Advancing Stargardt Disease Treatments: 20+ Companies Poised to Accelerate the Therapeutic Pipeline | DelveInsight

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.

Request a sample and discover the recent advances in Stargardt disease drugs @ Stargardt Disease Pipeline Report 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: 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 For further information on the Stargardt disease pipeline therapeutics, reach out @ Stargardt Disease Therapeutics Related Reports Stargardt Disease Epidemiology Forecast Stargardt Disease Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and Stargardt disease epidemiology trends. Stargardt Disease Market Stargardt Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Stargardt disease companies, including Kubota Pharmaceuticals, Nanoscope Therapeutics, Alkeus Pharmaceuticals, Belite Bio, Astellas Pharma, among others.

Age-related Macular Degeneration Market Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key AMD companies including Regeneron Pharmaceuticals, Novartis, Roche, Opthea Limited, Kodiak Sciences Inc., REGENXBIO, Alkahest Inc, Graybug Vision, Ribomic USA Inc, Outlook Therapeutics, Inc., Unity Biotechnology, Inc, PanOptica, Inc.

, Clearside Biomedical, Alexion Pharmaceuticals, AstraZeneca, Evergreen Therapeutics, Alkeus Pharmaceuticals, Stealth BioTherapeutics, CellCure Neurosciences, Regenerative Patch Technologies, Allegro Ophthalmics, Annexon Biosciences, NGM Biopharmaceuticals, Ionis Pharmaceuticals, Apellis Pharmaceuticals, Iveric Bio, Gyroscope Therapeutics, Luxa Biotechnology, Gemini Therapeutics , among others. Retinitis Pigmentosa Market Retinitis Pigmentosa Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key retinitis pigmentosa companies including Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, Gensight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Ocugen, Bionic Sight, jCyte, Endogena Therapeutics, ProQR Therapeutics, Aldeyra Therapeutics , among others. Retinitis Pigmentosa Pipeline Retinitis Pigmentosa Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key retinitis pigmentosa companies, including MeiraGTx, Aldeyra Therapeutics, Inc.

, SparingVision, Frontera Therapeutics, ProQR Therapeutics, Bionic Sight, Endogena Therapeutics, Nacuity Pharmaceuticals, Kiora Pharmaceuticals, jCyte, Ocugen, Neurotech USA, Nanoscope Therapeutics, ONL Therapeutics, PulseSight Therapeutics, ViGeneron, Beacon Therapeutics, among others. DelveInsight’s Pharma Competitive Intelligence Service : Through its CI solutions, DelveInsight provides its clients with real-time and actionable intelligence on their competitors and markets of interest to keep them stay ahead of the competition by providing insights into the latest therapeutic area-specific/indication-specific market trends, in emerging drugs, and competitive strategies. These services are tailored to the specific needs of each client and are delivered through a combination of reports, dashboards, and interactive presentations, enabling clients to make informed decisions, mitigate risks, and identify opportunities for growth and expansion.

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