Choroideremia - Pipeline Insight, 2025
Description
DelveInsight’s, Choroideremia - Pipeline Insight, 2025,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Choroideremia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Choroideremia (CHM) is an uncommon heredodystrophy with an estimated prevalence of 1 in 50,000 patients. This disorder mainly involves males because of its X-linked inheritance pattern1 and is dependent on mutations in the CHM gene. This gene is known to be related to membrane transportation protein in the retina and retinal pigment epithelium (RPE).
Choroideremia is an X-linked recessive inherited disorder due to mutation in the CHM gene (OMIM 303390), which is placed on chromosome X at position q21.2. CHM messenger RNA (mRNA) is responsible for the creation of the Rab escort protein (REP)-1 which is ubiquitously expressed. This protein has 653 amino acids and is involved in intracellular migration of organelles and molecules. Especially, REP-1 manages the process aimed at attaching the unprenylated Rab proteins to the geranyl-geranyl transferase 2 enzyme.
""Choroideremia - Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Choroideremia pipeline landscape is provided which includes the disease overview and Choroideremia treatment guidelines. The assessment part of the report embraces, in depth Choroideremia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Choroideremia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
AAV2-hCHM: Spark Therapeutics
Spark Therapeutics is advancing an open-label, dose-escalating Phase I/II trial designed to assess the safety and preliminary efficacy of subretinal administration of investigational SPK-7001. Each of the investigational research programs uses an adeno-associated viral (AAV) vector developed and manufactured by the Spark team and their collaborators.
Further product details are provided in the report……..
Choroideremia: Therapeutic Assessment
This segment of the report provides insights about the Choroideremia drugs segregated based on following parameters that define the scope of the report, such as:
Choroideremia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Choroideremia therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Choroideremia drugs.
Choroideremia Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Choroideremia (CHM) is an uncommon heredodystrophy with an estimated prevalence of 1 in 50,000 patients. This disorder mainly involves males because of its X-linked inheritance pattern1 and is dependent on mutations in the CHM gene. This gene is known to be related to membrane transportation protein in the retina and retinal pigment epithelium (RPE).
Choroideremia is an X-linked recessive inherited disorder due to mutation in the CHM gene (OMIM 303390), which is placed on chromosome X at position q21.2. CHM messenger RNA (mRNA) is responsible for the creation of the Rab escort protein (REP)-1 which is ubiquitously expressed. This protein has 653 amino acids and is involved in intracellular migration of organelles and molecules. Especially, REP-1 manages the process aimed at attaching the unprenylated Rab proteins to the geranyl-geranyl transferase 2 enzyme.
""Choroideremia - Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Choroideremia pipeline landscape is provided which includes the disease overview and Choroideremia treatment guidelines. The assessment part of the report embraces, in depth Choroideremia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Choroideremia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence R&D Choroideremia. The therapies under development are focused on novel approaches to treat/improve Choroideremia.
- In February 2019, Roche entered into a definitive merger agreement to acquire Spark Therapeutics and its clinical assets, including SPK-7001 for choroideremia in Phase I/II.
- 4D therapeutics is developing and investigating its lead product candidate 4D-110 in an ongoing Phase I dose-escalation clinical trial in patients with choroideremia related to mutations in the CHM gene.
AAV2-hCHM: Spark Therapeutics
Spark Therapeutics is advancing an open-label, dose-escalating Phase I/II trial designed to assess the safety and preliminary efficacy of subretinal administration of investigational SPK-7001. Each of the investigational research programs uses an adeno-associated viral (AAV) vector developed and manufactured by the Spark team and their collaborators.
Further product details are provided in the report……..
Choroideremia: Therapeutic Assessment
This segment of the report provides insights about the Choroideremia drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Choroideremia
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Intravenous
- Subcutaneous
- Molecule Type
- Small molecule
- Cell Therapy
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Choroideremia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Choroideremia therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Choroideremia drugs.
Choroideremia Report Insights
- Choroideremia Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Choroideremia drugs?
- How many Choroideremia drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Choroideremia?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Choroideremia therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Choroideremia and their status?
- What are the key designations that have been granted to the emerging drugs?
- Spark Therapeutics
- Molecular Therapeutics
- Curative Biotechnology
- Ray Therapeutics
- AAV2-hCHM
- 4D 110
- Metformin
- RAY 001
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Choroideremia: Overview
- What is Choroideremia?
- Types of Choroideremia
- Causes
- Epidemiology and Risk Factors
- Disease Management
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Choroideremia– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase I/II)
- Comparative Analysis
- AAV2-hCHM: Vaccitech
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- 4D 110: 4D Molecular Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical Stage Products
- Comparative Analysis
- RAY 001: Ray Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Choroideremia Key Companies
- Choroideremia Key Products
- Choroideremia- Unmet Needs
- Choroideremia- Market Drivers and Barriers
- Choroideremia- Future Perspectives and Conclusion
- Choroideremia Analyst Views
- Choroideremia Key Companies
- Appendix
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