REGENXBIO
REGENXBIO is a leading clinical-stage biotechnology company dedicated to improving lives through the curative potential of gene therapy. They focus on developing gene therapy product candidates across various therapeutic areas using their proprietary NAV® Technology Platform, which involves delivering genes to cells via adeno-associated virus (AAV) vectors. Their mission is to rapidly discover and develop innovative AAV therapeutics with the potential for long-lasting effects after a single administration, aiming to significantly alter the course of disease and improve patient outcomes.
Industries
Nr. of Employees
large (251-1000)
REGENXBIO
Rockville, Maryland, United States, North America
Products
Investigational AAV-based gene therapy candidates (retinal, metabolic, neurodegenerative, neuromuscular)
Pipeline of investigational AAV gene therapy candidates targeting ocular (retina), metabolic, central nervous system and neuromuscular indications, developed for single-administration long-term expression.
Investigational AAV-based gene therapy candidates (retinal, metabolic, neurodegenerative, neuromuscular)
Pipeline of investigational AAV gene therapy candidates targeting ocular (retina), metabolic, central nervous system and neuromuscular indications, developed for single-administration long-term expression.
Services
cGMP commercial-scale AAV manufacturing
Clinical- and commercial-grade manufacturing of AAV vectors at scales up to 2,000 L within a cGMP facility.
Process development and scale-up for AAV production
Process development services focused on suspension-based production, optimization of yields and product purity, and technology transfer to clinical/commercial manufacturing.
Licensing of proprietary AAV gene delivery platform
Licensing and partnership arrangements to enable external development of AAV therapeutics using an internally developed gene delivery platform.
Preclinical development and translational testing
Design and execution of in vivo efficacy and safety studies across multiple animal models to support IND-enabling packages.
Clinical development and trial execution
Clinical program design and management across Phase I–III trials, including pivotal studies and patient enrollment activities for gene therapy candidates.
cGMP commercial-scale AAV manufacturing
Clinical- and commercial-grade manufacturing of AAV vectors at scales up to 2,000 L within a cGMP facility.
Process development and scale-up for AAV production
Process development services focused on suspension-based production, optimization of yields and product purity, and technology transfer to clinical/commercial manufacturing.
Licensing of proprietary AAV gene delivery platform
Licensing and partnership arrangements to enable external development of AAV therapeutics using an internally developed gene delivery platform.
Preclinical development and translational testing
Design and execution of in vivo efficacy and safety studies across multiple animal models to support IND-enabling packages.
Clinical development and trial execution
Clinical program design and management across Phase I–III trials, including pivotal studies and patient enrollment activities for gene therapy candidates.
Expertise Areas
- AAV gene therapy development
- Vector engineering and capsid optimization
- Tissue-targeted delivery strategies
- Preclinical translational pharmacology and tox
Key Technologies
- Adeno-associated virus (AAV) vectors
- Capsid engineering and serotype panels
- Promoter selection for tissue-specific expression
- Suspension bioreactor manufacturing