Neurogene (NGNE) — Stock, Pipeline & Market Cap

Is this your company? Claim your profile to update info and connect with investors.

Claim profile

NGNE · Stock Price

USD 31.26+16.53 (+112.22%)

Market Cap: $470.0M

Historical price data

Market Cap: $470.0MPipeline: 1 drugFounded: 2024HQ: New York, United States

Sector Ranking#15 in RNA Therapeutics (non-mRNA)#52 in Rare & Orphan Diseases

Overview

Neurogene's mission is to develop life-changing genetic medicines for patients and families impacted by rare neurological diseases. Its key achievement is advancing NGN-401, a gene therapy for Rett syndrome, into the Embolden™ registrational trial, with promising early safety data. The company's strategy is built on its proprietary EXACT™ technology platform, designed to overcome overexpression toxicity, and a fully integrated approach that includes internal manufacturing to accelerate development.

Rare Neurological DiseasesNeurodevelopmental Disorders

Technology Platform

EXACT™ (Expression Attenuation via Construct Tuning) is a microRNA-based transgene regulation platform designed to control therapeutic protein expression within a safe, therapeutic window, aiming to overcome overexpression toxicity in gene therapy.

Pipeline

1

1 drug in pipeline

Drug	Indication	Stage	Watch
NL-201 + Pembrolizumab Injection [Keytruda]	Solid Tumor	Phase 1

Funding History

3

Total raised:$308M

IPO$125MUndisclosedNov 9, 2023

Series B$115MEcoR1 CapitalSep 15, 2021

Series A$68MEcoR1 CapitalJan 15, 2018

Opportunities

Success in the Rett syndrome registrational trial could unlock a high-value, unmet need market and serve as a powerful proof-of-concept for the EXACT platform.

This would enable expansion into other monogenic neurological disorders where precise gene dosing is critical, creating a multi-program pipeline.

Risk Factors

The company faces high clinical risk as its value is concentrated in a single lead program.

Failure in the ongoing pivotal trial, emergence of severe safety signals, or challenges in manufacturing or regulatory approval would have a catastrophic impact on valuation and viability.

Competitive Landscape

Neurogene's primary competition in Rett syndrome is Taysha Gene Therapies. Its key differentiator is the EXACT platform, designed to mitigate overexpression toxicity—a known risk in MECP2 gene therapy. Broader platform competition includes other gene regulation technologies from both biotech and large pharma.