Neurogene, Inc./$NGNE

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About Neurogene, Inc.

Neurogene Inc is a clinical-stage biotechnology company committed to overcoming the limitations of neurological diseases into treatable conditions, by harnessing its proprietary transgene regulation technology, EXACTTM (Expression Attenuation via Construct Tuning). It is building a differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet needs. The drug candidates in the company's product pipeline include NGN-401, which is in development for the treatment of Rett syndrome, using its EXACT platform, and NGN-101, a non-EXACT, conventional gene therapy development candidate for the treatment of CLN5 Batten disease, designed to deliver the CLN5 gene and packaged in an AAV9 capsid.

Ticker

$NGNE
Sector
Health
Primary listing
NASDAQ

Employees

107
Headquarters
New York, United States
Website
www.neurogene.com

Neurogene, Inc. Metrics

BasicAdvanced
$427M
-
-$4.30
-
-

Bulls say / Bears say

Neurogene completed dosing of the last five participants in its Phase 1/2 NGN-401 trial and received FDA agreement on key elements of the Embolden registrational trial design, paving a clear path to a potential registration study (Business Wire)
A head-to-head non-human primate study presented at ESGCT 2025 demonstrated intracerebroventricular (ICV) delivery of NGN-401 drives superior transgene expression in key brain regions versus intrathecal dosing, reinforcing the product’s differentiated mechanism of action (Neurogene IR)
Neurogene’s robust cash position with runway into mid-2027 alongside a peer-reviewed Science Translational Medicine publication validating the EXACT™ platform’s proof-of-concept provides strong financial flexibility and platform credibility (Business Wire)
FDA’s requirement for a 12-month primary endpoint in the Embolden registrational trial extends the study duration, increasing development costs and delaying potential approval beyond initial expectations (Business Wire)
High operating expenses resulted in a $22.6 million net loss in Q1 2025, underscoring significant cash burn that could necessitate further financing ahead of key registrational milestones (Business Wire)
Neurogene remains without any clinical efficacy readouts until the second half of 2025, leaving the stock vulnerable to binary trial risk and a lack of near-term data catalysts (Business Wire)
Data summarised monthly by Lightyear AI. Last updated on 11 Oct 2025.

Neurogene, Inc. News

AllArticlesVideos
Neurogene to Participate in H.C. Wainwright Genetic Medicines Virtual Conference

Neurogene to Participate in H.C. Wainwright Genetic Medicines Virtual Conference

Business Wire3 weeks ago
Neurogene Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Neurogene Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Business Wire3 weeks ago
Neurogene Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Neurogene Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Business Wire2 months ago
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