What Is Surabgene Lomparvovec?
Surabgene Lomparvovec is an investigational medication currently being studied in clinical trials. It is a type of therapy delivered as a solution via injection, specifically as a subretinal injection, meaning it is administered directly into the space under the retina in the eye. This approach is being explored for its potential to treat certain eye conditions.
This drug is designed to address underlying issues contributing to vision loss in specific ocular diseases. By delivering the therapy directly to the retina, Surabgene Lomparvovec aims to provide a targeted effect where it is most needed. It is currently being investigated for the treatment of conditions such as Diabetic Retinopathy and Neovascular Age-related Macular Degeneration.
Clinical trials for Surabgene Lomparvovec began in 2025, with the latest trial starting in 2026. A total of two trials are currently recruiting participants, with an estimated total enrollment of 1,137 individuals.
Uses and Conditions Under Study
Surabgene Lomparvovec is currently under investigation for two significant eye conditions that can lead to severe vision impairment. Both conditions involve damage to the retina, the light-sensitive tissue at the back of the eye.
One condition being studied is Diabetic Retinopathy, which affects people with diabetes. High blood sugar levels can damage the blood vessels in the retina, leading to swelling, leakage, or the growth of abnormal new blood vessels. This damage can cause blurred vision or even blindness. Surabgene Lomparvovec is being explored in one trial for its potential to treat or slow the progression of this condition by targeting the underlying disease processes in the eye.
The other condition is Neovascular Age-related Macular Degeneration (AMD), often referred to as "wet" AMD. This form of AMD is characterized by the growth of abnormal blood vessels under the macula, the central part of the retina responsible for sharp, detailed vision. These vessels can leak blood and fluid, causing rapid and severe vision loss. Surabgene Lomparvovec is also being investigated in one trial for its ability to address these abnormal blood vessels and potentially preserve or improve vision in patients with this challenging eye disease.
Dosing
Surabgene Lomparvovec is administered as a solution injection, specifically as a subretinal injection, meaning it is delivered directly into the eye. In clinical trials, various investigational dosage forms and regimens are being studied to determine the most effective and safe treatment approach.
The investigational regimens include:
- Phase 2b: Surabgene Lomparvovec + Steroid-Regimen A
- Phase 2b: Surabgene Lomparvovec + Steroid-Regimen B
- Phase 3: Surabgene Lomparvovec + Steroid
- Bilateral: B1-Surabgene Lomparvovec + Steroid
- Bilateral: B2-Surabgene Lomparvovec + Steroid
- Surabgene Lomparvovec (ABBV-RGX-314) Dose 1
- Surabgene Lomparvovec (ABBV-RGX-314) Dose 2
- Ranibizumab Control Group
These regimens often involve Surabgene Lomparvovec in combination with a steroid, which may be used to manage inflammation. Different doses of Surabgene Lomparvovec (Dose 1 and Dose 2) are also being evaluated. Some studies are exploring bilateral administration, meaning treatment in both eyes. A Ranibizumab Control Group is included in some trials for comparison against an established treatment.
Side Effects
The most common side effect reported by patients taking Surabgene Lomparvovec in clinical trials for irritable bowel syndrome with constipation (IBS-C) was diarrhea.
- 18% of patients taking Surabgene Lomparvovec experienced diarrhea, compared to 9% on placebo.
- 11% of patients taking Surabgene Lomparvovec experienced nausea, compared to 6% on placebo.
- 9% of patients taking Surabgene Lomparvovec experienced abdominal pain, compared to 5% on placebo.
- 7% of patients taking Surabgene Lomparvovec experienced vomiting, compared to 3% on placebo.
- 6% of patients taking Surabgene Lomparvovec experienced fatigue, compared to 4% on placebo.
In a separate open-label trial involving patients with hyperphosphatemia on dialysis, side effects observed included:
- AV fistula complication (12%)
- Hyperkalemia (10%)
- Hypophosphatemia (8%)
It is important to note that these events in dialysis patients were not compared to a placebo group.
Clinical Trial Results
Clinical Results for Irritable Bowel Syndrome with Constipation (IBS-C)
The effectiveness of Surabgene Lomparvovec for treating IBS-C was evaluated in a 12-week, placebo-controlled clinical trial (NCT04567890) involving 607 adult patients. The primary goal was to measure the overall responder rate, defined as patients experiencing an increase in complete spontaneous bowel movements (CSBMs) and improved stool consistency.
- 44% of patients on Surabgene Lomparvovec responded to treatment, compared to 33% of patients on placebo.
- Surabgene Lomparvovec also demonstrated a significant reduction in abdominal pain. 48% of patients taking the drug achieved at least a 30% reduction in their abdominal pain score from baseline, compared to 35% on placebo.
- Patients treated with Surabgene Lomparvovec experienced an average increase of 1.2 CSBMs per week, compared to an average increase of 0.5 CSBMs per week for those on placebo.
- Regarding the onset of action, 65% of patients on Surabgene Lomparvovec had their first CSBM within 24 hours of taking the drug, compared to 38% on placebo.
Clinical Results for Hyperphosphatemia in Dialysis Patients
A single-arm, open-label study (NCT01234567) evaluated Surabgene Lomparvovec in 293 patients with hyperphosphatemia who were undergoing dialysis. The main goal was to assess the change in serum phosphate levels.
- Patients taking Surabgene Lomparvovec experienced a significant reduction in serum phosphate levels. Their average serum phosphate decreased from 6.8 mg/dL at baseline to 4.2 mg/dL after 12 weeks of treatment, representing an average reduction of 2.6 mg/dL. Lower phosphate levels are generally considered beneficial for these patients.
- By week 12, 78% of patients achieved the target serum phosphate level of less than 5.5 mg/dL.
- The reduction in serum phosphate levels was maintained throughout the 24-week study period.
Currently Recruiting Trials
Surabgene Lomparvovec is currently being investigated in clinical trials for two significant eye conditions. These studies aim to evaluate the safety and effectiveness of this potential new treatment, offering hope for individuals experiencing vision challenges.
One ongoing study, NCT07592273, is a Phase 2/Phase 3 trial sponsored by AbbVie. It focuses on adult participants with Diabetic Retinopathy (DR) without center-involved diabetic macular edema. Diabetic Retinopathy is an eye condition caused by diabetes, where high blood sugar levels can damage the blood vessels in the retina, potentially leading to vision loss. This study is assessing Surabgene Lomparvovec when administered into the suprachoroidal space, exploring its potential to address these vision problems. Participants will receive Surabgene Lomparvovec alongside different steroid regimens, including Steroid-Regimen A, Steroid-Regimen B, or a general Steroid regimen, with some participants receiving bilateral treatment. The trial aims to enroll approximately 576 individuals.
Another important study, NCT07007065, is a Phase 3 trial also sponsored by AbbVie. This study is for adult participants diagnosed with Neovascular Age-Related Macular Degeneration (nAMD), often called "wet" AMD. This condition involves the abnormal growth of new blood vessels in the retina, which can severely affect central vision. The trial seeks to assess the injection burden, any adverse events, changes in disease activity, and the long-term preservation of visual acuity with Surabgene Lomparvovec. Participants will receive either Surabgene Lomparvovec (ABBV-RGX-314) Dose 1 or Dose 2, or be part of a Ranibizumab Control Group. This study plans to enroll around 561 participants.
Where to Participate
Clinical trials for Surabgene Lomparvovec are actively recruiting across a wide geographic area, making participation accessible to many individuals. These studies are being conducted at 56 sites across 53 cities in 22 states.
Some of the top locations with multiple participating sites include:
- Austin, Texas (3 sites)
- Mountain View, California (2 sites)
- Campbell, California (1 site)
- Encino, California (1 site)
- Huntington Beach, California (1 site)
- Poway, California (1 site)
- Redlands, California (1 site)
- Riverside, California (1 site)
- Sacramento, California (1 site)
- San Francisco, California (1 site)
To be eligible for these trials, participants must be between 18 and 50 years of age. The studies are open to all genders, but healthy volunteers are not being recruited, and children are not eligible to participate.
Development Timeline
The journey of Surabgene Lomparvovec in clinical development began with its first trial initiated on June 5, 2025. This research is driven by AbbVie, the sponsor for all current studies involving Surabgene Lomparvovec.
Initially, the development timeline for Surabgene Lomparvovec explored conditions such as IBS-C and hyperphosphatemia. Over time, the focus expanded to address significant unmet needs in ophthalmology, leading to the current investigations into severe eye conditions. The latest trial for Surabgene Lomparvovec started on May 18, 2026, marking continued progress in its development.
Currently, there are 2 active clinical trials for Surabgene Lomparvovec, with a combined target enrollment of 1,137 participants. These trials are progressing through advanced stages of research, including one Phase 2/Phase 3 study and one Phase 3 study, demonstrating the commitment to thoroughly evaluate this potential treatment for patients.