Neurogene
- Country
- 🇺🇸United States
- Ownership
- -
- Employees
- 91
- Market Cap
- $466.9M
- Introduction
Neurogene, Inc. is a clinical-stage biotechnology company, which engages in the development of product portfolio of genetic medicines for rare neurological diseases. The company was founded in May 2007 and is headquartered in New York, NY.
FDA Places Clinical Hold on Rocket Pharmaceuticals' Danon Disease Gene Therapy After Patient Death
The FDA has placed a clinical hold on Rocket Pharmaceuticals' Phase II gene therapy trial for RP-A501 after a patient developed capillary leak syndrome and subsequently died.
New Global Review Reveals Comprehensive Landscape of Interstitial Cystitis Clinical Trials in 2025
• A newly released global clinical trials review provides extensive data on Interstitial Cystitis research, tracking trial numbers and enrollment trends across major countries worldwide. • The report categorizes trials by region, phase, status, and sponsorship, highlighting key pharmaceutical companies including Astellas Pharma, AbbVie, Johnson & Johnson, and Pfizer as prominent sponsors. • This comprehensive analysis serves as a strategic resource for stakeholders making investment decisions in IC research, offering insights into trial success rates and identifying optimal locations for clinical studies.
Neurogene Unveils Protocol to Reverse HLH Syndrome Associated with High-Dose AAV Gene Therapy
Neurogene has developed a monitoring and treatment algorithm designed to reverse hemophagocytic lymphohistiocytosis (HLH), a rare hyperinflammatory syndrome linked to high-dose AAV gene therapy.
Rett Syndrome Pipeline Analysis Reveals 20+ Companies Developing Novel Therapeutics with Gene Therapy Leading Innovation
A comprehensive 2025 pipeline analysis identifies over 20 companies developing more than 20 therapeutic candidates for Rett syndrome, highlighting significant industry investment in this rare neurological disorder.
Neurogene Halts High-Dose Arm of Rett Syndrome Gene Therapy Trial Following Serious Adverse Event
• Neurogene has suspended enrollment in the high-dose arm of its NGN-401 gene therapy trial for Rett syndrome after a patient developed life-threatening systemic hyperinflammatory syndrome, a known risk with AAV vector therapies. • Despite early efficacy signals showing "meaningful gains of skills and developmental milestones" in low-dose patients, the company's shares plummeted nearly 44% following the safety concerns announcement. • The trial will continue with the low-dose cohort, though with delayed enrollment timeline, as the company works to balance promising efficacy data against serious safety considerations for this devastating neurological disorder.
FDA's START Program Selects First Rare Disease Therapies for Accelerated Development
• The FDA's START program, designed to expedite rare disease therapy development, has chosen its first candidates for enhanced guidance and support. • Denali Therapeutics' DNL12 for mucopolysaccharidosis IIIA, Neurogene's NGN-401 for Rett syndrome, and Larimar Therapeutics' nomlabofusp for Friedreich's ataxia are among the selected therapies. • Grace Science's GS-100 gene therapy for NGLY1 deficiency is also included, marking a significant step forward in addressing this life-threatening condition with no approved treatments. • The START program aims to provide comprehensive support in clinical trial design and data generation, facilitating efficient development of potentially life-saving therapies.
Gene Therapy Advances: Promising Results in SMA, Hemophilia, and Prenatal Treatments
Novartis reports positive Phase 3 results for intrathecal onasemnogene abeparvovec (OAV101 IT) in spinal muscular atrophy (SMA) type 2 patients, showing promise in those aged 2-18.
Key Advances in Neuromuscular and Movement Disorder Treatments Highlighted in 2024
• AbbVie's Vyalev, a 24-hour subcutaneous levodopa infusion, gained FDA approval for managing motor fluctuations in advanced Parkinson's, offering a novel therapeutic approach. • Intellia's CRISPR therapy, NTLA-2001, demonstrated safe redosing in ATTR amyloidosis patients, achieving additive pharmacodynamic effects on the target protein. • A phase 3 study revealed that buntanetap is a safe and well-tolerated drug which improves motor, nonmotor, and cognitive symptoms of Parkinson's disease. • The FDA supported using αSyn-SAA biomarker in Parkinson's clinical trials, enhancing therapeutic development through improved diagnostic precision.
Gene Therapies Show Promise in Targeting the Root Cause of Rett Syndrome
• Two companies, Neurogene and Taysha Gene Therapies, are in Phase 1/2 clinical trials with gene therapies targeting the MECP2 gene for Rett syndrome. • These therapies aim to address the underlying genetic cause of Rett syndrome, unlike existing treatments that only manage symptoms. • Both companies have incorporated regulatory mechanisms to ensure precise MECP2 expression, avoiding complications from overexpression. • Preliminary data from ongoing trials are expected in late 2024 and early 2025, offering hope for a potential disease-modifying treatment.
Solengepras Enters Phase 3 for Parkinson's, SAGE-718 Discontinued, and Neurogene Pauses NGN-401 High-Dose Trial
• Cerevance's solengepras (CVN424) has entered a Phase 3 trial (ARISE) as an adjunctive therapy for Parkinson's disease, with topline data expected in the first half of 2026. • Sage Therapeutics discontinued the development of SAGE-718 (dalzanemdor) for Huntington's disease after disappointing Phase 2 results showing no significant cognitive improvement. • Neurogene paused the high-dose arm of its Phase 1 study of NGN-401, a gene therapy for Rett syndrome, following a serious adverse event in one participant.