Neurogene Inc.
↗New York, NY
Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare, devastating neurological diseases. The company utilizes its proprietary EXACT (Expression Attenuation Cell Therapy) technology platform to deliver transgene levels within a narrow, therapeutic range, avoiding the toxicities associated with conventional gene therapy over-expression.
Their primary focus is on addressing unmet needs in disorders like Rett syndrome and CLN5 Batten disease. By combining internal manufacturing capabilities with advanced transgene regulation technology, Neurogene aims to provide potentially curative treatments for complex genetic conditions that have historically been difficult to target safely.
CLASSIFICATION
SIZE & FINANCIALS
Employees:51-200
Revenue:$0M (Pre-revenue)
Founded:2018
Ownership:public
Status:operating
FUNDING
Stage:Post-IPO
Total Raised:$285M
Investors:EcoR1 Capital, Redmile Group, Samsara BioCapital, Cormorant Asset Management, Casdin Capital
STOCK
Exchange:NASDAQ
Ticker:NGNE
Market Cap:$245M
PIPELINE
Stage:Phase 2
Lead Drug Stage:Phase 1/2
Modalities:Gene therapy, AAV, EXACT Technology
Active Trials:2
Trial Phases:Phase 1: 2 | Phase 2: 2
FDA Approvals:0
EMA Approvals:0
CORPORATE STRUCTURE
Key Partnerships:University of Edinburgh (Rett syndrome research), Neoleukin Therapeutics (Reverse merger partner)
COMPETITION
Position:Emerging
Competitors:Taysha Gene Therapies, Acadia Pharmaceuticals, Ultragenyx, REGENXBIO
LEADERSHIP
Key Executives:
Rachel McMinn, Ph.D. - Founder and CEO
Christine Mikail - President
Scientific Founders:Rachel McMinn, Ph.D.
Board Members:Rachel McMinn, Stephen Squinto, William Slattery
LINKS
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