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Monoclonal Antibodies for Rare Diseases

Posted Jul 20 2011 8:00pm

Description of Invention:
Available for licensing are three monoclonal antibodies (mAb) that bind with high specificity and affinity to the tumor cell surface antigen tyrosine kinase-like orphan receptor 1 (ROR1). ROR1 is expressed in chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL), two incurable B-cell malignancies that are designated as rare diseases by NIH’s Office of Rare Diseases Research. Therapeutics for rare diseases can qualify for orphan drug status and receive expedited review by the FDA. Currently, there are no therapeutic mAbs that target CLL or MCL but not healthy cells.

Investigators from the National Cancer Institute developed chimeric antibodies that selectively target ROR1 malignant B-cells but not normal B-cells. Additionally, this technology allows for mAb derivatives with potentially higher pharmacokinetic and/or pharmacodynamic activity, including humanized mAb in an IgG and IgM format, antibody-drug conjugates, immunotoxins, and bispecific antibodies. These three mAbs have been characterized in vitro for mediating antibody-dependent cellular cytotoxicity, complement-dependent cytotoxicity, apoptosis, and internalization. Results show that these mAbs bind with high specificity and affinity to three different epitopes on human ROR1, and ROR1-expressing primary CLL cells from untreated CLL patients and MCL cell lines. Moreover, as these antibodies selectively target ROR1, they can also be used to diagnose B-cell malignancies.

  • Antibody treatments for B-CLL and MCL
  • Diagnostics for B-CLL and MCL

  • Therapeutics that can qualify for an orphan drug status by the FDA and receive expedited FDA review
  • Antibodies that selectively target malignant B-cells and not healthy cells

Development Status:
The technology is currently in the pre-clinical stage of development.

Christoph Rader (NCI)
Jiahui Yang (NCI)

Patent Status:
HHS, Reference No. E-039-2011/0
US, Application No. 61/418,550 filed 01 Dec 2010

Relevant Publication:
  1. Yang J et al. Therapeutic potential and challenges of targeting receptor tyrosine kinase ROR1 with monoclonal antibodies in B-cell malignancies. PLoS ONE 2011;6(6):e21018. Epub 2011 Jun 15. [ PMID: 21698301 ]

Licensing Status:
Available for licensing.

Collaborative Research Opportunity:
The National Cancer Institute, Center for Cancer Research, Experimental Transplantation and Immunology Branch is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize anti-ROR1 monoclonal antibodies and their derivatives. Please contact Dr. Christoph Rader at (301) 451-2235 or for more information.

For Licensing Information Please Contact:
Jennifer Wong
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-4633
Fax: 301-402-0220

Ref No: 2283

Updated: 07/2011

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