enGene is a clinical-stage biotechnology company focused on developing genetic medicines through its proprietary Dually Derivatized Oligochitosan (DDX) platform. The company's DDX technology enables the penetration of mucosal tissues and delivery of various types of genetic cargo, including DNA and RNA, to transform cells into therapeutic production factories that generate proteins and RNA for localized treatment effects without systemic toxicities. The company's lead product candidate, detalimogene voraplasmid (formerly EG-70), is being evaluated in a pivotal Phase 2 LEGEND study for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ. In preliminary data from the LEGEND study, detalimogene demonstrated a 71% complete response rate at any time point, with 67% response rate at three months and 47% at six months, while being well-tolerated with no treatment-related discontinuations. The company's product candidates are designed to deliver localized treatment specifically to target sites without the immunogenicity and systemic effects typical of viral vector-based genetic medicines. As of April 2024, enGene had USD 264.8 million in cash and cash equivalents.
Key functionality of the DDX platform includes its ability to enable carriers to penetrate mucosal tissue and deliver a wide range of cargo sizes and types, turning mucosal cells into therapeutic production factories that generate proteins and RNAs for desired local effects. The platform is designed to support localized treatment specifically to the target site without the immunogenicity and systemic effects typical of viral vector-based genetic medicines. The company is positioning detalimogene as a patient and practice-friendly option that can seamlessly integrate into medical practice.
Key customers and partnerships
The company's pipeline development has been supported through collaborations with several pharmaceutical and investment firms.
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