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The platform, developed by University College London and Heidelberg Engineering, demonstrated accuracy and precision in early tests.

AI-powered Eye2Gene uses multimodal imaging to diagnose inherited retinal disease

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

Ocular Therapeutix announced the company’s conference participation in the upcoming annual OIS Retina Innovation Summit, the American Society of Retina Specialists (ASRS), and Women in Ophthalmology (WIO) meetings.

The company will be presenting 2 sessions at OIS Retina 2025 in Long Beach, California:

Tuesday, July 29, 2025, 11 AM to 12:35 PM PT

Presenter: Sanjay Nayak, MBBS, PhD, Chief Strategy Officer

Tuesday, July 29, 2025, 11 AM to 1:35 PM PT

Presenter: Peter K. Kaiser, MD, Chief Development Officer

The company will have a presentation and a poster at ASRS 2025 in Long Beach:

Presentation: Longitudinal Quantitative Ultra-Widefield Retinal Leakage Assessment in Diabetic Retinopathy Following a Single Axitinib Hydrogel (OTX-TKI) in the HELIOS Trial

Thursday, July 31, 2025, 11:35 AM to 12:15 PM PT

Session: Diabetic Retinopathy Symposium 2

Poster: Macular Volumetric Fluid Outcomes Following Treatment with Intravitreal Axitinib Hydrogel (OTX-TKI) in Non-Proliferative Diabetic Retinopathy

Thursday, July 31, 2025, 9:30 AM to 6:30 PM PT

The company is presenting 1 poster at WIO 2025 in Amelia Island, FL:

Poster: Macular Fluid Volume and Retinal Vascular Leakage Analysis Following a Single Axitinib Intravitreal Hydrogel in Diabetic Retinopathy

Saturday, August 9, 2025, 11 AM to noon ET and Sunday, August 10, 2025, 9:45 to 10:45 AM ET

Ocular Therapeutix is a biopharmaceutical company with its investigational candidate, AXPAXLI (OTX-TKI). AXPAXLI is an axitinib intravitreal hydrogel currently in phase 3 clinical trials for treating wet age-related macular degeneration.

Globenewswire.com. Published 2025. Accessed July 22, 2025. 

https://www.globenewswire.com/news-release/2025/07/22/3119284/0/en/Ocular-Therapeutix-to-Participate-in-Upcoming-Scientific-Conferences.htmlhttps://www.globenewswire.com/news-release/2025/07/22/3119284/0/en/Ocular-Therapeutix-to-Participate-in-Upcoming-Scientific-Conferences.html

Ocular Therapeutix to showcase its drug delivery solutions at upcoming ophthalmology conferences.

Ocular Therapeutix announces upcoming conference presentations

A recent report published by Turkish investigators showed that mucormycosis infection should not be overlooked in the differential diagnosis in patients with diabetes who present with sudden vision loss and cranial nerve palsies,¹ according to Irem Sena Sarac, MD, first author and ophthalmology resident in the Department of Ophthalmology, Prof Dr Cemil Tascioglu Sehir Hastanesi City Hospital, Istanbul.

Sarac and colleagues explained that rhino-orbital-cerebral mucormycosis (ROCM) is a severe, life-threatening fungal infection caused by opportunistic fungi of the order Mucorales, most often 

 This infection typically occurs in patients with diabetes or those who are immunocompromised.

The fungus spores can be inhaled or enter the body through sites of burns or trauma.

 Uncontrolled diabetes, hematologic malignancies, and immunosuppressive therapy are the major risk factors for ROCM infection.

 Once established in the body, the results are often vascular thrombosis and tissue necrosis.

 The most common clinical presentations of mucormycosis are ROCM and pulmonary mucormycosis.

A woman aged 64 years with diabetes arrived at the emergency department reporting dizziness, headache, and decreased vision in the left eye that had persisted over the course of 1 week.

On initial examination in the ophthalmology department, the best-corrected visual acuities in the right and left eyes, respectively, were 6/60 and no light perception.

Although the anterior chamber of the right eye was normal, funduscopy showed retinal hemorrhages and exudates consistent with diabetic retinopathy, and the optic disc appeared normal.

The pupil in the left eye was nonreactive to light. Ptosis, mild proptosis, and chemosis in the left eye were observed, with no eyelid edema or hyperemia. The extraocular motility of that eye was limited in all directions. The anterior segment was normal. Fundus examination showed optic disc edema, retinal hemorrhages, and exudates consistent with diabetic retinopathy, and retinal pallor with a cherry-red spot at the fovea consistent with central retinal artery occlusion. On day 2 of hospitalization, eyelid edema and hyperemia developed in the left eye.

Laboratory testing showed leukocytosis with neutrophil predominance, elevated C-reactive protein, hyperglycemia, and elevated hemoglobin A

. Noncontrast orbital and paranasal sinus computed tomography scans showed left orbital cellulitis, proptosis, and severe ethmoidal and maxillary sinusitis.

was identified by histopathologic examination. No black necrotic tissue was apparent in the nasal and sinus mucosa and skin. Blood cultures showed no growth. Cranial magnetic resonance imaging showed maxillary and ethmoidal sinusitis and increased T2 signal intensity surrounding the left optic nerve, consistent with inflammation, the investigators described.

Management included antifungal treatment (ie, increasing dosages of liposomal amphotericin B), left orbital exenteration, and endoscopic sinus surgery. One week after exenteration, the C-reactive protein value decreased, and a week later, imaging showed progressed sinusitis followed by another sinus surgery, after which the disease stabilized.

The next round of blood cultures grew pan-resistant 

During follow-up, no signs of infection or inflammation were observed in the right eye or orbit. Despite intensive treatment and surgery, the patient died on day 64 of the hospitalization.

Sarac and colleagues commented, “Mucormycosis is an angiotropic fungal infection that invades the internal elastic lamina of blood vessels, leading to angioinvasion, hemorrhagic necrosis, and vascular thrombosis, particularly affecting arteries, veins, and lymphatics, through both mechanical compression and toxin-mediated damage.

 Vision loss may be due to central retinal artery occlusion or orbital apex involvement affecting the optic nerve function.

 In our case, central retinal artery occlusion was suspected to have occurred either due to compression of the ophthalmic artery from soft tissue invasion of the orbital apex or as a direct result of angioinvasion by the fungus.”

Considering this patient’s course, the investigators concluded, “Mucormycosis infection should be considered as a differential diagnosis in diabetic patients presenting with sudden vision loss and cranial nerve palsies. Mucormycosis is a rapidly progressing, devastating infection that requires rapid diagnosis and management by a multidisciplinary team.”

Sarac I, Aksu Y, Yildiz D, et al. Rhino-orbital mucormycosis presenting with central retinal artery occlusion: a case report. Cureus.2025;17:e88020. doi:10.7759/cureus.88020.

Katsitadze L, Javakhishvili G, Burkadze G, et al.

Mucormycosis in a diabetic patient: a case report from Georgia

Bitar D, Van Cauteren D, Lanternier F, et al.

Increasing incidence of zygomycosis (mucormycosis), France, 1997-2006

Benlamkaddem S, Zdaik G, Doughmi D, et al.

Rhino-orbital cerebral mucormycosis: a fatal evolution

Sethi NK, Chadha C, Bajaj M, Moond H. Central retinal vein occlusion as primary ocular manifestation of rhino-orbital-cerebral mucormycosis: A case report. 

. 2022;704451-3. DOI:10.4103/ijo.IJO_1318_22

Mucormycosis emerges as a critical concern in diabetic patients with sudden vision loss, highlighting the need for prompt diagnosis and treatment.

Canonical

clinical

Role of mucormycosis infection on vision in diabetic patient

Ocugen recently announced new members of the Retina Scientific Advisory Board (SAB) and executive leadership team.

These updates support Ocugen's efforts to pursue its goal of 3 biologics license applications (BLA) in the next 3 years.

“As our novel modifier gene therapy programs continue to progress and demonstrate positive data in the clinic, it is now more important than ever to solidify and optimized Retina SAB reflecting the absolute best guidance in this space as we move closer to commercialization,” Shankar Musunuri, PhD, MBA, chairman, CEO, and cofounder of Ocugen, said in a press release. “Additionally, it’s imperative that we ensure our leadership team includes top talent to most effectively execute our corporate strategy.”

Three new members of the Retina SAB are retina surgeons Jeffrey S. Heier, MD; Peter K. Kaiser, MD; and Arshad N. Khanani, MA, MA, FASRS. Heier, Kaiser, and Khanani join SAB chair Lejla Vajzovic, MD, FASRS; and existing SAB members David S. Boyer, MD; and Carl D. Regillo, MD, FACS.

One of the leading retinal clinical researchers in the US, Heier, is the director of the vitreoretinal service and director of retina research at Ophthalmic Consultants of Boston, Massachusetts. Heier’s research focuses on new treatments for exudative and nonexudative macular degeneration, diabetic macular edema, venous occlusive disease, vitreoretinal surgical techniques and instrumentation, and diagnostic imaging of the retina.

Kaiser comes to the SAB with a resume of authoring 7 textbooks, 30 book chapters, and more than 400 peer-reviewed manuscripts. Associate editor of 

, Kaiser is also the Chaney Family Endowed Chair in Ophthalmology Research at the Cleveland Clinic’s Cole Eye Institute, and professor of ophthalmology at Case Western Reserve School of Medicine. Additionally, he is the director of the Center for Ocular Research and Evaluation (CORE).

Managing partner, director of clinical research, and director of fellowship at Sierra Eye Associates, and clinical professor at the University of Nevada, Reno School of Medicine, Khanani has been recognized among the top 10 researchers globally on the Ophthalmologist Power List 2025. In 2021, Khanani founded the Clinical Trials at the Summit meeting and has received numerous awards, including the Macular Society’s Lawrence J. Singerman Medal in 2025 and the American Society of Retina Specialists Presidential Award.

In addition to the new SAB members, Ocugen has made new leadership appointments, including a newly created chief development officer (CDO) position.

Joining Ocugen in the new CDO role, Vijay Tammara, PhD, brings more than 32 years of global regulatory leadership experience with knowledge in biotechnology, biosimilars, 505(b)(2), and complex regulatory submissions. He has made contributions toward the approval of 9 marketing authorizations (MAs), BLAs and new drug applications (NDAs), 2 biosimilar MAs in emerging markets, 3 abbreviated new drug applications, 12 orphan drug designations, 7 qualified infections drug product designations, and filed over 62 investigational NDAs with clearance in the first review cycle with no clinical holds.

 Tammara has held senior advisory and leadership roles at the FDA and life sciences companies, including Sanofi, Wyeth/Pfizer, and Merck, and led regulatory strategy and operations across the US, EU, Latin America, and Asia.

Following the retirement of Mike Shine, Abhi Gupta, MBA, has been named executive vice president, commercial and business development, bringing more than 20 years of experience across commercial strategy, gene therapy, and corporate development in the biopharmaceutical industry. Gupta has led commercialization planning for rAAV gene therapies and played a key role in Pfizer’s $5.5B gene therapy portfolio across neuromuscular, hematologic, and cardiovascular indications. He previously served as senior vice president and head of cell and gene therapies at Syneos Health and held leadership roles at Pfizer, Regeneron, and Johnson & Johnson.

“Ocugen is honored to partner with this distinguished group of advisers and delighted to welcome Dr Tammara and Mr Gupta as the company sharpens its patient-centric focus to potentially deliver paradigm-changing gene therapies,” the company said in a press release.

Ocugen. Ocugen, Inc. Announces New Appointments to Retina Scientific Advisory Board & Executive Leadership Team. GlobeNewswire News Room. Published July 21, 2025. Accessed July 21, 2025. 

https://www.globenewswire.com/news-release/2025/07/21/3118634/0/en/Ocugen-Inc-Announces-New-Appointments-to-Retina-Scientific-Advisory-Board-Executive-Leadership-Team.html

Ocugen announces new SAB members and leadership changes

For decades, I had to deliver the difficult message to patients with inherited retinal diseases (IRDs) that there was no treatment available for them. The cruel reality was that their condition would continue to cause irreversible vision loss.

 In 2017, voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics) was the first in human gene therapy to receive US approval for Leber congenital amaurosis (LCA) due to biallelic 

mutation. This ushered in a new era of gene therapy across medicine. LCA is one of hundreds of genetically characterized IRDs. The population with LCA is limited; about 1000 to 2000 individuals in the US are affected.

 Although I still communicate to patients with other IRDs that no treatment is available for their conditions, I now can say one may be on the horizon. Optogenetics offers a promising mutation-agnostic approach to gene therapy, helping us deliver hope to more patients.

Degenerative retinal disease progressively damages the photoreceptors responsible for capturing light and transmitting visual signals to the brain. Generally—and because of associated and progressive vision loss—IRDs are considered a degenerative disease. 

Studies show that IRDs account for about 20% to 25% of the world’s blindness in the working-age population.

 The most common IRD is retinitis pigmentosa (RP).

RP is generally characterized by retinal pigmentary changes and a gradual loss of night and peripheral vision. Black peripheral retinal “bone spicules” are a classic clinical feature, but pigmentary changes may be subtle to absent. Patients often progress to complete blindness.

 mutation—is another type of IRD clinically characterized by yellow flecks in the macula and retinal midperiphery, progressing to macular “beaten bronze” atrophic changes, leading to central vision loss,

 and severely impacting daily tasks like reading and recognizing faces.

Despite the prevalence and impact of RP and Stargardt disease, treatment options remain limited. This is especially true for individuals with advanced vision loss.

Optogenetics uses gene therapy strategies to introduce light-sensitive transmembrane proteins called opsins into the retina. Unlike gene replacement therapies targeting specific genetic mutations, optogenetics is both mutation- and disease-agnostic. Optogenetic strategies use remaining retinal cells (eg, bipolar and ganglion cells) to regain light sensitivity and restore a degree of functional vision,

 opening new possibilities for individuals with severe vision loss and potentially transforming the management of conditions such as RP and Stargardt disease.

The field of optogenetics is evolving. Current therapies in development include, in order of most advanced clinical status to preclinical therapies, MCO-010 (Nanoscope Therapeutics), RST-001 (RetroSense/Allergan), GS030 (GenSight Biologics), BS01 (Bionic Sight), and RTx-015 (Ray Therapeutics). These therapies differ with respect to their cellular targets, kinetics, and spectral and light sensitivity (

The most advanced optogenetic in development is MCO-010. Multicharacteristic opsin (MCO) is a transgene for a high-performing opsin that sensitizes bipolar cells to light.

 It is sensitive across the visible light spectrum, has rapid kinetics for tracking movement, and responds to ambient light levels. It is delivered by a proprietary adeno-associated virus (AAV2) in a single office-based intravitreal injection and, unlike some other treatments in development, does not require a light-intensifying device to mimic photoreceptor function (

). In a phase 1/2a clinical study, intravitreal treatment with MCO-010 has demonstrated restoration of vision in patients with advanced RP.

In RESTORE (NCT04945772), a randomized, multicenter, sham-controlled phase 2 clinical trial of 27 patients with severe vision loss from advanced RP, no better than logMAR +1.9 (20/1600 Snellen equivalent, counting fingers), MCO-010 demonstrated a clinically meaningful improvement in vision. Patients received either a single intravitreal injection (IVT) of a high- or low-dose MCO-010 or sham IVT. At the 52-week primary end point, approximately 40% of patients who received MCO-010 experienced a mean change in best-corrected visual acuity (BCVA) improvement from baseline of at least +0.3 logMAR, equivalent to 3 lines of Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity improvement, which was statistically significant in both the high- and low-dose MCO-010 groups compared with the sham (

Vision gains were durable, with sustained results at the 76-week key secondary end point. Vision improvement relative to baseline continued to be observed through 2.5 years, including about a 5× greater BCVA area under the curve at the end of 126 weeks in both groups treated with MCO-010 vs those treated with sham. The difference was statistically significant. MCO-010 was well tolerated, and there were no serious or severe adverse events, including severe inflammation, vasculitis, and hypotony. Although inflammation was seen in some patients, it was effectively treated with topical steroids. Based on these results, Nanoscope is preparing a biologics licensing application for marketing approval through the FDA. Additionally, a phase 4 RP postapproval study will begin later in 2025.

What makes MCO-010 particularly exciting is its visible light sensitivity and fast on-and-off kinetics. Unlike other optogenetic therapies that require external laser-stimulation goggles or a light amplification device, MCO-010 works with ambient light. This significantly enhances the therapy’s practicality for patients in their everyday lives.

Although the focus has been on RP, the use of MCO-010 for Stargardt disease is also under investigation. Early clinical data in this population show promising improvements in visual acuity and functional vision. In the open-label, phase 2 STARLIGHT study (NCT05417126) enrolling 6 individuals with severe vision loss (visual acuity no better than 20/800) from advanced Stargardt disease, MCO-010 continued to show a positive safety profile with improvements in visual function across multiple parameters such as visual acuity, visual field, and patient-reported outcome measures. The findings in STARLIGHT support continued development. A randomized, controlled phase 3 registrational study will begin dosing this year. MCO-020 (Nanoscope Therapeutics) is under an investigational new drug (IND)–ready phase for geographic atrophy (GA).

Companies like Ray Therapeutics and GenSight Biologics are also advancing optogenetic programs that target retinal ganglion cells. A phase 1 study (NCT06460844) of RTx-015 (Ray Therapeutics) for RP is under way to evaluate the safety and preliminary efficacy of a single uniocular intravitreal injection. Up to 3 dose cohorts are planned. RTx-021 (Ray Therapeutics) is under an IND-enabling study phase for GA and Stargardt disease. BS01 (Bionic Sight) and GS030 (GenSight Biologics) combine optogenetic therapy with goggles enabling laser stimulation of the transduced ganglion cells. GenSight has initiated a dose-escalation study to evaluate the safety and tolerability of GS030 in patients with RP and plans to investigate the potential for GS030 to treat GA thereafter.

Each optogenetics approach has unique advantages. As the field progresses, we will better understand how best to tailor these therapies for different retinal conditions.

Paradigm shift and potential for a brighter future

Optogenetics represents a potential paradigm shift for retina specialists. Historically, our role was limited to monitoring disease progression and offering low-vision support for patients with IRDs. An approved optogenetic therapy may deliver an opportunity to restore some functional vision in some patients with significant vision loss.

From a clinical perspective—and if an optogenetic strategy is approved—we will need to study and understand who the most suitable candidates for optogenetic therapy will be and who will most likely benefit. Additionally, managing patient expectations and providing comprehensive counseling on potential outcomes will be essential.

Integrating potential optogenetic treatments into clinical practice will require ongoing education and collaboration within the ophthalmology community. Retina specialists will be key in posttreatment care, monitoring patients for efficacy and safety and collaborating with low-vision specialists to maximize functional outcomes.

The prospect of offering mutation-agnostic vision restoration to a population that has long been without options is exciting. For the countless patients with RP, Stargardt disease, and other IRDs without current available treatment, we may be able to provide hope and brighter days based on randomized controlled clinical trial evidence.

Retinal degeneration. Chapter: Stem cell therapy and retinal regeneration. Science Direct. Accessed March 18, 2025. https://www.sciencedirect.com/topics/neuroscience/retinal-degeneration

Hu ML, Edwards TL, O’Hare F, et al. Gene therapy for inherited retinal diseases: progress and possibilities. 

. 2021;104(4):444-454. doi:10.1080/08164622.2021.1880863

Crewe JM, Morlet N, Morgan WH, et al. Mortality and hospital morbidity of working-age blind. 

. 2013;97(12):1579-1585. doi:10.1136/bjophthalmol-2013-303993

Liew G, Michaelides M, Bunce C. A comparison of the causes of blindness certifications in England and Wales in working age adults (16-64 years), 1999-2000 with 2009-2010. 

 2014;4(2):e004015. doi:10.1136/bmjopen-2013-004015

Inherited retinal dystrophies including retinitis pigmentosa. Royal National Institute of Blind People. Reviewed October 12, 2022. Accessed March 18, 2025. https://www.rnib.org.uk/your-eyes/eye-conditions-az/retinitis-pigmentosa

Diagnosis, clinical trials and treatments for inherited retinal diseases. Mayo Clinic. October 14, 2023. Accessed March 18, 2025. https://www.mayoclinic.org/medical-professionals/ophthalmology/news/diagnosis-clinical-trials-and-treatments-for-inherited-retinal-diseases/mac-20556115

Stargardt disease. National Eye Institute. Updated December 4, 2024. Accessed March 18, 2025. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/stargardt-disease

Stargardt disease. Royal National Institute of Blind People. Updated February 1, 2024. Accessed March 18, 2025. https://www.rnib.org.uk/your-eyes/eye-conditions-az/stargardt-disease

Wood EH, Kreymerman A, Kowal T, et al. Cellular and subcellular optogenetic approaches towards neuroprotection and vision restoration. 

. 2023;96:101153. doi:10.1016/j.preteyeres.2022.101153

Wright W, Gajjeraman S, Batabyal S, et al. Restoring vision in mice with retinal degeneration using multicharacteristic opsin. 

. 2017;4(4):041505. doi:10.1117/1.NPh.4.4.041505

Batabyal S, Gajjeraman S, Pradhan S, Bhattacharya S, Wright W, Mohanty S. Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice. 

2021;28(3-4):162-176. doi:10.1038/s41434-020-00200-2

Batabyal S, Kim S, Carlson M, et al. Multi-characteristic opsin therapy to functionalize retina, attenuate retinal degeneration, and restore vision in mouse models of retinitis pigmentosa. 

. 2024;13(10):25. doi:10.1167/tvst.13.10.25

Mahapatra S, Batabyal S,et al. A synthetic opsin restores vision in patients with severe retinal degeneration. 

. 2025;33(5):2279-2290. doi:10.1016/j.ymthe.2025.03.031

Allen C. Ho, MD, FACS, FASRS, is director of retina research, Mid Atlantic Retina, Wills Eye Hospital, and professor of ophthalmology, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia. Ho is a consultant and/or receives grant funding from Adverum Biotechnologies, Apellis Pharmaceuticals, AsclepiX Therapeutics, Astellas Pharma, Clearside Biomedical, Genentech/Roche, Gyroscope Therapeutics, Kodiak Sciences, Lineage, Nanoscope Therapeutics, and Regenxbio.

A mutation-agnostic gene therapy approach offers new possibilities for patients with advanced retinal disease.

Optogenetics for retinal disease

An Indonesian study of the characteristics of the retinal vasculature found that the retinal vessel tortuosity (VT) was significantly higher and the fractal dimension (FD) was significantly lower in patients with 

 Lead author Fariztah Sukainah Nur Fathimah, MD, from the Department of Ophthalmology, Faculty of Medicine, Universitas Airlangga, Surabaya, East Java, Indonesia, and colleagues reported their findings in the 

International Journal of Retina and Vitreous.

Because diabetes is so rampant worldwide, the push is on to be able to identify DR earlier than what is currently possible, with the goal of saving vision.

Abigail Lewis, editorial intern

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