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AAVCAGsCD59

Phase 1

Dry Age-related Macular Degeneration | Monoclonal antibody | Ophthalmology |Johnson & Johnson|Last Updated: Mar 30, 2025

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment17

FDA Designations

No designations recorded

Clinical trial landscape

AAVCAGsCD59 · 1 trial · 1 indication

Phase 1 1
NCT03144999Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59Dry Age-related Macular Degeneration
COMPLETED17 Analytics
PHASE1COMPLETED
Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59
Dry Age-related Macular DegenerationUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of participants experiencing ocular and systemic adverse events as graded by CTCAE v4.0
26 Weeks

Measure intraocular inflammation, ocular changes and systemic side effects following a single intraocular injection of AAVCAGsCD59

Secondary Endpoints

Evaluate the change in area of GA in eyes with dry AMD
26 Weeks
Evaluate the rate of growth of GA in eyes with dry AMD
26 Weeks
Incidence of conversion of dry AMD to wet AMD
26 Weeks
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeOTHER

Treatment Arms

ArmTypeDescription
Low DoseEXPERIMENTALAAVCAGsCD59
Mid DoseEXPERIMENTALAAVCAGsCD59
High DoseEXPERIMENTALAAVCAGsCD59

Interventions

NameTypeDescription
AAVCAGsCD59BIOLOGICALAAVCAGsCD59 is administered as a single intravitreal injection in an office setting
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Eligibility Criteria

Age Range50 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Men or women 50 years of age or older * Advanced dry AMD with GA in the study eye * BCVA Snellen equivalent of 20/80 or worse in the study eye using ETDRS charts at a starting distance of 4m after the first 3 patients are enrolled and demonstrate favorable safety data * Total ...

Countries:United States
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Frequently asked questions about AAVCAGsCD59

What is AAVCAGsCD59 used for?

AAVCAGsCD59 is an investigational gene therapy being studied for the treatment of advanced dry age-related macular degeneration (AMD). It is administered to patients aged 50 years and older. The drug is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

What does AAVCAGsCD59 target?

AAVCAGsCD59 is a gene therapy designed to deliver the CD59 gene, which encodes a protein that inhibits the complement system, a part of the immune response implicated in the progression of dry age-related macular degeneration. By expressing CD59, the therapy aims to protect retinal cells from complement-mediated damage.

Who is developing AAVCAGsCD59?

AAVCAGsCD59 is being developed by Johnson & Johnson (NYSE: JNJ). The company is conducting clinical research on this gene therapy for the treatment of advanced dry age-related macular degeneration.

What phase is AAVCAGsCD59 in?

AAVCAGsCD59 is in Phase 1 clinical development. A single Phase 1 trial has been completed, involving 17 participants with dry age-related macular degeneration. The drug remains investigational and is not yet approved for commercial use.

What clinical trials is AAVCAGsCD59 in?

AAVCAGsCD59 has one completed clinical trial registered as NCT03144999, titled 'Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59.' This Phase 1 study enrolled 17 participants in the United States and evaluated the therapy in patients with dry age-related macular degeneration.

Is AAVCAGsCD59 the same as AAVCAGsCD59?

Yes, AAVCAGsCD59 is the drug name used in clinical trial registrations and research. No alternative names have been reported for this investigational gene therapy.