Published in Retina

On Gene Therapy, New Clinical Trials, and Retina in the Military

John W. Kitchens, MD

John W. Kitchens, MD

Mitchell J. Goff, MD

Mitchell J. Goff, MD

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Sit down with John W. Kitchens, MD, and Mitchell J. Goff, MD, to learn about developments in gene therapies, recruiting for clinical trials, and more!

On this episode of Retina Mentor Moments, John W. Kitchens, MD, sits down with Mitchell J. Goff, MD, to discuss developments in gene therapies for retinal diseases.
Dr. Goff is a partner at Rocky Mountain Retina Consultants and an adjunct assistant professor of ophthalmology and visual science at the University of Utah in Salt Lake City, Utah.

Retina care in the military

Dr. Goff served as an officer in the United States Army, where he was responsible for the retina and uveitis service for 5 years at the Brooke Army Medical Center in San Antonio, Texas. This military health facility is also where he pursued his internship and ophthalmology residency via the Health Professions Scholarship Program (HPSP). He explained that the program offered high-quality education with excellent staff and high standards for the trainees.
A common misconception about healthcare in the military is that physicians primarily treat wounded soldiers; however, when there isn’t active conflict, the majority of the patients they see are in fact retirees and their dependents. Consequently, the patient population he treated during residency was predominantly older patients, which is similar to who he treats now.

Treating conflict-related injuries

In 2005, Dr. Goff contributed to a paper published in Ophthalmology that evaluated the number, cause, and type of intraocular foreign body (IOFB) injuries that occurred during the Iraq War.1
Based on data from 55 US military personnel, they found that IOFB injuries in a military setting tended to be caused by explosive devices, which often caused multiple foreign bodies and simultaneous injuries to other body systems. In addition, due to the lack of availability of specialty care in the combat theater, there was often a delay in removal of the foreign body.

To learn about humanitarian deployments Dr. Goff was involved in, watch the full interview!

Recruiting for clinical trials

As patient recruiting is the backbone of any clinical trial, Dr. Goff prioritizes recruiting at least one patient from his practice in every trial that he sees promise in, with the goal of making a difference in their medical care by embracing innovative therapies.
This is particularly beneficial for patients struggling with the financial burden of retina treatments, as clinical trials allow them to gain access to the gold standard of care (or potentially even better) at no cost to them.
For example, Dr. Kitchens highlighted that patients involved in the clinical trials for intravitreal complement inhibitors for geographic atrophy (GA) had access to the drugs 4 to 5 years before they were approved and made available to the general public.

Investigational gene therapies for retinal diseases

As one of a handful of retina surgeons certified to conduct genetic research in the state, Dr. Goff noted that he has enrolled a few patients in the clinical trial for ABBV-RGX-314, a gene therapy candidate being investigated as a potential one-time treatment for wet age-related macular degeneration (AMD), diabetic retinopathy, and other chronic retinal conditions.2
In his opinion, while still in its early stages, gene therapy is among the most promising therapies due to its potential to be a durable treatment with acceptable risk profiles. Though, he is still careful in evaluating the long-term effects of gene therapy injections.
There are currently three ongoing clinical trials for ABBV-RGX-314 for the treatment of wet AMD, including:2
  • ATMOSPHERE (NCT04704921)
    • Pivotal phase 2b/3 trial
    • Evaluating two doses of ABBV-RGX-314 delivered subretinally compared to an active comparator
    • Primary endpoint: Mean change in best-corrected visual acuity (BCVA) relative to ranibizumab
  • ASCENT (NCT05407636)
    • Pivotal phase 3 trial
    • Evaluating two doses of ABBV-RGX-314 delivered subretinally compared to an active comparator
    • Primary endpoint: Mean change in BCVA relative to aflibercept
  • AAVIATE (NCT04514653)
    • Multicenter, open-label, randomized, active-controlled, dose-escalation phase 2 trial
    • Evaluating the safety and efficacy of ABBV-RGX-314 delivered in the suprachoroidal space in 115 participants (aged 50 to 89)
    • Primary endpoint: Mean change in vision measured via BCVA at Week 40 from baseline compared to patients receiving monthly ranibizumab injections
    • Secondary endpoints: Mean change in central retinal thickness (CRT) and number of anti-VEGF injections after injection of ABBV-RGX-314
    • Doses: Three doses / dose combinations were divided into six cohorts
      • Cohort 1: Dose 1 (2.5 x 1,011 genome copies per eye [GC / eye])
      • Cohorts 2 and 3: Dose 2 (5 x 1,011 GC / eye)
      • Cohorts 4 to 6: Dose 3 (1 x 1012 GC / eye)
So far, RegenxBio has reported that ABBV-RGX-314 was well-tolerated among all patients from the three dose levels with no drug-related serious adverse events.2 Of note, dose level 3 in AAVIATE has demonstrated the highest reduction in treatment burden, with zero cases of intraocular inflammation observed in patients who received prophylactic short-course topical steroids.
Pivotal data evaluating the safety and efficacy of the subretinal delivery of ABBV-RGX-314 in patients with wet AMD are expected in 2026, and AbbVie and RegenxBio are in the process of planning the phase 3 clinical program for suprachoroidal delivery to treat diabetic retinopathy.3

Watch the interview to hear about real-world data on the extended treatment duration of second-generation anti-VEGF drugs!

Conclusion

While further research is required, gene therapy shows promise as a game-changing treatment that may significantly reduce the treatment burden for patients with wet AMD.
  1. Thach AB, Ward TP, Dick JSB, et al. Intraocular foreign body injuries during Operation Iraqi Freedom. Ophthalmology. 2005 Oct;112(10):1829-1833. doi:10.1016/j.ophtha.2005.04.024
  2. Delaney-Gesing A. RegenxBio reports positive interim data in gene therapy trial for wet AMD. Glance by Eyes On Eyecare. January 23, 2024. Accessed May 30, 2025. https://glance.eyesoneyecare.com/stories/2024-01-23/regenxbio-reports-positive-interim-data-in-gene-therapy-trial-for-wet-amd/.
  3. AbbVie and REGENXBIO announce updates on the ABBV-RGX-314 clinical program. AbbVie. January 13, 2025. Accessed May 30, 2025. https://news.abbvie.com/2025-01-13-AbbVie-and-REGENXBIO-Announce-Updates-on-the-ABBV-RGX-314-Clinical-Program.
John W. Kitchens, MD
About John W. Kitchens, MD

John W. Kitchens, MD, received his undergraduate degree from the University of Evansville, and his Doctor of Medicine degree from Indiana University School of Medicine. He served his ophthalmology residency at the University of Iowa Hospital. Dr. Kitchens completed his fellowship and was the chief resident at Bascom Palmer Eye Institute in Miami.

Dr. Kitchens enjoys speaking both nationally and internationally about new treatments for age-related macular degeneration (AMD), diabetes, and vascular disease. Dr. Kitchens has developed several innovative surgical techniques and has been awarded the American Society Retina Specialists “Rhett Buckler” Award on three different occasions.

More by John W. Kitchens, MD
John W. Kitchens, MD
Mitchell J. Goff, MD
About Mitchell J. Goff, MD

Mitchell J. Goff, MD, was born and raised in Ogden, Utah. He graduated magna cum laude from Weber State University with a degree in Business Administration and Finance and went on to earn his medical degree from the Medical College of Wisconsin.

Dr. Goff served as an officer in the United States Army, where he completed his internship and ophthalmology residency at Brooke Army Medical Center in San Antonio, Texas. He subsequently completed his vitreoretinal surgery fellowship training at California Pacific Medical Center in San Francisco, California.

Dr. Goff was responsible for the retina and uveitis service at Brooke Army Medical Center, where he trained ophthalmology residents in a large combined residency program that included the Army, the Air Force, and the University of Texas, San Antonio. During this time, he also cared for active duty service members who were wounded while on deployments to Iraq and Afghanistan.

Dr. Goff returned home to Utah and joined Rocky Mountain Retina Consultants, where he is now a partner. He is an adjunct assistant professor of Ophthalmology and Visual Sciences at the University of Utah. He has been actively involved in clinical research, serving as an investigator on over 60 large-scale clinical trials, many of which have led to the approval of new medical and surgical treatments for retinal disease.

He is one of a very small number of retina surgeons certified to conduct genetic research in the state of Utah. He has published numerous scientific articles and book chapters on diabetic retinopathy, macular degeneration, retinal occlusive disease, retinal detachment repair, and ocular trauma. He is a regular speaker on a national and international level, and a consultant for several pharmaceutical companies.

He is a member of the American Board of Ophthalmology, a Fellow of the American Society of Retina Specialists (FASRS), a member of the Utah Medical Association, the Utah Ophthalmology Association, and a Fellow of the American Academy of Ophthalmology.

More by Mitchell J. Goff, MD
Mitchell J. Goff, MD
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