Proof of concept in human of inhibiting MASP2 for treatment of human diseases was already achieved by antibodies for the MASP2 protein in a number of diseases, including chronic diseases of the kidney and COVID 19, substantially de-risking LN-008 development. LN-008 is a siRNA inhibiting the MASP2 gene of the lectin pathway of the complement cascade, appropriately chemically modified (for efficient delivery to the liver). LN-008 is initially in development for underserved chronic kidney diseases: IgA Nephropathy and Lupus Nephritis.
LN-Cast will be initially used as improved cell therapy to treat cancer indications. Please link here for a brief technical review of the Castling Technology. The revolutionary tools available for gene editing (Gene Editing Technologies – GETs – such as CRISPR-Cas or TALEN, but not limited to those) as a technical platform.The pleiotropic effects that each individual miRNA has on multiple (up to hundreds) genes within a specific molecular pathway, thus regulating whole gene networks associated with a certain biological state of a cell.Broad and pivotal roles that microRNAs (miRNAs) play in cell biology and the recognition that miRNAs can be manipulated to treat diseases.Lepton’s current primary goal is improvement of Adoptive Cell Transfer (ACT), that is a cellular immunotherapy, a form of treatment that uses the cells of our immune system to eliminate cancer. LN-Cast is Lepton’s proprietary breakthrough general technology platform to manipulate microRNAs for the development of cell therapies for treatment of a range of serious diseases.
We currently focus on two programs that, each, opens-up opportunities to a multitude of treatments for different indications based on one gene or one technology. Leverage extensive expertise, know-how and intellectual property in manipulation of non-coding RNA (ncRNA) regulation to generate and develop breakthrough medicines that improve and change patients’ lives.
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