The Mission

My mission is to develop a new class of gut-targeted therapies that restore natural biological function rather than managing symptoms. I am building a programmable gene regulation platform based on CRISPR-Cas9 using a non-DNA-cutting CRISPRa approach to selectively activate endogenous gene expression in intestinal epithelial cells. The initial focus is lactose intolerance, where the objective is to reactivate physiological expression of the LCT gene in the gut epithelium, thereby restoring the body’s own capacity to produce lactase at the site of digestion. This approach is based on the hypothesis that re-establishing endogenous gene activity may provide a more physiological and potentially durable alternative to exogenous enzyme supplementation. Delivery strategies are being explored using lipid nanoparticle systems inspired by clinically validated technologies such as Spikevax, adapted for intestinal targeting and epithelial uptake. Overall, this work aims to establish a modular framework for targeted gene regulation in the gastrointestinal tract, with lactose intolerance serving as an initial proof-of-concept for a broader class of functional gut disorders.

The Challenge

Many gastrointestinal conditions are currently managed through symptomatic, repetitive, or external interventions rather than by addressing the underlying biological dysfunction. Lactose intolerance is a clear example: patients typically rely on dietary restriction, lactase enzyme supplements, or lifestyle adjustments. These approaches can be useful, but they are temporary, variable, and dependent on continuous patient adherence. More broadly, the gastrointestinal tract remains a highly challenging therapeutic frontier. There is a need for technologies that can act locally, precisely, and controllably within the gut, while avoiding unnecessary systemic exposure. For gene-based medicines, the unmet need is not only the treatment of lactose intolerance, but the lack of validated platforms for safe, targeted, and reversible gene regulation in intestinal tissues.

The solution

I am developing a gut-targeted gene regulation platform based on CRISPR-Cas9 using a CRISPRa (non-cutting) approach to activate endogenous therapeutic gene expression in intestinal epithelial cells. My first application is lactose intolerance, achieved by reactivating the LCT gene to restore natural lactase production. Unlike current lactase enzyme supplements, which provide temporary and variable symptom relief, this approach aims to restore endogenous biological function and reduce dependence on repeated dosing. Due to the rapid turnover of the gut epithelium, the expected effect is durable but not necessarily permanent, enabling a controllable and potentially repeatable therapeutic modulation. This platform can be extended to other gastrointestinal diseases, shifting treatment from external supplementation to endogenous gene regulation.