Progress
PatientsAt X4, we are driven to successfully advance innovative therapeutics that truly make a difference in the lives of people with rare immunodeficiencies and few or no treatment options.
What’s it Like Living with Chronic Neutropenia?
Kevin’s Story
Vanessa’s Story
What’s it Like Living with WHIM Syndrome?
Gaby & Willow
Courtney’s Story
As seen on Medical Stories: Luke’s Story
Note: This segment was sponsored by X4 Pharmaceuticals, but was not created or produced by the company.
Our Clinical Trials
Chronic Neutropenic Disorders
- Chronic neutropenia is a rare blood condition defined by a decrease in blood neutrophil counts lasting more than three months; people with chronic neutropenia are at higher risk of developing infections and certain cancers and having a reduced quality of life.
- Following successful completion of Phase 1b and Phase 2 clinical trials exploring the use of once-daily oral mavorixafor in the treatment of certain chronic neutropenic disorders, we have initiated a pivotal, global Phase 3 clinical trial.
- The Phase 3 4WARD trial (NCT06056297) aims to evaluate the efficacy, safety, and tolerability of oral once-daily mavorixafor (with or without G-CSF) in people with congenital or acquired primary autoimmune and idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. For more information, please visit 4WARDstudy.com.
Community and Care Resources
Private Facebook group for people living with WHIM Syndrome. Search under Groups for WHIM Syndrome Community
The Immune Deficiency Foundation (IDF)
Not-for-profit patient advocacy organization that provides information and resources for people with primary immunodeficiencies (PIs)
International Patient Organization for Primary Immunodeficiencies (IPOPI)
IPOPI is an international not-for-profit organization that advocates for people with primary immunodeficiencies worldwide
The Jeffrey Modell Foundation (JMF)
Not-for-profit organization devoted to early and precise diagnosis, meaningful treatments and ultimately cures for people with primary immunodeficiencies.
National Neutropenia Network (NNN)
Not-for-profit organization that promotes awareness, education and research for people with severe chronic neutropenia and their families
National Organization for Rare Disorders (NORD)
A not-for-profit patient advocacy organization dedicated to improving the lives of people living with rare diseases and the organizations that serve them