CGTLive's 2024 Pillars of Progress: Top News in Ophthalmology

For all of 2024, our team was following the clinical development of targeted and novel engineered approaches to the treatment of patients with various medical disorders. These efforts included holding in-depth conversations with experts in the clinical care of these individuals, as well as in cell and gene therapy development, culminating in our coverage of each step of progress that the most exciting cellular and genetic treatments have made along the pipeline.

From major data publications and presentations to FDA decisions and medical meetings, the team spent all year bringing the latest information to the website's front page.

Among our areas of focus in 2024 has been ophthalmology. The major news items appeared among the top pieces our team produced—but sometimes smaller stories reach those heights as well because of their clinical impact, their inventive mechanisms, or otherwise. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in ophthalmology over the course of 2024.

Here, we'll highlight some of the most-read content on CGTLive's ophthalmology page this year. Click the buttons to read further into these stories.

Ocugen Doses First Patient With Retinitis Pigmentosa Gene Therapy in Phase 3 Trial

“Patients with RP associated with mutations in multiple genes currently have no therapeutic options. As a retinal surgeon, I am encouraged by the therapeutic potential of OCU400 to provide long-term benefit to RP patients. OCU400 is a novel modifier gene therapy approach that could initiate a paradigm shift in the treatment of RP and to field of ophthalmology.”
—Lejla Vajzovic, MD, FASRS

In June, Ocugen dosed the first patient with retinitis pigmentosa (RP) in its phase 3 liMeliGhT trial (NCT06388200) of OCU400 gene therapy. The phase 3 trial will last 1 year and have a sample size of 150 participants, at least 8 years of age, half with RHO gene mutations and half that have gene agnostic RP.

Phase 3 Trial of Gene Modifier Therapy for Retinitis Pigmentosa Ready For 2024

“This news brings us even closer to fulfilling our mission to bring our first-in-class, gene-agnostic therapies to market and provide access to patients globally. We look forward to beginning the Phase 3 clinical trial, which we plan to initiate in early 2024.”
—Shankar Musunuri, PhD

In late December of 2024, Ocugen aligned with the FDA on key aspects of its proposed phase 3 trial design to further assess its gene modifier therapy, OCU400, in patients with retinitis pigmentosa (RP) caused by RHO (rhodopsin) and other gene mutations. Ocugen and the FDA agreed on aspects of the trial including the study endpoint, patient enrollment strategy, and study duration (1 year).

Nanoscope Therapeutics’ Retinitis Pigmentosa Gene Therapy MCO-010 Improves Visual Acuity in Randomized Controlled Clinical Trial

“The durable statistically significant vision improvement achieved at multiple time points during the 2-year study is highly noteworthy. This degree of improvement has never been observed in a randomized, controlled trial of a highly heterogeneous severe vision-loss patient population. These findings deliver hope to patients and ophthalmologists that a solution is close to being found. We are finally on the brink of an impactful in-office, mutation-agnostic gene therapy for people with severe vision loss.”
—Allen C. Ho, MD, FACS, FASRS

Nanoscope Therapeutics’ MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy, has improved visual acuity in patients with retinitis pigmentosa (RP) in comparison to patients who received a placebo in data from the phase 2b RESTORE clinical trial (NCT04945772). The data were presented at the American Academy of Ophthalmology 2024 Annual Meeting, held October 18 to 21 in Chicago, Illinois.

MCO-010 Phase 2b Data Supports BLA Submission for Retinitis Pigmentosa

“We observed significant vision restoration in many patients with severe vision loss, including those who were completely blind. Many patients treated with MCO-010 derived a clinically meaningful benefit measurable on the primary visual function test, and this effect was confirmed by a parallel improvement in functional vision assessments. If approved, MCO-010 is poised to make a positive, meaningful impact on the lives of patients affected by this debilitating condition.”
—David Boyer, MD

In March 2024, the Phase 2b RESTORE trial (NCT04945772) evaluating MCO-010 (sonpiretigene isteparvovec; Nanoscope Therapeutics) in patients with retinitis pigmentosa (RP) met its primary and secondary end points, significantly improving best-corrected visual acuity (BCVA) with no serious adverse events (AEs). The company previously received positive feedback from the FDA that change from baseline in a measure of visual acuity in low vision patients could be an appropriate primary efficacy assessment in an adequate and well-controlled study to support a biologic license application (BLA) approval.

Nanoscope Announces BLA Submission Plans for Retinitis Pigmentosa Gene Therapy MCO-010 Following Meeting With FDA

“We are pleased with the positive interactions we have had with FDA as a result of the exceptional expertise and tireless commitment of the Nanoscope team. Our shared goal is to change lives, and together, we have advanced MCO-010 to the point of BLA submission. With every step forward, we are focused on the patients who are waiting for meaningful sight restoration. Our team looks forward to continuing the important work we have begun, along with our partners, to bring this therapy to patients who have significant unmet need.”
—Sulagna Bhattacharya, B. Tech, MBA

In October, Nanoscope Therapeutics stated its plans to file a BLA for MCO-010 (sonpiretigene isteparvovec), its investigational ambient-light activatable MCO gene therapy intended to treat RP, following a meeting with the FDA that it characterized as “productive”. The company noted its intent to pursue a rolling BLA submission for MCO-010 in severe vision loss due to RP, with reference to the fast track designation that the program previously has received from the FDA.