[Federal Register Volume 83, Number 169 (Thursday, August 30, 2018)]
[Notices]
[Pages 44281-44282]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-18779]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


Government-Owned Inventions; Availability for Licensing

AGENCY: National Institutes of Health, HHS.

ACTION: Notice.

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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; 
[email protected]. 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. 301-496-2644. 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unpublished patent applications.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 (Complement Hyperactivation, Angiopathic thrombosis, 
and Protein-Losing Enteropathy), 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

[[Page 44282]]

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; [email protected].

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; [email protected].

    Dated: August 18, 2018.
Suzanne M. Frisbie,
Deputy Director, Technology Transfer and Intellectual Property Office, 
National Institute of Allergy and Infectious Diseases.
[FR Doc. 2018-18779 Filed 8-29-18; 8:45 am]
BILLING CODE 4140-01-P