Neurogene Inc. is a biotechnology company focused on developing innovative gene therapies for rare neurological diseases. The company aims to address the underlying genetic causes of these conditions by employing cutting-edge science and technology to create targeted treatments that have the potential to improve the lives of patients. Through its research and development efforts, Neurogene seeks to advance the understanding and treatment of neurogenetic disorders, offering hope to individuals and families affected by these challenging ailments. Read More
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 positive, updated interim clinical data in the pediatric cohort (ages 4-10 cohort) from its ongoing Phase 1/2 trial evaluating NGN-401 gene therapy for the treatment of females with Rett syndrome.
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 the first participant was dosed in the Embolden™ registrational clinical trial of NGN-401, a gene therapy in development for the treatment of Rett syndrome. NGN-401 has the potential to be a best-in-class and first-in-class gene therapy for Rett syndrome, a neurodevelopmental disorder with significant unmet medical need.
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 the Compensation Committee of the Company’s Board of Directors approved the grant of non-qualified stock options to purchase an aggregate of 1,185 shares of the Company’s common stock to one new employee (the “Inducement Grant”) on November 4, 2025 (the “Grant Date”). The Inducement Grant has been granted pursuant to the Company’s 2025 Inducement Plan (the “Plan”). The Inducement Grant was granted as an inducement material to this individual entering into employment with Neurogene in accordance with Nasdaq Listing Rule 5635(c)(4).
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 the Company has completed discussions with the U.S. Food and Drug Administration (FDA) on its Embolden™ registrational trial protocol and plans to initiate dosing in the fourth quarter of 2025. In response to strong interest from the Rett syndrome community, the trial is expected to enroll across 13 sites.
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 H.C. Wainwright Genetic Medicines Virtual Conference.
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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 the Compensation Committee of the Company’s Board of Directors approved the grant of non-qualified stock options to purchase an aggregate of 9,390 shares of the Company’s common stock to four new employees (the “Inducement Grants”) on October 1, 2025 (the “Grant Date”). The Inducement Grants have been granted pursuant to the Company’s 2025 Inducement Plan (the “Plan”). The Inducement Grants were granted as an inducement material to these individuals entering into employment with Neurogene in accordance with Nasdaq Listing Rule 5635(c)(4).
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 the Compensation Committee of the Company’s Board of Directors approved the grant of non-qualified stock options to purchase an aggregate of 47,040 shares of the Company’s common stock to six new employees (the “Inducement Grants”) on September 3, 2025 (the “Grant Date”). The Inducement Grants have been granted pursuant to the Company’s 2025 Inducement Plan (the “Plan”). The Inducement Grants were granted as an inducement material to these individuals entering into employment with Neurogene in accordance with Nasdaq Listing Rule 5635(c)(4).
FDA launches Rare Disease Evidence Principles to guide therapy reviews, offering new pathways for approval while raising questions on development impact.
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 second quarter 2025 financial results and highlighted recent corporate updates.
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 the Compensation Committee of the Company’s Board of Directors approved the grant of non-qualified stock options to purchase an aggregate of 27,480 shares of the Company’s common stock to seven new employees (the “Inducement Grants”) on August 6, 2025 (the “Grant Date”). The Inducement Grants have been granted pursuant to the Company’s 2025 Inducement Plan (the “Plan”). The Inducement Grants were granted as an inducement material to these individuals entering into employment with Neurogene in accordance with Nasdaq Listing Rule 5635(c)(4).
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 the Compensation Committee of the Company’s Board of Directors approved the grant of non-qualified stock options to purchase an aggregate of 4,290 shares of the Company’s common stock to two new employees (the “Inducement Grants”) on July 3, 2025 (the “Grant Date”). The Inducement Grants have been granted pursuant to the Company’s 2025 Inducement Plan (the “Plan”). The Inducement Grants were granted as an inducement material to these individuals entering into employment with Neurogene in accordance with Nasdaq Listing Rule 5635(c)(4).
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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 details of Embolden™, the Company’s registrational clinical trial designed to evaluate NGN-401 gene therapy for the treatment of females with Rett syndrome in participants ages 3 years and older. The Company has written agreement from the U.S. Food and Drug Administration (FDA) on key aspects of the registrational trial’s design.
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 the Compensation Committee of the Company’s Board of Directors approved the grant of non-qualified stock options to purchase an aggregate of 39,345 shares of the Company’s common stock to four new employees (the “Inducement Grants”) on June 5, 2025 (the “Grant Date”). The Inducement Grants have been granted pursuant to the Company’s 2025 Inducement Plan (the “Plan”). The Inducement Grants were granted as an inducement material to these individuals entering into employment with Neurogene in accordance with Nasdaq Listing Rule 5635(c)(4).
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 oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting describing a monitoring and treatment algorithm intended to reverse the rare, severe hyperinflammatory syndrome hemophagocytic lymphohistiocytosis (HLH) that has been associated with systemic exposure to high doses of adeno-associated virus (AAV) gene therapy (>1E14 vg/kg).
