AI Drug Discovery for Pharma and Biotech

Drug discovery

0

drugs

With orphan designations

Overview

Infectious posterior uveitis (IPU) is a rare but vision-threatening inflammation of the choroid and retina caused by pathogens such as Toxoplasma gondii, herpesviruses, syphilis, tuberculosis, or fungal/parasitic infections [1][6][11]. It presents with floaters, photopsia, and vision loss, often with retinal infiltrates, vasculitis, or macular edema [1][16]. Diagnosis requires targeted microbiological testing, and treatment combines antimicrobial therapy with corticosteroids to mitigate inflammation and prevent complications like retinal detachment or glaucoma [11][18].

Population

  • Primarily affects adults aged 20–50 years [5][16], with a US prevalence of ~23.4 per 100,000 [2].

  • Higher risk in immunocompromised individuals (e.g., HIV, rheumatologic disease) [2][16] and regions with endemic infections (e.g., Central/South America for toxoplasmosis) [5][16].

Burden

  • Accounts for ~15% of blindness in the US/Europe [17], with posterior uveitis linked to worse vision-related quality of life than anterior forms [4].

  • Direct annual costs average $12,149 per US patient (vs. $7,834 for anterior uveitis) [4].

  • Chronic inflammation causes cumulative damage (e.g., cataracts, glaucoma) in >50% of cases [1][16].

Therapies

  • Antimicrobials: Pathogen-specific agents (e.g., pyrimethamine-sulfadiazine for toxoplasmosis, antivirals for HSV/VZV) [11][18].

  • Steroids: Low-dose systemic corticosteroids initiated 48–72 hours after antimicrobials to control inflammation [18].

  • Surgical Intervention: Required for complications like retinal detachment or refractory vitreous opacities [1][16].

Categories: rare ophthalmic disorders

Research Papers

329 drug discovery papers related to Infectious posterior uveitis, with 4 first-in-class and 0 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

329 drug discovery papers related to Infectious posterior uveitis, with 4 first-in-class and 0 next-in-class early-stage therapies forecasted to outperform the average preclinical success rate. Recent publications:

2026-06-13 | Real-World Anatomical Outcomes of Suprachoroidal Triamcinolone Acetonide Injections in a Large Retinal Practice over a 4-Year Period.

To evaluate the clinical role, durability, anatomical response, and safety for suprachoroidal triamcinolone acetonide (SCTA) (Xipere) in routine clinical practice. Data were manually extracted from health records at a high-volume retina practice in Cleveland, Ohio. All patients with noninfectious inflammatory macular edema undergoing SCTA (≥1 billing code) between October 25, 2021, and July 17, 2025, were included. Patients with known systemic autoimmune or infectious associations were excluded. Unique eyes were defined by MRN + laterality, with "OU" entries split into OD/OS. Patient demographics, underlying diagnosis categorization, time between first and second injection (for eyes with ≥2 injections) were summarized. Pre- versus post-treatment central retinal thickness (CRT) and intraocular pressure (IOP) were compared via paired t-tests. A total of 177 patients (195 eyes) received 340 SCTA injections for macular edema associated with an underlying diagnosis of intermediate uveitis (10%), pseudophakic cystoid macular edema (31%), and posterior uveitis (59%). The time between first and second injection was approximately 5 months [SD ± 72.5; median 144 (min 35, max 427)]. From baseline to follow-up, mean CRT decreased by 103 µm [95% CI: (-122,-83.2), P < 0.001], while IOP increased minimally [mean difference 0.7 mmHg, 95% CI: (-0.02, 1.43), P = 0.057]; and was medically managed when elevated. In this real-world cohort, findings suggest that SCTA achieves robust anatomical improvement, a favorable IOP profile, and prolonged durability across patients with inflammation-related macular edema.

Open article ↗



2026-04-15 | Retinal pigment epithelium drives macrophage migration during Toxoplasma gondii infection in vitro.

Ocular toxoplasmosis is a leading cause of infectious posterior uveitis worldwide. The retinal pigment epithelium (RPE), a key barrier and immunomodulatory layer in the eye, is directly targeted by Toxoplasma gondii during infection. However, its role in orchestrating the local immune response remains unclear. To investigate whether RPE cells actively drive macrophage migration during T. gondii infection in vitro, and to identify associated cytokine profiles. Adult retinal pigment epithelial cells (ARPE)-19 and primary RPE cells were exposed to tachyzoites, soluble antigens or conditioned supernatants. Macrophage migration was assessed using Transwell® and under-agar assays. Cytokines were quantified by cytometric bead array. Both ARPE-19 and primary RPE exhibited chemotaxis toward parasite antigens (0.12 - 0.5 μg), and enhanced interleukin-6 (IL-6), IL-10 and tumor necrosis factor-α (TNF-α) secretion. Co-culture with RAW 264.7 macrophages further amplified cytokine production. Primary RPE from infected animals occluded 90% of Transwell® pores within 24h. IL-6 and IL-10 levels strongly correlated with migratory activity (r = 0.82 and 0.77, respectively). RPE cells are not passive targets but active participants in the ocular immune response to T. gondii. By secreting IL-6 and IL-10, they establish a chemotactic environment that recruits macrophages. These insights identify the RPE-cytokine-macrophage axis as a potential therapeutic target in ocular toxoplasmosis.

Open article ↗



2026-03-13 | Combined PCR Positivity for Mycobacterium tuberculosis Complex and Nontuberculous Mycobacterium Species in Patients Treated for Tubercular Panuveitis.

Intraocular dual infection by Mycobacterium tuberculosis complex (MTBC) and non-tuberculous mycobacteria (NTM) is rarely reported. This study describes the clinical features, aetiology, and treatment outcomes of patients with dual infection from MTBC and NTM-related uveitis. A retrospective study was conducted on 146 clinically suspected tubercular uveitis (TBU) patients who underwent diagnostic or therapeutic pars plana vitrectomy as plan of laboratory diagnosis-based management between June 2023 and December 2024. Undiluted vitreous samples were analysed with nested MTBC-NTM multiplex real-time PCR assay kit. Six out of 146 patients (4.11%) were found infected with dual infection from MTBC and NTM. All six cases showed severe clinical presentation characterised by bilateral panuveitis (5/6 patients), with significant structural sequelae. Optic atrophy was universal and occurred more frequently than in isolated TBU, while complicated cataract and hypotony-related maculopathy were also common. Despite the dual etiology, all patients achieved inflammatory resolution and visual recovery (mean BCVA improving from 20/400 to 20/100) following standard anti-tubercular therapy (ATT) and corticosteroids, with no recurrence during follow-up. Concurrent MTBC and NTM infection drives a distinct, aggressive form of panuveitis characterized by optic nerve pallor, anterior segment complications of complicated cataract or uveitic membranes and maculopathy. Despite this severity, standard ATT remained an effective first-line strategy, likely either due to therapeutic cross-coverage against NTM or MTBC being the primary driver of uveitis in the present study. The concurrent positivity also highlights the need for vitreous molecular profiling and further research in co-infections in infectious posterior uveitis.

Open article ↗



2026-06-13 | Real-World Anatomical Outcomes of Suprachoroidal Triamcinolone Acetonide Injections in a Large Retinal Practice over a 4-Year Period.

To evaluate the clinical role, durability, anatomical response, and safety for suprachoroidal triamcinolone acetonide (SCTA) (Xipere) in routine clinical practice. Data were manually extracted from health records at a high-volume retina practice in Cleveland, Ohio. All patients with noninfectious inflammatory macular edema undergoing SCTA (≥1 billing code) between October 25, 2021, and July 17, 2025, were included. Patients with known systemic autoimmune or infectious associations were excluded. Unique eyes were defined by MRN + laterality, with "OU" entries split into OD/OS. Patient demographics, underlying diagnosis categorization, time between first and second injection (for eyes with ≥2 injections) were summarized. Pre- versus post-treatment central retinal thickness (CRT) and intraocular pressure (IOP) were compared via paired t-tests. A total of 177 patients (195 eyes) received 340 SCTA injections for macular edema associated with an underlying diagnosis of intermediate uveitis (10%), pseudophakic cystoid macular edema (31%), and posterior uveitis (59%). The time between first and second injection was approximately 5 months [SD ± 72.5; median 144 (min 35, max 427)]. From baseline to follow-up, mean CRT decreased by 103 µm [95% CI: (-122,-83.2), P < 0.001], while IOP increased minimally [mean difference 0.7 mmHg, 95% CI: (-0.02, 1.43), P = 0.057]; and was medically managed when elevated. In this real-world cohort, findings suggest that SCTA achieves robust anatomical improvement, a favorable IOP profile, and prolonged durability across patients with inflammation-related macular edema.

Open article ↗



2026-04-15 | Retinal pigment epithelium drives macrophage migration during Toxoplasma gondii infection in vitro.

Ocular toxoplasmosis is a leading cause of infectious posterior uveitis worldwide. The retinal pigment epithelium (RPE), a key barrier and immunomodulatory layer in the eye, is directly targeted by Toxoplasma gondii during infection. However, its role in orchestrating the local immune response remains unclear. To investigate whether RPE cells actively drive macrophage migration during T. gondii infection in vitro, and to identify associated cytokine profiles. Adult retinal pigment epithelial cells (ARPE)-19 and primary RPE cells were exposed to tachyzoites, soluble antigens or conditioned supernatants. Macrophage migration was assessed using Transwell® and under-agar assays. Cytokines were quantified by cytometric bead array. Both ARPE-19 and primary RPE exhibited chemotaxis toward parasite antigens (0.12 - 0.5 μg), and enhanced interleukin-6 (IL-6), IL-10 and tumor necrosis factor-α (TNF-α) secretion. Co-culture with RAW 264.7 macrophages further amplified cytokine production. Primary RPE from infected animals occluded 90% of Transwell® pores within 24h. IL-6 and IL-10 levels strongly correlated with migratory activity (r = 0.82 and 0.77, respectively). RPE cells are not passive targets but active participants in the ocular immune response to T. gondii. By secreting IL-6 and IL-10, they establish a chemotactic environment that recruits macrophages. These insights identify the RPE-cytokine-macrophage axis as a potential therapeutic target in ocular toxoplasmosis.

Open article ↗



2026-03-13 | Combined PCR Positivity for Mycobacterium tuberculosis Complex and Nontuberculous Mycobacterium Species in Patients Treated for Tubercular Panuveitis.

Intraocular dual infection by Mycobacterium tuberculosis complex (MTBC) and non-tuberculous mycobacteria (NTM) is rarely reported. This study describes the clinical features, aetiology, and treatment outcomes of patients with dual infection from MTBC and NTM-related uveitis. A retrospective study was conducted on 146 clinically suspected tubercular uveitis (TBU) patients who underwent diagnostic or therapeutic pars plana vitrectomy as plan of laboratory diagnosis-based management between June 2023 and December 2024. Undiluted vitreous samples were analysed with nested MTBC-NTM multiplex real-time PCR assay kit. Six out of 146 patients (4.11%) were found infected with dual infection from MTBC and NTM. All six cases showed severe clinical presentation characterised by bilateral panuveitis (5/6 patients), with significant structural sequelae. Optic atrophy was universal and occurred more frequently than in isolated TBU, while complicated cataract and hypotony-related maculopathy were also common. Despite the dual etiology, all patients achieved inflammatory resolution and visual recovery (mean BCVA improving from 20/400 to 20/100) following standard anti-tubercular therapy (ATT) and corticosteroids, with no recurrence during follow-up. Concurrent MTBC and NTM infection drives a distinct, aggressive form of panuveitis characterized by optic nerve pallor, anterior segment complications of complicated cataract or uveitic membranes and maculopathy. Despite this severity, standard ATT remained an effective first-line strategy, likely either due to therapeutic cross-coverage against NTM or MTBC being the primary driver of uveitis in the present study. The concurrent positivity also highlights the need for vitreous molecular profiling and further research in co-infections in infectious posterior uveitis.

Open article ↗



Access all drug discovery articles and probability of success in trials forecasts:

Access all drug discovery articles and probability of success in trials forecasts:

Drug Discovery Landscape

0 orphan drug designations.

0 orphan drug designations.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.