REGENXBIO Analysis: $358M Raised
What is REGENXBIO?
Pioneering gene therapy with NAV® Technology
Employees
201-500
Founded
2009
Valuation
358.37M
Revenue Growth Rate
45.3%
Product Features & Capabilities
- NAV® Technology Platform
- Gene therapy product candidates for retinal diseases
- Gene therapy product candidates for metabolic diseases
- Gene therapy product candidates for neurodegenerative diseases
Use Cases
Develop gene therapies for retinal diseases like wet AMD; Create treatments for Duchenne muscular dystrophy; Address MPS II (Hunter Syndrome) with gene therapy; Utilize over 100 novel AAV vectors in clinical programs; Manufacture gene therapy products in-house
Investment Focus
REGENXBIO is primarily focused on developing gene therapy product candidates in the following therapeutic areas: retinal diseases, metabolic disorders, and neurodegenerative conditions. Their mission is to leverage the curative potential of gene therapy to improve lives.
Other Considerations
Awarded Washington Post's Top Work Places in 2021, 2022 & 2023; Developed partnerships with various organizations in gene therapy; Engaged in robust clinical programs across multiple therapeutic areas
Key Innovations
REGENXBIO has developed several key innovations and products in the biotech sector, particularly in gene therapy for serious health conditions. Notable products include:
- ABBV-RGX-314 - A gene therapy candidate for treating wet age-related macular degeneration (AMD) and diabetic retinopathy, utilizing the NAV® AAV8 vector to deliver a gene encoding a monoclonal anti-VEGF antibody fragment. It is currently in pivotal and Phase II stages of development.
- RGX-202 - Designed for the treatment of Duchenne muscular dystrophy, RGX-202 employs the NAV® AAV8 vector to deliver a transgene for a novel microdystrophin.
- RGX-121 - Aimed at treating MPS II (Hunter Syndrome), RGX-121 uses the NAV® AAV9 vector to deliver the human iduronate-2-sulfatase gene to the central nervous system. It is also in the pivotal stage, with a Biologics License Application (BLA) filing expected in 2024.
These innovations reflect REGENXBIO's commitment to advancing gene therapy solutions.
Awards Recognition
REGENXBIO has received several recognitions in the biotech field, including being awarded the Washington Post's Top Workplaces in 2021, 2022, and 2023. Additionally, they received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for their RGX-121 gene therapy for Hunter syndrome.
Latest Funding Round
REGENXBIO recently secured a strategic partnership with Nippon Shinyaku, which was announced in March 2025. This partnership includes an upfront payment of $110 million and potential milestone payments totaling up to $700 million, bringing the total value of the partnership to $810 million.
Regulatory Approvals
- RGX-121 - A Biologics Licensing Application (BLA) has been submitted for RGX-121, which is on track for potential FDA approval in the second half of 2025. The company has also had a successful pre-BLA meeting with the FDA to support an accelerated approval pathway for this treatment.
- RGX-202
- RGX-111 - The FDA granted Rare Pediatric Disease Designation to RGX-111, an investigational gene therapy product.
These approvals and designations highlight REGENXBIO's commitment to advancing gene therapies for various conditions.
Partnerships
REGENXBIO has established several significant partnerships in the biotech industry, including:
- Nippon Shinyaku - REGENXBIO entered a strategic partnership with Nippon Shinyaku to develop and commercialize gene therapies RGX-121 and RGX-111 for MPS diseases.
- AbbVie - REGENXBIO collaborates with AbbVie on the development of RGX-314, a gene therapy for retinal diseases such as wet AMD and diabetic retinopathy.
- Biogen - REGENXBIO has granted Biogen an exclusive worldwide research license for its NAV AAV8 and AAV9 vectors, which are used in the development of gene therapies.
- Neurimmune - REGENXBIO partnered with Neurimmune to develop novel vectorized human antibodies for chronic neurodegenerative diseases.
These collaborations highlight REGENXBIO's commitment to advancing gene therapy treatments through strategic partnerships.
Find more companies like REGENXBIO
See something that needs updating? Suggest edits to this profile.
Financial Overview
$358MTotal Raised
REGENXBIO recently secured a strategic partnership with Nippon Shinyaku, which was announced in March 2025. This partnership includes an upfront payment of $110 million and potential milestone payments totaling up to $700 million, bringing the total value of the partnership to $810 million.
