Neurogene Inc.

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Neurogene Inc.

Neurogene’s mission is to turn devastating neurological diseases into treatable conditions. For more information, visit www.neurogene.com. laws and regulations.

About Neurogene

The mission of Neurogene is to turn devastating neurological diseases into treatable conditions to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and by designing products to maximize potency and purity for an optimized efficacy and safety profile. The company’s novel and proprietary EXACT gene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Terms of Use & Community Rules
We think social media is a great way to share what excites us and what we are working on. We look forward to engaging with families and friends of the rare disease community and invite you to be an active participant in the conversation. As a biotechnology company, we operate in a highly regulated industry and are required to adhere to certain laws and regulations. Therefore, all comments submitted to our page must be monitored and reviewed and may be subject to removal. To help ensure that your comments will be taken into consideration and posted, please adhere to the following community rules. We do not allow:
•Offensive, defamatory or obscene language;
•Any reference to pharmaceutical products, treatments or questions related to medical advice;
•Information to pitch products or services; or
•Any adverse events or side effects possibly due to a medication. Any adverse event concerns should be reported immediately to http://www.fda.gov/medwatch or by calling 1-800-FDA-1088. Comments must clearly relate to Neurogene Inc.’s discussion topics. Any posts or comments submitted become the property of Neurogene Inc. and can be removed at our discretion. This page is intended for U.S. residents only, 18 years of age or older, and is governed by U.S. All comments shared are not necessarily reflective of Neurogene Inc. We reserve the right to update our community rules or remove this page, or any of its content or comments, at any time. Any information provided here is for informational purposes and is not a substitute for medical advice from a healthcare professional. Always visit or speak to a qualified healthcare professional regarding your medical condition. All decisions regarding patient care must be handled by a healthcare professional and be made based on the unique needs of each patient. While Neurogene Inc. makes reasonable efforts to include accurate and up-to-date information on this site, Neurogene Inc. makes no representations or warranties as to the accuracy and assumes no liability for any errors or omissions in the content of this site. Read more concerning our Privacy Policy and Terms of Use on our website: https://www.neurogene.com.

03/19/2026

Do you or someone you know need a quick overview to help answer the question, “What is Rett syndrome, exactly?” The Rett Syndrome Research Trust has a great resource page with a number of helpful resources. https://reverserett.org/about-rett-syndrome/

03/11/2026

Neurogene is a proud sponsor of the International Rett Syndrome Foundation’s Rett Forward Master Class. The course contains three modules to help break down key topics around navigating clinical trials and emerging therapies for . Take a look. https://www.rettsyndrome.org/rett-forward/

03/05/2026

We’ve updated our website! The “Patients and Families” section on Neurogene.com contains resources, our letters to the community, perspectives from families, and more information about our clinical program in . Take a look! https://www.neurogene.com/patients-and-families/

03/03/2026

Neurogene was honored and grateful to have Kate, Alyssa & Rick, parents of boys with Rett syndrome, share their perspectives in recognition of . They helped us understand the experiences of boys with . Check out the images for their main message and some great photos from the call.

02/26/2026

Today, Neurogene announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to NGN-401, our investigational gene therapy for Rett syndrome. Learn more about this designation by reading our letter to the community: https://www.neurogene.com/communication-corner/

02/23/2026

This week is , and we will once again “Light up for Rare” to show our support of the Rare Disease community. Learn how you can join us as we . https://rarediseases.org/rare-disease-day/light-up-a-monument/monument-map/

02/20/2026

Today is National Caregivers Day, which was established to celebrate professional caregivers, such as home health aides, nurses, and therapists, and family caregivers who provide daily health support to loved ones. Caregivers do essential work, so join us in taking time today to !

02/17/2026

Did you know rare diseases impact more than 300 million people globally. February 28 is , a day dedicated to raising awareness of , and the people who live with them.

The Official Campaign Video of Rare Disease Day 2026- Learn more and get involved!👉 https://www.rarediseaseday.org/

02/12/2026

Rett syndrome has an estimated incidence of 1 out of every 10,000 live female births, making it one of more than 6,000 rare diseases that impact people around the world. On February 28, , we join the community in raising awareness.

02/09/2026

Neurogene has created a summary of previously shared interim clinical trial data (Nov. 2025) from our Phase 1/2 clinical trial evaluating NGN-401, our investigational gene therapy for Rett syndrome. View a summary of the update here: https://bit.ly/3YWsEWO

11/26/2025

During this season of gratitude, we are thankful for you and the entire Rett syndrome community, our partners, healthcare providers, researchers, and all others who work tirelessly on behalf of the community. We are grateful to be able to focus on the future of life-changing genetic medicines and to continue working side by side with the families impacted by neurological diseases.

11/10/2025

We are excited to announce that Neurogene’s registrational clinical trial, Embolden™, for our investigational gene therapy for Rett syndrome, NGN-401, is now enrolling and the first participant has been dosed. Visit https://www.neurogene.com/patients-and-families/about-rett-syndrome/ to read our letter to the community about Embolden.

Address

535 W 24th Street, 5th Floor
New York, NY
10011

Telephone

+18772375020

Website

http://www.neurogene.com/, https://www.linkedin.com/company/neurogeneinc/

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