WHIM Syndrome - Pipeline Insights, Recent Developments, Major Players and Review

The WHIM syndrome (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) is a rare disease after its significant symptoms. It is widely expressed on leukocytes and significantly impacts immune system homeostasis and organogenesis. The prevailing opinion holds that the reduced neutrophil count in the peripheral blood is due to the failure of myeloid cells to exit the bone marrow due to an abnormal CXCR4-based chemokine response rather than a fault in production. As a result, this disease is distinct from other congenital neutropenias because it is characterized by stimulating substances such as granulocyte-colony stimulating factors.

Precision medicine is exemplified by new treatments for the condition that use medications to inhibit CXCR4 function. Because CXCR4 and its ligand CXCL12 are involved in a wide range of infectious, inflammatory, autoimmune, and malignant disorders, the study of WHIM syndrome sheds light on both the physiologic and pathological functions of these molecules. By 30, more than 80% of patients have developed extensive HPV-induced warts that are sometimes difficult to treat and typically begin on the hands and feet.

Report Key Takeaways

Recommended Medications/Treatments for WHIM Syndrome

  • Plerixafor is an FDA-approved drug for mobilizing hematopoietic stem cells (HSCs) that have shown preliminary safety and efficacy in WHIM syndrome phase I clinical studies. In the mechanism-based therapy of WHIM syndrome, long-term treatment with plerixafor was proven to be efficacious and safe. Suppose CXCR4-specific nanobodies have the potential to be used as treatments for CXCR4-related disorders such as WHIM syndrome. It would appear necessary to compare and combine their efficacy with that of plerixafor.
  • Gene editing may represent a potential curative method for WHIM syndrome. Chromothripsis deletion of the disease allele in HSCs resulted in the clinical treatment of a patient as CXCR4 haploinsufficiency promotes engraftment of transplanted HSCs in mice.

Recent Developments Related to WHIM Syndrome

The Food and Drug Administration (FDA) has officially granted a Rare Pediatric Disease (RPD) Designation for mavorixafor, developed by X4 Pharmaceuticals, Inc. It’s claimed as the principal source of the effectiveness of WHIM syndrome. Mavorixafor is under comprehensive research to analyze its impact on the syndrome and how effectively it recovers the patient.

Major Players Working on WHIM Syndrome

X4 Pharmaceuticals, Myrtelle, Moderna, Molbio Diagnostics, Janssen, and Novartis are among the few leading companies that are investing heavily in their research and development programs working on the WHIM syndrome.

 

Key Reasons to Purchase This Report

  • The pipeline insight report gives a thorough overview of the syndrome in the discussion, emphasizing the disease basics, causes, diagnosis, complications, and effective treatments.
  • The report sheds light on the modern treatment practices, drugs in the development stage, and performance analysis for each available therapy coupled with a regional outlook for the disease in the discussion.
  • Trend analysis with comparative analysis of past and current trends, along with projections about the probable trends likely to influence the global landscape.
  • Accurate information and accurate statistics related to the historical and present patient pool for the target disease and scenario for all the major countries across the globe.

All the details featured in the report will help the reader understand the subject under discussion and have a clear view of its dynamics at the domestic and global levels.

A bone marrow biopsy can indicate myelokathexis, which, when combined with other symptoms, strongly suggests WHIM syndrome. Molecular genetic testing is performed to detect the syndrome in most of the patients.

The increase of mature neutrophils in the bone marrow causes WHIM syndrome, which results in neutropenia.

The WHIM syndrome mutation is inherited as an autosomal dominant characteristic, meaning that only one copy of a faulty gene is required for the condition to manifest. The faulty gene might be inherited from either parent, or it can be caused by a new mutation in the person who is affected.

WHIM syndrome is extremely uncommon, with just about 100 cases described in the medical literature worldwide to date.

Warts, hypogammaglobulinemia, infections, and myelokathexis are the four symptoms that most diagnosed individuals have encountered.

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Published Date:  Jun 2022
Category:  Pharmaceuticals
Report ID:   60573
Report Format:   PDF
Pages:   90
Rating:    4.7 (16)
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