Neurogene reports progress in gene therapy regulation technology

NEW YORK - Neurogene Inc. (NASDAQ: NGNE), a clinical-stage biotech company currently valued at $156 million, has announced the publication of a study in Science Translational Medicine detailing its EXACT™ transgene regulation technology. The company’s stock has experienced significant volatility, declining 78% over the past year, though InvestingPro analysis suggests the stock may be oversold based on technical indicators. The preclinical research indicates that EXACT™ can control gene therapy expression to therapeutic levels without causing toxicity in models of Rett syndrome, a rare neurological disorder.

The study’s findings have supported the launch of a Phase 1/2 clinical trial for NGN-401, Neurogene’s gene therapy candidate for Rett syndrome. While the company maintains a strong liquidity position with a current ratio of 20.8 and more cash than debt on its balance sheet, InvestingPro data indicates rapid cash burn remains a key challenge. Rett syndrome is a debilitating condition caused by mutations in the MECP2 gene. Traditional gene therapies for such disorders can result in variable and potentially toxic levels of gene expression due to unregulated delivery.

EXACT™, which stands for "Expression Attenuation Circuit Technology," is designed to deliver controlled levels of therapeutic genes. It uses a self-regulating circuit within the gene therapy vector that responds to cellular microRNA levels, ensuring that each cell only produces the necessary amount of the therapeutic protein.

The preclinical studies have shown that NGN-401 extended median survival in a male Rett syndrome model and was well-tolerated across all doses in a female model. It also demonstrated better safety and efficacy compared to conventional gene therapies in non-human primates.

Neurogene’s CEO, Rachel McMinn, Ph.D., emphasized the significance of EXACT™ in enabling gene therapy for dosage-sensitive diseases like Rett syndrome, where precise gene expression is crucial.

NGN-401 has received several designations from the U.S. Food and Drug Administration (FDA), including Fast Track and orphan drug designations, which may expedite its development and review. It has also received similar recognitions from the European Medicines Agency (EMA) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

The press release notes that the company has constructed a gene therapy manufacturing facility in Houston, Texas, to produce NGN-401 and support its clinical development.

The press release also contains forward-looking statements regarding the potential of EXACT™ and NGN-401. These statements are based on current expectations and involve risks and uncertainties that could cause actual results to differ.

This article is based on a press release statement and provides an overview of the key points without endorsing the claims or potential of the technology. According to InvestingPro analysis, Neurogene appears undervalued at current levels, with analysts setting price targets significantly above the current trading price. Discover comprehensive insights and 12 additional ProTips about NGNE, along with detailed financial analysis in the Pro Research Report, available exclusively to InvestingPro subscribers.

In other recent news, the departure of Dr. Peter Marks from the FDA has caused significant concern in the biotech sector. Marks, who played a critical role in overseeing vaccine and biotech drug approvals, resigned after being asked to do so by a Health and Human Services official. His exit has raised worries about potential changes in the FDA’s approach to drug approvals, which could impact the speed and efficiency of future vaccine and drug development. This development has particularly affected companies like Moderna, Novavax, and Sarepta Therapeutics, among others, as their shares experienced a notable decline.

Meanwhile, Stifel analysts have maintained a Buy rating on Neurogene, with a price target of $46. Neurogene’s recent update for the fourth quarter of 2024 indicates that its development plans are on track, particularly with its NGN-401 therapy for Rett syndrome. The company reported promising initial clinical data and a strong safety profile for the therapy, which is currently in a phase 1/2 study. Furthermore, Neurogene’s financial health appears robust, with cash reserves projected to support operations until the second half of 2027. Investors will be closely watching for a regulatory update on NGN-401 in the first half of 2025.

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