Breaking Barriers
IN GENETIC MEDICINE
Expanding access through localized, non-viral genetic medicine
Our goal is to broaden the reach of genetic medicine by advancing non-viral treatments that can be delivered locally to the site of disease. Detalimogene voraplasmid (“detalimogene” and previously EG-70)–currently being studied for non-muscle invasive bladder cancer (NMIBC)–represents a new generation, a first-in-class, localized gene therapy with the potential to treat patients in a more targeted and convenient way.
Detalimogene
Our investigational therapy for NMIBC detalimogene, is a novel gene therapy that delivers a genetic blueprint to help the body recognize and attack bladder cancer. Developed using our proprietary Dually Derivatized Oligochitosan® (DDX) platform, detalimogene is part of a growing pipeline of innovative, non-viral genetic medicines advancing across urology and other serious diseases.
DDX
We designed our DDX platform to deliver genetic medicines directly to the bladder and other organs with the goal of creating new ways to address diseases with high treatment burden. By delivering therapeutic cargoes directly to these cells, we aim to achieve the desired effect and avoid the immune response and systemic side effects often seen with viral-based gene therapies.
Overcoming the limitations of viral genetic medicine through innovation and research
Mucosal tissues, such as the bladder, lungs and gut, make up a large part of the human body.
Although viral-based genetic medicines have shown great promise in helping patients, these therapies have left behind many patients suffering from illnesses that manifest in mucosal tissues, due to their inability to cross biological barriers. Mucosal tissues, such as the bladder, lungs and gut, make up a large part of the human body and serve as a critical first line of defense against pathogens—including viruses. This natural barrier function, while vital for protecting the body, can also make it difficult for locally administered therapies to reach and effectively treat diseases in mucosal tissues.
In addition to these delivery challenges, viral-based genetic medicines can carry the risk of systemic toxicities, limiting their use in certain patient populations or treatment settings.
We believe that non-viral genetic medicines have the potential to overcome limitations seen with viral genetic medicines, including limited efficacy, the inability to re-dose patients, safe handling and cold storage considerations, challenging administration paradigms and complex and expensive manufacturing processes.
Therapies being developed using the DDX platform are designed to turn mucosal cells into localized “factories” that produce therapeutic proteins and RNAs (ribonucleic acids)—molecules that play a key role in regulating gene expression and directing protein production—directly at the site of disease. This tissue-targeted approach potentially enables potent, localized effects while minimizing the immunogenicity and systemic effects often associated with viral-based genetic medicines.
Overcoming the limitations of viral genetic medicine through innovation and research
Mucosal tissues, such as the bladder, lungs and gut, make up a large part of the human body.
Although viral-based genetic medicines have shown great promise in helping patients, these therapies have left behind many patients suffering from illnesses that manifest in mucosal tissues, due to their inability to cross biological barriers. Mucosal tissues, such as the bladder, lungs and gut, make up a large part of the human body and serve as a critical first line of defense against pathogens—including viruses. This natural barrier function, while vital for protecting the body, can also make it difficult for locally administered therapies to reach and effectively treat diseases in mucosal tissues.
In addition to these delivery challenges, viral-based genetic medicines can carry the risk of systemic toxicities, limiting their use in certain patient populations or treatment settings.
We believe that non-viral genetic medicines have the potential to overcome limitations seen with viral genetic medicines, including limited efficacy, the inability to re-dose patients, safe handling and cold storage considerations, challenging administration paradigms and complex and expensive manufacturing processes.
Therapies being developed using the DDX platform are designed to turn mucosal cells into localized “factories” that produce therapeutic proteins and RNAs (ribonucleic acids)—molecules that play a key role in regulating gene expression and directing protein production—directly at the site of disease. This tissue-targeted approach potentially enables potent, localized effects while minimizing the immunogenicity and systemic effects often associated with viral-based genetic medicines.
Video
NMIBC and the Potential of Non-Viral Gene Therapy with Tom Jayram, MD
Tom Jayram, MD, discusses NMIBC management and the challenge of preserving quality of life while sparing the bladder and maintaining function, highlighting how non-viral gene therapy options may offer a more practical treatment approach for patients.
Video
Sandy’s Story: Navigating NMIBC and the Importance of Expanding Treatment Options
Sandy, an NMIBC survivor and advocate, shares her personal journey from diagnosis through treatment and bladder removal, highlighting the life-altering reality of losing an organ and her desire for new treatment options that can help future patients save their bladders.
Expanding genetic medicine to underserved diseases, including NMIBC
Non-muscle invasive bladder cancer (NMIBC) is a disease with a high burden for patients and limited treatment options.
The annual instance and impact of bladder cancer in the United States is alarming,
That translates to roughly 64,000 to 72,000 new NMIBC cases diagnosed in the U.S. annually.1
Alarmingly, half of patients treated for NMIBC experience recurrence and/or progression after first-line standard of care treatment with Bacillus Calmette-Guérin (BCG) and are subsequently considered BCG-unresponsive.2 Those who are unresponsive to BCG therapy are potentially subject to full removal of the bladder as a curative but life-altering next step.
Detalimogene—a potential new treatment
We are focused on developing a potential new treatment option—detalimogene, which is currently being evaluated in the pivotal Phase 2 LEGEND Clinical Trial for patients with BCG-unresponsive, high-risk NMIBC with carcinoma in situ (CIS) and patients with papillary-only NMIBC (without CIS). 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.
Based on its encouraging preliminary clinical activity, safety and ease of administration, we believe detalimogene has the potential to become a preferred option for a broad range of NMIBC settings.
Expanding genetic medicine to underserved diseases, including NMIBC
Non-muscle invasive bladder cancer (NMIBC) is a disease with a high burden for patients and limited treatment options.
The annual instance and impact of bladder cancer in the United States is alarming,
That translates to roughly 64,000 to 72,000 new NMIBC cases diagnosed in the U.S. annually.1
Alarmingly, half of patients treated for NMIBC experience recurrence and/or progression after first-line standard of care treatment with Bacillus Calmette-Guérin (BCG) and are subsequently considered BCG-unresponsive.2 Those who are unresponsive to BCG therapy are potentially subject to full removal of the bladder as a curative but life-altering next step.
Detalimogene—a potential new treatment
We are focused on developing a potential new treatment option—detalimogene, which is currently being evaluated in the pivotal Phase 2 LEGEND Clinical Trial for patients with BCG-unresponsive, high-risk NMIBC with carcinoma in situ (CIS) and patients with papillary-only NMIBC (without CIS). 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.
Based on its encouraging preliminary clinical activity, safety and ease of administration, we believe detalimogene has the potential to become a preferred option for a broad range of NMIBC settings.
KEY ATTRIBUTES OF DETALIMOGENE:
Promising Clinical ActivityObserved anti-tumor responses in clinical trials suggest robust complete response rates for patients with high-risk NMIBC.
Favorable Safety ProfileClinical studies have shown a generally well-tolerated safety profile, supporting its potential use in a broad set of clinical contexts.
Ease of Use for Urologists and PatientsAs an investigational, non-viral gene therapy, detalimogene requires no specialized handling or cold-chain storage—making it a potential “off-the-shelf” therapy that is well-suited for busy urology clinics.
High-risk NMIBC typically requires long-term management, often overseen by community urologists. The clinical goal is to manage recurrent disease with a combination of minimally invasive surgical and non-surgical approaches, while avoiding or delaying radical cystectomy (bladder removal surgery). Detalimogene is designed to activate a localized immune response in the bladder while minimizing the toxicities often associated with systemic immunotherapies. By driving expression of two double-stranded RNAs (ribonucleic acids)—molecules that play a key role in regulating gene expression and directing protein production—as well as the adaptive immune system through expression of the cytokine IL-12. This “one-two punch” has the potential to induce a potent immune response locally at the site of the tumor.
References:
1. National Cancer Institute. (n.d.). Surveillance, Epidemiology and End Results Program. Bladder cancer—Cancer Stat Facts. Retrieved August 11, 2025, from https://seer.cancer.gov/statfacts/html/urinb.html
2. Alhunaidi O, Zlotta AR. The use of intravesical BCG in urothelial carcinoma of the bladder. Ecancermedicalscience. 2019 Feb 26;13:905. doi: 10.3332/ecancer.2019.905
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.
