Highest-materiality recent filing
Neurogene raises ~$134.8M in public offering of common stock and pre-funded warrants
Issued 3.5M shares at $30.00 and pre-funded warrants for 666,666 shares at $29.999999.
47 developmental milestones gained across 10 participants (avg 4.7); 100% improved CGI-I and gained ≥1 milestone.
Neurogene completes dosing in NGN-401 Rett trial; no HLH, topline expected 2H 2027
Dosed 25 participants in Embolden trial (target 20, ITT up to 24) due to strong demand from Rett community.
Neurogene reports Q1 net loss $30.9M; ~90% dosed in Rett gene therapy trial, cash runway to Q1 2028
Net loss $30.9M for Q1 2026 vs $22.6M in Q1 2025; R&D expenses $25.2M driven by NGN-401 trial and CMC costs.
Neurogene reports FY2025 net loss $90.4M, cash $269M; NGN-401 dosing on track
Net loss $90.4M in FY2025 vs $75.1M in FY2024; cash $269M funds through Q1 2028.
Neurogene's NGN-401 receives FDA Breakthrough Therapy designation for Rett syndrome
FDA granted Breakthrough Therapy designation for NGN-401, a gene therapy for Rett syndrome.
Neurogene doses multiple participants in Embolden Rett trial; interim data mid-2026
Dosed multiple participants in Embolden registrational trial in Q4 2025; expect to complete dosing Q2 2026.
Net loss of $21.0M for Q3 2025 vs $20.2M in Q3 2024; R&D expenses $17.2M, G&A $6.6M.
Neurogene Reports Positive Interim Phase 1/2 Data for NGN-401 Gene Therapy in Rett Syndrome
All 8 pediatric participants (ages 4-10) showed functional gains across spectrum of disease severity.
Neurogene doses first participant in Embolden registrational trial of NGN-401 for Rett syndrome
First participant dosed in single-arm, open-label, baseline-controlled registrational trial of NGN-401 gene therapy for Rett syndrome.
Neurogene FDA agreement on Rett gene therapy registrational trial; Q4 2025 dosing start
Completed FDA discussions on Embolden registrational trial protocol for NGN-401; first participant dosing planned in Q4 2025 across 13 U.S. sites.
Net loss of $22.0M for Q2 2025 vs $18.5M in Q2 2024; R&D expenses $19.4M vs $15.7M.
FDA agreed on single-arm, open-label, baseline-controlled design; N=18 proposed; primary endpoint: CGI-I improvement plus gain of developmental milestone.
Neurogene reports Q1 net loss of $22.6M; cash $292.6M; NGN-401 trial on track
Net loss of $22.6M for Q1 2025 vs $16.9M Q1 2024; R&D expenses rose to $17.8M from $13.5M.
Neurogene raises BBA director nomination threshold to 14.9%, exchanges shares for prefunded warrants
BBA director nomination threshold raised from 12.5% to 14.9% of voting common stock via amendment to July 2023 letter agreement.
Neurogene posts FY2024 net loss $75.1M, cash $312.4M, NGN-401 gets EMA PRIME, trial updates
Cash and marketable securities $312.4M as of Dec 31, 2024; runway into H2 2027 to support NGN-401 registrational trial and pipeline.
Neurogene patient dies after high-dose NGN-401; FDA allows trial to continue at lower dose
Patient dosed Nov 5 with 3E15 vg NGN-401 died from hyperinflammatory syndrome linked to high AAV exposure.
Patient dosed on Nov 5 with 3E15 vg dose developed life-threatening systemic hyperinflammatory syndrome; is in critical condition.
Neurogene Q3 net loss $20.2M; positive NGN-401 data; $200M private placement
Q3 net loss $20.2M vs $14.6M YoY; cash & investments $139M as of Sep 30, 2024.
All 4 low-dose participants achieved a 2-point improvement on clinician-rated CGI-I scale from baseline.
Neurogene raises $200M in oversubscribed PIPE; funds into H2 2027
Gross proceeds of ~$200M from sale of 1.835M shares at $50/share and 2.165M pre-funded warrants at $49.999 each.
Neurogene Q2 net loss $18.5M; NGN-401 gets RMAT and START FDA designations
Cash and equivalents $153.9M as of June 30, 2024; expected to fund operations into 2H 2026.
Neurogene receives FDA RMAT designation for NGN-401 gene therapy in Rett syndrome
RMAT designation based on preliminary clinical evidence from ongoing Phase 1/2 trial showing potential to address unmet medical needs.
Neurogene doses first patient in high-dose cohort of NGN-401; safety data positive
First patient in Cohort 2 received high-dose NGN-401 in May 2024; well-tolerated to date.
Neurogene's NGN-401 Rett syndrome gene therapy selected for FDA START Pilot Program
NGN-401 one of only three CBER programs chosen for FDA's START Pilot Program.
All three pediatric patients (ages 4-7) treated with single ICV dose show mild, transient AEs; no serious adverse events.
Neurogene Reports Positive Initial Safety Data from Phase 1/2 Gene Therapy Trial for Rett Syndrome
NGN-401 generally well-tolerated in three patients with multiple months of follow-up.
Neurogene reports FY2023 net loss $36.3M; Rett gene therapy trial expanded, cash runway to H2 2026
Cash and investments $197.2M at Dec 31, 2023; reverse merger and $95M private placement completed December 2023.
Neurogene expands Rett syndrome gene therapy trial, adds high-dose cohort
Cohort 1 expanded from 5 to 8 patients; staggered dosing removed for parallel enrollment.
Neurogene reports ~$200M cash; DSMB clears third patient in Rett syndrome trial
DSMB cleared third pediatric patient for dosing in NGN-401 Phase 1/2 for Rett syndrome; dosing expected early Q1 2024.
Neoleukin completes reverse merger with Neurogene; raises ~$95M PIPE; interim CEO/CFO depart
Merger closed Dec 18, 2023: Neoleukin renamed Neurogene Inc.; all Neurogene stock converted at 0.0756:1 exchange ratio.
Neoleukin Therapeutics to effect 1-for-5 reverse stock split on Sept 25 to regain Nasdaq compliance
Reverse stock split at 1-for-5 ratio effective 12:01 a.m. ET on Sept 25, 2023; ticker NLTX unchanged, new CUSIP 64049K203.
Neoleukin to merge with Neurogene; Neurogene stockholders to own ~84% of combined co.
Neoleukin stockholders will own ~16% of combined company; Neurogene stockholders ~84% on as-converted basis.
Neoleukin cuts 70% of workforce, CEO to resign as company seeks strategic alternatives
Board approved 70% workforce reduction and re-prioritization to seek strategic alternatives to maximize shareholder value.
Neoleukin initiates strategic alternatives review, cuts workforce by 70%, CEO Drachman to step down
Engaged SVB Securities to explore sale, merger, asset divestiture, licensing, or other transaction to maximize shareholder value.
On February 15, 2024, Neurogene Inc. (the “Company”) appointed Donna Cochener as Senior Vice President, General Counsel.
the Board appointed the following four individuals to the Board: Rachel McMinn, Robert Baffi, Cory Freedland and Keith Woods.
Martin Babler, M. Cantey Boyd, Erin Lavelle and Todd Simpson resigned from Neoleukin’s board of directors
the Board appointed the following four individuals to the Board: Rachel McMinn, Robert Baffi, Cory Freedland and Keith Woods.
Rachel McMinn was also appointed as Chair of the Board.
Martin Babler, M. Cantey Boyd, Erin Lavelle and Todd Simpson resigned from Neoleukin’s board of directors
the Board appointed Rachel McMinn as the Company’s Chief Executive Officer
the Board appointed the following four individuals to the Board: Rachel McMinn, Robert Baffi, Cory Freedland and Keith Woods.
Martin Babler, M. Cantey Boyd, Erin Lavelle and Todd Simpson resigned from Neoleukin’s board of directors
the Board appointed the following four individuals to the Board: Rachel McMinn, Robert Baffi, Cory Freedland and Keith Woods.
Martin Babler, M. Cantey Boyd, Erin Lavelle and Todd Simpson resigned from Neoleukin’s board of directors
appointed Rachel McMinn as the Company’s Chief Executive Officer and Christine Mikail as the Company’s President and Chief Financial Officer
Max materiality 0.90 · Median 0.65 · Most common event other_material