We designed our breakthrough Dually Derivatized Oligochitosan® (DDX) platform to transform how gene therapies are accessed by patients and utilized by clinicians.
Breaking Barriers
in Genetic Medicine
Unlocking the promise of gene therapy for more patients with localized delivery of non-viral treatments
Overcoming the limitations of viral gene therapy
Mucosal tissues, such as the bladder, lungs, and gut, make up a large part of the human body.
Although gene therapies have shown great promise in helping patients, these therapies have left behind many patients suffering from illnesses that manifest in mucosal tissues because of biological barriers: For example, viral-based gene therapies are difficult to administer to a target tissue without also causing systemic toxicities.
Medicines developed with our DDX platform potentially overcome this and other limitations of viral-based gene therapies, including limited efficacy, the inability to re-dose patients, safe handling and cold storage considerations, challenging administration paradigms, and complex and expensive manufacturing processes.
Expanding gene therapy to underserved diseases, including NMIBC
Non-muscle invasive bladder cancer (NMIBC) is a disease with a high burden for patients and limited treatment options.
Patients suffering from NMIBC with carcinoma in situ (Cis) who are unresponsive to the standard of care, Bacillus Calmette-Guerin (BCG), face high rates of disease progression and recurrence, and are subject to full removal of the bladder as a curative but life-altering next step. We can improve the standard of care for patients with BCG-resistant NMIBC with Cis if we can safely and effectively deliver gene therapies to the bladder to recruit and train the immune system.
enGene’s therapeutic candidate in NMIBC, EG-70 (detalimogene voraplasmid), is a novel non-viral gene therapy designed to elicit a local immune response following bladder instillation at the site of disease.
EG-70 activates the innate immune system by driving expression of two double-stranded RNAs,as well as the adaptive immune system through expression of the cytokine IL-12. This “one-two punch” approach has the potential to induce a potent immune response locally at the site of the tumor, resulting in greater therapeutic benefit while reducing undesirable systemic toxicity. Based on its preliminary efficacy and safety profile and ease of administration by urologists, we believe EG-70 can benefit patients across a range of clinical settings.
