Choroideremia Pipeline Insight

DelveInsight’s, "Choroideremia - Pipeline Insight, 2022,” 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

  • Global coverage

Choroideremia Understanding

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, 2022" 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.

Choroideremia Emerging Drugs

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

There are approx. 5+ key companies which are developing the therapies 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 & 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.

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

Choroideremia Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

Key Questions

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?

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

List of Table

Table 1: Total Products for Choroideremia

Table 2: Late Stage Products

Table 3: Mid Stage Products

Table 4: Early Stage Products

Table 5: Pre-clinical & Discovery Stage Products

Table 6: Assessment by Product Type

Table 7: Assessment by Stage and Product Type

Table 8: Assessment by Route of Administration

Table 9: Assessment by Stage and Route of Administration

Table 10: Assessment by Molecule Type

Table 11: Assessment by Stage and Molecule Type                                              

Table 12: Inactive Products

List of Figures

Figure 1: Total Products for Choroideremia

Figure 2: Late Stage Products                             

Figure 3: Mid Stage Products

Figure 4: Early Stage Products

Figure 5: Preclinical and Discovery Stage Products

Figure 6: Assessment by Product Type                                                                       

Figure 7: Assessment by Stage and Product Type

Figure 8: Assessment by Route of Administration

Figure 9: Assessment by Stage and Route of Administration

Figure 10: Assessment by Molecule Type

Figure 11: Assessment by Stage and Molecule Type

Figure 12: Inactive Products

• AAV2-hCHM
• 4D 110
• Metformin
• RAY 001

 

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