Welcome to our dedicated page for Neurogene news (Ticker: NGNE), a resource for investors and traders seeking the latest updates and insights on Neurogene stock.
Neurogene Inc (NGNE) is a clinical-stage biotechnology pioneer developing gene therapies for neurological disorders through its proprietary EXACT gene regulation platform. This page provides investors and researchers with timely updates on the company's scientific advancements, regulatory progress, and strategic partnerships.
Access consolidated news coverage including clinical trial milestones, research publications, and executive commentary. Stay informed about developments in gene therapy applications for rare neurological conditions, with updates vetted for accuracy and relevance.
Key content categories include treatment pipeline updates, FDA communications, collaborative research initiatives, and technology patent announcements. All materials maintain strict compliance with financial disclosure regulations while prioritizing accessibility for diverse audiences.
Bookmark this page for streamlined tracking of Neurogene's progress in advancing precision gene therapies. Check regularly for official press releases and third-party analysis of the company's groundbreaking work in neurological disease treatment.
Neurogene has announced an upcoming oral presentation at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in New Orleans, focusing on monitoring and treating hemophagocytic lymphohistiocytosis (HLH) in gene therapy patients.
Key highlights:
- The presentation will address HLH, a rare inflammatory condition linked to high-dose AAV gene therapy
- CEO Rachel McMinn emphasizes the importance of sharing effective approaches for monitoring and treating HLH
- The company's NGN-401 gene therapy trial for Rett syndrome uses a 1E15 vg dose level (E13 vg/kg range)
- No HLH cases have been reported at this dose level
Additional participation includes:
- A fireside chat on reviving hope in deprioritized cell and gene therapy programs
- A panel discussion on FDA's START Pilot Program insights
Neurogene (NASDAQ: NGNE) has published peer-reviewed data in Science Translational Medicine demonstrating the effectiveness of its EXACT™ transgene regulation technology in preclinical models of Rett syndrome. The study supports their ongoing Phase 1/2 clinical trial of NGN-401, their gene therapy candidate.
The research shows EXACT's capability to regulate gene therapy expression to therapeutic and tolerable levels, addressing a key challenge in treating Rett syndrome, which requires careful regulation of MeCP2 levels. The technology is designed to overcome limitations of conventional gene therapy, which can result in toxic levels of transgene expression.
In preclinical studies, NGN-401 demonstrated:
- Extended median survival in male knockout models
- Good tolerability across all doses in female murine models
- Well-tolerated results in non-human primates
- More controlled MECP2 mRNA levels compared to conventional gene therapy
Neurogene (NASDAQ: NGNE) has released its Q4 and full year 2024 financial results, highlighting progress in its NGN-401 Phase 1/2 trial for Rett syndrome. The company reported encouraging interim efficacy data, with NGN-401 being well-tolerated at the 1E15 vg dose in pediatric patients.
Key financial metrics include cash position of $312.4 million, providing runway into H2 2027. R&D expenses increased to $60.9 million in 2024 from $44.4 million in 2023, while G&A expenses rose to $22.6 million from $11.2 million. Net loss for 2024 was $75.1 million.
Notable developments include:
- Received PRIME designation from European Medicines Agency
- Protocol amended to use 1E15 vector genomes dose for future trial participants
- Expanded age range to include patients >11 years
- Plans to provide regulatory update on registrational trial in H1 2025
- Additional interim clinical data expected in H2 2025
Neurogene (NASDAQ: NGNE), a clinical-stage company focused on developing genetic medicines for rare neurological diseases, has announced its participation in three upcoming healthcare conferences in March.
The company will participate in:
- The TD Cowen 45th Annual Health Care Conference on March 3 at 9:50 a.m. ET
- The Leerink Global Healthcare Conference on March 10 at 1:00 p.m. ET
- The Stifel Virtual CNS Forum on March 19 at 9:30 a.m. ET
Management will engage in fireside chats at all three events, with additional investor meetings scheduled for the TD Cowen and Leerink conferences. Live webcasts of the fireside chats will be available through Neurogene's website's Investor Relations section, with replays available for a time.
Neurogene (NASDAQ: NGNE) reported Q3 2024 financial results and updates. The company announced positive interim data from its NGN-401 gene therapy trial for Rett syndrome at the 1E15 vg dose, showing consistent improvements across multiple domains. Following a serious adverse event, the 3E15 vg dose was discontinued. Net loss increased to $20.2 million from $14.6 million year-over-year. R&D expenses rose to $16.3 million from $11.6 million, while G&A expenses increased to $5.9 million from $3.6 million. The company raised approximately $200 million through a private placement, extending cash runway into second half of 2027.
Neurogene (Nasdaq: NGNE) reported a serious adverse event (SAE) in its Phase 1/2 clinical trial of NGN-401 gene therapy for Rett syndrome. A participant who received the high dose (3E15 vg) experienced a life-threatening systemic hyperinflammatory syndrome and is in critical condition. The FDA reviewed the safety data and allowed continuation of the trial using only the lower dose (1E15 vg). No other treatment-related SAEs have been reported in the five participants who received the low dose and two participants who received the high dose. The company has paused further use of the high dose and will not enroll additional participants at that level.
Neurogene (NASDAQ: NGNE) reported positive interim data from its Phase 1/2 trial of NGN-401 gene therapy for Rett syndrome. The first four low-dose pediatric participants showed significant improvements, with all experiencing a 2-point improvement in the Clinical Global Impression-Improvement scale and 28-52% improvement in the Rett Syndrome Behavior Questionnaire. Key developments include improvements in sleep, constipation, and dysphagia, along with consistent skill gains and developmental milestones. The therapy demonstrated a favorable safety profile at low doses. The company plans to complete enrollment in the low-dose pediatric Cohort 1 in Q4 2024 and provide updates on the registrational trial design in H1 2025.
Neurogene has secured an oversubscribed $200 million PIPE financing with participation from major healthcare funds. The company is selling 1,835,000 common stock shares at $50.00 per share and 2,165,042 pre-funded warrants at $49.999 each. The funding, expected to close around November 5, 2024, will extend the company's runway into second half of 2027. The proceeds will support NGN-401's registrational study for Rett syndrome, CMC scale-up, and EXACT™ gene therapy pipeline development. The company will present interim clinical data from the NGN-401 Phase 1/2 trial on November 11, 2024.
Neurogene Inc. (Nasdaq: NGNE) announced it will present interim efficacy data from the low-dose cohort of its ongoing Phase 1/2 clinical trial of NGN-401 gene therapy for pediatric patients with Rett syndrome. The company will host a webcast on November 11, 2024 at 4:30 p.m. ET to review the data, which will include safety information from both low- and high-dose cohorts.
Additionally, these results will be presented in a late-breaking poster during the 53rd Annual Child Neurology Society (CNS) Meeting on November 12 at 12:30 p.m. PT in San Diego, CA. The poster, titled 'NGN-401, a Novel Regulated Gene Therapy for Rett Syndrome: Preliminary Results from the First-in-Human Study,' will be presented by Dr. Bernhard Suter from Texas Children's Hospital and Baylor College of Medicine.
Neurogene Inc. (Nasdaq: NGNE) reported its Q2 2024 financial results and corporate updates. Key highlights include:
1. NGN-401 gene therapy for Rett syndrome received RMAT designation and was selected for FDA's START Program, potentially accelerating development.
2. First patient dosed in high-dose Cohort 2 of NGN-401 trial in May, with favorable early safety profile.
3. Interim efficacy data from low-dose Cohort 1 expected in Q4 2024.
4. Cash position of $153.9 million as of June 30, 2024, expected to fund operations into H2 2026.
5. Q2 2024 net loss of $18.5 million, compared to $11.9 million in Q2 2023.
6. R&D expenses increased to $15.7 million in Q2 2024 from $10.3 million in Q2 2023.
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