enGene’s investigational therapy, detalimogene voraplasmid (“detalimogene” and previously EG-70) is a novel, non-viral gene therapy being studied in the LEGEND Clinical Trial to determine if it may help the body destroy cancer cells to ultimately either delay, or ideally, prevent bladder removal for people with non-muscle invasive bladder cancer (NMIBC).
Using our Dually Derivatized Oligochitosan® (DDX) platform, detalimogene is designed to elicit a local immune response following bladder instillation at the site of disease, which may result in therapeutic benefit while reducing undesirable systemic toxicity.
The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations to detalimogene, recognizing its potential to address serious unmet medical needs. These programs provide opportunities for expedited development and review.
Pipeline
LEGEND Clinical Trial
Detalimogene Voraplasmid (intravesical)
- Completed
- Active
Cohort
Pre‑clinical
Phase 1
Pivotal
COHORT 1: Pivotal:
BCG‑Unresponsive non‑muscle invasive bladder cancer (NMIBC) with CIS +/- papillary disease
COHORT 2a: BCG‑Naïve NMIBC with CIS*
COHORT 2b: BCG‑Exposed NMIBC with CIS*
COHORT 3: BCG‑Unresponsive NMIBC, Papillary‑Only*
DDX® Platform
Urological targets
ALL COHORTS ARE CURRENTLY IN PHASE 2 CLINICAL TRIALS.
*These additional cohorts of the LEGEND study provide further opportunity to demonstrate the potential use of detalimogene in clinical settings with unmet need.
All NMIBC cohorts refer to high‑risk NMIBC unless otherwise specified.
Expected data update, timelines, enrollment targets, and anticipated milestones reflect enGene management’s current estimate and are subject to change.
Detalimogene Clinical Program
THE LEGEND CLINICAL TRIAL
enGene’s investigational therapy, detalimogene voraplasmid (“detalimogene” and previously EG-70) is a novel, non-viral gene therapy being studied in the LEGEND Clinical Trial for patients with non-muscle invasive bladder cancer (NMIBC) whose cancer is considered high risk and has not responded to Bacillus Calmette-Guérin (BCG) therapy or are BCG-naïve or BCG-exposed, before bladder removal.
Patients suffering from high-risk NMIBC whose cancer is unresponsive to the standard of care, face high rates of disease recurrence (50–70%) and are often subject to full removal of the bladder (cystectomy) as a curative, but life-altering next step. Treating NMIBC is a multi-year journey managed mainly by community urologists, with the goal of treating multiple recurrences non-surgically to potentially delay radical cystectomy.
In 2025, bladder cancer was estimated to cause over 17,000 deaths in the United States. Each year, approximately 85,000 people are diagnosed with bladder cancer, and 75% to 85% of those cases are NMIBC. That translates to roughly 64,000 to 72,000 new NMIBC cases diagnosed in the U.S. annually.
Detalimogene Clinical Program
THE LEGEND CLINICAL TRIAL
enGene’s investigational therapy, detalimogene voraplasmid (“detalimogene” and previously EG-70) is a novel, non-viral gene therapy being studied in the LEGEND Clinical Trial for patients with non-muscle invasive bladder cancer (NMIBC) whose cancer is considered high risk and has not responded to Bacillus Calmette-Guérin (BCG) therapy or are BCG-naïve or BCG-exposed, before bladder removal.
Patients suffering from high-risk NMIBC whose cancer is unresponsive to the standard of care, face high rates of disease recurrence (50–70%) and are often subject to full removal of the bladder (cystectomy) as a curative, but life-altering next step. Treating NMIBC is a multi-year journey managed mainly by community urologists, with the goal of treating multiple recurrences non-surgically to potentially delay radical cystectomy.
In 2025, bladder cancer was estimated to cause over 17,000 deaths in the United States. Each year, approximately 85,000 people are diagnosed with bladder cancer, and 75% to 85% of those cases are NMIBC. That translates to roughly 64,000 to 72,000 new NMIBC cases diagnosed in the U.S. annually.
Advancing a powerful non-viral gene therapy option for patients with NMIBC
More than half a million new cases of bladder cancer are diagnosed each year globally and most new cases (75–85%) are non-muscle invasive bladder cancer (NMIBC). Patients with Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS), as well as those with BCG-unresponsive papillary-only NMIBC, are particularly underserved and have limited treatment options. Patients who fail BCG generally resort to having a radical cystectomy (bladder removal)—a life-altering, invasive and challenging procedure that significantly impairs patients’ quality of life.
Detalimogene has the potential to improve the standard of care for patients with NMIBC because of its optimized delivery to the bladder, safety in handling and ease of administration. It is designed with both patients and clinicians in mind—making treatment easier, faster and more practical for busy urology practices. Each dose takes just one hour of dwell time and requires no ultra-cold chain storage or special handling during preparation and administration. Unlike some therapies, there’s no need for urine bleaching or lengthy preparation steps, and no induction phase to manage. Because it’s made for off-the-shelf use, detalimogene fits naturally into existing clinic workflows, with minimal training needed for staff. It’s gene therapy built for the real world.
Proposed Mechanism of Action for an Investigational, Non-Viral Gene Therapy in NMIBC
How does detalimogene work?
Detalimogene is a novel, non-viral gene therapy designed to elicit a local immune response following the delivery to the bladder urothelium. This approach has the potential to induce a potent local immune response at the site of the tumor, resulting in greater therapeutic benefit while reducing undesirable systemic toxicity.
Detalimogene is currently being studied in the Phase 2 LEGEND Clinical Trial. At this point in time, enGene does not provide Expanded Access* to investigational medicine outside of the clinical trial program. In the event that enGene updates this policy, the Company webpage will be updated with additional details.
Additional information about the LEGEND Clinical Trial, including eligibility criteria, can be found on the LEGEND website or at clinicaltrials.gov.
*Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
*Investigational medical products have not yet been approved or cleared by the U.S. FDA and the U.S. FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.
Our programs, including detalimogene and the DDX® platform are investigational and have not been approved by the U.S. FDA or any other regulatory agency.
Once your bladder cells receive the study drug (1), the code-carrying molecules teach your bladder cells how to produce small proteins (2). Proteins tell your immune system its attention is needed in the bladder to fight the cancer (3). The immune system then launches an attack against the cancer cells (4).
How does detalimogene work?
Detalimogene is a novel, non-viral gene therapy designed to elicit a local immune response following the delivery to the bladder urothelium. This approach has the potential to induce a potent local immune response at the site of the tumor, resulting in greater therapeutic benefit while reducing undesirable systemic toxicity.
Detalimogene is currently being studied in the Phase 2 LEGEND Clinical Trial. At this point in time, enGene does not provide Expanded Access* to investigational medicine outside of the clinical trial program. In the event that enGene updates this policy, the Company webpage will be updated with additional details.
Additional information about the LEGEND Clinical Trial, including eligibility criteria, can be found on the LEGEND website or at clinicaltrials.gov.
Once your bladder cells receive the study drug (1), the code-carrying molecules teach your bladder cells how to produce small proteins (2). Proteins tell your immune system its attention is needed in the bladder to fight the cancer (3). The immune system then launches an attack against the cancer cells (4).
Our programs, including detalimogene and the DDX® platform are investigational and have not been approved by the U.S. FDA or any other regulatory agency.
*Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
*Investigational medical products have not yet been approved or cleared by the U.S. FDA and the U.S. FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.
