2018-18779. Government-Owned Inventions; Availability for Licensing

AGENCY:

National Institutes of Health, HHS.

ACTION:

Notice.

SUMMARY:

The invention listed below is owned by an agency of the U.S. Government and is available for licensing to achieve expeditious commercialization of results of federally-funded research and development. Foreign patent applications are filed on selected inventions to extend market coverage for companies and may also be available for licensing.

FOR FURTHER INFORMATION CONTACT:

Yogikala Prabhu, Ph.D., 301-761-7789; [e-mail]. Licensing information and copies of the patent applications listed below may be obtained by communicating with the indicated licensing contact at the Technology Transfer and Intellectual Property Office, National Institute of Allergy and Infectious Diseases, 5601 Fishers Lane, Rockville, MD 20852; tel. [phone]. A signed Confidential Disclosure Agreement will be required to receive copies of unpublished patent applications.

SUPPLEMENTARY INFORMATION:

Technology description follows.

Methods of Diagnosing and Treating CHAPLE, a Newly Identified Orphan Disease Description of Technology

This technology is directed towards a potential treatment for a new disease, CHAPLE (C omplement H yperactivation, A ngiopathic thrombosis, and P rotein-L osing E nteropathy), identified by NIAID researchers. CHAPLE is associated with GI symptoms and vascular thrombosis and is caused by loss-of-function variants in the gene encoding the complement regulatory protein CD55. The disease is caused by enhanced activation of the complement pathway and complement-mediated induction of intestinal lymphangiectasia and protein-losing enteropathy. There is no current therapy for the newly described heritable genetic disorder and the symptoms are poorly controlled. CHAPLE is similar to other complement activating diseases that can be fatal, particularly for patients who develop severe thrombosis. Recent off-label use of a complement inhibiting drug, eculizumab (CD55 inhibitor) was shown to provide a dramatic benefit in patients with CHAPLE disease with an immediate correction of gastrointestinal protein loss. Thus, identification of CD55 deficiency in CHAPLE patients, and the possibility to use complement inhibitory drugs provide opportunities for treatment.

This technology is available for licensing for commercial development in accordance with 35 U.S.C. 209 and 37 CFR part 404, as well as for further development and evaluation under a research collaboration.

Potential Commercial Applications

• Diagnostic.
• Therapeutic.

Competitive Advantages

• There is no therapy currently approved for CHAPLE disease, and patients face a debilitating and often time fatal course of the disease.
• Anti-complement drugs (including eculizumab) has the potential to treat CHAPLE disease.

Development Stage

• Pre-clinical.
• Clinical.

Inventors

Dr. Michael J. Lenardo (NIAID), Dr. Helen Su (NIAID), Ahmet Ozen (NIAID), William A. Comrie (NIAID), Mr. Rico C. Ardy (CeMM, Austria), and Dr. Kaan Boztug (CeMM, Austria).

Intellectual Property

HHS Reference No. E-251-2016/0, U.S. Provisional Patent Application Number 62/394,630, filed September 14, 2016, and PCT/US2017/051413 filed September 13, 2017.

Licensing Contact

Yogikala Prabhu, Ph.D., 301-761-7789; [e-mail].

Collaborative Research Opportunity

The National Institute of Allergy and Infectious Diseases is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize the use of Eculizumab or other complement inhibitory drugs for the treatment of CHAPLE. For collaboration opportunities, please contact Yogikala Prabhu, Ph.D., 301-761-7789; [e-mail].

Dated: August 18, 2018.

Suzanne M. Frisbie,

Deputy Director, Technology Transfer and Intellectual Property Office, National Institute of Allergy and Infectious Diseases.