• Optical coherence tomography (OCT)-guided nano-enhanced optical delivery (NOD) delivered multi-characteristic opsin (MCO) genes to precisely selected regions of the retina without a viral vector
• A single treatment produced retinal and cortical light responses that were sustained for up to 52 weeks in a mouse model of complete photoreceptor degeneration
• No detectable toxicity was observed across retinal structure, cell viability, intraocular pressure or electrophysiological function, and minimal vector DNA was detected in non-target tissues

DALLAS, Oct. 21, 2026 /PRNewswire/ -- Nanoscope Therapeutics, Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced the publication of preclinical data in Frontiers in Pharmacology. The paper, titled "Optical coherence tomography guided targeted non-viral optical delivery of opsin-encoding genes into the retina," describes an imaging-guided, non-viral platform for delivering opsin genes to targeted regions of the degenerating retina.

Retinal degenerative diseases, including geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and retinitis pigmentosa (RP), destroy the light-sensing photoreceptors while leaving the inner retinal neurons largely intact. In early to moderate stages of GA, degeneration is often focal or multifocal rather than uniform, and delivering a therapeutic gene to those specific lesions, rather than across the whole retina, has remained a significant technical challenge. The published work utilized the NOD platform, which combines PEGylated plasmonic gold nanorods with low-power near-infrared laser light to transiently permeabilize cell membranes, allowing MCO-plasmids to enter e targeted inner retinal neurons. Because the gene delivery laser is integrated with OCT imaging, the operator can visualize the retina and direct delivery to selected regions of interest.

In rd1 mice, which undergo rapid and complete photoreceptor degeneration, OCT-guided delivery produced robust MCO expression in inner retinal cells with no disruption of retinal structure or change in retinal thickness. Treated animals showed electroretinography and visually evoked potential responses that were significantly greater than untreated controls, and these responses were sustained through 52 weeks. In a focal laser-injury model designed to mimic GA, localized MCO delivery restored microfocal electroretinography responses in the damaged region within one week. Biodistribution analysis at 12 months found vector DNA in brain, gonads, kidney, spleen, heart, lung, liver, and mesentery comparable to control levels.

"GA is a focal disease, and until now the field has largely treated the retina as a single target. Pairing OCT guidance with non-viral delivery lets us put the therapeutic gene precisely where the photoreceptors have been lost, and the year-long durability of the cortical responses tells us those neurons keep working. Because the platform is not constrained by viral packaging limits, the same approach could carry larger payloads, including gene-editing constructs," said Samarendra Mohanty, PhD, co-founder, president and chief scientific officer of Nanoscope.

About the NOD Platform 
Nano-enhanced optical delivery (NOD) is a non-viral gene delivery method that uses functionalized plasmonic gold nanorods and near-infrared light to transiently permeabilize the cell membrane, allowing plasmid DNA to enter targeted cells. Combined with OCT imaging guidance, NOD is designed to deliver genes to specific regions of the retina, without the packaging-size limits or immunogenicity associated with viral capsids. The platform is investigational and has not been evaluated in humans. The intellectual property behind the NOD method and device has been secured globally by Nanoscope.

About MCO Platform
Nanoscope's multi-characteristic opsin (MCO) technology utilizes a proprietary genetically engineered synthetic opsin to enable optimized performance in terms of high light sensitivity across a broad spectrum and fast kinetics. After Nanoscope's Co-Founder, President, and Chief Scientific Officer, Samarendra Mohanty, envisioned and invented the technology, his team has spent over a decade evaluating its performance across multiple animal models and human diseases. Delivery of MCO genes transforms the surviving inner retinal cells to sense light, enabling them to utilize the remaining visual circuitry following photoreceptor loss.

About Nanoscope Therapeutics
Nanoscope Therapeutics is developing disease-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the U.S. Food and Drug Administration (FDA) has accepted and filed the Company's Biologics License Application (BLA) for MCO-010 (MOGENRY) for the treatment of retinitis pigmentosa (RP) with severe vision loss. Japan New Drug Application (JNDA) for MOGENRY is currently under PMDA review for IRDs. If approved, MOGENRY has the potential to become the standard of care for patients with RP and other IRDs, administered as a one-time, in-office injection without the need for genetic testing. The Company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (SD) (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2026. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD, along with RMAT designation for SD, and EMA Orphan designations covering non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies. MCO-010 has also received Sakigake and Orphan designations for inherited retinal dystrophies (IRDs) in Japan and an Orphan designation for IRDs in Saudi Arabia. A Phase 2 program for MCO in geographic atrophy is expected to start in 2026, and other IND-ready programs include Leber congenital amaurosis (LCA). Learn more at NanosTherapeutics.com and follow @NSTherapeutics on X and on LinkedIn. 

Contact
Nanoscope Therapeutics, Inc.
+1 (817) 857-1186
PR@nanostherapeutics.com

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