RNA interference (RNAi), sometimes called gene silencing, is an approach to therapeutics in certain diseases that are caused by the production of abnormal proteins or an overproduction of proteins, which can be treated by turning off or silencing the gene. One of the companies working on the cutting edge of this field is Pasadena, California-based Arrowhead Pharmaceuticals, which is expecting 2020 to be a real inflection point.

The first wave of a new class of anti-aging drugs have begun human testing. These drugs won’t let you live longer (yet) but aim to treat specific ailments by slowing or reversing a fundamental process of aging. The drugs are called senolytics—they work by removing certain cells that accumulate as we age.

Rezafungin is a novel molecule in the echinocandin class of antifungals, which its developer Cidara believes will be an improvement among current fungal infections treatments. Rezafungin is being developed as a first-line treatment of candidemia and invasive candidiasis.

Zach, his family and other people suffering from rare diseases will be guests of honor at an event commemorating Rare Disease Day on Leap Day – the rarest day of the year – at Carnegie Hall. Rocket Pharmaceuticals, Inc., a company that researches genetic solutions for diseases like Zach’s, is organizing the event for the second year.

Q: Your experimental protocol was given as third-line treatment, is that correct? Most patients were on their third line of treatment, but inclusion criteria were beyond first-line standard of care.

A number of ingestible technologies have been developed for the diagnosis of gastrointestinal (GI) diseases, including the use of conventional tools, such as endoscopy, radiological imaging, or biochemical tests. Ingestible diagnostics are particularly useful for the exploration of the small bowelⁱⁱ, since gastroscopy can only reach a limited area of the small intestine and alternative methods with further reach, such as balloon enteroscopy, require specific expertise and infrastructure.