rights to lead candidate farabursen, an oligonucleotide targeting autosomal dominant polycystic kidney disease set to start phase III testing on a path to a potential accelerated approval. And now, there’s no need to, as Regulus found a buyer for the whole company in a deal with Novartis AG valued at about $1.7 billion.
In an indication that has proved difficult for biopharma to conquer, Regulus Therapeutics Inc. disclosed further positive data from its ongoing phase Ib study testing RGLS-8429 in autosomal dominant polycystic kidney disease and laid out its plans to move straight into a phase III trial later this year, with the potential for an accelerated U.S. approval.
Cardiovascular disease is among the leading causes of death in individuals with type 2 diabetes. The role that microRNA 210 (miR-210) plays in endothelial cells and in diabetes-driven endothelial dysfunction is not clearly understood. Its potential as a therapeutic target was investigated.
Glioblastoma multiforme (GBM) recurs in most patients despite the aggressive therapies they receive. Novel advances allow the development of targeted therapies to treat tumors of the brain. Researchers from the Johns Hopkins School of Medicine have applied bioinformatics plus forward thinking on microRNA biology to advance targeted therapies for GBM.
Research into the regulation of gene expression experienced a significant breakthrough with the discovery of microRNA, small RNA molecules that do not code for proteins but control their translation. This finding has earned its discoverers – Victor Ambros and Gary Ruvkun – the 2024 Nobel Prize in Physiology or Medicine “for the discovery of microRNA and its role in post-transcriptional gene regulation.”
