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Ixo-vec

Phase 3

Neovascular Age-Related Macular Degeneration (nAMD) | Gene therapy | Ophthalmology |Adverum Biotechnologies, Inc.|Last Updated: Aug 5, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindACTIVE_CONTROLLEDDMC
Total Trials2
Total Enrollment595

FDA Designations

RMATFAST_TRACK

Clinical trial landscape

Ixo-vec · 3 trials · 3 indications

Phase 3 2Phase 2 1
NCT07482176Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS)Neovascular Age-Related Macular Degeneration (nAMD)
RECRUITING284 Analytics
NCT06856577Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular DegenerationNeovascular Age-Related Macular Degeneration (nAMD)
ACTIVE NOT_RECRUITING311 Analytics
PHASE3RECRUITING
Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS)
Neovascular Age-Related Macular Degeneration (nAMD)Unlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration
Neovascular Age-Related Macular Degeneration (nAMD)Unlock trial analytics

Study Endpoints

Primary Endpoints

Mean Change from Baseline in BCVA Based on an Average at Weeks 52 and 56
Baseline, Week 52 and Week 56

BCVA will be measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart.

Mean change from Baseline in Best-Corrected Visual Acuity (BCVA) based on an average at Weeks 52 and 56
Baseline, Week 52 and Week 56

BCVA will be measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart.

Incidence of ocular adverse events (AEs)
Baseline through Week 28

The number of participants who experience an ocular adverse event will be summarized.

Incidence of ocular serious adverse events (SAEs).
Baseline through Week 28

The number of participants who experience ocular serious adverse event will be summarized.

Incidence of non-ocular serious adverse events (SAEs).
Baseline through Week 28

The number of participants who experience non-ocular serious adverse event will be summarized.

Secondary Endpoints

Mean Number of Aflibercept IVT Injections Received
Week 4 through Week 56
Percentage of Participants with Worsened BCVA
Through Week 56
Percentage of Participants with Improved BCVA
Through Week 56
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Ixo-vecEXPERIMENTALParticipants will receive 3 monthly loading doses of aflibercept 2 mg IVT, a single IVT injection of Ixo-vec 6 x 10\^10 vg/eye at Week 1, and sham injections every 8 weeks.
AfliberceptACTIVE_COMPARATORParticipants will receive 3 monthly loading doses of aflibercept 2 mg IVT, a sham injection at Week 1, and aflibercept 2 mg IVT every 8 weeks.
Ixo-vec dosing in Contralateral EyeEXPERIMENTALPopulation of Ixo-vec-Experienced or Ixo-vec Naïve Participants.

Interventions

NameTypeDescription
Ixo-vecGENETICIxo-vec will be administered intravitreally.
AfliberceptDRUGAflibercept will be administered intravitreally.
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Eligibility Criteria

Age Range50 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites79

Inclusion Criteria: 1. Able and willing to provide informed consent (or have a legally authorized representative who is able and willing to provide informed consent) prior to any study assessments and procedures and comply with the study requirements and visits. 2. Male or female with a diagnosis o...

Countries:United States
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Recent Changes (Last 90 Days)

LOWAug 5, 2026NCT07630649lastUpdatePostDate: changed
LOWJul 15, 2026NCT07482176lastUpdatePostDate: changed
LOWJul 15, 2026NCT07482176lastUpdatePostDate: changed
LOWJun 16, 2026NCT07482176lastUpdatePostDate: changed
LOWJun 16, 2026NCT07482176lastUpdatePostDate: changed
LOWJun 16, 2026NCT07482176lastUpdatePostDate: changed
LOWJun 16, 2026NCT07482176lastUpdatePostDate: changed

Frequently asked questions about Ixo-vec

What is Ixo-vec used for?

Ixo-vec, also known as ixoberogene soroparvovec, is an investigational gene therapy being developed for neovascular age-related macular degeneration (nAMD), including wet AMD. It is designed to treat this condition, which affects the retina and can lead to vision loss.

What does Ixo-vec target?

Ixo-vec is a gene therapy, a class of treatment that delivers genetic material to cells. It is being studied for neovascular age-related macular degeneration (nAMD), a condition characterized by abnormal blood vessel growth in the eye.

Who is developing Ixo-vec?

Ixo-vec is being developed by Adverum Biotechnologies, Inc., a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for neovascular age-related macular degeneration.

What phase is Ixo-vec in?

Ixo-vec is currently in Phase 3 clinical trials for neovascular age-related macular degeneration (nAMD). It has received FDA designations including Regenerative Medicine Advanced Therapy (RMAT) and Fast Track, reflecting its potential to address a serious condition.

What clinical trials is Ixo-vec in?

Ixo-vec is being studied in two Phase 3 trials: NCT06856577, with 311 participants, and NCT07482176 (AQUARIUS), with 284 participants. Both are active in the United States, enrolling adults aged 50 and older with nAMD or wet AMD.

Is Ixo-vec the same as ixoberogene soroparvovec?

Yes, Ixo-vec is the same as ixoberogene soroparvovec. Both names refer to the same investigational gene therapy being developed for neovascular age-related macular degeneration (nAMD).