Drug Development of Choroideremia Pipeline Report Analysis | DelveInsight

The need for a new therapeutic option is acute. Clinical research is underway investigating the gene therapies for the effective treatment of Choroideremia.


Los Angeles, USA, Sept. 08, 2021 (GLOBE NEWSWIRE) -- Drug Development of Choroideremia Pipeline Report Analysis | DelveInsight

The need for a new therapeutic option is acute. Clinical research is underway investigating the gene therapies for the effective treatment of Choroideremia.

DelveInsight’s “Choroideremia Pipeline Insight” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in the Choroideremia pipeline landscapes. It comprises Choroideremia pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Choroideremia therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Choroideremia pipeline products.     

Some of the key takeaways from the Choroideremia Pipeline Report  

  • Major companies such as NightstaRx Ltd/Biogen, 4D Molecular Therapeutics, Curative Biotech, Therapeutics, Spark therapeutics/Roche, and others are developing potential drug candidates to improve the Choroideremia treatment scenario. 
  •  In June 2021, NightstaRx Ltd announced that the results from Phase III did not satisfy the primary endpoint and secondary efficacy endpoints. Therefore, they will evaluate the results before confirming the next step of the study.
  • 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.
  • 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.

Get an overview of pipeline landscape @ Choroideremia Clinical Trials Analysis 

Choroideremia is a rare genetic disorder of vision that is more common in men. It is an X-linked recessive condition that gradually degrades the choroid, retinal pigment epithelium (RPE), and photoreceptors.

Choroideremia Emerging Drugs

  • BIIB111: NightstaRx Ltd

NightstaRx Ltd initiated the development of BIIB111 for the treatment of Choroideremia. BIIB111 is a gene therapy-based treatment consisting of AAV2 vectors with recombinant human DNA designed to deliver REP1 protein to the retina.

  • 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.

For further information, refer to the detailed report @ Choroideremia Pipeline Therapeutics 

Scope of Choroideremia Pipeline Drug Insight    

  • Coverage: Global 
  • Major Players: 4+ Key Players
  • Prominent Players: NightstaRx Ltd/Biogen, Molecular Therapeutics, Curative Biotech, Spark Therapeutics/Roche, and many others.   
  • Key Drugs Profiles: 4+ Products
  • Phases:  

·       Choroideremia Therapies Late-stage (Phase III)  
·       Choroideremia Therapies Mid-stage (Phase II)
·       Choroideremia Therapies Early-stage (Phase I) 
·       Choroideremia Preclinical stage and Discovery candidates     
·       Discontinued and Inactive candidates  

  • Mechanism of Action:

·       Gene transference
·     CHM protein expression stimulants; Gene transference
·     Gluconeogenesis inhibitors
·     CHM protein expression stimulants; Gene transference       

  • Molecule Types:   

·     Peptides
·     Monoclonal antibodies
·    Small molecules 
·    Polymer 
·    Gene therapy

  • Route of Administration:

·     Parenteral 
·      Intravitreal
·      Oral
·      Subretinal

  • Product Types:

·       Monotherapy
·       Combination
·       Mono/Combination 

Key Questions regarding Current Choroideremia Treatment Landscape and Emerging Therapies Answered in the Pipeline Report  

  • What are the current options for Choroideremia treatment?
  • How many companies are developing therapies for the treatment of Choroideremia? 
  • How many are Choroideremia emerging therapies in the early-stage, mid-stage, and late development stages to treat Choroideremia? 
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and significant licensing activities that will impact the Choroideremia market? 
  • Which are the dormant and discontinued products and the reasons for the same?
  • What is the unmet need for current therapies for the treatment of Choroideremia?  
  • What are the current novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing Choroideremia therapies? 
  • What are the critical designations that have been granted for the emerging therapies for Choroideremia? 
  • How many patents are granted and pending for the emerging therapies to treat Choroideremia?   

Table of Contents

1Choroideremia Report Introduction
2Choroideremia Executive Summary
3Choroideremia Overview
4Choroideremia Pipeline Therapeutics
5Choroideremia Therapeutic Assessment
6Choroideremia – DelveInsight’s Analytical Perspective In-depth Commercial Assessment
7Choroideremia Late Stage Products (Phase III)  
7.1 BIIB111: NightstaRx Ltd/Biogen
8Choroideremia Mid Stage Products (Phase II)
9Choroideremia Early Stage Products (Phase I & I/II)
9.14D-110: 4D Molecular Therapeutics
10Choroideremia Preclinical and Discovery Stage Products
10.1Metformin: Curative Biotech
11Choroideremia Inactive Products
12Choroideremia Key Companies
13Choroideremia Key Products
14Choroideremia Unmet Needs
15Choroideremia Market Drivers and Barriers
16Choroideremia Future Perspectives and Conclusion
17Choroideremia Analyst Views
18 Appendix
19About DelveInsight

Get a customized pipeline report @ Choroideremia Drugs Pipeline Report  

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