Gene therapy could end frequent eye injections for wet AMD

NCT ID NCT04704921

Disease control Sponsor: AbbVie Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. The treatment aims to help the eye produce its own medicine to stop leaky blood vessels, potentially replacing the need for repeated eye injections. About 671 participants who have already responded to standard anti-VEGF therapy will receive either the gene therapy or continued injections. The trial will measure vision changes and safety over up to 98 weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: RGX-314 (surabgene lomparvovec) gene therapy
What this could lead to: If successful, this one-time gene therapy could reduce or eliminate the need for repeated eye injections in people with wet AMD, making treatment much less burdensome.
What could go wrong: This is still an early-to-mid-stage trial, so the treatment may not work as hoped or could cause unexpected side effects. Long-term safety and effectiveness are not yet proven.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

671 people

The number who actually took part.

Started

Dec 2020

Expected to finish

Nov 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

50 to 89 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 eye. 5. Willing and able to provide written, signed informed consent for this study 6. Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry Inclusion Criteria (Bilateral Treatment Substudy)\*: 1. An ETDRS BCVA letter score between ≤ 83 and ≥ 40 in both eyes 2. Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in both eyes 3. Must be pseudophakic (at least 12 weeks postcataract surgery) in both eyes 4. Willing and able to provide written, signed informed consent for this study 5. Newcomers must have active disease in the study eye; crossover participants must have active disease in the eye not treated in the main study Exclusion Criteria: 1. CNV or macular edema in the study eye secondary to any causes other than AMD 2. Subfoveal fibrosis or atrophy in the study eye, as determined by CRC 3. Any condition in the investigator's opinion that could limit VA improvement in the study eye 4. Active or history of retinal detachment, or current retinal tear that cannot be treated, in the study eye 5. Advanced glaucoma or history of secondary glaucoma in the study eye 6. History of intraocular surgery in the study eye within 12 weeks prior to randomization 7. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1 8. Prior treatment with gene therapy 9. Recent myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months Exclusion Criteria (Bilateral Treatment Substudy)\*: 1. CNV or macular edema in either eye secondary to any causes other than AMD 2. Subfoveal fibrosis or atrophy in either eye 3. Any condition in the investigator's opinion that could limit VA improvement in either eye 4. Active or history of retinal detachment, or current retinal tear that cannot be treated in either eye 5. Advanced glaucoma or history of secondary glaucoma in either eye 6. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months 7. History of intraocular surgery in either eye within 12 weeks prior to randomization (Week -2) 8. History of intravitreal therapy in either eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to screening 9. Prior treatment with gene therapy (\*) For previously treated crossover participants, criteria apply to the eye not treated in the main study only.

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Conditions

The condition(s) this trial relates to.

age-related macular degeneration choroidal neovascularization eye disorder macular degeneration nemaline myopathy retinal degeneration retinal disorder wet macular degeneration

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AMD Choroidal neovascularization CNV NAMD Neovascular age-related macular degeneration Neovascular AMD WAMD Wet age related macular degeneration Wet AMD

Contacts and locations

Locations

Advanced Research, LLC /ID# 275451

Deerfield Beach, Florida, 33064-1342, United States
Associated Retinal Consultants /ID# 256156

Royal Oak, Michigan, 48073, United States
Atrium Health Wake Forest Baptist Medical Center /ID# 270767

Winston-Salem, North Carolina, 27157, United States
Austin Clinical Research, LLC /ID# 256043

Austin, Texas, 78750-2298, United States
Austin Research Center for Retina /ID# 256148

Austin, Texas, 78705, United States
Austin Retina Associates - Austin /ID# 256053

Austin, Texas, 78705, United States
Barnet Dulaney Perkins Eye Center - Sun City /ID# 256055

Sun City, Arizona, 85351, United States
Bascom Palmer Eye Institute - University of Miami /ID# 256072

Miami, Florida, 33136, United States
Baylor Scott & White Surgicare /ID# 256122

Fort Worth, Texas, 76104, United States
Black Hills Regional Eye Institute /ID# 256161

Rapid City, South Dakota, 57701, United States
California Eye Specialists Medical Group Inc. /ID# 256079

Pasadena, California, 91107, United States
California Retina Consultants - Santa Barbara /ID# 256017

Santa Barbara, California, 93103, United States
Charles Retina Institute /ID# 256016

Germantown, Tennessee, 38138, United States
Charleston Neuroscience Institute /ID# 256235

Ladson, South Carolina, 29456, United States
Cincinnati Eye Institute- Edgewood /ID# 256132

Edgewood, Kentucky, 41017-3415, United States
Cleveland Clinic Main Campus /ID# 256064

Cleveland, Ohio, 44195, United States
Colorado Retina Associates /ID# 256121

Lakewood, Colorado, 80228, United States
Cumberland Valley Retina Consultants - Hagerstown /ID# 256151

Hagerstown, Maryland, 21740, United States
Duke Eye Center /ID# 256076

Durham, North Carolina, 27705, United States
Erie Retina Research /ID# 256154

Erie, Pennsylvania, 16507, United States
Eye Associates of New Mexico /ID# 256075

Albuquerque, New Mexico, 87109, United States
Florida Retina Consultants /ID# 265661

Lakeland, Florida, 33805, United States
Georgia Retina - Marietta /ID# 256142

Marietta, Georgia, 30060, United States
John A. Moran Eye Center /ID# 256068

Salt Lake City, Utah, 84132, United States
John-Kenyon American Eye Institute -New Albany /ID# 256065

New Albany, Indiana, 47150-3620, United States
Johns Hopkins Hospital /ID# 256015

Baltimore, Maryland, 21287, United States
Long Island Vitreoretinal Consultants /ID# 256074

Great Neck, New York, 11021, United States
Marietta Eye Clinic /ID# 268163

Marietta, Georgia, 30060, United States
Mayo Clinic - Minnesota /ID# 256051

Rochester, Minnesota, 55905, United States
Mid Atlantic Retina /ID# 256013

Philadelphia, Pennsylvania, 19107, United States
Northern California Retina Vitreous Associates Medical Group, Inc /ID# 256298

Mountain View, California, 94040-4101, United States
Ochsner Medical Center - Jefferson Highway /ID# 270524

New Orleans, Louisiana, 70121, United States
Ophthalmic Consultants of Boston /ID# 256014

Boston, Massachusetts, 02114, United States
Orange County Retina Medical Group /ID# 256073

Santa Ana, California, 92705, United States
Pacific Northwest Retina /ID# 256155

Bellevue, Washington, 98004, United States
Retina Associates - Lenexa /ID# 256080

Lenexa, Kansas, 66215, United States
Retina Associates of Cleveland-Middleburg Heights /ID# 256052

Cleveland, Ohio, 44130, United States
Retina Associates of Michigan /ID# 266198

Grand Blanc, Michigan, 48439, United States
Retina Associates of New Orleans /ID# 272440

Metairie, Louisiana, 70006-2940, United States
Retina Associates of Utah /ID# 256044

Salt Lake City, Utah, 84107, United States
Retina Center NW, PLLC /ID# 256157

Silverdale, Washington, 98383, United States
Retina Center Of Texas (Rct) - Southlake /ID# 256056

Southlake, Texas, 76092, United States
Retina Consultants - The Woodlands /ID# 256018

The Woodlands, Texas, 77384, United States
Retina Consultants Of Texas /ID# 268611

San Antonio, Texas, 78240, United States
Retina Consultants of Hawaii /ID# 256049

‘Aiea, Hawaii, 96701, United States
Retina Consultants of Orange County /ID# 256152

Fullerton, California, 92835, United States
Retina Consultants of San Diego /ID# 256021

Poway, California, 92064-2530, United States
Retina Consultants of Southern Colorado /ID# 256069

Colorado Springs, Colorado, 80909, United States
Retina Consultants of Texas - Beaumont /ID# 266279

Beaumont, Texas, 77707, United States
Retina Group of New England - Waterford /ID# 256071

Waterford, Connecticut, 06385-1215, United States
Retina Northwest, PC /ID# 256140

Portland, Oregon, 97221, United States
Retina Partners Midwest, PC /ID# 256045

Indianapolis, Indiana, 46290, United States
Retina Research Institute of Texas /ID# 256141

Abilene, Texas, 79606-1224, United States
Retina Specialty Institute /ID# 256153

Pensacola, Florida, 32503, United States
Retina Vitreous Assoc Med Grp /ID# 256299

Beverly Hills, California, 90211, United States
Retina Vitreous Associates of Florida - St. Petersburg /ID# 256050

St. Petersburg, Florida, 33711-1141, United States
Retina Vitreous Center, Research /ID# 256067

Edmond, Oklahoma, 73013, United States
Retina Vitreous Consultants - Monroeville /ID# 271654

Monroeville, Pennsylvania, 15146, United States
Retina and Vitreous of Texas /ID# 263961

Bellaire, Texas, 77401, United States
Retina-Vitreous Surgeons of Central New York - Liverpool /ID# 266274

Liverpool, New York, 13088, United States
Retinal Consultants Medical Group /ID# 256047

Sacramento, California, 95825, United States
Retinal Diagnostic Center /ID# 256137

Campbell, California, 95008, United States
Retinal Research Institute /ID# 256019

Phoenix, Arizona, 85053, United States
Salehi Retina Institute /ID# 263485

Huntington Beach, California, 92647, United States
Sierra Eye Associates /ID# 256020

Reno, Nevada, 89502, United States
Southeast Retina Center /ID# 256022

Augusta, Georgia, 30909, United States
Southern Vitreoretinal Associates /ID# 256158

Tallahassee, Florida, 32308, United States
Southwest Retina Research Center /ID# 256136

Durango, Colorado, 81303, United States
Springfield Clinic /ID# 266225

Springfield, Illinois, 62702-3749, United States
Stone Oak Surgery Center /ID# 266199

San Antonio, Texas, 78240, United States
Texas Retina Associates - Dallas /ID# 256133

Dallas, Texas, 75231, United States
The Retina Care Center /ID# 256144

Baltimore, Maryland, 21209, United States
The Retina Group Of Washington - Chevy Chase /ID# 276039

Chevy Chase, Maryland, 20815, United States
The Retina Institute /ID# 266587

St Louis, Missouri, 46214, United States
The Retina Partners - Encino /ID# 256054

Encino, California, 91436, United States
Thomas Eye Group PC /ID# 268159

Sandy Springs, Georgia, 30328-4411, United States
UC Irvine/Gavin Herbert Eye Institute /ID# 256145

Irvine, California, 92697, United States
UCLA Doheny Eye Center /ID# 256120

Pasadena, California, 91105, United States
University Retina and Macula Associates /ID# 256077

Oak Forest, Illinois, 60452, United States
University Retina and Macula Associates /ID# 256078

Lemont, Illinois, 60439, United States
University of Arkansas for Medical Sciences /ID# 271290

Little Rock, Arkansas, 72205, United States
University of California, San Francisco /ID# 256130

San Francisco, California, 94143, United States
University of Illinois at Chicago /ID# 256300

Chicago, Illinois, 60607, United States
University of Wisconsin-Madison, Department of Ophthalmology and Visual Sciences /ID# 256159

Madison, Wisconsin, 53705-3644, United States
Verum Research, LLC /ID# 266585

Eugene, Oregon, 97401, United States
VitreoRetinal Surgery PLLC DBA Retina Consultants of Minnesota /ID# 256160

Edina, Minnesota, 55435, United States
Vitreoretinal Associates, P.A. /ID# 256150

Gainesville, Florida, 32607, United States
Wagner Macula & Retina Center - Norfolk /ID# 256134

Norfolk, Virginia, 23502, United States
West Coast Retina /ID# 256448

San Francisco, California, 94107, United States

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