Brazil has established an accelerated pathway for approval of drugs for ultra-rare diseases, encouraging development activity – but the definition of qualifying drugs is narrow. Expansion to include more treatments could boost clinical trials activity and companies’ overall development presence.

A new publication offers up proof of concept that the genetic signatures of microorganisms found in blood and tumor tissue could improve precision medicine cancer diagnostics. The study also bolsters the notion that targeting the microbiome could lead to innovative cancer therapies.

A better understanding of heme trafficking could lead to drug targets in metabolic diseases, anemias, porphyrias, hemolytic diseases such as sickle cell disease – even parasitic infections.

A series of basic research insights around the cell death process called ferroptosis could form the underpinnings of a new class of treatments aimed at drug resistant tumors.

Recent clinical data using antisense drugs in neurological diseases are encouraging signs that targeting genetic drivers of ALS with antisense oligonucleotides or other gene-altering drugs could halt progression of several forms of the disease.

No NASH treatments are on the market yet, but Phase III data readouts are emerging, mid-stage drugs are demonstrating their capabilities and combinations are in view.