Neurogene to Present Interim Clinical Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome
October 21 2024 - 7:30AM
Business Wire
Company to host webcast to review data on
November 11 at 4:30 p.m. ET
Late-breaker poster to be presented during
Child Neurology Society Meeting on November 12
Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded
to bring life-changing genetic medicines to patients and families
affected by rare neurological diseases, today announced that it
will host a webcast to 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 on November
11, 2024 at 4:30 p.m. ET. Safety data from the low- and high-dose
cohorts will also be shared.
These data will also be presented in a late-breaking poster
presentation during the 53rd Annual Child Neurology Society (CNS)
Annual Meeting on November 12 at 12:30 p.m. PT in San Diego,
CA.
Neurogene’s Analyst and Investor Webcast/Conference
Call
The live webcast and conference call information will be
accessible from the Investor Relations section of Neurogene’s
website under Events, where the webcast replay will also be
available for a limited time.
Neurogene’s CNS Meeting Presentation
- Poster Title: NGN-401, a Novel Regulated Gene Therapy
for Rett Syndrome: Preliminary Results from the First-in-Human
Study
- Presenter: Bernhard Suter, M.D., Medical Director of the
Blue Bird Circle Rett Center at Texas Children’s Hospital,
Associate Professor of Pediatrics and Neurology at Baylor College
of Medicine, and principal investigator in the NGN-401 clinical
trial
About Neurogene The mission of Neurogene is to treat
devastating neurological diseases 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 designing products to maximize
potency and purity for an optimized efficacy and safety profile.
The Company’s novel and proprietary EXACT transgene regulation
platform technology allows for the delivery of therapeutic levels
while limiting transgene toxicity associated with conventional gene
therapy. Neurogene has constructed a state-of-the-art gene therapy
manufacturing facility in Houston, Texas. CGMP production of
NGN-401 was conducted in this facility and will support pivotal
clinical development activities. For more information, visit
www.neurogene.com.
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version on businesswire.com: https://www.businesswire.com/news/home/20241018666990/en/
Company: Cara Mayfield Vice President, Corporate Affairs
cara.mayfield@neurogene.com Investor: Melissa Forst Argot
Partners Neurogene@argotpartners.com
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