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ABBV-RGX-314 Dose 1

Phase 3

AMD | Gene therapy | Ophthalmology |AbbVie Inc.|Last Updated: Aug 19, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindACTIVE_CONTROLLEDDMC
Total Trials1
Total Enrollment735

FDA Designations

No designations recorded

Clinical trial landscape

ABBV-RGX-314 Dose 1 · 2 trials · 8 indications

Phase 3 1Phase 2 1
NCT05407636Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMDAMD
ACTIVE NOT_RECRUITING735 Analytics
PHASE3ACTIVE NOT_RECRUITING
Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD
AMDUnlock trial analytics

Study Endpoints

Primary Endpoints

Mean change from baseline in Best Corrected Visual Acuity (BCVA)
At Week 54

To evaluate the noninferiority of ABBV-RGX-314 relative to aflibercept in mean change from Baseline BCVA at Week 54

Bilateral Treatment Substudy: Incidence of ocular AEs and any SAEs
Week 50

AEs and SAEs through Week 50

Part 1: Proportion of participants achieving a 2-step or greater improvement in DR in the study eye per the ETDRS-DRSS on 4 widefield digital stereoscopic fundus photography at Week 48
At Week 48

To evaluate the effect of ABBV-RGX-314 on DR by the ETDRS DRSS at Week 48.

Part 2: Mean change from baseline in Best Corrected Visual Acuity (BCVA) in the study eye at Week 54.
At Week 54

To evaluate the effect of ABBV-RGX-314 on BCVA at Week 54.

Secondary Endpoints

Incidences of ocular and overall AEs
Through Week 54
Proportion of participants with improved BCVA
Week 54
Proportion of participants with worsened BCVA
Week 54
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ABBV-RGX-314 Dose 1EXPERIMENTALABBV-RGX-314 Dose 1 administered via subretinal delivery one time.
ABBV-RGX-314 Dose 2EXPERIMENTALABBV-RGX-314 Dose 2 administered via subretinal delivery one time.
Control ArmACTIVE_COMPARATORAflibercept administered via intravitreal injection approximately every 8 weeks
Part 1: Observation Control ArmNO_INTERVENTIONObservation Control
Part 1: ABBV-RGX-314 Treatment Arm (Dose 1)EXPERIMENTALABBV-RGX-314 Dose 1
Part 1: ABBV-RGX-314 Treatment Arm (Dose 2)EXPERIMENTALABBV-RGX-314 Dose 2
Part 1: ABBV-RGX-314 Treatment Arm (Dose 3) and Topical SteroidEXPERIMENTALABBV-RGX-314 Dose 3 and Topical Steroid
Part 2: ABBV-RGX-314 Treatment Arm (Dose 4) and Topical SteroidEXPERIMENTALABBV-RGX-314 Dose 4 and Topical Steroid
Part 2: Aflibercept ControlACTIVE_COMPARATORControl treatment arm

Interventions

NameTypeDescription
ABBV-RGX-314 Dose 1GENETICAAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)
ABBV-RGX-314 Dose 2GENETICAAV8 vector containing a transgene for anti-VEGF Fab (Dose 2)
Aflibercept (EYLEA®)BIOLOGICAL2.0 mg (0.05 mLsolution) administered by intravitreal injection approximately every 8 weeks after 3 monthly injections
ABBV-RGX-314 Dose 3GENETICAAV8 vector containing a transgene for anti-VEGF fab (Dose 3)
Topical SteroidDRUGTopical Steroid
ABBV-RGX-314 Dose 4GENETICAAV8 vector containing a transgene for anti-VEGF fab (Dose 4)
AfliberceptBIOLOGICALAflibercept
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Eligibility Criteria

Age Range50 Years to 89 Years
SexALL
Healthy VolunteersNo
Study Sites181

Inclusion Criteria: 1. Age ≥ 50 years and ≤ 89 years 2. An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye 3. Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in the study eye previously treated with anti-VEGF 4. Must be pseudophakic (at least 12 weeks postc...

Countries:United StatesCanadaFranceGermanyHungaryItalyJapanPuerto RicoSpainUnited Kingdom
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Recent Changes (Last 90 Days)

LOWAug 20, 2026NCT04567550primaryCompletionDate: changed
LOWAug 20, 2026NCT04567550primaryCompletionDate: changed
LOWAug 20, 2026NCT04567550primaryCompletionDate: changed
LOWAug 20, 2026NCT04567550primaryCompletionDate: changed
MEDIUMAug 3, 2026NCT05407636Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 3, 2026NCT05407636Status: RECRUITING → ACTIVE_NOT_RECRUITING

Frequently asked questions about ABBV-RGX-314 Dose 1

What is ABBV-RGX-314 Dose 1 used for?

ABBV-RGX-314 Dose 1 is an investigational gene therapy being developed for the treatment of age-related macular degeneration (AMD) and diabetic retinopathy (DR). It is administered in the suprachoroidal space and is currently in Phase 3 clinical development for these ophthalmologic conditions.

How does ABBV-RGX-314 Dose 1 work?

ABBV-RGX-314 Dose 1 is a gene therapy designed to deliver a gene that encodes for a therapeutic protein. While the specific molecular target is not disclosed, the therapy is intended to provide a durable treatment effect for retinal diseases by enabling the eye to produce the therapeutic agent locally.

Who is developing ABBV-RGX-314 Dose 1?

ABBV-RGX-314 Dose 1 is being developed by AbbVie Inc., a biopharmaceutical company traded on the New York Stock Exchange under the ticker symbol ABBV. The therapy is part of AbbVie's ophthalmology pipeline and is currently in Phase 3 clinical trials.

What phase is ABBV-RGX-314 Dose 1 in?

ABBV-RGX-314 Dose 1 is in Phase 3 clinical development. It is an investigational gene therapy and has not been approved by regulatory authorities. The therapy is being evaluated in active clinical trials for diabetic retinopathy and neovascular age-related macular degeneration.

What clinical trials is ABBV-RGX-314 Dose 1 in?

ABBV-RGX-314 Dose 1 is being studied in two active clinical trials. NCT05407636 is a Phase 3 pivotal study in participants with neovascular AMD, enrolling 735 participants across multiple countries. NCT04567550 is a Phase 2 study in diabetic retinopathy with and without center-involved diabetic macular edema, enrolling 139 participants in the United States.

Is ABBV-RGX-314 Dose 1 the same as RGX-314?

ABBV-RGX-314 Dose 1 is a specific dose of the gene therapy RGX-314. The trials listed under ABBV-RGX-314 Dose 1 evaluate RGX-314 administered in the suprachoroidal space, and the drug is referred to by both names in clinical research.