Choroideremia is a rare genetic eye disorder that causes progressive vision loss and eventual blindness, primarily in males. According to Niamh Wynne et al., 2024, choroideremia (CHM) is an X-linked inherited retinal degeneration with an estimated prevalence of 1 in 50,000 individuals. The current drug pipeline includes gene therapies and other targeted choroideremia therapeutic products aimed at preserving retinal function. As per the choroideremia pipeline analysis by Expert Market Research, the outlook appears highly promising.
Major companies involved in the choroideremia pipeline analysis include NightstaRx Ltd., Kiora Pharmaceuticals, Inc., and others.
Leading drugs currently in the pipeline include KIO-301, RTx-015, 4D-110, and others.
Advancements in gene therapy, increasing clinical trials of AAV-based vectors, and rising regulatory support are expected to drive significant growth in the choroideremia drug pipeline over the coming years.
The Choroideremia Pipeline Analysis Report by Expert Market Research gives comprehensive insights into choroideremia therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for choroideremia. The choroideremia report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The choroideremia pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with choroideremia treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to choroideremia.
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Choroideremia is a rare, inherited retinal disorder that causes progressive vision loss due to degeneration of the choroid, retinal pigment epithelium, and photoreceptors. It primarily affects males and is caused by mutations in the CHM gene, leading to gradual blindness, typically starting in childhood and progressing into adulthood.
Choroideremia treatment currently focuses on managing vision loss. Gene therapy shows promise by targeting the underlying genetic mutation. Although no cure exists, research suggests gene therapy could slow disease progression and preserve vision over time. In October 2023, a Phase 3 trial evaluated timrepigene emparvovec gene therapy for choroideremia. While it did not meet the FDA's primary efficacy endpoint, the study demonstrated vision improvement in some patients, supporting further research.
According to Niamh Wynne et al., 2024, choroideremia (CHM) is a rare X-linked retinal disorder with an incidence of approximately 1 in 50,000 individuals. As per Vision Center, symptoms typically begin with night blindness around age 10, followed by progressive peripheral vision loss during adolescence. The disease advances over decades, leading to tunnel vision, impaired color and depth perception, and eventually complete blindness by 50 to 70 years. Continuous research is driving advancements in targeted therapeutic development.
This section of the report covers the analysis of choroideremia drug candidates based on several segmentations, including:
By Phase
The pipeline assessment report covers 50+ drug analyses based on phase:
By Drug Class
The choroideremia pipeline analysis report covers 50+ drug analyses based on drug classes:
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration.
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase I accounts for 60% of the total choroideremia clinical trials, reflecting strong early-stage research activity. Phase II and III each hold a 20% share, showing steady progression toward advanced development. The promising distribution across phases highlights ongoing innovation, which is expected to significantly enhance treatment options and positively impact the choroideremia market landscape.
The drug molecule categories covered under the choroideremia pipeline analysis include small molecules, monoclonal antibodies, gene therapies, peptides, and polymer. The choroideremia report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for choroideremia. Photoswitch-based therapies are emerging as a promising drug class in the choroideremia treatment pipeline. For instance, KIO-301 by Kiora Pharmaceuticals is under clinical investigation. It acts by rendering retinal ganglion cells responsive to light, offering a potential therapeutic solution for patients with advanced retinal degeneration. This approach bypasses lost photoreceptors and may restore visual function through intravitreal administration, with preliminary trials indicating improved visual perception and safety.
The EMR report for the choroideremia pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed choroideremia therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in choroideremia clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for choroideremia. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of choroideremia drug candidates.
KIO-301 is currently undergoing a Phase I/II dose-escalating clinical trial, sponsored by Kiora Pharmaceuticals, Inc., to assess its safety, tolerability, and efficacy in patients with retinitis pigmentosa and choroideremia. The study is examining intravitreal administration of KIO-301, a molecular photoswitch designed to restore vision by reactivating dormant retinal cells. The objective of this phase is to evaluate its therapeutic potential in inherited retinal diseases. KIO-301 is demonstrating promising improvements in visual function and brain activity, supporting expanded clinical development.
RTx-015, developed by Ray Therapeutics, Inc., is currently undergoing evaluation in a Phase 1 clinical trial for patients with retinitis pigmentosa or choroideremia. The objective of this phase is to assess the safety and preliminary efficacy of a single, uniocular intravitreal injection of RTx-015. This investigational optogenetic gene therapy is targeting retinal ganglion cells to potentially restore vision. Approximately 18 patients are being enrolled across four escalating dose cohorts. Participants are receiving one injection and are being monitored for five years.
4D-110 is a recombinant adeno-associated virus (AAV) gene therapy currently being evaluated in a Phase 1 dose-escalation study and is sponsored by 4D Molecular Therapeutics. This study is assessing the safety, tolerability, and preliminary efficacy of a single intravitreal injection of 4D-110 in male patients with genetically confirmed Choroideremia. 4D-110 is delivering a functional CHM gene directly into retinal cells to potentially halt or reverse vision loss. The trial is focusing on three escalating dose levels and is aiming to determine the optimal therapeutic window while monitoring adverse effects.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Choroideremia Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for choroideremia. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into choroideremia collaborations, regulatory environments, and potential growth opportunities.
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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