Regenxbio Inc
NASDAQ:RGNX
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Regenxbio Inc
NASDAQ:RGNX
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Thunder Bridge Capital Partners IV Inc
NASDAQ:THCP
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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).
RGX-202: Dosing in the pivotal Duchenne study completed; top-line pivotal data expected early Q2 2026 and company plans a midyear pre-BLA meeting with FDA targeting accelerated approval.
Phase I/II results: Company highlighted a 7.4% average functional improvement versus cTAP at 18 months and no SAEs/AESIs reported to date in the Phase I/II Duchenne cohort.
Retina programs: Subretinal wet AMD pivotal readouts (Atmosphere/Ascent) due Q4 2026; NAVIGATE diabetic retinopathy Phase IIb/III site activations underway with first dosing expected next quarter and a $100M AbbVie milestone upon first dose.
MPS programs update: CRL received for RGX-121 and clinical holds for RGX-111 and RGX-121 following a SAE with vector integration; company says holds are addressable and is preparing a Type A meeting and BLA resubmission.
Safety differentiation: Management attributes favorable safety to product purity (>80% full capsids), construct design, and proactive immune‑suppression regimen; no thrombocytopenia or liver injury reported in their Phase I/II Duchenne patients.
Finances & runway: Cash, equivalents and marketable securities were $241 million at 12/31/25 (versus $245 million a year prior); company expects cash to fund operations into early 2027, potentially extended into H2 2027 if AbbVie milestone and additional royalty monetization funds materialize.
Commercial readiness & partnerships: Company is scaling manufacturing and co-development with AbbVie; continues royalty monetization and highlights potential near-term commercial transition if late-stage readouts are positive.
