enGene is pioneering advancements in gene therapy through its innovative Dually Derivatized Oligochitosan® (DDX) platform, which facilitates the localized delivery of non-viral gene therapies. This platform is designed to penetrate mucosal tissues, allowing for the direct administration of therapeutic agents such as DNA and RNA to target areas, thereby transforming mucosal cells into production sites for proteins and RNAs. This localized approach aims to mitigate the systemic side effects and immunogenicity often associated with viral-based gene therapies, making it particularly beneficial for treating diseases that manifest in mucosal tissues, such as non-muscle invasive bladder cancer (NMIBC).

One of enGene's key therapeutic candidates is detalimogene voraplasmid, a non-viral gene therapy specifically developed for NMIBC. This treatment is designed to elicit a localized immune response by instilling the therapy directly into the bladder, targeting the tumor site. By activating both the innate and adaptive immune systems, detalimogene aims to enhance therapeutic efficacy while minimizing systemic toxicity. The potential of this therapy is underscored by its preliminary safety and efficacy profiles, as well as its ease of administration by urologists, positioning it as a promising option for patients with limited treatment alternatives, particularly those unresponsive to standard therapies like Bacillus Calmette-Guerin (BCG).

Canada

enGene Inc. has developed a highly flexible nucleotide (DNA and RNAi) delivery technology that targetsmucosal tissues and can be used to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels. Our platform technology has the ability to significantly impact diseases of mucosal tissues such as the gastrointestinal tract, lung and bladder as well as provide systemic release of proteins from the gut to treat diabetes, anemia, hemophilia and others.

enGene

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