C11550: Novel Target for Treatment of Autoimmune DiseasesNovelty:
Inhibition of this novel target has the potential to treat autoimmune diseases, as well as enhancing immunity against pathogen infections and tumor formation.
Value Proposition:
Currently, most immunosuppressive therapies non-specifically inhibit the immune system and leave the body susceptible to infection. Inhibition of this novel target addresses this perilous side effect by directly targeting the effectors responsible for human immune response and promotion of autoimmune diseases, while leaving the rest of the immune system intact to fight infections through enhanced cellular immunity pathways. Other advantages include:
• Novel target for drug development for patients with Th2 mediated autoimmune diseases like asthma, lupus etc..
• Drug development applications can be expanded to a range of non-viral and viral diseases.
• Implications in promoting anti-tumor immunity and improved efficacy of vaccines.
• Proof of concept studies in mice asthma model.
Technical Details:
Johns Hopkins University researchers have discovered a novel target which when inhibited selectively addresses Th2-mediated autoimmune disorders while enhancing Th1 mediated immune response to pathogen and tumor activity. The target is one of the many downstream components involved in the mammalian target of rapamycin (mTOR) pathway, a master regulator of T-cell differentiation. Knockout mice were engineered in which the protein was specifically deleted in CD4+ and CD8+ T cells. The results of biochemical experiments indicate that these cells adopt a Th1 phenotype even under Th2 skewing conditions. The cells inability to adopt a Th2 phenotype made the knockout mice resistant to induced allergic asthma. Additional studies suggest that these mice have increased anti-viral and anti-tumor immunity.
Looking for Partners:
To commercialize the technology by developing inhibitors to the novel target for long term treatment of asthma, as a combination with vaccines to boost their efficacy to prevent infection and promote anti-tumor immunity
Stage of Development:
Pre-Clinical
Data Availability:
Under NDA/CDA
Publications/Associated Cases:
Not available at this time