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Neurogene, Inc. - NGNE STOCK NEWS

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 company focused on leveraging gene therapy to treat severe neurological diseases. With headquarters in Seattle, Washington, Neurogene's mission is to transform the lives of patients with complex neurological conditions through innovative genetic solutions. The company utilizes its proprietary EXACT gene regulation technology, which offers precise control over gene expression, enhancing the efficacy and safety of its treatments.

Neurogene's lead product candidate, NL-201, is designed to mimic the therapeutic benefits of cytokines interleukin (IL)-2 and IL-15. This de novo protein aims to treat various types of cancer, including renal cell carcinoma and melanoma. The company is dedicated to developing immunotherapies for cancer, inflammation, and autoimmunity disorders using advanced protein design technology.

Founded in 2003 and initially named Aquinox Pharmaceuticals, Inc., the company rebranded to Neoleukin Therapeutics, Inc. in August 2019, before evolving into the specialized entity it is today, Neurogene, Inc. The company has established significant partnerships and collaborations within the biotech industry, aiming to leverage these relationships to accelerate the development and commercialization of their gene therapy solutions.

Neurogene's financial health is bolstered by a robust pipeline of transformative therapies and a dedicated team of experts. The company continues to innovate and expand its research and development efforts, aiming to bring life-changing therapies to patients who currently have limited treatment options.

For the latest updates on Neurogene, Inc. (NGNE), including financial performance, new research, and other significant developments, visit the News section on StockTitan.

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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.

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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.

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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.

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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.

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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.

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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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Neurogene (NASDAQ: NGNE) announced that its investigational gene therapy NGN-401 for Rett syndrome has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. This designation, based on preliminary clinical evidence from the ongoing Phase 1/2 trial, indicates NGN-401's potential to address unmet medical needs in Rett syndrome.

The RMAT designation provides benefits such as accelerated approval pathways, frequent FDA communications, and eligibility for priority review. This follows NGN-401's previous selection for the FDA's START Pilot Program, further highlighting its therapeutic potential. Neurogene expects to share interim efficacy data from the low-dose cohort in Q4 2023, with additional data, including from the high-dose cohort, in H2 2025.

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Neurogene (NASDAQ: NGNE), a clinical-stage company focused on developing genetic medicines for rare neurological diseases, announced its participation in the BMO 2024 Genetic Medicines Summit. The event will take place on Tuesday, July 9, at 8:30 a.m. ET. Neurogene's management will be involved in a panel discussion titled 'Gene Therapy in Rare Disease: Unmet Need + Lack of Alternatives = Commercial Viability?' and will also engage in investor meetings. This participation underscores Neurogene's commitment to advancing gene therapy as a viable treatment option for rare diseases.

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Neurogene (Nasdaq: NGNE) announced its addition to the Russell 3000® Index, effective July 1, 2024. The annual Russell indexes reconstitution includes the 4,000 largest U.S. stocks as of April 30, 2024, ranked by market capitalization. Neurogene's inclusion will last for one year, and the company will also join the Russell 2000® and Russell Microcap® indexes. CEO Rachel McMinn highlighted the company's progress, including its upcoming interim clinical efficacy data for the NGN-401 gene therapy trial for Rett syndrome, expected in Q4 2024. The NGN-401 program was also selected for the FDA’s START Pilot Program.

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Neurogene announced that the first patient in the high-dose cohort of their NGN-401 gene therapy trial for Rett syndrome has been dosed. Early data shows that high-dose NGN-401 is well-tolerated. Additionally, interim safety data from the low-dose cohort, shared at the IRSF ASCEND 2024 Summit, indicates a favorable safety profile with no new treatment-related adverse events (AEs). The company plans to release interim efficacy data from the low-dose cohort in Q4 2024 and is working with the FDA under its START Pilot Program to expedite NGN-401's development.

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FAQ

What is the current stock price of Neurogene (NGNE)?

The current stock price of Neurogene (NGNE) is $23.2 as of December 11, 2024.

What is the market cap of Neurogene (NGNE)?

The market cap of Neurogene (NGNE) is approximately 341.7M.

What does Neurogene, Inc. do?

Neurogene, Inc. is a clinical-stage biotechnology company focused on developing gene therapies for severe neurological diseases.

What is Neurogene's lead product candidate?

Neurogene's lead product candidate is NL-201, a de novo protein designed to mimic the activity of cytokines IL-2 and IL-15 for cancer treatment.

Where is Neurogene, Inc. headquartered?

Neurogene, Inc. is headquartered in Seattle, Washington.

What technology does Neurogene use for its treatments?

Neurogene uses its proprietary EXACT gene regulation technology for precise control over gene expression in its therapy development.

When was Neurogene, Inc. founded?

Neurogene, Inc. was founded in 2003.

What was the former name of Neurogene, Inc.?

The company was formerly known as Aquinox Pharmaceuticals, Inc. before changing its name to Neoleukin Therapeutics, Inc. in 2019.

What types of diseases does Neurogene aim to treat?

Neurogene aims to treat severe neurological diseases, cancer, inflammation, and autoimmunity disorders.

What is the EXACT gene regulation technology?

The EXACT gene regulation technology is a proprietary system developed by Neurogene to offer precise control over gene expression, enhancing the safety and efficacy of gene therapies.

Does Neurogene have any partnerships?

Yes, Neurogene has established significant partnerships and collaborations within the biotech industry to advance its research and development efforts.

How can I stay updated about Neurogene, Inc.?

You can stay updated about Neurogene, Inc. by visiting the News section on StockTitan for the latest financial performance, new research, and other significant developments.

Neurogene, Inc.

Nasdaq:NGNE

NGNE Rankings

NGNE Stock Data

341.66M
13.39M
10.69%
110.72%
7.04%
Biotechnology
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