Data demonstrate EXACT has potential to overcome limitations of conventional gene therapy for dosage-sensitive disorders Findings published in Science Translational Medicine NEW YORK / Apr 02, 2025 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced a peer-reviewed... Read More

Remains on track to provide regulatory update on registrational trial plans with NGN-401 for Rett syndrome in the first half of 2025 Continues to expect to share additional interim clinical data from NGN-401 Phase 1/2 trial in the second half of 2025 Received PRIME designation for NGN-401 from the European Medicines Agency Cash runway into the second half of 2027 NEW YORK / Mar 24, 2025 / Business Wire / Neurogene Inc.... Read More

NEW YORK / Feb 24, 2025 / Business Wire / Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the following conferences: TD Cowen 45th Annual Health Care Conference Format: Management will participate in a fireside chat and investor meetings Date:... Read More

Announced positive interim clinical data with 1E15 vg dose of NGN-401 gene therapy trial for Rett syndrome Neurogene to advance NGN-401 at 1E15 vg dose Expects to provide an update on registrational trial design in first half of 2025 Strong cash position provides runway into the second half of 2027 NEW YORK / Nov 18, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing... Read More

NEW YORK / Nov 18, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced an update on its ongoing Phase 1/2 open-label clinical trial evaluating NGN-401 gene therapy for the treatment of Rett syndrome. As previously disclosed, on November 11, 2024, Neurogene became aware... Read More

All participants experienced a 2-point improvement in the clinician-rated Clinical Global Impression-Improvement (CGI-I) scale from baseline All participants improved in the caregiver-completed Rett Syndrome Behavior Questionnaire (RSBQ), ranging from 28 to 52 percent improvement from baseline All participants with disruptions in sleep, constipation, and dysphagia at baseline demonstrated objective improvements Gains in... Read More

Financing included participation from top-tier healthcare funds Proceeds expected to fund Company into the second half of 2027 Company to host webcast to review interim clinical data from Phase 1/2 trial of NGN-401 gene therapy for Rett syndrome on November 11 at 4:30 p.m. ET NEW YORK / Nov 04, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to... Read More

Company to host webcast to review data on November 11 at 4:30 p.m. ET Late-breaker poster to be presented during Child Neurology Society Meeting on November 12 NEW YORK / Oct 21, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that it will host a webcast to present... Read More

NGN-401 gene therapy for Rett syndrome received RMAT designation from FDA based on preliminary clinical evidence indicating the potential to address unmet medical needs NGN-401 selected for FDA START Program, also designed to accelerate development Interim NGN-401 efficacy data from Cohort 1 remains on track for 4Q:24 NEW YORK / Aug 09, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to... Read More

Designation based on preliminary clinical evidence from ongoing NGN-401 clinical trial that shows potential to address unmet medical needs in Rett syndrome RMAT designation provides an opportunity for an Accelerated Approval pathway under the 21st Century Cures Act, and is in addition to NGN-401’s selection by the FDA for the START Pilot Program NEW YORK / Aug 07, 2024 / Business Wire / Neurogene Inc. (NASDAQ: NGNE), a... Read More

NEW YORK / Jul 02, 2024 / Business Wire / Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the BMO 2024 Genetic Medicines Summit. Format: Management will participate in the panel, “Gene Therapy in Rare Disease: Unmet Need + Lack of Alternatives =... Read More

NEW YORK / Jul 01, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Neurogene will be added to the Russell 3000 ® Index, effective at the open of U.S. equity markets today, Monday, July 1, 2024. The annual Russell indexes reconstitution captures the 4,000 largest... Read More

High-dose NGN-401 has been well-tolerated, and low-dose NGN-401 continues to show a favorable safety profile Interim safety data presented at the International Rett Syndrome Foundation (IRSF) ASCEND Summit NEW YORK / Jun 18, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today... Read More

NEW YORK / Jun 05, 2024 / Business Wire / Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the following conferences: Goldman Sachs 45th Annual Global Healthcare Conference Format: Management will present a corporate overview and participate in... Read More

NGN-401 is one of only three CBER programs chosen by FDA NGN-401 was selected based on potential for clinical benefits and clinical development program readiness START Program provides sponsors enhanced communications with FDA to accelerate development of rare disease therapies NEW YORK / Jun 03, 2024 / Business Wire / Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to... Read More

Presented favorable safety data from Phase 1/2 NGN-401 gene therapy trial for Rett syndrome at ASGCT Annual Meeting Received Australian HREC approval for NGN-401 trial Remains on track to provide interim NGN-401 efficacy data from Cohort 1 in 4Q:24 Strong balance sheet with cash runway into 2H:26 NEW YORK / May 10, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing... Read More

NGN-401 has been generally well-tolerated by first three patients dosed, with three to nine months of follow-up No signs or symptoms of overexpression toxicity, including in one patient with a mild variant predicted to result in residual MeCP2 expression Neurogene remains on track to provide interim efficacy data from the trial in 4Q:24 NEW YORK / May 07, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage... Read More

NGN-401 has been generally well-tolerated by three patients with multiple months of follow-up NEW YORK / Apr 22, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett... Read More

Expanded Phase 1/2 gene therapy trial for Rett syndrome to inform future registrational study design; Company remains on track to share interim clinical data in 4Q:24 Strong financial position with runway into 2H:26 following reverse merger and private financing in December 2023 NEW YORK / Mar 18, 2024 / Business Wire / Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines... Read More

Ongoing Phase 1/2 clinical trial for NGN-401 to include additional patients in Cohort 1 and a dose-escalation cohort Both previously planned updates are expected to provide a more robust dataset to inform future registrational trial design Removal of staggered dosing in Cohort 1 expected to enable the anticipated completion of Cohort 1 dosing in the second half of 2024 Third patient dosed in Cohort 1 NEW YORK / Mar 04, 2024... Read More

NEW YORK / Feb 29, 2024 / Business Wire / Neurogene Inc. (NASDAQ: NGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the following investor conferences: TD Cowen 44 th Annual Health Care Conference Format: Management will participate in a CNS... Read More

NEW YORK / Jan 16, 2024 / Business Wire / Neurogene Inc. (NASDAQ: NGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the appointment of Julie Jordan, M.D., as Chief Medical Officer. Dr. Jordan brings to Neurogene over 20 years of industry and clinical expertise involving the design and... Read More