DelveInsight’s Choroideremia pipeline report provides comprehensive insights into the Choroideremia market’s current clinical development scenario and growth prospects. The report provides a detailed description of the drug, including the drug’s mechanism of action, clinical studies, NDA approvals, and product development activities involving the technology.
Key Takeaways from the Choroideremia Pipeline Report
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The Choroideremia pipeline comprises emerging therapies in the different clinical stages, including BIIB111, SPK-7001, 4D-110, Metformin, and others.
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Key companies working to improve the Choroideremia treatment landscape include NightstaRx Ltd, Spark Therapeutics, 4D Molecular Therapeutics, Curative Biotech, among others.
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The Choroideremia pipeline therapies in the late stages of development include BIIB111 (NightstaRx Ltd), SPK-7001 (Spark Therapeutics), and others.
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4D-110 is being developed and investigated in the phase I Choroideremia clinical trials by 4D Molecular Therapeutics.
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Metformin (Curative Biotech) is still in the early phase of development, i.e., preclinical stage for Choroideremia.
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Choroideremia Overview
Choroideremia is a rare genetic vision disorder that affects more men than women. It is an X-linked recessive condition that causes the choroid, retinal pigment epithelium (RPE), and photoreceptors to degrade gradually. Females are the majority of carriers and are mostly unaffected, though they may experience minor symptoms such as blurred vision later in life.
Choroideremia Symptoms
The disease usually manifests itself in childhood, around the age of five or six. The child develops night blindness, or the inability to see objects in dim lighting, followed by loss of peripheral vision due to choroid blood vessel degeneration. Loss of color vision as a result of macula degeneration is another common symptom.
Choroideremia Treatment
Choroideremia currently has no treatment or cure. However, depending on the intensity of the symptoms, the patient may be offered symptom-specific treatment. However, because Choroideremia is a genetic condition, gene therapy and small molecule medications may be viable treatment choices for Choroideremia patients.
Choroideremia Pipeline Analysis: Drug Profiles
BIIB111: NightstaRx Ltd
Product Description
NightstaRx Ltd developed BIIB111 for the treatment of Choroideremia. BIIB111 is a gene therapy treatment consisting of an AAV2 vector with recombinant human DNA meant to deliver REP1 protein to the retina.
Phase III
NCT03496012: NightstaRx Ltd. began a Phase III trial of retinal gene therapy for Choroideremia using an Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1) in December 2017.
Choroideremia Pipeline Therapies and Key Companies
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BIIB111: NightstaRx Ltd
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SPK-7001: Spark Therapeutics
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4D-110: 4D Molecular Therapeutics
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Metformin: Curative Biotech
Find out more about the emerging drugs @ Choroideremia Emerging Therapies and Companies
Choroideremia Therapeutics Assessment
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By Product Type
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Monotherapy
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Combination Therapy
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By Stage
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Discovery
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Pre-Clinical
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Phase I
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Phase II
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Phase III
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Pre-registration
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By Route of Administration
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Inhalation
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Intravenous
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Oral
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Subcutaneous
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By Molecule Type
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Small Molecule
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Stem Cell Therapy
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Gene Therapy
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Targets
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Immune System
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Multiple Kinase
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By Mechanism of Action
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Protease Inhibitors
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Immunomodulatory
Scope of the Report
Coverage: Global
Choroideremia Key Players: NightstaRx Ltd, Spark Therapeutics, 4D Molecular Therapeutics, Curative Biotech, among others
Choroideremia Pipeline Therapies: BIIB111, SPK-7001, 4D-110, Metformin, and others
Table of Contents
1. |
Introduction |
2. |
Executive Summary |
3. |
Choroideremia: Overview |
4. |
Choroideremia Analytical Perspective In-depth Commercial Assessment |
5. |
Choroideremia Pipeline Therapeutics |
6. |
Choroideremia Late Stage Products (Phase III) |
7. |
Choroideremia Mid Stage Products (Phase II) |
8. |
Choroideremia Early Stage Products (Phase I & I/II) |
9. |
Choroideremia Preclinical Products |
10. |
Choroideremia Therapeutic Assessment |
11. |
Choroideremia Inactive Products |
12. |
Choroideremia Company-University Collaborations (Licensing/Partnering) Analysis |
13. |
Choroideremia Key Companies |
14. |
Choroideremia Key Products |
15. |
Choroideremia Unmet Needs |
16. |
Choroideremia Market Drivers and Barriers |
17. |
Choroideremia Future Perspectives and Conclusion |
18. |
Choroideremia Analyst Views |
19. |
Appendix |
20. |
About DelveInsight |
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Key questions answered in the Choroideremia Pipeline Report
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What are the therapy options for Choroideremia?
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How many pharmaceutical companies are working on therapies for Choroideremia?
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Which of these firms’ medications are the most popular?
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What is the total number of Choroideremia drugs produced by each company?
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How many Choroideremia medicines are currently in the early, mid, or late stages of development?
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How many of the therapies now in development can be used alone or in conjunction with others?
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What are the most crucial industry-industry and industry-academy cooperation, mergers and acquisitions, and licensing strategies that impact Choroideremia?
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