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<Sectiontitle="Abstract"id="Abstract">
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<pid="obenindescription">We are proud to introduce <PreCyse/>, our next-generation Prime Editing technology aimed at developing an innovative gene therapy for cystic fibrosis, specifically targeting the most common mutation, <b>F508del</b> of the CFTR gene. PreCyse is designed to address key limitations in current treatments, including limited size, speed, precision, and long-term effectiveness. Our approach integrates <b>PrimeGuide</b>, a highly optimized Prime Editing system, with <b>AirBuddy</b>, a novel lipid nanoparticle (LNP) delivery platform. The <b>SORT LNPs</b> used in AirBuddy are optimized for pulmonary delivery, offering precise organ targeting and structural stability throughout the inhalation process. As a Postdoc at the Broad Institute of MIT and Havard—where Prime Editing was first pioneered—observed, “<i>Everyone is looking for smaller prime editors</i>” and PrimeGuide embodies this vision by utilizing a smaller, more efficient editing complex. Unlike current treatments, which often require frequent administration and provide only temporary relief, PreCyse is developed as a monthly applied therapy with the potential for longer-lasting results, reducing the frequency of use and improving patient outcomes. By lowering costs and improving accessibility, PreCyse aims to offer a more advanced and user-friendly cure for cystic fibrosis. </p>
<pid="obenindescription">We are proud to present <PreCyse/>, a next-generation Prime Editing technology, as innovative gene therapy approach for cystic fibrosis
specifically targeting the most common mutation <b>F508del</b> of the CFTR gene. Cystic fibrosis is a severe disorder that primarily affects the lungs
and for which only short-term symptomatic treatments exist. PreCyse aims to provide long-term relief by delivering a small genetic payload with speed
and precision. Our approach integrates <b>PrimeGuide</b>, a highly optimized Prime Editing system, with <b>AirBuddy</b>, a novel lipid nanoparticle
(LNP) delivery platform. The <b>SORT LNPs</b> used in AirBuddy are optimized for pulmonary delivery, offering precise organ targeting and structural
stability throughout the inhalation process. Small payload size is key for effective delivery and compatibility with viral vectors, which have a
limited capacity. PrimeGuide embodies this vision by utilizing a smaller, more efficient editing complex. Current treatments often require daily
administration whereas PreCyse is currently developed as a monthly applied therapy. In addition, Prime Editing holds the promise to offer a causal cure,
when mutations are corrected in stem cells. Our approach aims to reduce medication frequency while improving patient outcomes.
By lowering costs and improving accessibility, PreCyse aspires to offer an advanced and user-friendly cure for cystic fibrosis.</p>
