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Lepton and TAmiRNA collaborate on microRNA immune therapy discovery

Lepton Pharmaceuticals LTD (Lepton), a biopharmaceutical company, focused on discovery and development of novel treatments for unmet medical needs, exploiting the subtle silencing of coding RNA expression by noncoding RNA molecules microRNA (miRNA) and small interfering RNA, and TAmiRNA GmbH, a research and development company specialized in the discovery and validation of microRNA biomarkers to improve patients’ life, announce the reinforcement of their collaboration by signing a second agreement to discover the epigenetic pathways that affect the activity of microRNAs thereby improving genetically modified immune cells therapy.

Lepton together with Freiburg University is developing a technology (the Castling Technology) for enhancement of immune cells (T-cells or CAR T-cells, B-cells, natural killer (NK) cells, T-regulatory cells, macrophages, stem cells and their lineage cell types) efficacy by modifying expression of miRNAs, which impact immune cells activity upon contact with and activation by a cancer or virus targets. The Castling Technology addresses drawbacks of immune cells therapy to improve therapeutic effect. In particular, one of the major drawbacks of T-cell or CAR-T-cell-based immunotherapies is the cancer cells microenvironment’ s attempt to inhibit the T- cell’s effect, resulting in dysfunctional T-cell states such as anergy, tolerance, and exhaustion.  Exhaustion is a common issue in CAR-T cells (as well as other immune cells) that devastates their functions and is a major limitation to their efficacy whether autologous or allogeneic, and in the application of CAR immune cells for treatment of solid tumors. The Castling technology is a breakthrough novel approach that utilizes gene editing technologies like CRISPR/Cas and epigenetic regulations like DNA methylation to block inhibitory effects of cancer cells on immune cells activity by manipulation of specific miRNAs.  The identity and the number of such miRNAs may differ depending on tumor type and target cells, thus enabling a tailored made treatment to different types of solid tumors as well as blood cancers.

TAmiRNA is specialized in the discovery and validation of microRNA biomarkers to improve patients’ life. microRNAs are evolutionary conserved and act as crucial regulators of tissue function by regulating cell differentiation and tissue homeostasis. MicroRNA transcription and biological activity change in cells during the onset and progression of diseases. Passive or active release of miRNAs into the circulation enables non-invasive detection in liquid biopsies. This has led to the investigation and application of microRNAs as disease biomarkers and potential pharmaceuticals. As part of this research and development, TAmiRNA has been developing algorithms based on the logic of combination of microRNAs expression levels, cellular epigenetic changes as well as clinical parameters (expressing adverse events of diseases such as cancer and infectious diseases or effect of treatment response). The application of TAmiRNA´s sophisticated algorithms for selection of appropriate microRNAs in various cancer diseases suitable to be specially designed to Lepton’s Casting Technology, has created clear synergism between the two companies. This synergism, already results in developing of the first version of improved CAR T-cells for treatment of leukemia by Lepton.

“We are very pleased to collaborate with TAmiRNA GmbH, a pioneer in development of microRNA-based diagnostics. We believe microRNAs are going to make a very important impact in the field of diagnostics and pharmaceuticals. We are hopeful that this collaboration will position both TAmiRNA and Lepton as leading international players in development of microRNA-based diagnostics and pharmaceuticals” said Daniel Zurr, Ph.D., CEO, Lepton Pharmaceuticals

“We are excited to announce our new technology partnership with Lepton Pharmaceuticals. This collaboration marks a significant milestone in our mission to apply microRNAs in novel healthcare solutions. By combining our cutting-edge technologies for genome-wide quantification of microRNAs with Lepton Pharmaceutical´s expertise on CAR T-cells and genome engineering, we aim to accelerate drug discovery and bring innovative treatments to market.  Together, we are poised to make a substantial impact on global health and wellbeing.” Said Dr. Matthias Hackl, CEO&CSO, TAmiRNA

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