Every 8-K that Neurogene (NGNE) has filed with the SEC in the last 24 months is listed below, newest first, and each one links through to the document itself with the summary and the scores our analysis gives it.
A 8-K covers material events a company has to report between its quarterly reports, so if you follow NGNE and want that one kind of document rather than the whole filing history, this is the page to keep. The company's other filings, of every form, are on the full NGNE filings page.
Neurogene Inc. reported second quarter 2026 results and provided extensive updates on its NGN-401 gene therapy for Rett syndrome. Phase 1/2 data as of June 16, 2026 showed that 100% of 10 participants improved on the CGI-I scale and gained at least one developmental milestone, with a total of 47 milestones and an average of 4.7 per participant, and no plateau or loss of milestones through 30 months. NGN-401 at the 1E15 vg dose was generally well-tolerated across 35 treated participants as of August 10, 2026. Enrollment and dosing in the Embolden registrational trial (N=25) are complete, with topline data anticipated in the second half of 2027 and BLA-enabling PPQ runs expected to complete by year-end 2026.
Neurogene strengthened its balance sheet with an oversubscribed public follow-on offering generating approximately $144 million in gross proceeds and about $134.8 million in net proceeds, extending its cash runway into the first quarter of 2029. Cash, cash equivalents and short-term investments were $225.4 million as of June 30, 2026, or about $360.2 million on a pro forma basis including the offering. For the quarter, R&D expenses were $25.5 million versus $19.4 million a year earlier, G&A expenses were $11.2 million versus $6.7 million, and net loss was $34.5 million versus $22.0 million, reflecting higher development, stock-based compensation and pre-commercial spending.
Neurogene Inc. entered into an underwriting agreement to complete a public offering of its common stock and pre-funded warrants. The company is issuing 3,500,000 shares of common stock at $30.00 per share and pre-funded warrants to purchase up to 666,666 shares at $29.999999 each. Underwriters exercised in full a 30‑day option to buy an additional 624,999 shares at the public offering price, less underwriting discounts and commissions. Net proceeds from the offering, including the exercised option, are expected to be approximately $134.8 million after fees and expenses. The company states that these proceeds, together with existing cash and cash equivalents, should fund operating and capital needs into the first quarter of 2029.
Neurogene Inc. reported updated, positive Phase 1/2 results for NGN-401, its investigational gene therapy for females with Rett syndrome. Across 10 participants, 47 developmental milestones were gained, averaging 4.7 per participant, and 100% improved on the Clinical Global Impression-Improvement scale and gained at least one milestone.
Milestones accumulated in a progressive, developmentally ordered sequence with rapid onset, as median time to first clinical improvement was two months and gains deepened by 95% from 6 to 12 months and 147% from 6 to at least 12 months. No plateau or loss of milestones was seen through up to 30 months of follow-up, and NGN-401 at the 1E15 vg dose remained generally well-tolerated, with only mild or moderate treatment-related adverse events and no new treatment-related serious adverse events. Neurogene has completed dosing of 25 participants in the Embolden registrational trial, which uses a composite responder endpoint, and expects topline data in the second half of 2027.
Neurogene Inc. has completed dosing in Embolden, its registrational trial of NGN-401, an investigational one-time gene therapy for Rett syndrome. The company exceeded its initial 20-participant target by dosing 25 patients after strong community demand, with a pre-specified intent-to-treat population of 24 for the primary analysis.
At the 1E15 vg dose, NGN-401 has been generally well-tolerated; as of June 7, 2026, 35 treated participants across trials showed no cases of hemophagocytic lymphohistiocytosis. Interim Phase 1/2 data in 10 participants reported multidomain, durable functional gains, with 35 developmental milestones achieved and 88% improving on the CGI-I scale.
Embolden’s primary endpoint requires a 33% minimum response rate, or 8 of 24 participants, at 12 months to support a planned Biologics License Application. Neurogene expects updated Phase 1/2 data in mid-2026 and topline Embolden results in the second half of 2027.
Neurogene Inc. reported the results of its 2026 annual stockholder meeting. Stockholders representing 11,387,407 common shares, about 72.92% of voting power, were present, establishing a quorum. Two Class III directors, Robert Baffi and Rohan Palekar, were elected to serve until the 2029 annual meeting.
Stockholders approved, on an advisory basis, the compensation of the company’s named executive officers and ratified Deloitte & Touche LLP as independent auditor for the year ending December 31, 2026. In a separate advisory vote, investors expressed a preference for annual advisory votes on executive pay, and the company plans to hold them every year.
Neurogene Inc. reported first quarter 2026 results while advancing its lead Rett syndrome gene therapy, NGN-401. About 90% of participants have been dosed in the Embolden registrational trial, and dosing is expected to be completed in the second quarter of 2026. NGN-401 at the 1E15 vg dose has been generally well-tolerated across the Phase 1/2 trial and Embolden, with no cases of hemophagocytic lymphohistiocytosis reported as of May 11, 2026.
Interim Phase 1/2 data as of October 30, 2025 showed multidomain, durable functional gains across fine motor, gross motor and communication, with 35 developmental milestones gained and 88% of participants achieving improved CGI-I scores. Caregiver testimonials highlighted meaningful improvements in daily living and quality of life.
Financially, Neurogene held $243.2 million in cash, cash equivalents and short-term investments as of March 31, 2026, which is expected to fund operations through the first quarter of 2028. R&D expenses rose to $25.2 million from $17.8 million year over year, driven mainly by NGN-401 clinical and CMC costs, while G&A expenses held at $8.2 million. Net loss widened to $30.9 million from $22.6 million as the company invested in its Rett program and early commercial-readiness activities.
Neurogene Inc. expanded its Board of Directors to seven members and appointed its President and Chief Financial Officer, Christine Mikail, J.D., as a Class II director whose term runs until the 2028 annual stockholder meeting.
The company also appointed Christy Shafer as Chief Commercial Officer to lead commercial strategy and launch readiness for NGN-401, its gene therapy candidate for Rett syndrome. Both leaders bring extensive experience in rare neurological diseases, strategic transactions and commercialization as Neurogene advances its registrational Embolden trial and prepares for potential future commercialization.
Neurogene Inc. reported a full year 2025 net loss of $90.4 million, widening from $75.1 million in 2024, as it increased investment in its Rett syndrome gene therapy program NGN-401.
Research and development expenses rose to $75.0 million from $60.9 million, and general and administrative expenses increased to $28.3 million from $22.6 million, mainly due to higher clinical trial and employee-related costs. The company ended 2025 with $269.0 million in cash, cash equivalents and short-term investments, which it expects to fund operations through the first quarter of 2028.
Clinically, Neurogene fully enrolled its Embolden registrational trial for NGN-401 in Rett syndrome, with more than half of participants dosed and dosing completion targeted for the second quarter of 2026. Interim Phase 1/2 data at the 1E15 vg dose showed multidomain, durable functional gains across motor and communication domains, with NGN-401 generally well-tolerated and no cases of hemophagocytic lymphohistiocytosis.
NGN-401 received FDA Breakthrough Therapy designation based on interim Phase 1/2 data, and the FDA confirmed commercial manufacturing scale will match clinical scale, avoiding comparability studies. Neurogene plans to begin a Process Performance Qualification campaign and present updated Phase 1/2 data, including at least 12 months of follow-up for all 10 participants, in mid-2026, while continuing early commercial-readiness and payor engagement activities.
Neurogene Inc. announced that the U.S. FDA has granted Breakthrough Therapy designation to NGN-401, its investigational, one-time AAV9 gene therapy for Rett syndrome. The designation is based on interim Phase 1/2 data as of October 30, 2025, showing clinically meaningful, durable functional improvements across multiple Rett domains with continued skill acquisition over time.
NGN-401 uses Neurogene’s proprietary EXACT™ transgene regulation technology and is delivered via intracerebroventricular administration to broadly target the brain and nervous system. The therapy is being studied in the Embolden™ registrational trial, with dosing expected to complete in the second quarter of 2026 and additional interim Phase 1/2 data planned for mid-2026. NGN-401 also holds multiple other U.S., European and U.K. regulatory designations intended to support development for this serious rare disease.
Neurogene Inc. filed a current report to let investors know it has issued a new press release and updated its corporate presentation. On January 12, 2026, the company released a press statement providing recent corporate updates and anticipated milestones for the coming year, which is included as Exhibit 99.1. On the same date, Neurogene posted an updated corporate presentation on its website, furnished as Exhibit 99.2. The company notes that this information is being furnished, not filed, under the Exchange Act, meaning it is not automatically subject to certain liability provisions or incorporated into other SEC filings unless specifically referenced.
Neurogene Inc. (NGNE) reported that it issued a press release with financial results for the quarter ended September 30, 2025 and posted an updated corporate presentation. Both materials, dated November 13, 2025, are furnished with this report as Exhibits 99.1 (press release) and 99.2 (presentation). The information under Item 2.02 and these exhibits is furnished and not deemed filed under the Exchange Act.
Neurogene Inc. (NGNE) furnished an Item 7.01 update announcing positive, updated interim clinical data from the pediatric (ages 4–10) cohort of its ongoing Phase 1/2 trial of NGN-401 gene therapy for females with Rett syndrome. The company also posted a related corporate presentation. A press release (Exhibit 99.1) and the presentation (Exhibit 99.2) were furnished, not filed, on November 12, 2025.
Neurogene Inc. (NGNE) reported a clinical milestone: the first patient has been dosed in the Embolden registrational trial of NGN-401 for Rett syndrome. The company furnished a press release and an updated corporate presentation highlighting ongoing enrollment and dosing activity.
The materials were provided as Exhibit 99.1 (press release) and Exhibit 99.2 (corporate presentation). The information was furnished under Item 7.01 and is not deemed “filed” for purposes of Section 18 of the Exchange Act or incorporated by reference, except as specifically referenced.
Neurogene Inc. reported that it has completed discussions with the U.S. Food and Drug Administration on the protocol for its anticipated registrational trial in Rett syndrome and plans to dose the first participant in that trial in the fourth quarter of 2025. The company also highlighted preclinical data indicating that intracerebroventricular (ICV) administration appears superior to intrathecal lumbar (IT-L) delivery in reaching key brain regions involved in Rett syndrome, with these data being presented as a poster at the European Society of Gene & Cell Therapy Annual Congress in Seville, Spain. Neurogene issued a press release and posted an updated corporate presentation on its website, both dated October 9, 2025.
On August 11, 2025, Neurogene Inc. reported that it issued a press release announcing financial results for the quarter ended June 30, 2025 and posted an updated corporate presentation on its website. The Form 8-K attaches Exhibit 99.1 (press release) and Exhibit 99.2 (corporate presentation) and states these exhibits are being furnished, not filed, so they are not subject to Section 18 liabilities or deemed incorporated by reference. The filing text does not include the actual earnings figures; the report is signed by Christine Mikail, President and Chief Financial Officer.