X4 Pharmaceuticals, Inc.

Genzyme Corporation

Beth Israel Deaconess Medical Center, Inc.

Georgetown University

Robert D Arbeit

Paula Marie Ragan

Kieran James Crowley

Michael B Atkins

David McDermott

James W Mier

The present invention relates to methods of treating patients with advanced forms of cancer, such as clear cell renal cell carcinoma, in which X4P-001 is administered in order to reduce angiogenic escape that typically occurs with TKI therapy. The methods demonstrate surprising results, including regression of tumor size and cell number, with comparatively little toxicity.

Methods for treating cancer

The present invention relates to methods of treating patients with advanced forms of cancer, such as unresectable or metastatic renal cell carcinoma or kidney cancer, in which X4P-001 or a pharmaceutically acceptable salt thereof is administered as monotherapy or in combination with an immune checkpoint inhibitor, such as nivolumab. The methods demonstrate surprising results, including regression of disease, with comparatively little toxicity.

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.

Methods for treating immunodeficiency disease

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West Newton, MA, United States of America

Robert D Arbeit