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ABBV-RGX-314

Phase 2

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment671
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04704921Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMDPHASE2 ACTIVE NOT_RECRUITING 671Dec 29, 2020Nov 1, 2027Apr 28, 202689 United States
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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 — 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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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT04704921Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 24, 2026NCT04704921studyFirstPostDate: changed