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.

Our Strategy

Focus initially on two projects that, each, is expected to reach proof of concept (POC) in a short period of time (8-10 months) and, each, opens up opportunities to a multitude of treatments for different indications based on one gene or one technology. Achieving such POCs will substantially increase the valuation of the Company and paves the way for many potential collaborative and licensing agreements on many different products (see Pipeline).

LN-008, a siRNA molecule to inhibit the gene MASP2. This program takes advantage of:

  1. An inherent advantage 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.
  3. Capitalizing on the potential fast FDA approval for the LN-008 in its first indication, treating COVID-19 patients.

LN-Cast, Lepton’s proprietary breakthrough general platform to manipulate microRNAs. This program takes advantage of:

  1. The new and revolutionary Crisper-Cas technology[i] that allows to precisely perform gene editing.
  2. The recent recognition that miRNAs not only are major players in many diseases, but also can be manipulated by the disease

LN- Cast will be initially used as improved cell therapy to treat cancer indications.

Our Current Focus

LN-008 is a siRNA inhibiting the MASP2 gene  developed, initially, as prophylactic /treatment for COVID-19 disease. Its development was prompted by the COVID-19 pandemic that created an acute need to develop drugs to combat the appalling consequences of the infection, particularly in high – risk patients, independently of the rapid mutations of the virus. LN-008 provides a safe approach for prevention of severe COVID-19 complications, by inhibiting components of lectin pathway of the complement cascade, a part of the immune system, overactivation of which is responsible for the vasculitis, hypercoagulation and long-term cardiovascular complications of the COVID-19 disease.  Proof of concept in human of inhibiting MASP2 for treatment of COVID-19 was already achieved by antibodies for the MASP2 protein. 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.  A patent application was filed.

Lepton estimates initiating clinical trials with the LN-008 within approximately one year under the Coronavirus Treatment Acceleration Program (CTAP) of the FDA Emergency Use Authorization (EUA) authority.   Early progress and potential EUA, will likely facilitate substantially de-risked development and approval of LN-008 for treating several serious underserved diseases (see Pipeline), a remarkable benefit to the Company.

LN-Cast, Lepton’s miRNA based Castling platform technology, is a proprietary, breakthrough technology for manipulation/engineering of miRNA expression by application of gene editing technologies (in particular CRISPER-CAS) to create products that modify gene expression ex vivo for use in cellular therapy, such as adoptive cell transfer -mediated therapies. The method is in essence, counter-manipulating/ modifying the MicroRNAs (miRNAs)’ expression patterns created by the disease, by editing their genetic loci, ex vivo in cells, such as T-cells, in order to enhance their functionality by simultaneously up-regulating a desired (“beneficial”) miRNA and shutting down/ down-regulating an undesired (“harmful”) miRNA in a single editing event.

A patent application was filed to cover the technology and for several miRNA combinations pursued by Lepton.

[i] https://explorebiology.org/learn-overview/genetics/crispr-cas:-from-bacterial-adaptive-immunity-to-a-genome-editing-revolution