• Target disease: Post kala-azar dermal leishmaniasis (PKDL) and cutaneous leishmaniasis (CL)
  • Main partners (since project start): Amatsi Aquitaine, France; GeneDesign Inc, Japan; Nagasaki University, Japan; National Institutes of Health, USA; Ohio State University, USA; Osaka University, Japan;  PK/Pdesign Sas, France; US Food and Drug Administration, USA.
  • Project Start: June 2014
  • Funding: Department for International Development (DFID), UK; Global Health Innovative Technology (GHIT) Fund, Japan; Special Programme for Research and Training in Tropical Diseases (WHO-TDR).


Overall objective:

  • Demonstrate the suitability of an immunomodulator to stimulate the innate immune system to fight the parasitic infection responsible for CL


CpG-D35 is being developed as a combination therapy for the treatment of complicated cutaneous leishmaniasis and post-kala-azar dermal leishmaniasis (PKDL) in partnership with GeneDesign. Leishmania parasites are able to persist in host cells by evading or exploiting immune, mechanisms. Modulating the immune response with CpG oligonucleotides may improve the effectiveness of chemotherapies. This project aims to produce an immunomodulator to stimulate the innate immune system to fight parasitic infection, as an adjunct to drug therapy. 


A pre-clinical package enabling a first-in-human study (Phase I) will be performed in 2019. A meeting with the UK Medicines and Healthcare Products Regulatory Agency is planned for 2019 to discuss the pre-clinical development plan and clinical package.

Final results of the preclinical in vivo efficacy study showed an improved outcome for CpG-D35, either alone or in combination with pentavalent antimony (glucantime). These results supported the completion of the pre-clinical package and initiation of the preparation of clinical supplies for a Phase I study.  

Two studies, one in vitro and one in vivo, were initiated in 2016. The in vivo study aims to demonstrate if CpG-D35 – whether alone or in combination with antimonials chemotherapy – will lead to improved leishmania infection outcomes, compared with antimonials alone. Results are expected by mid-2017. The in vitro study aims to assess the stimulatory capability of CpG-D35 in both peripheral blood mononuclear cells and whole blood samples from patients with both CL, due to different leishmania species, and PKDL patients and to determine which host genes are modulated in these two conditions. Results are expected by the end of 2017.

IND-enabling pre-clinical safety prerequisites and service providers for entry into Phase I proof-of-concept clinical trials were identified.


Last update: August 2019