STARGARDT DISEASE THERAPEUTICS MARKET ANALYSIS

Global stargardt disease therapeutics market is estimated to be valued at US$ 213.5 Mn in 2023, and is expected to exhibit a CAGR of 31.7% during the forecast period (2023-2030).

Analysts’ Views on Global Stargardt Disease Therapeutics Market:

Global stargardt disease therapeutics market is emerging and willwitness growth in the near future due to the involvement of key players who are launching new technology and innovations to the field. These key players are investing heavily in research and development to find effective treatments and therapies for stargardt disease. Growing awareness and support from various organizations and government initiatives is expected to drive growth of the stargardt disease therapeutics market.

Figure 1. Global Stargardt Disease Therapeutics Market Share (%), By Drug Type, 2023

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Global Stargardt Disease Therapeutics Market– Drivers

• Increasing positive clinical trials result for the treatmemt of stargardt Disease: Increasing positive clinical trials for the treatment of stargardt disease is promising and offers hope for patients suffering from this rare genetic disorder. Stargardt disease is characterized by the progressive degeneration of the macula that leads tosevere vision loss. The positive results from these clinical trials indicate a potential breakthrough in managing and potentially reversing the effects of this debilitating condition. For instance, on August 9, 2023, Nanoscope Therapeutics Inc., a clinical-stage biotechnology company that focuses on gene therapies for retinal degenerative diseases, announced the key results from its Phase 2 clinical trial, STARLIGHT. As per the result, the patients treated with MCO-010 demonstrated clinically meaningful improvements in best-corrected visual acuity (BCVA).

Figure 2. Global Stargardt Disease Therapeutics Market Share(%), By Region, 2023

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Global Stargardt Disease Therapeutics Market- Regional Analysis

Among region, North America is estimated to hold a dominant position in the global stargardt disease therapeutics market over the forecast period. North America is estimated to hold 40.90% of the market share in 2023. Global stargardt disease therapeutics market is expected to witness significant growth in the near future due to introduction of synHSV platform for the treatment of stargardt disease. The synHSV is capable of delivering up to 30x the payload of AAV. It can deliver big genes, genomic genes, and multiple genes, and thus, unlocking the promise of polygenic medicine. The synHSV was launched by REPLAY, a U.S.-based genome writing company that reprogrammes biology by writing and delivering big DNA.

Global Stargardt Disease Therapeutics Market– Impact of Coronavirus (COVID-19) Pandemic

Since the COVID-19 virus outbreak in December 2019, the disease spread to over 100 countries across the globe, and the World Health Organization declared it a public health emergency on January 30, 2020.

COVID-19 affected the economy in three main ways: by directly affecting the production and demand of drugs, by creating disruptions in distribution channels, and through its financial impact on firms and financial markets. Due to nationwide lockdowns, several countries such as China, India, Saudi Arabia, the U.A.E., Egypt, and others faced problems regarding transportation of things from one place to another.

Global Stargardt Disease Therapeutics Market- Segmentation

Global Stargardt disease therapeutics market report is segmented into by drug type, by age group, by distribution channel, and by region.

• By Drug Type, the market is segmented into Emixustat, LBS-008, and Others (Gildeuretinol). Out of which, the emixustat segment is expected to hold a dominant position in the global stargardt disease therapeutics market during the forecast period due to increasing positive clinical trial results and broader plans for the emixustat by the company.
• By Age Group, the market is segmented into Children (Below 17 Years) and Adult (Above 17 Years). Out of which, Children (Below 17 Years) segment is expected to dominate the market over the forecast period due to increasing children patient pool as compared to the adult patient group.
• By Distribution Channel, the market is segmented into Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies. Out of which,  hospital pharmacies segment is  expected to dominate the market over the forecast period andue to  increasing management of the stargardt disease by the healthcare professionals.

Among all the segmentation, the drug type segment is expected to dominate the market over the forecast period due to increasing launch of the drugs, collaboration for the market expansion, and also bringing new technology to meet the unmet needs.

Global Stargardt Disease Therapeutics Market- Cross Sectional Analysis

Rising incidence rate in the North America region is expected to boost demand for stargardt disease therapeutics in North America region. It is estimated prevalence in the U.S. is 10 to 12.5 per 100,000. Cornish et al. estimated its annual incidence in the U.K. is 0.110 to 0.128 per 100 000 individuals.

Global Stargardt Disease Therapeutics Market: Key Developments

In October 2022, ATP, a life sciences venture firm, is the most recent startup to make its debut with a novel approach to genetic editing. With US$ 50 million in funding, U.S.-based Ascidian Therapeutics claims that its RNA “exon editing” approach could match the durability of gene therapy while avoiding some of the risks that come with editing DNA. The platform is designed to correct for mutations in exons,the regions of DNA that contain information needed to make proteins

Global Stargardt Disease Therapeutics Market: Key Trends

• Funding for the novel treatment of stargardt disease: The funding for the stargardt disease research is the solution to the current challenges, and to generate various new opportunities. By funding stargardt disease research, it may be possible to develop ground-breaking cures and therapies for those who suffer from this crippling affliction. Furthermore, funding for research can also lead to a better understanding of the underlying causes of stargardt disease, paving the way for early detection methods and preventive measures. For instance, in February 2022, SpliceBio, a biotechnology company, received US$ 54.2 Mn (€50M) in series A funding to the boundaries of gene therapy vectors. With this new funding in hand, SpliceBio has firmly established its gene therapy focus, and is creating a pipeline of treatments for genetic blindness conditions. SpliceBio’s lead program has its crosshairs on Stargardt disease, a condition caused by mutations in a retinal gene called ABCA4.

Global Stargardt Disease Therapeutics Market: Restraint

• Unavailiability of prior treatment option : Since there is currently no cure for stargardt disease, patients can benefit from visual aids like glasses, magnifiers, and wearable technology as well as auditory choices like Siri, talk-to-text, and audiobooks. Patients are still unable to adapt for light sensitivity, see clearly with 20/20 vision, or drive a car even with these tools. This promotes the creation of novel treatments for Stargardt disease. Researchers are actively exploring potential treatments such as gene therapy, stem cell therapy, and retinal implants to address the limitations faced by stargardt disease patients. These innovative approaches aim to restore vision and improve the quality of life for individuals affected by this condition.

Global Stargardt Disease Therapeutics Market - Key Players

Major players operating in the global stargardt disease therapeutics market include Kubota Pharmaceutical Holdings Co., Ltd., Stargazer Pharmaceuticals Inc., Iveric Bio, Inc., Sanofi S.A., Alkeus Pharmaceuticals Inc., Astellas Pharma Inc., CHABiotech CO., Ltd, ReVision Therapeutics, Inc., Lin BioScience, Inc., Biogen Inc., Hoffmann-La Roche AG, Ocugen, Inc., Ascidian Therapeutics, Nanoscope Therapeutics Inc., Aequus Pharmaceuticals Inc., Fera Pharmaceuticals, LLC, Insmed Incorporated, Belite Bio, Inc., and SpliceBio.

Global Stargardt Disease Therapeutics Market– Definition

Stargardt disease (STGD) is the most common childhood recessively inherited macular dystrophy. The condition has a genetic basis due to mutations in the ABCA4 gene, on chromosome 1, that encodes a retinal transported protein. It results from the accumulation of visual cycle kinetics-derived byproducts in the retinal pigmented epithelium (RPE) with secondary photoreceptor dysfunction and death. The research team in Oxford uses a technology called CRISPR to target specific errors in the faulty gene ABCA4, that causes stargardt disease.  The team hopes to correct the code, by targeting DNA and RNA so that enough normal ABCA4 protein is made to restore diseased macular cells to health.