Earnings summaries and quarterly performance for Neurogene.
Executive leadership at Neurogene.
Board of directors at Neurogene.
Research analysts covering Neurogene.
Recent press releases and 8-K filings for NGNE.
Neurogene Updates on NGN401 Clinical Progress and Outlook
NGNE
New Projects/Investments
Guidance Update
Product Launch
- Neurogene's lead program, NGN401 for Rett syndrome, is progressing in its Embolden registrational trial, with multiple participants dosed in Q4 2025.
- The company anticipates completing enrollment and dosing for the Embolden trial in Q2 2026 and will present additional Phase 1/2 clinical data mid-2026, including a minimum of 12 months of data on all participants.
- Interim Phase 1/2 data for NGN401 demonstrated clinically meaningful, deepening, and durable improvements across key Rett syndrome domains, with 100% of pediatric participants showing functional improvements.
- Neurogene maintains a strong balance sheet providing runway through Q1 2028, expected to fund operations through key milestones such as the Embolden data readout and BLA submission.
- The market for Rett syndrome is estimated as a multi-billion dollar opportunity, with 15,000-20,000 patients in major markets.
Jan 14, 2026, 3:30 PM
Neurogene Provides Update on NGN-401 for Rett Syndrome and Upcoming Milestones
NGNE
Product Launch
New Projects/Investments
Guidance Update
- Neurogene is advancing NGN-401, a potential best-in-class gene therapy for Rett Syndrome, targeting a multi-billion-dollar market with no disease-modifying treatment available.
- Interim Phase 1/2 data for NGN-401 showed 100% functional improvements across core disease domains and 35 total developmental milestones gained with durability up to 24 months.
- The company is conducting the single registrational Embolden trial for NGN-401, with enrollment underway and key elements aligned with the FDA for broad age range approval.
- Anticipated 2026 milestones include completing enrollment in the Embolden trial by 2Q'26 and presenting 12+ months Phase 1/2 data for all 10 participants by mid-2026.
- Neurogene's strong cash balance is expected to fund operations through the Embolden data readout, BLA submission, and key pre-launch activities until 1Q'28.
Jan 14, 2026, 3:30 PM
Neurogene Provides Update on NGN401 for Rett Syndrome at JPM Healthcare Conference
NGNE
New Projects/Investments
Guidance Update
Product Launch
- Neurogene's lead program, NGN401 for Rett syndrome, is currently dosing patients in the Embolden registrational trial, with multiple participants dosed in Q4 2025.
- The company expects to complete enrollment and dosing for the Embolden trial in Q2 2026 and present additional clinical data from the phase 1/2 trial mid-2026, including a minimum of 12 months of data on all participants.
- Interim phase 1/2 clinical data for NGN401 demonstrated clinically meaningful, deepening, and durable improvements across key Rett syndrome domains, with 100% of participants showing functional improvements.
- Neurogene maintains a strong balance sheet providing a financial runway through Q1 2028, which is expected to fund operations through key milestones including Embolden data readout and BLA submission.
- The company is rapidly advancing towards commercialization for NGN401, targeting a multi-billion-dollar market opportunity in Rett syndrome, with preparations including converting clinical sites and internal manufacturing.
Jan 14, 2026, 3:30 PM
Neurogene Provides Update on NGN-401 Rett Syndrome Program
NGNE
Product Launch
New Projects/Investments
Guidance Update
- Neurogene's lead program, NGN-401 for Rett syndrome, is currently dosing patients in the Embolden registrational trial, with enrollment and dosing expected to be completed in the second quarter of 2026.
- Interim clinical data from the Phase I/II trial for NGN-401 demonstrated clinically meaningful, deepening, and durable improvements across key Rett syndrome domains, with 100% of participants showing functional improvements in one or more domains. The 1E15 vector genome dose was generally well tolerated.
- The company anticipates presenting new safety and efficacy data from the Phase I/II trial mid-2026, which will include a minimum of 12 months of data on all 10 participants.
- Neurogene maintains a strong balance sheet, providing a financial runway through the first quarter of 2028, which is expected to fund operations through key milestones including the Embolden data readout and BLA submission.
Jan 14, 2026, 3:30 PM
Neurogene Provides Update on NGN-401 Achievements and 2026 Milestones
NGNE
New Projects/Investments
Guidance Update
- Neurogene dosed multiple participants in the Embolden™ registrational trial for NGN-401 in Q4 2025, with completion of dosing expected in Q2 2026.
- The company plans to present updated interim safety and efficacy data from the Phase 1/2 trial for NGN-401 in mid-2026, including at least 12 months of data for all participants.
- Neurogene anticipates a cash runway through Q1 2028 and is undertaking early commercial-readiness activities for NGN-401.
Jan 12, 2026, 12:00 PM
Neurogene Provides Update on NGN-401 for Rett Syndrome
NGNE
Product Launch
New Projects/Investments
Revenue Acceleration/Inflection
- Neurogene is rapidly advancing NGN-401, a potential one-time gene therapy for Rett Syndrome, towards commercialization, targeting a multi-billion-dollar market. The company's mission is to develop life-changing genetic medicines for people impacted by devastating neurological diseases.
- Interim Phase 1/2 clinical trial data for NGN-401 demonstrated clinically meaningful and durable improvements across core disease domains in 100% of participants, with 35 total developmental milestones gained and no plateau out to 24 months. The 1E15 vg dose continues to be generally well-tolerated.
- Neurogene has received FDA alignment on the Embolden registrational trial for NGN-401, which is currently enrolling participants (multiple dosed in Q4 2025) and aims for a single trial covering ages ≥ 3 years. Completion of Embolden dosing is expected in Q2 2026, with 12+ months Phase 1/2 data for all 10 participants planned for mid-2026.
- NGN-401 has received significant regulatory designations, including FDA's RMAT, EMA's PRIME, and participation in the FDA's START program, positioning it as a potential best-in-class or first-in-class gene therapy for Rett syndrome.
Jan 12, 2026, 12:00 PM
Neurogene Provides Update on NGN-401 Gene Therapy for Rett Syndrome
NGNE
New Projects/Investments
Guidance Update
- Neurogene has dosed multiple participants in its Embolden registrational trial for NGN-401 in the fourth quarter of 2025, with completion of dosing anticipated in the second quarter of 2026.
- The company plans to present interim safety and efficacy data from the Phase 1/2 trial for NGN-401 in mid-2026, including at least 12 months of follow-up for all participants.
- Neurogene reported a cash runway expected through the first quarter of 2028.
- NGN-401, an investigational gene therapy for Rett syndrome, has received multiple regulatory designations, including the FDA's RMAT and EMA's PRIME.
Jan 12, 2026, 12:00 PM
Neurogene Reports Third Quarter 2025 Financial Results and Clinical Program Updates
NGNE
Earnings
Guidance Update
New Projects/Investments
- Neurogene Inc. reported $265.4 million in cash, cash equivalents, and short-term investments as of September 30, 2025, with a projected cash runway through the first quarter of 2028.
- For the third quarter of 2025, the company recorded a net loss of $21.0 million, with Research & Development (R&D) expenses of $17.2 million and General & Administrative (G&A) expenses of $6.6 million.
- The company announced positive interim data from its NGN-401 Phase 1/2 gene therapy trial for Rett syndrome, showing 35 total developmental milestones/skills acquired across eight participants, and has dosed the first participant in the Embolden registrational trial, with complete enrollment expected in three to six months.
Nov 13, 2025, 10:11 PM
Neurogene Reports Positive Interim Data for NGN-401 Gene Therapy Trial
NGNE
New Projects/Investments
- Neurogene Inc. announced positive, updated interim clinical data from its ongoing Phase 1/2 trial evaluating NGN-401 gene therapy for the treatment of females with Rett syndrome in the pediatric cohort (ages 4-10).
- As of the October 30, 2025 data cutoff, all 8 pediatric participants showed functional gains, acquiring an aggregate of 35 developmental milestones/skills across core clinical domains, with durable improvements observed up to 24 months post-treatment.
- NGN-401 at the 1E15 vg dose was generally well-tolerated, with all treatment-related adverse events being mild (Grade 1) or moderate (Grade 2) in severity, and no evidence of hemophagocytic lymphohistiocytosis (HLH).
Nov 12, 2025, 10:11 PM
Neurogene Presents Interim NGN-401 Clinical Data and Embolden Trial Update
NGNE
Product Launch
New Projects/Investments
Guidance Update
- Neurogene reported positive interim Phase 1-2 clinical data for NGN-401 in Rett syndrome, showing 35 skills gained in total across multiple domains in eight pediatric patients with follow-up ranging from six to 24 months, and all gains have been durable with no skills lost.
- Six of eight patients in the Phase 1-2 trial met the responder definition for the Embolden registrational trial, surpassing the 35% threshold required for a successful outcome.
- NGN-401 was generally well tolerated, with all related events being mild (grade 1) or moderate (grade 2) and resolving, and no evidence of a hyperinflammatory syndrome.
- Enrollment for the Embolden registrational trial is projected to be completed in the next three to six months, and the company has sufficient cash to fund operations through the first quarter of 2028.
- Neurogene believes NGN-401 demonstrates approximately 50% more skills gained in fewer patients compared to Taysha's TSHA-102, highlighting more complex and durable skill acquisition.
Nov 12, 2025, 9:40 PM
Quarterly earnings call transcripts for Neurogene.
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