DelveInsight’s, “Choroideremia – Pipeline Insight, 2023,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in the 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.
Choroideremia Key Highlights
- Key Companies working in the Choroideremia pipeline include Spark Therapeutics, Molecular Therapeutics, Curative Biotechnology, Ray Therapeutics, and many others
- Key Therapies included in the Choroideremia market include AAV2-hCHM and many others
Choroideremia Overview
Choroideremia is a rare, X-linked recessive form of hereditary retinal degeneration that affects roughly 1 in 50,000 males. The disease causes a gradual loss of vision, starting with childhood night blindness, followed by peripheral vision loss and progressing to loss of central vision later in life. Progression continues throughout the individual’s life, but both the rate of change and the degree of visual loss are variable among those affected, even within the same family.
Choroideremia is caused by a loss-of-function mutation in the CHM gene which encodes Rab escort protein 1 (REP1), a protein involved in the lipid modification of Rab proteins. While the complete mechanism of disease is not fully understood, the lack of a functional protein in the retina results in cell death and the gradual deterioration of the retinal pigment epithelium (RPE), photoreceptors, and the choroid.
Major Players in Choroideremia
There are approx. 5+ key companies are developing therapies for Choroideremia. The companies which have their Choroideremia drug candidates in the most advanced stage, i.e phase I/II include Spark Therapeutics
Phases
DelveInsight’s Report covers around 5+ products under different phases of clinical development like
- 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 and inactive candidates
Route of Administration
Choroideremia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Oral
- Intravenous
- Subcutaneous
Molecule Type
Products have been categorized under various Molecule types such as
- Small molecule
- Cell Therapy
- Peptides
- Polymer
- Small molecule
- Gene therapy
Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Key Companies Working in the Choroideremia Pipeline
- Spark Therapeutics
- Molecular Therapeutics
- Curative Biotechnology
And many others
Choroideremia Therapies Covered and Analyzed in the Report
- AAV2-hCHM
And many others
Learn more about the Key Companies and Emerging Therapies in the Choroideremia Pipeline
Table of Contents
- Key Insights
- Choroideremia Introduction
- Executive Summary of Choroideremia
- Disease Background and Overview
- Epidemiology and patient population
- Choroideremia Emerging Therapies
- Choroideremia Market Outlook
- Market Access and Reimbursement of Therapies
- Market Drivers
- Market Barriers
- Appendix
- Report Methodology
- DelveInsight Capabilities
- Disclaimer
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