Neurogene Inc.

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.

At Neurogene, we strive to not just listen to families in the rare disease community, but to truly integrate their persp...
08/27/2026

At Neurogene, we strive to not just listen to families in the rare disease community, but to truly integrate their perspectives into the core of our work. That’s why we are excited to announce the formation of our new Caregiver Advisory Council – a group of six parents from the community. We are truly grateful to each of them for agreeing to participate and share their insights as parents of sons and daughters ranging from 5 to 29 years old. We look forward to their involvement as we shape our future to align with the needs of families across the community.

We are looking forward to meeting with the Rett syndrome community on Wednesday, September 9th at 12 p.m. ET, and we hop...
08/24/2026

We are looking forward to meeting with the Rett syndrome community on Wednesday, September 9th at 12 p.m. ET, and we hope you’ll join us! Thank you to the International Rett Syndrome Foundation for hosting and providing the opportunity to share updates. Zoom registration link available below.

Registration is now open for the next Rett in Focus webinar!

Join IRSF on Wednesday, September 9th, at 12 p.m. ET for Rett in Focus: Neurogene Community Update, featuring information from Neurogene Inc. about NGN-401, its investigational gene therapy for Rett syndrome.

Building on updates shared at the ASCEND 2026 National Summit, Neurogene will discuss recent developments in the NGN-401 clinical program and what they anticipate will come next. Families can also hear about new educational resources and community initiatives, including Neurogene Cares and its Caregiver Advisory Council.

The webinar will include a moderated Q&A based on questions submitted in advance during registration.

Register: https://us02web.zoom.us/webinar/register/WN_-Qxmq0ksSZugZHt_iMK5BQ

We were proud to sponsor and attend the recent Day at Rady Children’s Hospital in San Diego, California. It is always ...
08/17/2026

We were proud to sponsor and attend the recent Day at Rady Children’s Hospital in San Diego, California. It is always an honor to spend time with families. Thanks to the International Rett Syndrome Foundation for creating these important events for caregivers. Keep an eye out for a RettEd Day near you: https://www.rettsyndrome.org/families/rett-education/retted-days/

We’re proud to be a supporting sponsor of the International Rett Syndrome Foundation’s upcoming Days in San Diego, Cin...
08/14/2026

We’re proud to be a supporting sponsor of the International Rett Syndrome Foundation’s upcoming Days in San Diego, Cincinnati, Grand Rapids and Miami. Each session offers a unique opportunity to learn from experts, connect with your local Rett community and gain tools to support your loved one. Take a look at the IRSF website for more information and to register for an upcoming RettEd Day.

Do you want to learn about the investigational gene therapy, NGN-401? Our website has more details, including a summary ...
07/23/2026

Do you want to learn about the investigational gene therapy, NGN-401? Our website has more details, including a summary of the recently released data (as of June 2026) from our clinical program evaluating NGN-401 for the treatment of Rett syndrome. https://bit.ly/42wU5bh

We were honored to attend the International Rett Syndrome Foundation’s ASCEND meeting last week where we heard from so m...
07/06/2026

We were honored to attend the International Rett Syndrome Foundation’s ASCEND meeting last week where we heard from so many families impacted by . Thank you to everyone who stopped by our booth to say hi and share your thoughts with us!

Neurogene has summarized the recently released clinical trial data, as of June 2026, from our Phase 1/2 clinical trial e...
06/29/2026

Neurogene has summarized the recently released clinical trial data, as of June 2026, from our Phase 1/2 clinical trial evaluating NGN-401, our investigational gene therapy for Rett syndrome. View a summary of the update at https://www.neurogene.com/patients-and-families/

Attending ASCEND this week? Stop by our booth to receive your Neurogene Cares Family Care Kit and join in a fun activity...
06/29/2026

Attending ASCEND this week? Stop by our booth to receive your Neurogene Cares Family Care Kit and join in a fun activity for the whole family. The kit contains useful items to have while traveling, including a waterproof bag, water bottle, bib, tissues, alcohol wipes and more, so you have these items on hand. Learn more about our Neurogene Cares program: https://www.neurogene.com/patients-and-families/ -cares

We are proud to announce “Neurogene Cares,” our new outreach program designed to support families beyond our research by...
06/26/2026

We are proud to announce “Neurogene Cares,” our new outreach program designed to support families beyond our research by addressing practical and meaningful needs. To kick off the program, our employees and the International Rett Syndrome Foundation’s CEO, Laura Hameed, recently joined together to pack Family Care Kits – a waterproof bag, water bottle, bib, tissues, alcohol wipes and more – for families traveling to the upcoming IRSF ASCEND conference. Learn more and share your ideas for our next Neurogene Cares activity: https://www.neurogene.com/patients-and-families/ -cares

06/25/2026

Neurogene uses ICV (intracerebroventricular) delivery for the investigational gene therapy, NGN-401 for Rett syndrome.

Watch our new video to learn what happens during an ICV procedure for Rett syndrome. https://www.neurogene.com/patients-and-families/

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