Regenxbio Inc
NASDAQ:RGNX
Regenxbio Inc
REGENXBIO, Inc. is a clinical-stage biotechnology company, which engages in the development, commercialization, and licensing of recombinant adeno-associated virus gene therapy. The company is headquartered in Rockville, Maryland and currently employs 372 full-time employees. The company went IPO on 2015-09-17. The Company’s gene therapy product candidates are designed to deliver functional genes to address genetic defects in cells, enabling the production of therapeutic proteins or antibodies that are intended to impact disease. Its gene therapy product candidates use adeno-associated virus (AAV) vectors from its gene delivery platform, called NAV Technology Platform. Its product candidate includes RGX-314, RGX-111, RGX-121, RGX-202, RGX 181 and RGX-381. RGX-314 is meant for the treatment of wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR) and other anti-VEGF treated conditions. The company is also developing product candidates, such as RGX-111 and RGX-121, to address the neurological symptoms of two severe genetic lysosomal storage diseases, Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II). RGX-202 is its product candidate for the treatment of duchenne muscular dystrophy (DMD).
REGENXBIO, Inc. is a clinical-stage biotechnology company, which engages in the development, commercialization, and licensing of recombinant adeno-associated virus gene therapy. The company is headquartered in Rockville, Maryland and currently employs 372 full-time employees. The company went IPO on 2015-09-17. The Company’s gene therapy product candidates are designed to deliver functional genes to address genetic defects in cells, enabling the production of therapeutic proteins or antibodies that are intended to impact disease. Its gene therapy product candidates use adeno-associated virus (AAV) vectors from its gene delivery platform, called NAV Technology Platform. Its product candidate includes RGX-314, RGX-111, RGX-121, RGX-202, RGX 181 and RGX-381. RGX-314 is meant for the treatment of wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR) and other anti-VEGF treated conditions. The company is also developing product candidates, such as RGX-111 and RGX-121, to address the neurological symptoms of two severe genetic lysosomal storage diseases, Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II). RGX-202 is its product candidate for the treatment of duchenne muscular dystrophy (DMD).
Pipeline Progress: REGENXBIO completed pivotal trial enrollment for Duchenne muscular dystrophy (RGX-202) ahead of plan and expects top-line data in early Q2 2026, with a BLA submission targeted for mid-2026.
MPS II Milestone: The RGX-121 gene therapy for MPS II is under FDA review with a PDUFA date of February 8, 2026; management remains confident about potential approval.
Retinal Programs: Enrollment is complete in global Phase III trials for wet AMD with AbbVie, covering over 1,200 patients, and the company is preparing for pivotal studies in diabetic retinopathy.
Financial Strength: Cash, cash equivalents, and marketable securities rose to $302 million as of September 30, 2025; current cash runway is projected into early 2027, with nondilutive financing options that could extend this further.
Manufacturing Readiness: Commercial batches of RGX-202 are already produced; the Rockville facility can manufacture up to 2,500 doses per year, supporting both Duchenne and MPS II launches.
