Pantherna´s scientific platform is based on two technological pillars, the PTXmRNA® and PTXΔLNP®, enabling optimized mRNA expression in the designated tissue for therapeutics purpose beyond vaccination. Novel PTXmRNA are destined for transient expression of the desired therapeutic protein of interest via various administration routes. Current investigational development program PAN004 employs a three lipid moiety to generate pharmacologically distinct PTXmRNA/cLNP002 for the intravenous route to target exclusively the vascular endothelium of the lung.
Various novel PTXΔLNP® formulations are currently under development to address a broad spectrum of therapeutic applications. Because of this, we also team up with academic expert partners to investigate their suitability for a given medical condition. We are convinced that mRNA technologies will become a powerful pharmaceutical modality and we are striving to identify new potent mRNA therapeutics for diseases with high unmet medical need, including rare disease.
Pantherna has developed cutting-edge genetic therapeutics through its PTXmRNA® and PTXΔLNP® technologies. PTXmRNA® is a proprietary technology by Pantherna, encompassing a sophisticated combination of flanking PTX_UTRs that have been carefully optimized to ensure robust target protein expression, specifically tailored for a diverse range of tissues and primary cell types in vivo. This tailored approach enhances target gene expression in the tissue and cell type of interest, emphasizing the company’s commitment to the development of potent mRNA therapies. In addition to the optimized UTRs, PTXmRNA® incorporates an optional optimized signal sequence for protein secretion, augmenting the efficiency of protein release from cells into the extracellular environment.
This feature further distinguishes PTXmRNA® from standard mRNA constructs, showcasing superior expression levels and demonstrating Pantherna’s dedication to pushing the boundaries of gene expression technology. Furthermore, PTXmRNA® is modified using state-of-the-art capping and base modification technologies ensuring high protein expression while reducing immune activation. Pantherna has secured intellectual property rights to protect the PTXmRNA® technology, ensuring the commercial safeguarding of its innovative features and unique capabilities. This commitment to IP protection not only signifies Pantherna’s leadership in mRNA therapeutics but also sets the stage for strong collaborations, partnerships, and the advancement of genetic medicine.
Complementing PTXmRNA® is PTXΔLNP®, an advanced technology that represents the next evolution in molecular therapeutics. PTXΔLNP® leverages next-generation PTXΔLNP® formulations designed to deliver PTXmRNA® with unprecedented precision to a diverse array of tissues and cell types. This precision is achieved through a modular lipid composition, allowing for the creation of novel LNPs with tailored properties.
These advanced PTXΔLNP® formulations cover “3-flavours” of LNPs, representing anionic (PTXaLNP®), neutral and neutralized (PTXnLNP®), or cationic (PTXcLNP®) surface charged formulations to cater to diverse therapeutic needs and preferences. PTXΔLNP® are ready for scalable manufacturing.
One of the key advantages of PTXΔLNP® is its versatility, applicable across different routes of administration. Whether intravenous, intramuscular, or other routes, PTXΔLNP® formulations offer consistent and efficient delivery of PTXmRNA®, underlining Pantherna’s dedication to providing solutions for a wide array of therapeutic applications. Much like PTXmRNA®, PTXΔLNP® also benefits from robust intellectual property protection. This combined approach of PTXmRNA® and PTXΔLNP® positions Pantherna at the forefront of genetic medicine, poised to shape the future of healthcare through pioneering advancements in mRNA therapies.