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New siRNA Nanotherapy for Inflammatory Bowel Diseases, targeting Janus kinases

Project description

SiRNA-based Janus kinase inhibition in the treatment of inflammatory bowel diseases

Current treatments for inflammatory bowel diseases (IBD) are compromised by low efficacy and tolerability. The EU-funded NEW DEAL project aims to develop novel and radical therapies with improved efficacy and increased safety for all IBD patients, using specific Janus kinase 1 and 3 (JAK1 and JAK3) inhibitors. Previous studies have shown that siRNA-based treatments offer better tolerability, especially in terms of immunogenicity, and the convenience of oral administration. Focusing on the clinical translation of a product based on JAK inhibitors, the project aims to validate inhibition selectivity and safety. Moreover, it will address local delivery of the siRNA to the inflamed gut by the innovative nanostructured lipid carriers with polymeric capsules for protection during transit along the gastrointestinal tract.


Inflammatory bowel diseases (IBD) are the second most common immune-mediated disorders in Europe, affecting more particularly young people. The current therapies, including antibodies, show three main drawbacks: efficacy, tolerability and convenience. NEW DEAL solution will offer radical therapeutic progress for all IBD patients, thanks to the improved efficacy and increased safety of the specific JAK3 inhibition, which has been proven in clinics to be a target of great interest, the better tolerability of siRNA in term of immunogenicity and the good convenience with oral administration. To achieve this challenge, we will address three objectives: i) Specifically inhibit JAK 3 in a highly selective and safer manner by the mean of siRNA carefully designed and validated, ii) Deliver the siRNA therapeutic locally to the inflamed gut, by combining innovative nanostructured lipid carriers enabling their transport across the mucus, the intestinal barrier and the plasma membrane of the target cells, with polymeric capsules for protecting siRNA nanotherapeutics during their transit along the GI tract, thus allowing an oral administration, and iii) Promote the clinical translation and the future industrial transfer of this new clinical product, by addressing manufacturing, safety and efficacy evaluation at the late preclinical stage, to generate a Regulatory Submission Package and a Clinical Development Plan. The NEW DEAL project brings together clinical experts on IBD, leading scientists in nanomedicine, RNAi biology and immunology, innovative SMEs with a strong background in nanosafety, design of capsules and regulatory issues and an established pharma company with historic expertise on gastroenterology medicinal products. If successful, NEW DEAL will open new avenues for siRNA-based therapy in IBD with oral administration.

Call for proposal


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Net EU contribution
€ 1 915 936,25
Rue leblanc 25
75015 Paris

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Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Other funding
€ 0,00

Participants (13)