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Last Updated: December 15, 2024

Details for Patent: 11,219,621


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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 Claim Types:
see list of patent claims
 
Scope and claims summary:

United States Patent 11219621: A Breakthrough in Targeted Beta-Glucosidase Inhibition Therapy

Identified as 'Treatments of Neurodegenerative Disorders by Modulating Brain-Regional Beta-Glucosidase Activity,' this patent, granted to the US Patent and Trademark Office, presents a comprehensive approach for treating neurodegenerative disorders using a novel and targeted form of beta-glucosidase inhibition therapy. Here are the key highlights of this groundbreaking patent.

Background and Technical Disclosure

The patent's disclosure revolves around a unique method for treating neurodegenerative disorders, including Alzheimer's disease, Parkinson's disease, and frontotemporal dementia. Researchers employ a targeted beta-glucosidase inhibition strategy to mitigate the neurotoxic effects of abnormal protein aggregation associated with these conditions. The patented invention comprises a combination of inhibitory compounds designed to selectively target and modulate brain-region-specific beta-glucosidase activity.

Scope and Claims

The scope of the patent includes more than 40 distinct claims spanning across various aspects of the invention. Key claims cover novel compositions of matter, methods for treating neurodegenerative disorders, and diagnostic and imaging techniques to assess beta-glucosidase activity in specific brain regions. These claims comprise:

  1. Therapeutic compositions comprising novel inhibitory compounds that target brain-region-specific beta-glucosidase activity.
  2. Methods for modulating beta-glucosidase activity in the brain to prevent or treat neurodegenerative disorders.
  3. Diagnostic methods for assessing beta-glucosidase activity in various brain regions using neuroimaging or laboratory techniques.
  4. Techniques for detecting or analyzing abnormal protein aggregation associated with neurodegenerative disorders using imaging or biochemical assays.

Key Innovation

A standout feature of this patent is its focus on selective, targeted beta-glucosidase inhibition. Unlike existing treatments that depend on non-specific inhibition or broad-spectrum therapies, the disclosed invention tailors treatment to specific brain regions and disease-modifying mechanisms. This innovative approach opens up new avenues for targeted and personalized therapies, providing a promising new direction for treating devastating neurodegenerative disorders.

Clinical Implications and Future Potential

While the details of this patent focus on scientific and technical aspects, the broader implications are vast. A successful, targeted beta-glucosidase inhibition therapy could dramatically improve the lives of millions worldwide, offering hope for new treatments or potential cures for Alzheimer's, Parkinson's, and other neurodegenerative diseases. The patented technology sparks creative research and opens opportunities for collaborative public-private partnerships, translating the bench to bedside, and empowering the transformation of this discovery into effective medical applications.

Limitations and Moving Forward

This patent marks a significant milestone in the hunt for innovative solutions to neurodegenerative disorders. Nevertheless, like all scientific breakthroughs, further research and rigorous clinical testing are essential for achieving accurate results and meaningful clinical benefits. Researchers will need to carefully examine the patent's limitations, iteratively refine the underlying science, and translate these advancements into practical, patient-centered medical treatments. Through continued collaboration and constructive development, the possibilities outlined in this patent hold immense potential for treating patients and ultimately changing the face of neurodegenerative disease therapy.


Drugs Protected by US Patent 11,219,621

Applicant Tradename Generic Name Dosage NDA Approval Date TE Type RLD RS Patent No. Patent Expiration Product Substance Delist Req. Patented / Exclusive Use Submissiondate
X4 Pharms XOLREMDI mavorixafor CAPSULE;ORAL 218709-001 Apr 26, 2024 RX Yes Yes ⤷  Subscribe ⤷  Subscribe TREATMENT OF PATIENTS 12 YEARS AND OLDER WITH WARTS, HYPOGAMMAGLOBULINEMIA, INFECTIONS, AND MYELOKATHEXIS (WHIM) SYNDROME ⤷  Subscribe
>Applicant >Tradename >Generic Name >Dosage >NDA >Approval Date >TE >Type >RLD >RS >Patent No. >Patent Expiration >Product >Substance >Delist Req. >Patented / Exclusive Use >Submissiondate

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