Engene Therapeutics Inc. · NASDAQ:ENGN
Engene Therapeutics — Technical Presentation (October 2026)
35 slides from Engene Therapeutics's technical presentation, Oct 15, 2026
Slide 1: Virtual KOL Event: Detalimogene voraplasmid with Surfactant
Non-Viral Genetic Medicine Virtual KOL Event: Detalimogene voraplasmid with Surfactant October 15, 2026
Slide 2: Disclaimers
Disclaimers Cautionary Statement Regarding Forward-Looking Statements This Presentation contains certain forward-looking statements within the meaning of the federal securities laws, including the Private Securities Litigation Reform Act of 1995, and "forward-looking information" within the meaning
Slide 3: Discussion Outline
Discussion Outline 01 02 03 04 Agenda & Opening Clinical Case Studies Preclinical Deep Dive Question & Answer Remarks Session Community Urology Key Detalimogene with Opinion Leaders Surfactant PRESENTER PRESENTERS PRESENTER PRESENTERS Ron Cooper Anthony Cheung, PhD Ron Cooper Susan Merrill, MD, FACS
Slide 4: Detalimogene: Investigational Next-Generation Therapy for NMIBC
Detalimogene: Investigational Next-Generation Therapy for NMIBC Expanding NMIBC Opportunity Scalable Non-Viral Platform More treatment options may increase sequencing and expand the Simplified storage / handling designed for broad community adoption treatable population Low-cost, readily available c
Slide 5: Key Takeaways: Eligible Patient Pool, Pricing, and Segmentation
Key Takeaways: Eligible Patient Pool, Pricing, and Segmentation • Eligible patient pool exceeds the annual BCG- ~20,000 eligible patients 2L Eligible Unresponsive incidence (i.e., 2L) 2L 3L Patients • Patients will sequence therapies creating a compounding effect on the eligible pool 2L 3L 4L • Rece
Slide 6: Clinical Case Studies
CLINICAL CASE STUDIES David Cahn, MD Suzanne Merrill, MD, FACS Colorado Urology Colorado Urology Director of Clinical Research Bladder Cancer Regional Leader
Slide 7: Colorado Urology
Colorado Urology Practice Overview COLORADO UROLOGY 30+ 18 ~130K UROLOGISTS LOCATIONS PATIENTS SERVED PART OF A LARGER NATIONAL GROUP United Urology Group: 200+ physicians and 50+ advanced practice providers CLINICAL RESEARCH CAPABILITIES REGIONAL EXPERTISE IN BLADDER CANCER Dedicated clinical resea
Slide 8: Case Study 1: Walter's patient overview
Case Study 1: Patient Overview, Medical History, Current Symptoms, and Personal Circumstances WALTER’S JOURNEY • 82-year-old Walter is retired • After his wife passed 2 years earlier, he moved into an apartment down the street from his daughter and 3 grandchildren • 80 pack-year history of smoking •
Slide 9: Walter's Journey
Walter’s Journey Presentation, Treatment Considerations, Recurrence, Next Steps JAN 2022 | INITIAL PRESENTATION AUG 2023–AUG 2024 | GEM / DOCE - Gross hematuria; positive cytology - Induction IVI ×6 CT normal - Monthly maintenance - Cystoscopy: diffuse erythema - Cystoscopies neg. between treatments
Slide 10: Case Study 2: Albert's patient overview
Case Study 2: Patient Overview, Medical History, Current Symptoms, and Personal Circumstances ALBERT’S JOURNEY 70-year-old newly diagnosed male Retired business executive who travels frequently and maintains active lifestyle • Advisory and board work • Visits grandchildren on both coasts • Plays gol
Slide 11: Albert's Journey
Albert’s Journey Presentation, Treatment Considerations, Recurrence, Next Steps JAN 2024 | INITIAL PRESENTATION JULY 2025 - NOV 2025 | GEM/DOCE - Gross hematuria - Induction IVI × 6 - Positive cytology; CT normal - 2x monthly maint. Cystectomy refused, Inlexzo - Cystoscopy: Multifocal lesions - Sign
Slide 12: Rationale Behind Detalimogene with Surfactant
Rationale Behind Detalimogene with Surfactant
Slide 13: Insights: Rationale, Evidence, and Next Steps for Surfactant Cohort
Insights: Rationale, Evidence, and Next Steps for Surfactant Cohort Scientific Preclinical Clinical Surfactant Efficacy and Selection Administration 13 Virtual KOL Event: Detalimogene with Surfactant October 2026
Slide 14: Scientific Rationale Supporting a Surfactant Bladder Rinse
Scientific Rationale Supporting a Surfactant Bladder Rinse Scientific Preclinical Clinical Surfactant Efficacy and Selection Administration 14 Virtual KOL Event: Detalimogene with Surfactant October 2026
Slide 15: Detalimogene: Designed for Bladder-Localized Immune Activation
Detalimogene: Designed for Bladder-Localized Immune Activation Synergistic Stimulation of Specific Innate and Adaptive Immune Responses Detalimogene Voraplasmid VA1 (RIG-I agonist dsRNA) Detalimogene Plasmid IL-12 eRNA11a nanoparticle (RIG-I agonist dsRNA) • Detalimogene delivers plasmid DNA encodin
Slide 16: Beyond Dose Escalation: Breaking the Sigmoidal Dose-Response
Beyond Dose Escalation: Breaking the Sigmoidal Dose-Response Urine IL-12 in Phase 1 LEGEND Detalimogene shows a sigmoidal dose–response, 1000 250ug/mL consistent with other NMIBC gene therapies 800ug/mL 2,500ug/mL Findings 100 • Sigmoidal dose-response in humans: Urine IL- 12 jumps from dose 250 to
Slide 17: Surfactants Increase Transfection of Gene Therapies
Surfactants Increase Transfection of Gene Therapies Preclinical Evaluation Before and After Surfactant Incorporation Cretostimogene1 Adstiladrin3 Cretostimogene-like2 virus: <5% transfection Adstiladrin-like virus4: Negligible transfection Bladders Harvested Bladder Surfactant (DDM) + Cretostimogene
Slide 18: Selection of Polidocanol for Surfactant Bladder Rinse
Selection of Polidocanol for Surfactant Bladder Rinse Scientific Preclinical Clinical Surfactant Efficacy and Selection Administration 18 Virtual KOL Event: Detalimogene with Surfactant October 2026
Slide 19: Selection Criteria: Commercial Availability and Extensive Safety Data
Selection Criteria: Commercial Availability and Extensive Safety Data ❑ Commercially Available: surfactant must already be commercially available to ensure immediate, uninterrupted supply ❑ Global Access: GMP-grade material must be manufactured at scale, at low cost of goods, and available in all re
Slide 20: Tested Multiple Agents Before Proceeding with Polidocanol
Tested Multiple Agents Before Proceeding with Polidocanol Part 1 Part 2 Transgene Expression in Bladder mIL12 protein expression in Bladder 6×10 5 500 Total Luciferase / Total Prot (mg) pg mIL-12 / mg total protein 400 4×10 5 300 200 2×10 5 N-9 supply chain 100 challenges 0 0 No 0.1% DDM 70kDa 150kD
Slide 21: Polidocanol is Commercially Available with Extensive Safety Data
Polidocanol is Commercially Available with Extensive Safety Data ✓ Commercially Available: surfactant must already be commercially available to ensure ❑ immediate, uninterrupted supply ✓ Global Access: GMP-grade material must be manufactured at scale, at low cost of goods, ❑ and available in all reg
Slide 22: Efficacy, Safety, and Administration for Detalimogene with Surfactant
Efficacy, Safety, and Administration for Detalimogene with Surfactant Scientific Preclinical Clinical Surfactant Efficacy and Selection Administration 22 Virtual KOL Event: Detalimogene with Surfactant October 2026
Slide 23: Surfactant Enables Detalimogene to Reach Deeper Urothelial Cells
Surfactant Enables Detalimogene to Reach Deeper Urothelial Cells Detalimogene Alone Detalimogene with Surfactant Deeper urothelial penetration lets nanoparticles reach intermediate and basal cells, which have significantly higher transfectability • Cells near the basal lamina are more metabolically
Slide 24: Surfactant Rinse Increases Gene Expression 10X and Improves Efficacy
Surfactant Rinse Increases Gene Expression 10X and Improves Efficacy Preclinical Murine Models Increases Gene Expression Improves Efficacy Curing Rate Time course of mIL-12 expression 1000 1000 50 No pre-Tx 0.25% polidocanol (5 min) % Survived at Study End mIL-12 / mgprotein 40 pg IL-12/mg protein p
Slide 25: Surfactant Rinse May Reduce Patient Burden by Reducing Dwell Time
Surfactant Rinse May Reduce Patient Burden by Reducing Dwell Time Surfactant Bladder Rinse Improves Gene Expression at Shorter Dwell Times Shortens Bladder Dwell Time 1000 Shorter Dwell Time pg IL-12/mg tissue protein 750 500 Patients: preferred in patients with 250 irritated or overactive bladders
Slide 26: Proven Anticancer Effect: Same Targets Upregulated by BCG
Proven Anticancer Effect: Same Targets Upregulated by BCG IL-12 IFN- VA1 (RIG-I agonist dsRNA) CXCL-10 RIG-I IL-12 eRNA11a Agonists (RIG-I agonist dsRNA) Effector molecules Established Effector Molecules • IL-12, IFN-γ and CXCL-10 drive anti-tumor immunity in the bladder, and are all upregulated by
Slide 27: Surfactant: Enhances IL-12 Expression & Downstream Target Engagement
Surfactant: Enhances IL-12 Expression & Downstream Target Engagement Increased and Prolonged IL-12 Sustained Enhancement of IFN-γ Sustained Enhancement of CXCL- Expression inmIL-12 Time course of Bladder expression Production 10 Production ✱✱✱ ✱✱ ✱✱ ✱✱ ✱ ✱✱ ✱✱ ✱✱ ✱ ✱ ✱ ✱ 1000 40 300 pg CXCL10 / mg p
Slide 28: Maintain Detalimogene Simplicity by Not Requiring Multiple Rinses
Maintain Detalimogene Simplicity by Not Requiring Multiple Rinses No Bladder Washout with Saline is Needed Before Detalimogene Administration No Impact to Detalimogene Stability No Impact to Detalimogene Potency Size (nm) 0.3 PDI 175 1000 ns 150 max max 0.2 pg mIL-12 / mg total protein 125 100 0.1 1
Slide 29: Surfactant: No Observed Bladder Toxicity or Systemic Exposure
Surfactant: No Observed Bladder Toxicity or Systemic Exposure Preclinical Evaluation in Murine pDNA in Plasmain Plasma mIL-12 pDNA Concentration 150 copies / mL of plasma Bladder weight 48h Post-Tx 145 Bladder Weight Bladder weightat at 48h 48h Post-Tx post-Tx 40 140 bladder weight (mg) 135 30 130 2
Slide 30: Non-Human Primates Model Validates Gene Expression and Safety Findings
Non-Human Primates Model Validates Gene Expression and Safety Findings Increases Gene Expression No systemic distribution of Surfactant + Detalimogene polidocanol following administration* Polidocanol in Plasma 10 BLQ: Below level of quantitation LLOQ: Lowest level of quantitation 8 ug/mL 6 Detalimo
Slide 31: Next Steps Studying Surfactant Bladder Rinse in a Clinical Trial
Next Steps Studying Surfactant Bladder Rinse in a Clinical Trial Scientific Preclinical Clinical Surfactant Efficacy and Selection Administration 31 Virtual KOL Event: Detalimogene with Surfactant October 2026
Slide 32: LEGEND Detalimogene + Polidocanol Surfactant Cohort
LEGEND Detalimogene + Polidocanol Surfactant Cohort First Patients Enrolled and Past Safety Run-In High-Risk NMIBC Study Design Complete In Progress BCG-unresponsive Safety Run-In HR NMIBC with No Dose Limiting Efficacy Run-In Up to n=80 CIS +/- Papillary Toxicities enrollment • Single-catheterizati
Slide 33: Detalimogene: Next Gen NMIBC Treatment Designed for Community Use
Detalimogene: Next Gen NMIBC Treatment Designed for Community Use Large Total NMIBC market forecasted to be >$20B Addressable Market Differentiated Total Demonstrated Clinical Activity, Favorable Safety, and a Streamlined Patient and Practice Experience Product Profile Lifecycle Surfactant Bladder R
Slide 34: Q&A
Q&A
Slide 35: Thank You
Thank You
enGene Therapeutics — Technical Presentation (October 2026) on 6ix