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

Optogenetics for 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

The US Food and Drug Administration (FDA) recently granted fast track designation to Sanofi’s SAR446597 for the treatment of geographic atrophy (GA).

SAR446597 is a one-time intravitreal gene therapy treatment that “delivers genetic material encoding 2 therapeutic antibody fragments that target and inhibit 2 critical components of the complement pathway: C1s in the classical pathway and factor Bb in the alternative pathway,” a press release said.

Through the dual targeting approach of SAR446597, treatment burden is significantly reduced by eliminating frequent intravitreal injections. SAR446597 “aims to address the underlying pathophysiology of complement-mediated retinal diseases through long-term expression of therapeutic proteins following a single intervention,” a press release stated.

A phase 1/2 clinical study to evaluate the safety, tolerability, and efficacy of SAR446597 is in the plans for Sanofi. The company is evaluating SAR402663 in a phase 1/2 study (

) for the treatment of neovascular wet age-related macular degeneration (nAMD). SAR402663 is also a one-time intravitreal gene therapy.

Affecting roughly 200 million people globally, AMD and GA are progressive retinal degeneration diseases. GA is an advanced form of AMD, characterized by lesions caused by the degeneration of retinal cells. GA affects approximately 1 million people in the US, more than 2.5 million in Europe, and more than 5 million worldwide, causing permanent vision loss and impacting quality of life.

Sanofi Winthrop Industrie. Press Release: Sanofi’s SAR446597 earns fast track designation in the US for geographic atrophy due to age-related macular degeneration. GlobeNewswire News Room. Published July 16, 2025. Accessed July 17, 2025. 

https://www.globenewswire.com/news-release/2025/07/16/3116124/0/en/Press-Release-Sanofi-s-SAR446597-earns-fast-track-designation-in-the-US-for-geographic-atrophy-due-to-age-related-macular-degeneration.html

Sanofi's SAR446597 receives FDA fast track designation, offering a promising one-time gene therapy for geographic atrophy and reducing treatment frequency.

clinical

FDA grants fast track designation to SAR446597

Apellis announced 5 abstracts that will be orally presented at the American 

Society of Retina Specialists (ASRS) Annual Scientific Meeting to be held July 30 through August 2 in Long Beach, California.

Presentation highlights include 48-month data from the OAKS and DERBY trials as well as the GALE open-label extension. OAKS and DERBY are multicenter, phase 3, randomized, double-masked trials evaluating pegcetacoplan injection (SYFOVRE) in patients with 

Caroline Baumal, MD, chief medical officer of Apellis, commented on the upcoming presentations in a press release from the company, saying, “Retina specialists continue to be excited about SYFOVRE—the most chosen treatment for GA—as demonstrated by the 5 oral presentations at this prestigious meeting. As leaders in GA, we are proud to present data that highlight SYFOVRE’s unique ability to slow GA progression in as few as 6 doses per year, offering hope to patients living with this progressive and irreversible disease.”

Presentations announced by the company include the following:

Baseline Characteristics Associated with Geographic Atrophy Progression in the OAKS and DERBY Trials

Presented by Srinivas R. Sadda, MD, at the Dry AMD Symposium 1 on Thursday, July 31, 2:16 to 2:19 PM PT


Early vs Delayed Pegcetacoplan Treatment for GA Secondary to AMD: 48-Month Results from OAKS, DERBY, and GALE Open-Label Extension

Presented by Roger A Goldberg, MD, MBA, at the Dry AMD Symposium 1 on Thursday, July 31, 2:19 to 2:22 PM PT


Clinical Utility of the Fellow Eye in Patients with Bilateral GA due to AMD: Long-Term Data from the Pegcetacoplan Clinical Program

Presented by Diana V. Do, MD, FASRS, at the Dry AMD Symposium 1 on Thursday, July 31, 2:22 to 2:25 PM PT


A Descriptive Analysis of Patients Who Received Pegcetacoplan and Anti-VEGF for the Treatment of Geographic Atrophy and Neovascular AMD

Presented by Nimesh A. Patel, MD, FASRS, at the Dry AMD Symposium 1 on Thursday, July 31, 2:38 to 2:41 PM PT


Impact of AREDS Oral Micronutrient Supplementation on Geographic Atrophy Progression: Insights from the Phase 3 OAKS and DERBY Trials

Presented by Paul Hahn, MD, PhD, FASRS, at the Dry AMD Symposium 2 on Thursday, July 31, 3:14 to 3:17 PM PT

Apellis Announces Five Abstracts Accepted for Oral Presentation at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting. Published July 15, 2025. Accessed July 15, 2025. 

https://investors.apellis.com/news-releases/news-release-details/apellis-announces-five-abstracts-accepted-oral-presentation-0

Presentation highlights include 48-month data from the OAKS and DERBY trials as well as the GALE open-label extension.

Canonical

Apellis announces 5 abstracts to be orally presented at the American Society of Retina Specialists (ASRS) annual meeting

Today Nanoscope Therapeutics announced its rolling submission of a biologics license application (BLA) to the US Food and Drug Administration (FDA) for MCO-010 for the treatment of vision loss caused by retinitis pigmentosa (RP).

Nanoscope’s BLA has been granted rolling review by the FDA, with the first modules already submitted. The completion of the full BLA submission is anticipated in early 2026 and is eligible for priority review under the program’s fast-track designation.

MCO-010 is Nanoscope’s lead investigational therapy designed to address the broad genetic diversity of RP. MCO-010 is a gene-agnostic gene therapy for retinal disease. This BLA marks the first BLA submitted for a gene-agnostic gene therapy for retinal disease, and if approved, MCO-010 would be the first gene-agnostic therapy to restore vision in legally blind patients with RP.

Affecting more than 100,000 people, leaving more than 25,000 legally blind, RP is one of the leading causes of blindness in the working-age population in the US.

“For the first time, patients who are considered to be on a path to permanent blindness may have a chance to regain sight,” said Sulagna Bhattacharya, CEO and cofounder of Nanoscope, in a press release. 

“We are deeply thankful to the FDA for their guidance as we remain steadfast in our mission to restore vision and bring light back into the lives of those living in darkness.”

The RESTORE phase 2b trial demonstrated MCO-010 met its best corrected visual acuity (BCVA) end points in both dose groups at 52 weeks vs sham-control. Vision gains from baseline were >0.3 LogMAR, a result equivalent to 3 or more lines on an eye chart.

Visual acuity gains have been observed in ongoing long-term follow-up through 3 years, with no serious adverse events reported in treated eyes. Nanoscope said further multi-year follow-up is scheduled to evaluate longer-term efficacy and safety.

“It’s the first time the FDA will be evaluating an application for a gene-agnostic therapy for inherited retinal disease,” Glenn Sblendorio, chairman of the board of directors for Nanoscope, said in a press release. 

“We feel confident in our clinical data, which we believe will mark the dawn of a new era in retinal care. Our goal is to bring back the light of hope for RP patients – and eventually for many other retinal degenerative conditions.”

Nanoscope Therapeutics. Nanoscope Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat Retinitis Pigmentosa. Prnewswire.com. Published July 14, 2025. Accessed July 14, 2025. https://www.prnewswire.com/news-releases/nanoscope-therapeutics-initiates-rolling-submission-of-biologics-license-application-to-fda-for-mco-010-the-first-gene-agnostic-therapy-to-treat-retinitis-pigmentosa-302503892.html

The completion of the full BLA submission is anticipated in early 2026 and is eligible for priority review under the program’s fast-track designation.

FDA grants Nanoscope rolling BLA submission of MCO-010

Boehringer Ingelheim recently announced the start of the THULITE phase 2 clinical study (

). THULITE will evaluate the efficacy, safety, and tolerability of BI 1815368, an oral medication for the treatment of 

Affecting more than 21 million people globally, DME is a leading cause of vision loss in people with diabetes. Current treatments for DME are limited, requiring frequent visits to a retina specialist for eye injections, and only addressing 1 eye at each visit.

“An oral therapy for DME could represent a major step forward in its management,” said Charles C. Wykoff, MD, PhD, principal investigator of the trial, director of research for Retina Consultants of Texas, and chair of research for Retina Consultants of America.

"A safe and effective systemic therapy would offer an important alternative to repeated intravitreal injections; it could enable more consistent initiation of care earlier in the disease process, improve treatment adherence, and optimize quality of life for patients."

This oral therapy would aim to prevent fluid leakage into the macula caused by DME by reducing the permeability of newly formed blood vessels. Through oral administration, unlike current treatments, BI 1815368 would allow for the potential of at-home treatment, while simultaneously addressing both eyes.

“DME is one of the most challenging complications of diabetes, and as vision worsens, people may lose their independence and rely more on loved ones,” said Dario Madani, CEO at PRO RETINA, members of which serve on Boehringer’s DME Clinical Program Steering Committee.

“Patients and caregivers may have to balance work, family obligations, and other diabetes complications, while managing their eye condition. We need treatment options that could reduce this burden.”

Part of Boehringer’s broader clinical program targeting diabetic retinal disease, BI 1815368 is the fourth investigational compound from Boehringer Ingelheim’s Eye Health portfolio to advance to a phase 2 clinical trial, a press release said. The broader program also includes BI 764524, which is currently being studied in the CRIMSON phase II trial (

“At Boehringer Ingelheim, we are working toward a future where earlier intervention leads to long-term outcomes that prevent vision loss. One way we aim to achieve this is by developing oral treatments, which offer patients the ability to be more in control of their condition,” said Patrick Bussfeld, MD, PhD, global head of medicine, eye health at Boehringer Ingelheim.

“By targeting the disease pathway systemically, we also have the potential to prevent DME in one eye while the other is being treated.”

Boehringer-ingelheim.com. Published 2025. Accessed July 7, 2025. 

https://www.boehringer-ingelheim.com/science-innovation/human-health-innovation/phase-ii-start-oral-treatment-diabetic-macular-edema

Boehringer Ingelheim initiates THULITE study, exploring an oral treatment for diabetic macular edema, aimed to improve patient care and convenience.

THULITE phase II study evaluates BI 1815368

Opus Genetics, Inc, announced that the company entered into an agreement with the Retinal Degeneration Fund (RD Fund), the venture philanthropy arm of the Foundation Fighting Blindness, through which Opus will receive up to $2 million in nondilutive funding to support the advancement of Opus’s OPGx-MERTK program. Through this program, gene therapies are developed to treat patients with retinitis pigmentosa caused by pathogenic variants in the 

George Magrath, MD, who is CEO of Opus Genetics, said, “This strategic funding enables us to advance the development of OPGx-MERTK, a promising program that aims to address a retinal degenerative condition with no currently approved therapies. OPGx-MERTK is currently in preclinical development, and we expect this financial support will allow us to move this asset toward Investigational New Drug enabling studies. We are grateful to the Foundation Fighting Blindness and the RD Fund for their continued partnership as we work to bring meaningful treatments to patients suffering from vision-threatening conditions.”

 gene cause a rod-cone dystrophy with early macular atrophy, with retinitis pigmentosa being the most common phenotype. Opus is developing OPGx-MERTK as a modern adeno-associated virus vector for the treatment of MERTK-associated inherited retinal disease, which affects approximately 600 individuals in the US.

This investment reflects the power of combining venture philanthropy, industry collaboration, and cutting-edge science to deliver meaningful progress for patients. Rusty Kelley, PhD, managing director of the RD Fund, commented, “Our alliance with Opus Genetics leverages the full strength of the Foundation Fighting Blindness community—our donors, mission, and team—alongside the proven expertise of Opus and gene therapy pioneers Drs Jean Bennett and Tomas Aleman, to accelerate life-changing treatments.”

With the addition of this funding, based on current projections, Opus believes that its cash on hand will now be sufficient to fund operations into the second half of 2026.

Through the program, gene therapies are developed to treat patients with retinitis pigmentosa caused by pathogenic variants in the MERTK gene.

Allen C. Ho, MD, FACS, FASRS

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