Claims for Patent: 11,219,621

Summary for Patent: 11,219,621

Title:	Methods for treating immunodeficiency disease
Abstract:	The present invention relates to methods of treating patients with WHIM syndrome or related disorders, such as myelokathexis, in which X4P-001 is administered in order to reduce the activity of CXCR4. The methods demonstrate surprising effectiveness, with comparatively little toxicity.
Inventor(s):	Arbeit Robert D., Ragan Paula Marie
Assignee:	X4 Pharmaceuticals, Inc.
Application Number:	US16804863
Patent Claims:	2. The method of claim 1 , wherein the X4P-001 or a pharmaceutically acceptable salt thereof is administered in a dose of about 400 mg per day in a single or divided dose.3. The method of claim 1 , wherein the patient exhibits warts.4. The method of claim 1 , wherein cells taken from the patient exhibit expression of a mutant form of CXCR4.5. The method of claim 1 , wherein cells taken from the patient exhibit increased expression of CXCR4.6. The method of claim 1 , further comprising the step of obtaining a biological sample from the patient and measuring the amount of a disease-related biomarker.7. The method of claim 6 , wherein the biological sample is a blood sample.8. The method of claim 7 , wherein the disease-related biomarker is circulating CXCR4 claim 7 , SDF-1α/CXCL12 claim 7 , or GRK3 (G protein coupled receptor kinase 3).9. The method of claim 2 , wherein the X4P-001 or a pharmaceutically acceptable salt or composition thereof is administered orally once per day.10. The method of claim 2 , wherein the X4P-001 or a pharmaceutically acceptable salt or composition thereof is administered orally twice per day.11. The method of claim 9 , wherein the patient is in a fasted state when the X4P-001 or a pharmaceutically acceptable salt thereof is administered.12. The method of claim 2 , wherein the patient originally exhibited ANC less than 600/μL and/or ALC less than 1000/μL before treatment with X4P-001 or a pharmaceutically acceptable salt thereof.13. The method of claim 2 , wherein the patient originally exhibited ANC less than 400/μL before treatment with X4P-001 or a pharmaceutically acceptable salt thereof on at least two independent blood samples collected over a period of up to 14 days.14. The method of claim 2 , wherein the patient originally exhibited ANC less than 400/μL and/or ALC less than 650/μL before treatment with X4P-001 or a pharmaceutically acceptable salt thereof on at least two independent blood samples collected over a period of up to 14 days.15. The method of claim 13 , wherein the method results in increases in ANC levels to at least about 600/μL on at least 85% of assessments.16. The method of claim 13 , wherein the method results in increases in ALC to at least about 1000/μL on at least 85% of assessments.17. The method of claim 13 , wherein the method results in improved levels of protective antibody in the patient in response to a vaccine.18. The method of claim 13 , wherein the method results in at least 50% less respiratory tract infections.19. The method of claim 13 , wherein the method results in increased levels of total circulating WBC claim 13 , neutrophils claim 13 , and/or lymphocytes to at least 1.4× baseline.