Neurogene Reports Full Year 2025 Financial Results and Highlights Recent Updates Neurogene (Nasdaq: NGNE) reported full-year 2025 results and program updates on March 24, 2026. Key highlights: 100% enrollment in the Embolden registrational trial with >50% dosed and on track to complete dosing in Q2 2026. NGN-401 received FDA Breakthrough Therapy designation. Cash, cash equivalents and short-term investments were $269.0 million, expected to fund operations through Q1 2028. R&D expense was $75.0M and G&A was $28.3M for 2025; net loss was $90.4M. PPQ is planned mid-2026 to support BLA readiness.

#NGNE Neurogene Reports Full Year 2025 Financial Results and Highlights Recent Updates

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Neurogene Announces FDA Breakthrough Therapy Designation for NGN-401 Gene Therapy for Rett Syndrome Neurogene (Nasdaq: NGNE) announced the FDA granted Breakthrough Therapy designation for NGN-401 for Rett syndrome, based on interim Phase 1/2 data through Oct 30, 2025 showing clinically meaningful, durable, multidomain functional improvements.NGN-401 is a one-time, intracerebroventricular gene therapy using EXACT transgene regulation; dosing in the Embolden registrational trial is expected to complete in Q2 2026. The program also holds RMAT and Rare Pediatric Disease designations and participated in FDA’s START Pilot Program.

#NGNE Neurogene Announces FDA Breakthrough Therapy Designation for NGN-401 Gene Therapy for Rett Syndrome

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Neurogene Reports Third Quarter 2025 Financial Results and Highlights Recent Updates “We significantly advanced our NGN-401 gene therapy clinical program for Rett syndrome over the past quarter, culminating in the recent dosing of the first participant in the Embolden registrational trial after initiating 12 of 13 sites that enable our plans to rapidly enroll the trial in the next three to six months,” stated Rachel McMinn, Ph.D., Founder and...

#NGNE Neurogene Reports Third Quarter 2025 Financial Results and Highlights Recent Updates

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Neurogene Reports Positive Interim Data in Pediatric Cohort from NGN-401 Gene Therapy Trial for Rett Syndrome Neurogene (Nasdaq: NGNE) reported positive interim Phase 1/2 data for its NGN-401 gene therapy in the pediatric Rett syndrome cohort (data cutoff Oct 30, 2025). All 8 pediatric participants showed functional gains across core domains with an aggregate 35 developmental milestones/skills gained and durable, multidomain improvements observed up to 24 months post treatment. Safety (N=10) at the 1E15 vg dose was generally well-tolerated; treatment-related adverse events were Grade 1–2 and no HLH was observed. Additional clinical data is planned for 2026, and management will discuss results at the Stifel 2025 Healthcare Conference webcast on Nov 12, 2025 at 4:40 p.m. ET.

#NGNE Neurogene Reports Positive Interim Data in Pediatric Cohort from NGN-401 Gene Therapy Trial for Rett Syndrome

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#NGNE Neurogene Announces First Participant Dosed in Embolden™ Registrational Trial of NGN-401 Gene Therapy for Rett Syndrome

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Neurogene Reports Second Quarter 2025 Financial Results and Highlights Recent Updates Neurogene (Nasdaq: NGNE) reported Q2 2025 financial results and provided updates on its NGN-401 gene therapy program for Rett syndrome. The company has completed dosing in its Phase 1/2 trial and received FDA agreement on key elements of the Embolden™ registrational trial, with trial initiation activities already underway.Financial highlights include a strong cash position of $274.5 million, providing runway into early 2028. Q2 2025 saw a net loss of $22.0 million, with R&D expenses of $19.4 million and G&A expenses of $6.7 million. The company plans to report updated clinical efficacy and safety data from the Phase 1/2 trial in H2 2025.

#NGNE Neurogene Reports Second Quarter 2025 Financial Results and Highlights Recent Updates

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Neurogene Announces Registrational Trial Design for Embolden™ Study of NGN-401 Gene Therapy for Rett Syndrome Neurogene (NASDAQ: NGNE) has announced key details of its Embolden™ registrational trial for NGN-401, a gene therapy treatment for Rett syndrome in females aged 3 years and older. The company has received FDA written agreement on critical aspects of the trial design.The registrational trial will be a single-arm, open-label study with a proposed enrollment of 18 participants. The primary endpoint combines improvement in Clinical Global Impression-Improvement (CGI-I) scale and gain of developmental milestone/skill, compared to baseline. The study will use the 1E15 vector genomes (vg) dose.In the Phase 1/2 trial, 10 participants have received treatment at the 1E15 vg dose, with no evidence of HLH/hyperinflammatory syndrome. Early data from the first four participants showed promising results, with participants collectively achieving 23 developmental milestones/skills across multiple domains.The company reports a strong financial position with $292.6 million in cash and investments as of March 31, 2025, extending their runway into early 2028. Updated clinical efficacy and safety data are expected in the second half of 2025.

#NGNE Neurogene Announces Registrational Trial Design for Embolden™ Study of NGN-401 Gene Therapy for Rett Syndrome

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Neurogene Announces Evidence-Based Monitoring and Treatment Intended to Reverse Rare Hyperinflammatory Syndrome Associated with High-Dose AAV Neurogene (NASDAQ: NGNE) presented an evidence-based monitoring and treatment algorithm at the ASGCT Annual Meeting for detecting and treating hemophagocytic lymphohistiocytosis (HLH), a rare hyperinflammatory syndrome associated with high-dose AAV gene therapy. The protocol focuses on monitoring three key indicators (the "three Fs"): elevated ferritin levels, fever, and falling blood counts.While HLH has been linked to AAV doses exceeding 1E14 vg/kg, Neurogene's Phase 1/2 trial of NGN-401 for Rett syndrome uses a lower dose in the E13 vg/kg range, where no HLH cases have been reported. The treatment algorithm includes high-dose corticosteroids as first-line treatment and anakinra as second-line treatment.

#NGNE Neurogene Announces Evidence-Based Monitoring and Treatment Intended to Reverse Rare Hyperinflammatory Syndrome Associated with High-Dose AAV

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Neurogene Reports First Quarter 2025 Financial Results and Highlights Recent Updates Neurogene (NASDAQ: NGNE) reported its Q1 2025 financial results and corporate updates. The company is advancing its NGN-401 gene therapy program for Rett syndrome, with plans to provide a registrational trial update in H1 2025 and additional interim clinical data in H2 2025. The Phase 1/2 trial includes pediatric females aged 4-10 (N=8) and older females aged 11+ (N=3). Financially, Neurogene reported cash and investments of $292.6M as of March 31, 2025, providing runway into H2 2027. Q1 2025 results showed R&D expenses of $17.8M (up from $13.5M in Q1 2024), G&A expenses of $8.2M (up from $5.2M), and a net loss of $22.6M (compared to $16.9M). The company also published proof-of-concept results for its EXACT™ transgene regulation technology in Science Translational Medicine.

#NGNE Neurogene Reports First Quarter 2025 Financial Results and Highlights Recent Updates

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Neurogene Announces Upcoming Oral Presentation on Monitoring and Treatment to Reverse Rare Complication of High Dose Gene Therapy at the ASGCT Annual Meeting Neurogene has announced an upcoming oral presentation at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in New Orleans, focusing on monitoring and treating hemophagocytic lymphohistiocytosis (HLH) in gene therapy patients. Key highlights: The presentation will address HLH, a rare inflammatory condition linked to high-dose AAV gene therapy CEO Rachel McMinn emphasizes the importance of sharing effective approaches for monitoring and treating HLH The company's NGN-401 gene therapy trial for Rett syndrome uses a 1E15 vg dose level (E13 vg/kg range) No HLH cases have been reported at this dose level Additional participation includes: A fireside chat on reviving hope in deprioritized cell and gene therapy programs A panel discussion on FDA's START Pilot Program insights

#NGNE Neurogene Announces Upcoming Oral Presentation on Monitoring and Treatment to Reverse Rare Complication of High Dose Gene Therapy at the ASGCT Annual Meeting

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Neurogene Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Updates Neurogene (NASDAQ: NGNE) has released its Q4 and full year 2024 financial results, highlighting progress in its NGN-401 Phase 1/2 trial for Rett syndrome. The company reported encouraging interim efficacy data, with NGN-401 being well-tolerated at the 1E15 vg dose in pediatric patients.Key financial metrics include cash position of $312.4 million, providing runway into H2 2027. R&D expenses increased to $60.9 million in 2024 from $44.4 million in 2023, while G&A expenses rose to $22.6 million from $11.2 million. Net loss for 2024 was $75.1 million.Notable developments include:Received PRIME designation from European Medicines AgencyProtocol amended to use 1E15 vector genomes dose for future trial participantsExpanded age range to include patients >11 yearsPlans to provide regulatory update on registrational trial in H1 2025Additional interim clinical data expected in H2 2025

#NGNE Neurogene Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Updates

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Pomerantz Law Firm Launches Investigation for Neurogene Inc. Investors Amidst Securities Concerns Pomerantz LLP has initiated an investigation into potential securities fraud involving Neurogene Inc., following troubling clinical trial updates that impacted stock performance.

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NEWS: ( NASDAQ: #NGNE ) Neurogene Inc. (NGNE) Investigation: Bronstein, Gewirtz & Grossman, LLC Encourages Investors to Contact the Firm to Learn More About the Investigation

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Pomerantz Law Firm Investigates Neurogene Inc. Following Investor Claims and Stock Decline The Pomerantz Law Firm is probing potential securities fraud involving Neurogene Inc. as investor concerns grow following a significant stock drop.

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#NGNE Neurogene Reports Third Quarter 2024 Financial Results and Highlights Recent Updates

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#NGNE Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome

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Breaking News: ( NASDAQ: #NGNE ) Neurogene Announces Oversubscribed $200 Million Private Placement

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