Stargardt Disease Pipeline 2020 by DelveInsight

(Albany, US) DelveInsight has launched a new report on Stargardt Disease Pipeline


“Stargardt Disease (STGD) Pipeline Insight, 2020” report by DelveInsight outlays comprehensive insights of present clinical development scenario and growth prospects across the Stargardt Disease (STGD) market. A detailed picture of the Stargardt Disease (STGD) pipeline landscape is provided, which includes the disease overview and Stargardt Disease (STGD) treatment guidelines. The assessment part of the report embraces in-depth Stargardt Disease (STGD) commercial assessment and clinical assessment of the Stargardt Disease (STGD) pipeline products from the pre-clinical developmental phase to the marketed phase. In the report, a detailed description of the drug is proffered including mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Stargardt Disease (STGD) collaborations, licensing, mergers and acquisition, funding, designations, and other product-related details.


Stargardt disease (STGD) is named after Karl Stargardt, a German ophthalmologist who first reported a case in his practice in 1901. It is also called Stargardt macular dystrophy, juvenile macular degeneration, or fundus flavimaculatus. It is a rare retinal disorder inherited in an autosomal recessive pattern. Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. It typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. Mutations in a gene called ABCA4 are the most common cause of Stargardt disease. This gene makes a protein that normally clears away vitamin A byproducts inside rods and cones (photoreceptors). Cells that lack the ABCA4 protein accumulate clumps of lipofuscin, a fatty substance that forms yellowish flecks. As the clumps of lipofuscin increase in and around the macula, central vision becomes impaired. Eventually, these fatty deposits lead to the death of photoreceptors and vision becomes further impaired. The most common symptom of Stargardt disease is variable, often slow loss of central vision in both eyes. The progression of symptoms in Stargardt disease is different for each person. People with an earlier onset of disease tend to have more rapid vision loss. Patients might notice gray, black, or hazy spots in the center of their vision, take longer than usual to adjust when moving from light to dark environments, their eyes may be more sensitive to bright light. Some people also develop color blindness later in the disease.


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Stargardt Disease Pipeline

Research is underway to identify novel treatment approaches that may prove to be a way to support such patients. Key players, such as Acucela Inc (Kubota), Alkeus Pharmaceuticals Inc, ProQR, Astellas Pharma Inc and others are involved in developing therapies for Stargardt’s disease. Acucela Inc. (now Kubota) recently announced that the European Medicines Agency (EMA) granted orphan designation to Acucela’s leading drug candidate Emixustat hydrochloride (“emixustat”) for the treatment of Stargardt disease. It is an oral drug which originally targeted the dry form of age-related macular degeneration (AMD) and works by slowing the buildup of toxic waste products that lead to retinal degeneration in a number of retinal conditions, including Stargardt disease. It is currently in Phase III trial development.


Astellas’s stem cell treatment (ASP7317) is designed to protect and regenerate photoreceptors in the retina that are damaged by eye diseases like Stargardt’s. It is currently in Phase II clinical trials and had been granted Orphan Drug Designation for Stargardt Disease. Other research suggests aggregation or “clumping” of vitamin A in the retina may be associated both with Stargardt’s disease and age-related macular degeneration (AMD). These clumpy deposits are known as “vitamin A dimers.“ Several pharmaceutical companies are developing drugs that target the problems associated with vitamin A dimers. For example,  Alkeus Pharmaceuticals’ ALK-001 is a modified form of vitamin A, which, when metabolized in the retina, results in much less waste. It is currently in Phase II clinical trials. ProQR is developing antisense oligonucleotides, or RNA therapies, to treat genetic diseases that are rare affecting the retina, called inherited retinal diseases, or IRDs. IRDs cause vision loss and blindness and the vast majority of these disorders currently have no treatment options. The company is developing QR-1011 for Stargardt disease against c.5461-10T>C mutation in ABCA4. The drug showed strong pre-clinical results and good efficacy in human minigene models and is being tested further. Gene replacement therapy is a logical means to halt and, possibly reverse, STGD. This therapy is based on introducing a subretinal injection of adeno-associated virus (AAV) vectors genetically engineered with ABCA4-DNA. Presently, there are 2 clinical trials investigating the use of StarGen, which is an equine infectious anemia virus lentiviral vector, in order to reduce A2E accumulation in STGD. The first is phase I/II clinical trial and has demonstrated safety. However, evidence of efficacy is limited in patients with severe disease. This trial had been terminated due to review of clinical development plans and priorities; sponsor decided to stop development of the product. The second trial is another ongoing safety and tolerance phase I/II clinical trial. Apart from the above mentioned companies, there are other few companies which have been focusing on the development of novel therapies for the treatment of Stargardt disease. Overall, a robust pipeline filled with novel therapies along with the rising prevalence of the disease will lead to a significant boost to the market revenue generated during the forecast period [2020–2030].


Emerging therapies included in the report

  • ASP7317: Astellas Pharma
  • QR-1011: ProQR


Stargardt Disease Report Scope

  • The Stargardt Disease (STGD) report provides an overview of therapeutic pipeline activity and therapeutic assessment of the products by development stage, product type, route of administration, molecule type, and MOA type for Stargardt Disease (STGD) across the complete product development cycle, including all clinical and nonclinical stages.
  • It comprises of detailed profiles of Stargardt Disease (STGD) therapeutic products with key coverage of developmental activities, including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product-related details
  • Detailed Stargardt Disease (STGD) research and development progress and trial details, results wherever available, are also included in the pipeline study.
  • Coverage of dormant and discontinued pipeline projects along with the reasons if available across Stargardt Disease (STGD)


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Table of content

1. Report Introduction

2. Stargardt Disease (STGD) 

3. Stargardt Disease (STGD) Current Treatment Patterns

4. Stargardt Disease (STGD) – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Stargardt Disease (STGD) Late Stage Products (Phase-III)

7. Stargardt Disease (STGD) Mid Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Stargardt Disease (STGD) Discontinued Products

13. Stargardt Disease (STGD) Product Profiles

14. Stargardt Disease (STGD) Key Companies

15. Stargardt Disease (STGD) Key Products

16. Dormant and Discontinued Products

17. Stargardt Disease (STGD) Unmet Needs

18. Stargardt Disease (STGD) Future Perspectives

19. Stargardt Disease (STGD) Analyst Review  

20. Appendix

21. Report Methodology


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