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REGENXBIO Inc. (RGNX)

REGENXBIO Inc. is a biotechnology company operating in the clinical stage, dedicated to developing gene therapy candidates. These therapies are designed to introduce genes into cells, aiming either to rectify genetic deficiencies or to stimulate the body's own cells to produce therapeutic proteins or antibodies to combat diseases. Central to its therapeutic development is the proprietary NAV Technology Platform, an adeno-associated virus-based gene delivery system. The company's primary investigational asset, RGX-314, is currently in Phase III clinical trials for treating wet age-related macular degeneration. Its pipeline also includes RGX-121 and RGX-111, both in Phase I/II trials for mucopolysaccharidosis type II and type I, respectively; RGX-181, which is in preclinical development for late-infantile neuronal ceroid lipofuscinosis type II disease; RGX-202, undergoing Phase I/II evaluation for Duchenne muscular dystrophy; and RGX-381, at the preclinical stage for addressing the ocular manifestations of CLN2 disease. In addition to its internal programs, REGENXBIO Inc. licenses its NAV Technology Platform to other biotech and pharmaceutical enterprises. It also maintains a collaborative and licensing agreement with Neurimmune AG for the advancement of innovative gene therapies. Founded in 2008, REGENXBIO Inc. maintains its headquarters in Rockville, Maryland.

Sector: Biological Products, (No Diagnostic Substances)

Industry: Biotechnology

REGENXBIO Inc. logo

REGENXBIO Inc.

NASDAQ:RGNXHealthcare
-2.83%$7.20
AH$7.20+0.00%vs close
Mkt Cap$475.29M
Float51.72M
O/S66.01M
Vol
$ Vol
RVOL
Inst Own63.7%
SI19.4%
Earnings
Dilution
REGENXBIO Inc. logoNASDAQ:RGNX$7.20-2.83%
Phase Distribution
18 lead-sponsored trials·7 active·4 completed
Phase 1/24
Phase 2/32
Phase 31
Not applicable11
(7 more as collaborator)
Status Overview
Recruiting2
Active, not recruiting5
Completed4
Stopped5
Expanded access1
Unknown1

Trials need funding. See this company's dilution risk and cash position.

Clinical Trials
Sponsor role
Status
Phase
  • Phase 3
    Active, not recruiting

    RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

    RGX-121-3102

    MPS II, Hunter Syndrome (MPS II)

    Enrollment
    2
    Start
    Nov 2025
    Primary Completion
    Apr 2028est.
    Completion
    May 2031est.
    NCT07236606
  • Phase 2/3
    Active, not recruiting

    CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

    RGX-121

    Mucopolysaccharidosis Type II (MPS II)

    Enrollment
    48
    Start
    Sep 2018
    Primary Completion
    Nov 2023
    Completion
    Aug 2025est.
    NCT03566043
  • Phase 2/3
    Active, not recruiting

    AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

    RGX-202

    Duchenne Muscular Dystrophy

    Enrollment
    65
    Start
    Jan 2023
    Primary Completion
    Sep 2026est.
    Completion
    Jun 2028est.
    NCT05693142
  • Phase 1/2
    Terminated

    A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

    AAV directed hLDLR gene therapy

    Homozygous Familial Hypercholesterolemia (HoFH)

    Enrollment
    9
    Start
    Mar 2016
    Primary Completion
    Nov 2020
    Completion
    Nov 2020
    NCT02651675
  • Phase 1/2
    Completed

    Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial

    RGX-314

    Neovascular Age-related Macular Degeneration, Wet Age-related Macular Degeneration

    Enrollment
    42
    Start
    Mar 2017
    Primary Completion
    Nov 2019
    Completion
    Jun 2021
    NCT03066258
  • Phase 1/2
    Suspended

    RGX-111 Gene Therapy in Patients With MPS I

    RGX-111

    Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome +1

    Enrollment
    21
    Start
    Apr 2019
    Primary Completion
    Jul 2027est.
    Completion
    Mar 2029est.
    NCT03580083
  • Phase 1/2
    Completed

    RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

    RGX-121

    Mucopolysaccharidosis Type II (MPS II)

    Enrollment
    6
    Start
    Mar 2021
    Primary Completion
    May 2023
    Completion
    May 2024
    NCT04571970
  • Observational
    Completed

    Mucopolysaccharidosis Type II Natural History

    -

    Mucopolysaccharidosis II

    Enrollment
    36
    Start
    Sep 2017
    Primary Completion
    Mar 2022
    Completion
    Mar 2022
    NCT03529786
  • Observational
    Unknown status

    A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

    Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy

    Homozygous Familial Hypercholesterolemia (HoFH)

    Enrollment
    8
    Start
    Sep 2019
    Primary Completion
    Sep 2024est.
    Completion
    Sep 2025est.
    NCT04080050
  • Observational
    Completed

    Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

    -

    Homozygous Familial Hypercholesterolemia (HoFH)

    Enrollment
    4
    Start
    Dec 2019
    Primary Completion
    Apr 2020
    Completion
    Apr 2020
    NCT04148001
  • Observational
    Withdrawn

    An Observational Study in Children With CLN2 Batten Disease

    -

    Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

    Enrollment
    0
    Start
    Mar 2021est.
    Primary Completion
    Oct 2023est.
    Completion
    Oct 2023est.
    NCT04462692
  • Observational
    Withdrawn

    A Retrospective, Natural History Study in Children With CLN2

    -

    Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

    Enrollment
    0
    Start
    Mar 2021est.
    Primary Completion
    Apr 2022est.
    Completion
    Apr 2022est.
    NCT04480476
  • Observational
    Withdrawn

    Mucopolysaccharidosis Type II Observational

    Observational

    Mucopolysaccharidosis II

    Enrollment
    0
    Start
    Mar 2022est.
    Primary Completion
    Jul 2025est.
    Completion
    Jul 2025est.
    NCT04591834
  • Observational
    Active, not recruiting

    Long-term Follow-Up for RGX-121

    Long-term Follow-Up

    Mucopolysaccharidosis II

    Enrollment
    27
    Start
    Mar 2021
    Primary Completion
    Oct 2028est.
    Completion
    Oct 2028est.
    NCT04597385
  • Observational
    Active, not recruiting

    AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD

    AAV8 DetectCDx

    Duchenne Muscular Dystrophy

    Enrollment
    200
    Start
    Dec 2022
    Primary Completion
    Jun 2026
    Completion
    Sep 2026est.
    NCT05683379
  • Observational
    Enrolling by invitation

    Long Term Follow-Up for RGX-111

    No Intervention

    Mucopolysaccharidosis I

    Enrollment
    21
    Start
    Jul 2023
    Primary Completion
    Sep 2027est.
    Completion
    Sep 2027est.
    NCT06103487
  • Observational
    Enrolling by invitation

    Long Term Follow-up for RGX-202

    No Intervention

    Duchenne Muscular Dystrophy

    Enrollment
    66
    Start
    May 2024
    Primary Completion
    Dec 2029est.
    Completion
    Dec 2029est.
    NCT06491927
  • Expanded access
    Temporarily not available

    Expanded Access to RGX-202

    RGX-202

    Enrollment
    -
    Start
    -
    Primary Completion
    -
    Completion
    -
    NCT07652606
Search ClinicalTrials.gov for “Regenxbio”