Leber Congenital Amaurosis

DALLAS, Oct. 13, 2025 /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 multiple presentations at the American Academy of Ophthalmology (AAO) 2025 Annual Meeting, to be held October 18-20, 2025 in Orlando, FL, as well as at Eyecelerator@AAO 2025, on October 16, 2025, also in Orlando. Nanoscope will showcase the vision-restoring proprietary Multi-Characteristic Opsin (MCO) platform for patients with severe vision loss from retinitis pigmentosa (RP) and Stargardt disease (SD), both debilitating conditions with no currently approved treatments. Presentation Details: Title: "Nanoscope Therapeutics Company Showcase" (Eyecelerator @AAO 2025) Session: Posterior/Retinal Date / Time: Thursday, October 16, 2025 | 1:40-1:45 p.m. EDT Location: W230 Presenter: Samuel Barone, MD, Chief Medical Officer, Nanoscope Therapeutics Title: "152-Week REMAIN Data from Extended Analysis of MCO-010 RESTORE Study of Optogenetic Therapy for Retinitis Pigmentosa" Session: Section XI: Late Breaking Developments, Part II Date / Time: Saturday, October 18, 2025 | 8:11-8:18 a.m. EDT Location: W415 Valencia Presenter: Allen C. Ho, MD, FACS, FASRS, Attending Surgeon and Director of Retina Research, Wills Eye Hospital; Chief Medical Advisor, Nanoscope Therapeutics. Title: "Visual Improvement Across Multiple Assessments with MCO-010 Optogenetic Therapy for Stargardt Disease" Session: Poster Panel Discussion: Retina, Vitreous Date / Time: Saturday, October 18, 2025 | 1:45-2:15 p.m. EDT Location: Hall WB4, Station 1 Presenter: Kenneth C. Fan, MD, Retina Consultants of Texas Title: "Optogenetic Therapy in Adults with Retinitis Pigmentosa and Stargardt Disease" Session: Therapeutic Approaches to Vision Restoration Date / Time: Monday, October 20, 2025 | 8:30-8:45 a.m. EDT Location: W311E Presenter: Samarendra Mohanty, PhD, President, and Chief Scientific Officer Nanoscope Therapeutics; and Stephen Tsang, MD, PhD, Columbia University, Irving Medical Center. "We look forward to sharing and discussing the latest MCO-010 data at AAO and Eyecelerator 2025," said Sulagna Bhattacharya, Chief Executive Officer. "These presentations underscore the continued strength of our clinical and regulatory foundation. With the rolling BLA for RP now underway and five new orphan designations granted by the European Medicines Agency, we are driving strong global momentum toward bringing MCO-010 to patients affected by blinding retinal diseases." About the MCO Platform MCO is a one-time, in-office, intravitreal disease-agnostic therapy platform designed to restore vision in patients with photoreceptor degeneration, including Retinitis Pigmentosa (RP), Stargardt disease (SD), and geographic atrophy (GA). By activating highly dense bipolar retinal cells to become light sensitive, MCO utilizes the remaining visual circuitry following photoreceptor death. MCO treatment does not require genetic testing, invasive surgery, or repeat dosing, enabling broad patient applicability within existing retina office workflows. 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 retinitis pigmentosa (RP) (NCT04945772), a rolling BLA submission to the FDA has been initiated. If approved, MCO-010 has the potential to be the standard of care for RP patients, 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 2025. 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 to cover non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies. A Phase 2 program for MCO in geographic atrophy (GA) is expected to start by the end of 2025. Other IND-ready programs include Leber congenital amaurosis (LCA). Contact: Nanoscope Therapeutics (817) 857-1186 [email protected] SOURCE Nanoscope Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Oct. 02, 2025 -- Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to NPI-001 (N-acetylcysteine amide) tablets, Nacuity’s proprietary, investigational therapy for the treatment of patients with retinitis pigmentosa (RP). “Breakthrough Therapy Designation represents objective assessment by the FDA that early clinical evidence supports the potential of NPI-001 tablets to deliver substantial treatment effects for patients with retinitis pigmentosa, a serious blinding disease,” said G. Michael Wall, Ph.D., Senior Vice President and Chief Scientific Officer of Nacuity Pharmaceuticals. “This recognition represents a key value-creating milestone for Nacuity and underscores our commitment to efficiently advancing NPI-001 toward late-stage development.” A drug may receive BTD if the drug is intended to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. In addition to the benefits of Fast Track Designation previously granted to NPI-001, a drug that receives BTD is eligible for intensive guidance on efficient drug development and organizational commitment from FDA. Often diagnosed in childhood or adolescence, retinitis pigmentosa (RP) is a heterogeneous group of inherited retinal diseases involving nearly 3,100 different mutations in more than 50 genes (Daiger et al., 2013), causing progressive night and peripheral vision loss. RP often leads to legal blindness and, in some cases, complete blindness. Forms of RP and related diseases include Usher syndrome, Leber congenital amaurosis and Bardet-Biedl syndrome. According to Foundation Fighting Blindness, an estimated 100,000 people in the U.S. have RP. There are no FDA-approved or standard treatments for RP. A curative gene therapy, voretigene neparvovec (LUXTURNA), is available for patients with the RPE65 mutation, which affects approximately 1-6% of patients with RP (Cross et al., 2022). NPI-001 is a proprietary, GMP-grade formulation of N-acetylcysteine amide (NACA) tablets being developed to target oxidative stress associated with diseases such as retinitis pigmentosa (RP). Preclinical studies indicate that NPI-001 boosts glutathione, the body’s most powerful endogenous antioxidant, to stop chemically aggressive oxygen molecules from damaging retinal cells (Wood et al., 2024). NPI-001 is formulated as a stable tablet, easy for patients to self-administer, with well-established manufacturing processes and convenient packaging options. In addition to Breakthrough Therapy Designation, NPI-001 has also been granted Fast Track and Orphan Drug Designations for the treatment of RP, the latter of which provides seven years of U.S. FDA regulatory exclusivity for the product upon regulatory approval. Nacuity Pharmaceuticals is a clinical-stage leader in innovative treatments for diseases and conditions involving oxidative stress. The company’s powerful, targeted therapies aim to attenuate oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has operations in Fort Worth, TX, USA, and Australia, and extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org). The content above comes from the network. if any infringement, please contact us to modify.

Opus Genetics plans to meet with the FDA in the fourth quarter to discuss the next steps for its gene therapy program. \n Opus Genetics has shared data on its gene therapy in children with an ultrarare eye disease, setting the biotech up to talk to the FDA about the next steps for the program.North Carolina-based Opus is developing the gene augmentation therapy for Leber congenital amaurosis type 5 (LCA5) patients. The candidate uses an AAV8 vector and the same promoter technology as Roche’s Luxturna to deliver a functional LCA5 gene. Opus’ goal is to address the photoreceptor impairment that causes vision loss in patients with LCA5, a condition that it says affects around 200 people in the U.S. Having reported data on three adults with LCA5 last year, Opus began enrolling adolescents with the rare disease to kick off its expansion into the pediatric population. The first three patients were aged 16 to 17 and had severe vision impairment at baseline.As of the data cutoff, Opus reported an average of a 0.3 logMAR improvement across the cohort. Each 0.1 logMAR shift indicates a one line change on an eye test chart. Two patients had improvements of 0.2 and 0.7 logMAR three months after treatment, with the smallest improvement seen in the patient with the best vision at baseline. The third patient had an improvement of 0.5 logMAR after one month. Opus compared the data favorably to the results seen in the first three adults to receive the therapy. The improvement in adults increased in the early part of the study, climbing (PDF) to a 0.48 logMAR change by the sixth month of the trial. Opus said the combined adult data support the durability of the vision improvements through 18 months.Shares were up more than 3% premarket on the data drop Tuesday morning. The top-line pediatric safety data shared by Opus largely support the encouraging signs the company reported in adults. No events were related to the study drug. One pediatric participant had a preexisting cataract that worsened at three months. Opus said the worsening was attributed to the surgical procedure used to deliver the gene therapy and didn’t obscure improvements in retinal sensitivity.Opus plans to meet with the FDA in the fourth quarter to discuss the next steps for the program. Before sharing the pediatric data, the company said an accelerated clinical development pathway to a filing for FDA approval could be acceptable if the efficacy seen in children is similar to the results in adults.