REGENXBIO Inc.
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 Inc.
9804 Medical Center Dr, Rockville, MD 20850
Patents
Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase
US-12121567-B2
View DetailsInjection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
US-12070510-B2
View DetailsTreatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS)
US-11986515-B2
View DetailsTreatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) produced by human neural or glial cells
US-11613739-B2
View Details
Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase
US-12121567-B2
View DetailsInjection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
US-12070510-B2
View DetailsTreatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS)
US-11986515-B2
View DetailsTreatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) produced by human neural or glial cells
US-11613739-B2
View DetailsProducts
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
Clinical- and commercial-grade manufacturing of AAV vectors at scales up to 2,000 L within a cGMP facility.
Process development services focused on suspension-based production, optimization of yields and product purity, and technology transfer to clinical/commercial manufacturing.
Licensing and partnership arrangements to enable external development of AAV therapeutics using an internally developed gene delivery platform.
Design and execution of in vivo efficacy and safety studies across multiple animal models to support IND-enabling packages.
Clinical program design and management across Phase I–III trials, including pivotal studies and patient enrollment activities for gene therapy candidates.
Clinical- and commercial-grade manufacturing of AAV vectors at scales up to 2,000 L within a cGMP facility.
Process development services focused on suspension-based production, optimization of yields and product purity, and technology transfer to clinical/commercial manufacturing.
Licensing and partnership arrangements to enable external development of AAV therapeutics using an internally developed gene delivery platform.
Design and execution of in vivo efficacy and safety studies across multiple animal models to support IND-enabling packages.
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
News & Updates
REGENXBIO has announced a strategic royalty monetization agreement that secures $150 million at closing, extending cash runway into early 2027, with additional potential non-dilutive funding opportunities.
The FDA has accepted and given priority review to the Biologics License Application (BLA) for RGX-121 for the treatment of MPS II, with a target action date of November 9, 2025.
The company reports that their Duchenne muscular dystrophy gene therapy RGX-202 is on track for BLA submission in mid-2026, with pivotal trial enrollment more than half complete and additional data expected in the first half of 2025.
Interim clinical data on RGX-202 for Duchenne muscular dystrophy presented by Dr. Aravindhan Veerapandiyan.
Clinical data presented by Dr. Carolina Tesi-Rocha on RGX-202 for Duchenne muscular dystrophy.
REGENXBIO has announced a strategic royalty monetization agreement that secures $150 million at closing, extending cash runway into early 2027, with additional potential non-dilutive funding opportunities.
The FDA has accepted and given priority review to the Biologics License Application (BLA) for RGX-121 for the treatment of MPS II, with a target action date of November 9, 2025.
The company reports that their Duchenne muscular dystrophy gene therapy RGX-202 is on track for BLA submission in mid-2026, with pivotal trial enrollment more than half complete and additional data expected in the first half of 2025.
Interim clinical data on RGX-202 for Duchenne muscular dystrophy presented by Dr. Aravindhan Veerapandiyan.
Clinical data presented by Dr. Carolina Tesi-Rocha on RGX-202 for Duchenne muscular dystrophy.