Companies in #Rare disease

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. With Phase 3 data showing a 'potentially best-in-class profile,' Arrowhead plans to submit plozasiran for FDA approval by year-end to rival Ionis's Tryngolza in severe hypertriglyceridemia.

Ashlins Pharma is a rare disease startup that has licensed a Chinese formulation of a well-known medicine and is pursuing a $50 million fundraise. Backed by Peter Thiel.