About Us


About Lepton

Lepton Pharmaceuticals Inc. focuses on subtle and precise silencing of coding RNA expression by small non-coding RNA molecules, working via the DROSHA/DICER/RISC pathways
• Small interfering RNA (siRNA), and
• MicroRNA (miRNA)
to develop therapies for underserved diseases.
US patent applications were filed to protect aspects of the proposed technology and potential drug products.

Our Mission

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. 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.

Cell Therapy Program

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. 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. This program takes advantage of:

  1. Broad and pivotal roles that microRNAs (miRNAs) play in cell biology and the recognition that miRNAs can be manipulated to treat diseases.
  2. 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.
  3. 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.

Please link here for a brief technical review of the Castling Technology.

LN-Cast will be initially used as improved cell therapy to treat cancer indications. See initial Cancer indications by linking here

SiRNA Program

LN-008 is a chemically modified siRNA molecule to inhibit the gene MASP2. This is Lepton’s first siRNA- based drug candidate, in development for a range of indications. This program takes advantage of:

  1. The inherent advantages of siRNA molecules over antibodies for the same gene/protein.
  2. Substantially increased odds of success for LN-008 since MASP2 antibodies already demonstrated POC in human in several diseases.

LN-008 is initially in development for underserved chronic kidney diseases: IgA Nephropathy and Lupus Nephritis.

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). 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.

It is well accepted that a siRNA in general and especially those that target the liver has inherent advantages over antibodies for the same target.