The company is focused on developing therapeutics for the treatment of rare diseases, including primary immunodeficiencies and cancer. X4P-001, the company's drug candidate, has completed a Phase 2 trial in patients with a rare genetic PI called Warts, while X4P-002 is a unique series of late lead molecules that penetrate the blood-brain barrier and will pursue early clinical development in glioblastoma multiforme. Additionally, the company is developing mavorixafor, a first-in-class CXCR4 inhibitor being developed as a once-daily, oral therapy for people with chronic neutropenic disorders. The company is currently evaluating the safety and efficacy of mavorixafor in a Phase 3 clinical trial for the treatment of people with WHIM syndrome and conducting a Phase 1b clinical trial of mavorixafor in people with idiopathic, cyclic, or congenital neutropenia.