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ABBV-RGX-314

Phase 2

AMD | Gene therapy | Ophthalmology |AbbVie Inc.|Last Updated: Apr 28, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindACTIVE_CONTROLLEDDMC
Total Trials1
Total Enrollment671

FDA Designations

No designations recorded

Clinical trial landscape

ABBV-RGX-314 · 1 trial · 9 indications

Phase 2 1
NCT04704921Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMDAMD
ACTIVE NOT_RECRUITING671 Analytics
PHASE2ACTIVE NOT_RECRUITING
Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD
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Study Endpoints

Primary Endpoints

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

BCVA measured by Early Treatment Diabetic Retinopathy Study (ETDRS)

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

Incidence of ocular AEs and any SAEs

Secondary Endpoints

Incidences of ocular and overall AEs over 54 weeks
Through Week 54
Incidences of ocular and overall AEs over 98 weeks
Through Week 98
Mean change from baseline in BCVA to Week 98 (ABBV-RGX-314 randomized participants) based on the ETDRS score
Week 98
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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_COMPARATORRanibizumab administered via intravitreal injection approximately every 28 days

Interventions

NameTypeDescription
ABBV-RGX-314GENETICAAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)
Ranibizumab (LUCENTIS®)BIOLOGICAL0.5 mg (0.05 mL of 10 mg/mL solution) administered by intravitreal injection approximately every 28 days
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Eligibility Criteria

Age Range50 Years to 89 Years
SexALL
Healthy VolunteersNo
Study Sites89

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 CNV secondary to AMD in the study eye previously treated with anti-VEGF 4. Must be pseudophakic (at least 12 weeks postcataract surgery) in the study e...

Countries:United States
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Frequently asked questions about ABBV-RGX-314

What is ABBV-RGX-314 used for?

ABBV-RGX-314 is an investigational gene therapy being developed for the treatment of age-related macular degeneration (AMD), specifically neovascular or wet AMD. It is administered via subretinal delivery as a one-time treatment. The drug is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.

What does ABBV-RGX-314 target?

ABBV-RGX-314 is a gene therapy designed to deliver a gene that encodes for an anti-angiogenic protein, which targets vascular endothelial growth factor (VEGF) to inhibit the abnormal blood vessel growth and leakage that characterize wet AMD. This mechanism aims to provide a durable, one-time treatment for the condition.

Who makes ABBV-RGX-314?

ABBV-RGX-314 is being developed by AbbVie Inc., a biopharmaceutical company listed on the New York Stock Exchange under the ticker symbol ABBV. The drug is currently in Phase 2 clinical trials for the treatment of neovascular age-related macular degeneration.

What phase is ABBV-RGX-314 in?

ABBV-RGX-314 is currently in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The drug is being studied in an active, not recruiting, Phase 2 trial for the treatment of wet age-related macular degeneration.

What clinical trials is ABBV-RGX-314 in?

ABBV-RGX-314 is being evaluated in a Phase 2 clinical trial with the identifier NCT04704921, titled 'Pivotal 1 Study of ABBV-RGX-314 Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD.' This active, not recruiting trial has an enrollment of 671 participants and is being conducted in the United States.

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

Yes, ABBV-RGX-314 is also known as RGX-314. The drug is referred to by both names in clinical trial documentation, with the Phase 2 study NCT04704921 explicitly noting that ABBV-RGX-314 is also known as RGX-314.