New York, USA, Nov. 19, 2024 (GLOBE NEWSWIRE) -- Advancing Stargardt Disease Treatments: 20+ Companies Poised to Accelerate the Therapeutic Pipeline | DelveInsight
Advancements in gene therapy is a key market driver for Stargardt disease as it offers the potential to target the underlying genetic mutations causing the condition. These therapies aim to restore or replace defective genes, potentially halting or reversing vision loss, attracting significant investment and interest in developing long-term treatments.
DelveInsight’s 'Stargardt Disease Pipeline Insight 2024' report provides comprehensive global coverage of pipeline Stargardt disease therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Stargardt disease pipeline domain.
Key Takeaways from the Stargardt Disease Pipeline Report
- DelveInsight’s Stargardt disease pipeline report depicts a robust space with 20+ active players working to develop 25+ pipeline Stargardt disease drugs.
- Key Stargardt disease companies such as Kubota Vision, Nanoscope Therapeutics, Ocugen, Belite Bio, Alkeus Pharmaceuticals, Biophytis, reVision Therapeutics, Inc, IIVERIC bio, Ascidian Therapeutics, Gene Vector Technologies, ReVision Therapeutics, Kiora Pharmaceuticals, and others are evaluating new Stargardt disease drugs to improve the treatment landscape.
- Promising pipeline Stargardt disease therapies such as Emixustat, MCO-010, OCU410ST, Tinlarebant, Gildeuretinol, BIO-201, REV-0100, Avacincaptad pegol, ACDN 01, JWK 006, KIO 301, and others are under different phases of Stargardt disease clinical trials.
- In September 2024, Nanoscope Therapeutics Inc., announced a productive End-of-Phase II (EOP2) meeting with the U.S. Food and Drug Administration (FDA) for its clinical program evaluating MCO-010 for the treatment of severe vision loss due to Stargardt Macular Degeneration (SMD), supporting advancement of MCO-010 to a Phase III registrational trial.
- In August 2024, Ocugen, Inc. announced that dosing is complete in the third cohort of its Phase I/II GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease.
- In July 2024, Alkeus Pharmaceuticals Inc. announced the presentation of interim data from its TEASE-3 study that showed early-stage Stargardt disease patients treated with gildeuretinol acetate exhibited no disease progression and remained asymptomatic while on therapy ranging between 2 and 6 years.
- In June 2024, Belite Bio, Inc. announced that its lead pipeline, Tinlarebant, has been granted Sakigake Designation by the Ministry of Health, Labour and Welfare in Japan (MHLW) for the treatment of STGD1.
- In April 2024, SalioGen Therapeutics, announced the nomination of a development candidate, SGT-1001, for the treatment of Stargardt disease, a rare and progressive inherited retinal disease. SGT-1001 is being developed as a one-time, non-viral therapy to slow or stop the progressive loss of central vision in people with Stargardt disease, regardless of the type of mutation.
- In January 2024, Ascidian Therapeutics announced that the FDA had cleared its application to begin the first RNA-editing clinical trial in the United States. This milestone will allow Ascidian to test its RNA exon editor ACDN-01 in patients with Stargardt disease and other retinal diseases caused by mutations in the ATP binding cassette transporter gene, ABCA4.
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The Stargardt disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Stargardt disease drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Stargardt disease clinical trial landscape.
Stargardt Disease Overview
Stargardt disease is a genetic eye disorder that typically causes progressive vision loss, particularly affecting central vision and impairing activities like reading and recognizing faces. It primarily impacts children and young adults and is one of the most common inherited forms of macular degeneration. Stargardt disease is caused by mutations in the ABCA4 gene, which leads to the buildup of a toxic byproduct called lipofuscin in the retinal cells, particularly in the macula.
Symptoms of Stargardt disease include blurry or distorted central vision, difficulty seeing in low-light conditions, and sometimes, color vision changes. These symptoms often worsen gradually, although the rate of progression varies among individuals.
Diagnosis typically involves a comprehensive eye examination that includes visual acuity testing, fundus photography to observe the retina, optical coherence tomography (OCT) to assess retinal layers, and autofluorescence imaging to detect lipofuscin accumulation. Genetic testing can confirm the presence of ABCA4 gene mutations and provide a definitive diagnosis.
Currently, there is no cure for Stargardt disease, but certain management strategies can help slow its progression and improve quality of life. Patients are often advised to avoid excessive sunlight exposure, as ultraviolet light may exacerbate retinal damage. Low-vision aids, like magnifying devices, can assist with daily activities. Research is ongoing into potential treatments, including gene therapies, stem cell therapies, and medications aimed at slowing lipofuscin accumulation in the retina.
Find out more about Stargardt disease drugs @ Stargardt Disease Analysis
A snapshot of the Pipeline Stargardt Disease Drugs mentioned in the report:
| Drugs | Company | Phase | MoA | RoA |
| Emixustat | Kubota Vision | III | Retinoid isomerohydrolase inhibitors | Oral |
| Tinlarebant | Belite Bio | III | RBP4 protein inhibitors | Oral |
| MCO-010 | Nanoscope Therapeutics | II | Eye protein replacements; Gene transference | Intravitreal |
| Gildeuretinol | Alkeus Pharmaceuticals/Columbia University | II | Dimerisation inhibitors; Vitamin A replacements | Oral |
| OCU410ST | Ocugen | I/II | Gene transference; Nuclear receptor subfamily 1 group F member 1 replacement | Sub retinal |
| REV-0100 | ReVision Therapeutics | Preclinical | Lipid modulators | Intravitreal |
| Macuneos | Biophytis | Preclinical | Peroxisome proliferator-activated receptor alpha agonists | Oral |
Learn more about the emerging Stargardt disease therapies @ Stargardt Disease Clinical Trials
Stargardt Disease Therapeutics Assessment
The Stargardt disease pipeline report proffers an integral view of the emerging Stargardt disease therapies segmented by stage, product type, molecule type, route of administration, and mechanism of action.
Scope of the Stargardt Disease Pipeline Report
- Coverage: Global
- Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
- Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical
- Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy
- Therapeutics Assessment By Mechanism of Action: Retinoid isomerohydrolase inhibitors, RBP4 protein inhibitors, Eye protein replacements; Dimerisation inhibitors; Vitamin A replacements, Gene transference, Nuclear receptor subfamily 1 group F member 1 replacement, Lipid modulators, Peroxisome proliferator-activated receptor alpha agonists
- Key Stargardt Disease Companies: Kubota Vision, Nanoscope Therapeutics, Ocugen, Belite Bio, Alkeus Pharmaceuticals, Biophytis, reVision Therapeutics, Inc, IIVERIC bio, Ascidian Therapeutics, Gene Vector Technologies, ReVision Therapeutics, Kiora Pharmaceuticals
- Key Stargardt Disease Pipeline Therapies: Emixustat, MCO-010, OCU410ST, Tinlarebant, Gildeuretinol, BIO-201, REV-0100, Avacincaptad pegol, ACDN 01, JWK 006, KIO 301, and others
Dive deep into rich insights for new Stargardt disease treatments, visit @ Stargardt Disease Drugs
Table of Contents
| 1. | Stargardt Disease Pipeline Report Introduction |
| 2. | Stargardt Disease Pipeline Report Executive Summary |
| 3. | Stargardt Disease Pipeline: Overview |
| 4. | Analytical Perspective In-depth Commercial Assessment |
| 5. | Stargardt Disease Clinical Trial Therapeutics |
| 6. | Stargardt Disease Pipeline: Late-Stage Products (Pre-registration) |
| 7. | Stargardt Disease Pipeline: Late-Stage Products (Phase III) |
| 8. | Stargardt Disease Pipeline: Mid-Stage Products (Phase II) |
| 9. | Stargardt Disease Pipeline: Early-Stage Products (Phase I) |
| 10. | Stargardt Disease Pipeline Therapeutics Assessment |
| 11. | Inactive Products in the Stargardt Disease Pipeline |
| 12. | Company-University Collaborations (Licensing/Partnering) Analysis |
| 13. | Key Companies |
| 14. | Key Products in the Stargardt Disease Pipeline |
| 15. | Unmet Needs |
| 16. | Market Drivers and Barriers |
| 17. | Future Perspectives and Conclusion |
| 18. | Analyst Views |
| 19. | Appendix |
For further information on the Stargardt disease pipeline therapeutics, reach out @ Stargardt Disease Therapeutics
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