enGene (NASDAQ:ENGN) Inc. is a clinical-stage biotechnology company focused on the development of gene-based therapies for cancer and ocular diseases. Founded in 2000 and headquartered in Cambridge, Massachusetts, with research facilities in Israel, enGene leverages proprietary drug‐delivery platforms to address high‐unmet‐need areas in oncology and ophthalmology. The company’s technology is designed to enhance the precision and durability of therapeutic agents by controlling the release and localization of nucleic acids in target tissues.
Central to enGene’s pipeline is the LOcal Drug EluteR (LODER®) platform, a biodegradable implant that provides sustained, site‐specific release of small interfering RNA (siRNA) within solid tumors. The lead oncology candidate, which targets mutant KRAS in pancreatic cancer, is advancing through clinical evaluation following promising safety and tolerability data. In parallel, enGene is developing a second LODER‐based therapy for head and neck cancer, as well as an ocular gene therapy program aimed at neovascular retinal disorders.
Beyond its implantable delivery system, enGene is advancing a suite of nanoparticle formulations designed to protect and transport messenger RNA (mRNA) and plasmid DNA to cells of interest. These formulations are being explored in preclinical studies for indications such as glioblastoma and age‐related macular degeneration. The company collaborates with leading academic institutions and contract research organizations across North America and Europe to accelerate translation of its proprietary platforms from bench to bedside.
Under the leadership of CEO Dan Rosenblum and a management team seasoned in pharmaceutical R&D, enGene is committed to expanding its global footprint and forging strategic partnerships. With operations spanning the U.S., Europe and Israel, the company aims to advance its novel therapeutics through clinical milestones and regulatory pathways, ultimately delivering targeted, durable treatments for patients with few options.
