Advances in Ophthalmic Drug Discovery and Delivery: From Targets to Treatments

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmacology".

Deadline for manuscript submissions: 30 September 2025 | Viewed by 1060

Special Issue Editor

Department of Pharmaceutical Sciences, University of North Texas Health Science Center (UNTHSC), Fort Worth, TX 76107, USA
Interests: eyes; ocular diseases; cataracts
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Vision loss remains a significant public health challenge, affecting millions worldwide and contributing to reduced quality of life, increased fall risk, cognitive decline, and overall mortality. As the global population ages, the prevalence of vision-threatening diseases, such as cataract, glaucoma, diabetic retinopathy, and age-related macular degeneration (AMD), continues to rise, highlighting the urgent need for innovative therapeutic solutions.

Recent advancements in ophthalmic treatments have provided new hope for patients. Anti-VEGF therapies have transformed the management of retinal diseases, significantly improving outcomes for conditions such as wet AMD and diabetic macular edema. More recently, complement inhibition has emerged as a promising strategy for geographic atrophy, an advanced form of AMD. Syfovre (pegcetacoplan) and Izervay (avacincaptad pegol) are two newly approved complement C5 inhibitors that work by blocking a key protein involved in inflammation and disease progression, offering the first FDA-approved treatments for dry AMD. Despite these breakthroughs, many challenges remain in drug delivery, durability, and efficacy, underscoring the need for continued innovation.

This Special Issue invites original research and comprehensive reviews focusing on advances in ophthalmic drug discovery and delivery. We welcome contributions on topics including novel drug targets, innovative delivery systems, sustained-release formulations, gene- and cell-based therapies, and artificial intelligence-driven drug development. By bringing together cutting-edge research, this Special Issue aims to foster new perspectives and transformative approaches in the fight against vision loss.

Dr. Hongli Wu
Guest Editor

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Keywords

  • retinal degeneration
  • glaucoma
  • cataract
  • diabetic retinopathy
  • drug target
  • drug delivery
  • drug candidate
  • gene therapy
  • stem cells
  • natural products
  • Artificial Intelligence

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Published Papers (3 papers)

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Research

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17 pages, 10957 KiB  
Article
Topical Application of a Collagen Mimetic Peptide Restores Peripapillary Scleral Stiffness Reduced by Ocular Stress
by Lauren K. Wareham, Ghazi O. Bou Ghanem, Kristin L. Clark, Eric Schlumpf, Brian J. Del Buono and David J. Calkins
Pharmaceuticals 2025, 18(6), 875; https://doi.org/10.3390/ph18060875 - 12 Jun 2025
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Abstract
Background: The biomechanical properties of ocular tissues are critical to physiological processes that span ocular development, aging, and disease. The structural integrity of these tissues is important in mediating how the eye responds to strain and stress that pose challenges to physiological homeostasis. [...] Read more.
Background: The biomechanical properties of ocular tissues are critical to physiological processes that span ocular development, aging, and disease. The structural integrity of these tissues is important in mediating how the eye responds to strain and stress that pose challenges to physiological homeostasis. In the posterior segment, the peripapillary sclera and lamina separate the intraocular chamber and the fluid-filled subarachnoid space. Degradation of each contribute to pathogenic progression in multiple conditions and are largely determined by the integrity and architecture of collagen fibers, especially type I collagen. Methods: We used atomic force microscopy to measure how stress induced by elevations in intraocular pressure impacts stiffness of the peripapillary sclera and glial lamina in the rat eye and whether changes in stiffness could be influenced by topical treatment of a reparative mimetic of type I collagen. Results: Four weeks of elevated intraocular pressure reduced Young’s modulus in peripapillary sclera and glial lamina, coincident with reduced anterograde transport along the optic projection to the brain. Reduction in tissue stiffness correlated with an increase in fragmented collagen. Topical application of collagen mimetic peptide during the period of elevation countered both. Conclusions: Collagen remodeling occurs in many ocular conditions that influence the peripapillary sclera and glial lamina, including glaucoma and myopia. Our results suggest that topical application of collagen mimetic peptides that intercalate with and repair collagen damaged by disease processes could serve to mitigate changes in tissue stiffness and integrity due to degraded collagen. Full article
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16 pages, 2458 KiB  
Article
Evaluating TnP as a Potential Therapeutic Agent for Retinopathy in Zebrafish Models
by João Gabriel Santos Rosa, Jefferson Thiago Gonçalves Bernardo, Yolanda Álvarez, Breandán Kennedy, Carla Lima and Monica Lopes-Ferreira
Pharmaceuticals 2025, 18(6), 840; https://doi.org/10.3390/ph18060840 - 4 Jun 2025
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Abstract
Background: The retina plays a vital role in vision, and its impairment can cause significant visual deficits. Current retinal disease treatments range from conventional anti-inflammatory drugs to advanced anti-VEGF therapies and monoclonal antibodies. TnP, a novel synthetic peptide in preclinical development, has [...] Read more.
Background: The retina plays a vital role in vision, and its impairment can cause significant visual deficits. Current retinal disease treatments range from conventional anti-inflammatory drugs to advanced anti-VEGF therapies and monoclonal antibodies. TnP, a novel synthetic peptide in preclinical development, has demonstrated therapeutic potential in chronic inflammatory conditions such as multiple sclerosis and asthma due to its immunomodulatory properties. Using zebrafish—which share significant genetic homology with humans—we investigated TnP’s effects on retinopathy models mimicking diabetic retinopathy (DR) through either cobalt chloride (CoCl2)-induced hypoxia or light-induced retinal damage (LIRD). Methods: We employed two retinal injury models (CoCl2-induced hypoxia and LIRD) and subjected them to TnP treatment, assessing the outcomes through visual–motor response testing and histological examination. Results: CoCl2 exposure impaired swimming activity, while light damage reduced the movement distance. Both models induced distinct retinal morphological changes. Although TnP failed to reverse most injury effects, it specifically restored the inner plexiform layer (IPL)’s thickness. Conclusions: Our findings suggest that TnP may enhance neuronal plasticity by promoting cell proliferation and synaptic connectivity. While showing promise as a therapeutic candidate for retinal and neurodegenerative disorders, TnP might achieve optimal efficacy when combined with complementary treatments. Full article
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9 pages, 3017 KiB  
Case Report
Intrascleral Tunnel Clamping of Fluocinolone Acetonide Implant: A Novel Scleral Fixation Technique
by Lucas Sejournet, Laurent Kodjikian, Thibaud Mathis, Alban Comet, Pierre Gascon and Frederic Matonti
Pharmaceuticals 2025, 18(6), 849; https://doi.org/10.3390/ph18060849 - 6 Jun 2025
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Abstract
Purpose: This retrospective observational study evaluates the efficacy and safety of a novel scleral fixation technique of the fluocinolone acetonide (FAc) implant in four consecutive patients with post-surgical macular edema (PSME). Case Presentation: Four patients with PSME underwent intrascleral tunnel clamping (ITC) of [...] Read more.
Purpose: This retrospective observational study evaluates the efficacy and safety of a novel scleral fixation technique of the fluocinolone acetonide (FAc) implant in four consecutive patients with post-surgical macular edema (PSME). Case Presentation: Four patients with PSME underwent intrascleral tunnel clamping (ITC) of the FAc implant due to lens defects. A 25-gauge sclerotomy was made 3.5 mm from the limbus and the implant was inserted into it until its end reached the edge of the sclera. Then, an 8-0 absorbable suture was passed through the sclera without penetrating the implant, thereby clamping the sclera around the FAc. All the patients showed improvements in best-corrected visual acuity (from a mean of 20/100 at baseline to 20/40) and central retinal thickness (from a mean of 534 µm at baseline to 318 µm) and with no recurrence of macular edema in most cases, without the need for further treatment. In addition, no anterior migration of the FAc implant or ocular hypertension was observed. This procedure effectively reduced the therapeutic burden for these patients. Although scleral fixation of the FAc implant has been described in small series of patients with successful results, this approach remains off-label. Conclusions: Although off-label, ITC of the FAc implant may offer a promising treatment option for patients who would otherwise remain untreated. Full article
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