Methods of Diagnosing and Treating CHAPLE, A Newly Identified Orphan Disease: Collaborative Research and Licensing Opportunity

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. 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 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. 

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: 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; prabhuyo@niaid.nih.gov


Collaborative Research and Licensing 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; prabhuyo@niaid.nih.gov.

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NIAID Technology Transfer and Intellectual Property Office

NIAID’s technology transfer office, TTIPO, is a one-stop resource for organizations interested in partnering with NIAID to access, develop, and manage the translation of research discoveries into medically beneficial products. TTIPO seeks to expand NIAID’s innovation pipeline with existing and new partners in areas such as newly emerging and re-emerging infectious diseases (e.g., dengue, Zika, Ebola, influenza, methicillin-resistant Staphylococcus aureus and HIV/AIDS), biodefense (e.g., smallpox and anthrax), and immune-mediated diseases (e.g., asthma and allergy).

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