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Despite setbacks in DME, Adverum Biotechnologies said it will continue to develop ADVM-022 for wet AMD.

Serious adverse reaction halts gene therapy for diabetic macular edema

Retina World Congress 2025 will take place May 8-11. As researchers prepare for their, presentation, we are highlighting the upcoming sessions that are scheduled for this meeting, which will be held in Fort Lauderdale, Florida.

Note: The following list of presentations are in alphabetical order by company. Updates will be added to this article as new information becomes available.

The company highlight Phase 2 results for migaldendranib (MGB), Ashvattha’s investigational subcutaneous treatment for wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). The presentation, entitled, "Emerging Data om Anti-angiogenic Nanomedicine (Migaldendranib MGB/D-4517.2) for AMD and DME" will be given by Michael Singer, MD on Sunday, May 11, 2025; 11:11 – 11:16 am ET.

Phase 2 clinical data demonstrated that MGB administration lowered production of subretinal fluid reducing or eliminating the need for intravitreal anti-VEGF treatment in both the study eye and fellow eye in wet AMD and DME subjects with bilateral disease. New data indicates that MGB may be the first safe systemic therapy for the treatment of wet AMD and DME without ocular adverse events.

Huma Qamar, Chief Medical Officer at Ocugen will represent Ocugen at Retina World Congress to share a presentation and join retinal surgeons and industry peers during a panel discussion.

Inherited and Rare Retinal Diseases Session
Moderated by Rishi P. Singh, MD, FASRS and Kourous A. Rezaei, MD on Thursday, May 8, 2025, 10:35 – 11:12 AM ET

Ocular Therapeutix will present information at the meeting in Fort Lauderdale regarding the axitinib intravitreal hydrogel clinical trials, the use of its candidate OTX-TKI for diabetic retinopathy, and the HELIOS clinical trial for diabetic retinopathy.

Axitinib Intravitreal Hydrogel: Update on Clinical Trials

Presented by Carl C. Awh, MD, FASRS, on Friday, May 9, 2025, 4:33 – 4:38 PM ET.

Presented by Dilsher S. Dhoot, MD, FASRS on Saturday, May 10, 2025, 8:44 – 8:49 AM ET

Volumetric Macular Fluid Analysis of the Impact of a Single Axitinib Intravitreal Injection (OTX-TKI) from the HELIOS Clinical Trial for Diabetic Retinopathy

Presented by Carl J. Danzig, MD on Saturday, May 10, 2025, 8:52 – 8:57 AM ET

The company will present further insights from Stage 1 of the Phase 3 DIAMOND program reinforcing the efficacy and safety profile of OCS-01 eye drops for DME will also be shared. OCS-01 is being developed to be the first non-invasive treatment for DME, addressing key unmet needs for earlier treatment intervention and for inadequate responders to the current standard of care.

Ashvattha Therapeutics to Present at ARVO, Retina World Congress, and AOS Annual Meeting. Ashvattha Therapeutics. April 15, 2025. Accessed April 16, 2025. https://www.globenewswire.com/news-release/2025/04/15/3061682/0/en/Ashvattha-Therapeutics-to-Present-at-ARVO-Retina-World-Congress-and-AOS-Annual-Meeting.html

Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2025 Annual Meeting and Retina World Congress. Ocugen Inc. April 29, 2025. Accessed April 30, 2025. https://www.globenewswire.com/news-release/2025/04/29/3070074/0/en/Ocugen-to-Present-on-Modifier-Gene-Therapy-Platform-at-Association-for-Research-in-Vision-and-Ophthalmology-2025-Annual-Meeting-and-Retina-World-Congress.html

Ocular Therapeutix™ to Participate in Upcoming Investor and Scientific Conferences. Ocular Therapeutix. April 29, 2025. Accessed April 29, 2025. https://www.globenewswire.com/news-release/2025/04/29/3070022/0/en/Ocular-Therapeutix-to-Participate-in-Upcoming-Investor-and-Scientific-Conferences.html

Oculis to Present its Innovative Late-Stage Pipeline Candidates at Upcoming Ophthalmology Conferences. Oculis Holding AG. May 1, 2025. Accessed May 1, 2025. https://www.biospace.com/press-releases/oculis-to-present-its-innovative-late-stage-pipeline-candidates-at-upcoming-ophthalmology-conferences

Companies working in the retina space have announced that data from clinical trials will be shared at this year’s meeting in Fort Lauderdale, Florida.

RWC 2025: Companies to share data at meeting in Fort Lauderdale

A recent longitudinal prospective study revealed that electroretinography (ERG)/pupillometry outperforms structural imaging in identifying patients at the highest risk of progressing to vision-threatening complications over 1 year. 

 and provided a post hoc analysis of 56 parameters from 4 testing modalities obtained in a multicenter US trial (

 Eighteen of these parameters were from the RETeval device’s 

 assessment protocol graded by manufacturer LKC Technologies, which was found to be the strongest predictor of DR progression.

“The DR score, based on a combination of ERG and pupillometry, offered the best predictive capability… These results suggest it is possible to improve the DR staging system, which in turn may enable better allocation of health care resources,” the study authors, led by C. Quentin Davis, PhD, of LKC Technologies, stated.

“This publication is a significant advancement in the quest to identify high-risk patients earlier and improve clinical outcomes,” said Dina Dubey, CEO of LKC Technologies, in the release. “The prospective study included patients with moderate to severe nonproliferative diabetic retinopathy (NPDR) and no center-involved DME at baseline, and it revealed that a single number—known as the DR Score – is the most powerful predictor of progression from NPDR to vision-threatening complications, including proliferative diabetic retinopathy, diabetic macular edema, or treatment.”

The trial used in the post hoc analysis for data collection was the prospective, double-masked, randomized, placebo-controlled TIME-2b study that evaluated the safety and efficacy of 48 weeks of subcutaneous AKB-9778 (a Tie2 activator) in patients with moderate to severe NPDR. Every 12 weeks, patients underwent a comprehensive ocular assessment, including a dilated slit lamp biomicroscopy, intraocular pressure, dilated indirect ophthalmoscopy, spectral-domain OCT, and 7-field FP. FA was conducted at weeks 0, 24, and 48. ERG/pupillometry, OCTA, and UWF-FA were performed using commercially available equipment at a subset of the 44 sites included in the study based on availability.

The DR Score of 26.9 demonstrated a relative risk of 5.6 for progression to vision-threatening conditions (

<.0001), surpassing other predictors including ultra-widefield fluorescein angiography (UWF-FA), optical coherence tomography angiography (OCTA), and fundus photography (FP). Comparatively, the UWF-FA total ischemia index ≥ 0.125 had a relative risk of 5.3 (

<.0001), the OCTA foveal avascular zone area ≥ 0.295 mm

<.05), and the fundus photograph-based diabetic retinopathy severity scale ≥ 27 had a relative risk of 2.1 (

<.05), each still proving statistical significance.

“There is mounting evidence that DR's effects on retinal neurons occur earlier and can progress somewhat independently of changes in retinal vasculature,” the study authors stated. “Thus, it is likely that combining measures of neuronal function with structural vascular information should enhance prediction of DR progression.

“This research suggests that the ERG provides value in defining how DR is staged and monitored, supporting mounting evidence that functional changes in the retina can precede observable vascular abnormalities,” said Mitchell Brigell, PhD, one of the study authors, in the release. “The findings clearly show the value of integrating objective functional testing into the management of diabetic retinopathy. This is also reflected in the inclusion of ERG in the American Academy of Ophthalmology’s Preferred Practice Pattern Guidelines for Diabetic Retinopathy.”

Limitations of the study include its unequal number of subjects across modalities, with fewer eyes tested with OCTA, UWF-FA, and ERG/pupillometry than FP due to equipment availability, in addition to the binary classification of high or low risk, according to the study authors.

 “Thus, performance differences may reflect sampling variation, although the reported uncertainties account for differences in sample size,” the authors stated.

Study identifies RETeval DR Score as the strongest predictor of diabetic retinopathy progression. LKC Technologies. August 4, 2025. Accessed August 12, 2025.

Davis CQ, Waheed NK, Brigell M. Predicting progression to vision-threatening complications in diabetic retinopathy. 

 2025;5(6). doi:10.1016/j.xops.2025.100859

A new study highlights the effectiveness of ERG/pupillometry in predicting diabetic retinopathy progression, surpassing traditional imaging methods.

Canonical

clinical

Study identifies electroretinography/pupillometry as top predictor of DR progression, vision-threatening complications

 casts a shadow of blindness over millions of lives worldwide: 288 million people are expected to be affected by 2040.

 In the United States alone, 2% to 3% of those older than 50 years are already impacted.

 The impact extends beyond lost vision, with profound emotional, social, and economic consequences, including decreased independence, reduced quality of life, and incidences of depression.

In individuals diagnosed with AMD, a remarkable compensatory mechanism emerges to mitigate the detrimental effects of central vision loss. It involves the utilization of a preferred retinal locus (PRL), a location within the macula, outside the foveal region, which serves as the primary site for visual perception in the absence of foveal function. However, this novel visual pathway comes at the cost of compromised visual acuity, as the PRL does not replicate the same quality of vision afforded by the fovea, resulting in a noticeable reduction in visual clarity and definition.

 Despite this trade-off, the establishment of a PRL represents a vital means of preserving some visual function in individuals with AMD, offering a measure of resilience in the face of progressive vision loss.

In order to restore vision for those affected by central vision loss, proposed solutions have included implantable devices and intraocular telescopes. While these advancements have shown promise, they have some drawbacks: cumbersome surgeries, limited field of vision, and the need for intense rehabilitation training.

 The status quo—standard monofocal IOLs when cataract surgery is required—may offer temporary improvement of vision, but ultimately fall short, as they exacerbate the very issue by tightly focusing light on the diseased fovea.

The optical design for improving vision in AMD

An extended macular vision (EMV) IOL is a lens specifically developed to address the distinct challenges of dry AMD. By employing advanced optics to enhance image clarity across the entire macula, this device is designed to enable patients with retinal pathologies to make better use of a PRL, thereby improving their visual function without extensive rehabilitation.

At our facility in Milan, we have adopted the use of the EyeMax Mono IOL (Sharpview Ophthalmology) to address the unique visual needs of patients with dry AMD and retinal diseases in general. This clinical experience formed the basis for our recently published peer-reviewed study in 

, in which we present our long-term safety and sustained visual results in eyes with macular disease and visually insignificant cataract.

This extended macular vision IOL is specifically designed to revitalize image quality in areas of the retina that may still be unaffected by disease. Its optical design allows the light to focus across the entire 10 degrees of the macula, unlike the standard IOL or even the crystalline lens that focus the image on the central 4 degrees only, an area that is often compromised by the retinal condition.

 Besides the different optical design, it is a standard monofocal IOL, which requires no modification to standard cataract surgery technique, using the same incision size, injector system, and surgical approach. As such, it shares the same safety profile as standard monofocal IOLs (note that the EyeMax Mono is CE-marked and not approved in the United States as of May 2025).

The study: Long-term benefits of EyeMax Mono IOL in AMD

We conducted a study that uniquely targeted 113 eyes with 

 and intermediate to severe AMD (of which 9.7% of eyes were developing extensive atrophy) to evaluate the long-term efficacy and safety of the EMV IOL with a mean follow-up period of 48.3 months.

 Nearly 95% of participants gained at least 1 line of corrected distance visual acuity (CDVA), with 35% experiencing a 3-line improvement (

). Notably, even eyes with extensive maculopathy, a condition previously deemed unresponsive to treatment, demonstrated a significant increase in CDVA (0.32 logMAR improvement). The study’s findings also showed that the visual gains persisted even after several years, with participants followed up for as long as 9 years still experiencing significant improvements.

Indeed, beyond the immediate observable outcomes, this EMV IOL potential is more fully realized in its long-term impact on patients’ visual function. While previous studies have elucidated its short-term efficacy and safety,

 research indicates that the PRLs may require up to 6 months to fully develop. It is within this extended time frame that the optical design value may be demonstrated: a sustained and significant visual improvement, which can only be fully appreciated through longitudinal observation.

Interestingly, patients with poorer baseline vision experienced the most significant improvements, suggesting that the optical design helps maximize the use of remaining healthy areas in the macula. This makes it particularly valuable for individuals with advanced macular disease. No specific intraoperative or postoperative complications were reported, and vision loss was uncommon, affecting only 2.7% of eyes. This is a low rate given that these patients are inherently prone to vision deterioration due to the progressive nature of the disease.

However, it is crucial to emphasise this point: the EyeMax Mono does not 

 AMD. Its value is in helping patients with AMD make better use of their remaining healthy retina, even in the presence of foveal atrophy. If macular atrophy reaches and covers the entire 10-degree central area, the benefits of the lens are eroded.

A second chance at sight: Rethinking AMD management

EMV IOL design has been shown to support the brain’s adaptation and the development of new visual pathways, in the face of retinal pathology progression. This is promising for younger patients who have had their central vision compromised by macular degeneration, offering a chance to regain some of the lost acuity. For instance, one remarkable case in our study was a patient diagnosed with AMD at a young age due to occupational sun exposure.

 Over the course of more than 6 years following implantation, this individual’s vision not only stabilised, but significantly improved.

Recent advancements in EMV IOLs represent a significant development in the management of AMD. Unlike more invasive implantable devices or intraocular telescopes— which may be better suited for patients with profound vision loss but require complex surgery and rehabilitation—an EMV IOL offers a simple and safe alternative that can be used earlier in the disease course. It bridges the gap between conventional monofocal lenses and more advanced (and difficult-to-use and implant) solutions, by enhancing image quality across the macula and supporting the brain’s ability to adapt to central vision loss. While it does not halt disease progression, our long-term data suggest that it can deliver sustained visual improvements in selected patients with AMD, particularly those with preserved peripheral macular function. Further prospective studies will help define its role more precisely, but current evidence supports its utility as a practical and effective option for patients with dry AMD undergoing cataract surgery.

1. Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. 

. 2014;2(2):e106-e116. doi:10.1016/S2214-109X(13)70145-1

2. Jager RD, Mieler WF, Miller JW. Age-related macular degeneration. 

 2008;358(24):2606-2617. doi:10.1056/NEJMra0801537

3. Crossland MD, Engel SA, Legge GE. The preferred retinal locus in macular disease. 

. 2011;31(10):2109-2114. doi:10.1097/IAE.0b013e31820d3fba

4. Grzybowski A, Wasinska-Borowiec W, Alio JL, Amat-Peral P, Tabernero J. Intraocular lenses in age-related macular degeneration. 

 2017;255(9):1687-1696. doi:10.1007/s00417-017-3740-8

5. Dunbar HMP, Dhawahir-Scala FE. A Discussion of commercially available intra-ocular telescopic implants for patients with age-related macular degeneration. 

 2018;7(1):33-48. doi:10.1007/s40123-018-0129-7

6. Kessel L, Erngaard D, Flesner P, Andresen J, Tendal B, Hjortdal J. Cataract surgery and age‐related macular degeneration. An evidence‐based update. 

 2015;93(7):593-600. doi:10.1111/aos.12665

7. Casparis H, Lindsley K, Kuo IC, Sikder S, Bressler NM. Surgery for cataracts in people with age-related macular degeneration. 

 2017; 2(2):CD006757. doi:10.1002/14651858.CD006757.pub4

8. Badalà F, Bona E, Devincenzi G, Nouri-Mahdavi K. Long-term visual outcomes of an extended macular vision IOL in eyes with macular disease and visually insignificant cataract. Clin Ophthalmol. 2024;18:2765-2775. doi:10.2147/OPTH.S481570

9. Robbie SJ, Tabernero J, Artal P, Qureshi MA. Initial clinical results with a novel monofocal-type intraocular lens for extended macular vision in patients with macular degeneration. J Refract Surg.2018;34(11):718-725. doi:10.3928/1081597X-20180831-01

10. Hengerer FH, Auffarth GU, Robbie SJ, Yildirim TM, Conrad-Hengerer I. First results of a new hyperaspheric add-on intraocular lens approach implanted in pseudophakic patients with age-related macular degeneration. 

. 2018;2(9):900-905. doi:10.1016/j.oret.2018.02.003

11. Qureshi MA, Robbie SJ, Hengerer FH, Auffarth GU, Conrad-Hengerer I, Artal P. Consecutive case series of 244 age-related macular degeneration patients undergoing implantation with an extended macular vision IOL. 

 2018;28(2):198-203. doi:10.5301/ejo.5001052

12. Sui GY, Liu GC, Liu GY, et al. Is sunlight exposure a risk factor for age-related macular degeneration? a systematic review and meta-analysis. 

 2013;97(4):389-394. doi:10.1136/bjophthalmol-2012-302281

Federico Badalà, MD |E: direzione@microchirurgiaoculare.com

Badalà is an Italian ophthalmologist renowned for pioneering advanced surgical techniques in corneal transplantation, cataract surgery and macular treatments. He has trained in Italy and at the UCLA Stein Eye Institute in California. Dr Badalà practices in Milan at Micro Chirurgia Oculare.

Financial disclosure: Consultant to SharpView Ophthalmology

An implantable solution fills the treatment gap for AMD patients

The US Food and Drug Administration (FDA) has accepted Opus Genetics’ investigational new drug (IND) application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (

Best disease, or vitelliform macular dystrophy, is a rare, inherited retinal condition causing macular degeneration by mutations in the 

 gene, leading to progressive vision loss and, in some cases, blindness. It is characterized by retinal lesions, with symptoms including dimness of vision, metamorphopsia, or scotoma.

 channel, when activated by calcium (Ca2+) ions, controls chloride (Cl-) ion transfer into and out of the retinal pigment epithelium (RPE) cell. This function is crucial to maintaining homeostasis between rod and cone photoreceptors and RPE cells. Mutations in 

 disrupt this homeostasis and result in the breakdown of the rod and cone interphotoreceptor matrix (cIPM) and microvilli (cMV) connection with the RPE. Retinal lesions form containing vitelliform material between the RPE and Bruch’s membrane (BM)/choroid (CH). These vitelliform lesions disrupt and cause atrophy of the RPE. Without support from the RPE, photoreceptor cells critical for normal vision die, resulting in progressive vision loss.

OPGx-BEST1 uses Opus Genetics’ proprietary adeno-associated virus (AAV)-based gene therapy platform, designed to deliver a functional copy of the 

 gene directly to the RPE cells where the defective gene resides.

According to the company, following the IND clearance, it plans to initiate a phase 1/2 clinical trial in the second half of 2025. The trial will be a multicenter, open-label study that will evaluate the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related IRD

George Magrath, MD, CEO of Opus Genetics, commented on the IND clearance in a press release from the company, saying, “BEST1-related IRDs have no approved treatments today, leaving patients and families with uncertainty about the future of their vision. The OPGx-BEST1 trial will be our third ongoing clinical program, reflecting the depth of our pipeline and our commitment to advancing multiple therapies in parallel for patients with urgent, unmet needs.”

The company aims to obtain preliminary data from the phase 1/2 study by the first quarter of 2026.

Opus Genetics Announces FDA Clearance of IND Application for Gene Therapy Candidate OPGx-BEST1. Published August 18, 2025. Accessed August 19, 2025. 

https://ir.opusgtx.com/press-releases/detail/495/opus-genetics-announces-fda-clearance-of-ind-application-for-gene-therapy-candidate-opgx-best1

IRD Programs. Opus Genetics. Accessed August 19, 2025. 

https://opusgtx.com/pipeline-programs/ird-programs/

Best disease, or vitelliform macular dystrophy, is a rare, inherited retinal condition causing macular degeneration by mutations in the BEST1 gene, leading to progressive vision loss and, in some cases, blindness.

FDA clears IND for Opus Genetics’ OPGx-BEST1 gene therapy for inherited retinal disease

jCyte recently announced the dosing of the first patients enrolled in the JC02-88 study for the evaluation of famzeretcel (jCell; jCyte) for the treatment of retinitis pigmentosa (RP).

 Famzeretcel consists of allogeneic retinal progenitor cells (RPCs) administered via intravitreal injection.

“The initiation of the patient dosing in this trial marks an important milestone in our mission to bring a breakthrough treatment to the majority of RP patients who currently have limited treatment options,” John Sholar, chief executive officer of jCyte, said in a press release.

JC02-88 is a phase 2 trial evaluating the safety, tolerability, and efficacy of a single 8.8 million cell injection of famzeretcel. The famzeretcel dose being studied in the JC02-88 study is approximately 50% higher than the highest dose administered in previous jCyte clinical trials.

 Patients enrolled will receive either one famzeretcel dose or a sham treatment (control) and will be assessed for safety and vision changes over 6 months.

The trial is supported by the Gavin Herbert Eye Institute, the UCI GMP Facility, Lexitas, and the California Institute for Regenerative Medicine. A subsequent extension study is anticipated following the conclusion of the JC02-88 trial.

A couple of basics are needed to achieve a response to famzeretcel, 

 Based on the neurotrophic mechanism of famzeretcel, it was hypothesized in a phase 2b study that patients with RP need to have sufficient living photoreceptors to enable restoration of visual function.

 Photoreceptors cannot divide and replace dead adjacent photoreceptors. To benefit from neurotrophic factors, a photoreceptor may have lost its function but must still be alive. More specifically, sufficient living cone photoreceptors are needed to achieve a BCVA response to the famzeretcel injection.

Affecting an estimated 2 million people worldwide, with 100,000 in the United States, RP is a rare genetic disorder that leads to progressive loss of the rod and cone photoreceptors in the retina. Typically diagnosed in adolescence, it causes many patients to become legally blind by middle age.

“This is an exciting development for the RP community, and I am eager to see how this promising therapy advances toward providing a novel cell-therapy treatment for a patient population with vast unmet need,” Paul Sieving, MD, PhD, Neil and MJ Kelly Professor of Ophthalmology at UC Davis School of Medicine and immediate past-Director of the National Eye Institute at NIH, said in a press release.

jCyte Inc. Announces First Patients Treated in JC02-88 Trial for Retinitis Pigmentosa. August 18, 2025. Accessed August 18, 2025. 

https://www.businesswire.com/news/home/20250818947550/en/jCyte-Inc.-Announces-First-Patients-Treated-in-JC02-88-Trial-for-Retinitis-Pigmentosa

Charters L. Retinal progenitor cells: Targeting retinitis pigmentosa. 

https://www.ophthalmologytimes.com/view/retinal-progenitor-cells-targeting-retinitis-pigmentosa

jCyte initiates patient dosing in a new trial for famzeretcel, aiming to transform treatment options for retinitis pigmentosa.

First patients dosed in JC02-88 study

David S. Liao, MD, PhD, with Retina Vitreous Associates Medical Group in Los Angeles, California, spoke with 

 at the 2025 annual scientific meeting of the American Society of Retina Specialists (ASRS). This event was held in Long Beach, California, from July 30 through August 2, 2025.

Liao gave a presentation at the meeting titled, "Extended efficacy and safety of MCO-010 optogenetic therapy for RP: 126-week results from the RESTORE follow-up study (REMAIN). 

Note: The following conversation has been lightly edited for clarity.

Your presentation discusses the most recent developments in MCO-010 as a treatment for retinitis pigmentosa (RP). Can you share this data with us?

I had the opportunity to present on some long-term follow-up data for MCO-010 in patients with advanced retinitis pigmentosa. So patients with advanced RP, vision no greater than 20/1600 were enrolled in 3 treatment groups, low-dose, high-dose, and sham, and they were followed after a single intravitreal injection up to 100 weeks in the original study and up to 2 1/2 years in the follow-up study. The results really show there are some meaningful improvements in vision. They're durable. The safety profile was favorable. So we're looking forward to the medication continuing to be developed and available to our patients with advanced RP. 

There are several gene therapies for inherited retinal diseases that are in development. How does this candidate, MCO-010, approach treating RP?

As we know, there are so many gene therapies coming up for inherited retinal disease; optogenetics is kind of unique in means of its action. It tries to make nonphotoreceptors sensitive to light. So this can be used in patients with advanced disease. It can be used irrespective of their underlying genetic mutation, so very novel. MCO-010 is unique. It targets bipolar cells. It's sensitive across the visible light spectrum, and uses ambient light. So I think it's a very elegant solution. 

What are the next steps for this continued research?

So based on the long-term follow-up study, they've actually submitted a rolling FDA biological licensing application (BLA). So that will be ongoing. Of course, it will depend on the FDA, but hopefully we'll know some more information, perhaps later next year (2026). I know they are planning on doing some additional studies to get more data on how this treatment performs.

What hope does this MCO-010, as well as the other candidates that are in the pipeline, bring for patients with inherited retinal diseases?

For so long, as retina specialists, we've been seeing our patients with inherited retinal diseases. We've been telling them, "Oh, there'll be something in another 5 years. It'll be something in another 10 years." So something is on the horizon now. It's a meaningful step in vision. The patients, even though they had advanced vision loss, gained approximately 3 lines of Snellen equivalent vision. So this is promising. It's not going to be a cure-all for every patient. But this is another option, and really, it's going to be irrespective of your underlying genetic disease. So it will hopefully help a lot of these patients.

Victoria Johnson

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