Retinitis Pigmentosa Pipeline Insights, 2020

Retinitis Pigmentosa Pipeline Insights, 2020

“Retinitis Pigmentosa Pipeline Insight, 2020” report by DelveInsight outlays comprehensive insights of present clinical development scenario and growth prospects across the Retinitis Pigmentosa (RP) market. A detailed picture of the Retinitis Pigmentosa pipeline landscape is provided, which includes the disease overview and Retinitis Pigmentosa treatment guidelines.

DelveInsight launched a new report on Retinitis Pigmentosa Pipeline Insights, 2020


Some of the key facts of the report:-

Horama is developing a gene therapy candidate HORA-RPE65 in Phase III for the treatment of Autosomal Recessive RP (Non syndromic) RPE65 RP. It is an AAV 2/4 encapsidating the human RPE65 gene under the control of the human native RPE65 promoter. The company’s investigational candidate HORA-RPE65 provides the cell with a non-mutated copy of the human RPE65 gene, which can express functional RPE65 protein to halt or at least significantly delay retinal degeneration in patients with inherited retinal dystrophies caused by RPE65 gene mutations.

Nightstar Therapeutics (acquired by Biogen) is developing a sub-retinal AAV-based Adeno-Associated Viral Vector Encoding RP GTPase Regulator gene therapy (AAV8-RPGR) in Phase II/III for Non syndromic X-linked Retinitis Pigmentosa (XLRP). The company’s NSR-RPGR gene therapy consists of a standard AAV8 vector, including the codon-optimized human RPGR DNA. The drug candidate has already demonstrated positive outcomes in the initial Phase of the Phase II/III XIRIUS trial.

jCyte is evaluating jCell human retinal progenitor cells in a Phase II trial in in Adult patients with RP. The company’s investigational therapy uses adult retinal progenitor cells to rescue rods and cones from RP progression and possibly generate new retinal cells. The treatment requires a single intravitreal injection, which can be performed in with topical anesthetic. jCyte have already completed a phase I/IIa study to determine the therapy’s safety. Furthermore, the drug received US FDA Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designation.

Neurotech is currently evaluating Renexus (NT-501) in a Phase II in Participants with Syndromic RP Using Rates of Change in Cone Spacing and Density. Renexus (NT-501) Consists of encapsulated human cells genetically modified to secrete therapeutic doses of ciliary neurotrophic factor in to the back of eye for the treatment of RP. The drug candidate was granted orphan drug and fast-track designation by the US FDA for RP.

ReNeuron is currently leading a Phase IIa trial for the assessment of proprietary cGMP-produced, Subretinal, allogeneic, cryopreserved human retinal progenitor cells (hrpc) cell. The company received Orphan Drug in EU and US & Fast Track Designation for in US, for hrpc, for the treatment of RP.


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Scope of the Report

  • The Retinitis Pigmentosa report provides an overview of therapeutic pipeline activity and therapeutic assessment of the products by development stage, product type, route of administration, molecule type, and MOA type for Retinitis Pigmentosa across the complete product development cycle, including all clinical and nonclinical stages.
  • It comprises of detailed profiles of Retinitis Pigmentosa therapeutic products with key coverage of developmental activities, including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product-related details
  • Detailed Retinitis Pigmentosa research and development progress and trial details, results wherever available, are also included in the pipeline study.
  • Coverage of dormant and discontinued pipeline projects along with the reasons if available across Retinitis Pigmentosa .



Report highlights

  • A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Retinitis Pigmentosa .    
  • In the coming years, the Retinitis Pigmentosa market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market. 
  • The companies and academics that are working to assess challenges and seek opportunities that could influence Retinitis Pigmentosa R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • A detailed portfolio of major pharma players who are involved in fueling the Retinitis Pigmentosa treatment market. Several potential therapies for Retinitis Pigmentosa are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Retinitis Pigmentosa market size in the coming years.  
  • Our in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Retinitis Pigmentosa ) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities. 



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 Table of contents

1. Report Introduction

2. Retinitis Pigmentosa

2.1. Overview

2.2. History 

2.3. Retinitis Pigmentosa Symptoms

2.4. Causes


2.6. Retinitis Pigmentosa Diagnosis 

2.6.1. Diagnostic Guidelines

3. Retinitis Pigmentosa Current Treatment Patterns

3.1. Retinitis Pigmentosa Treatment Guidelines

4. Retinitis Pigmentosa – DelveInsight’s Analytical Perspective

4.1. In-depth Commercial Assessment

4.1.1. Retinitis Pigmentosa companies collaborations, Licensing, Acquisition -Deal Value Trends Assessment Summary

4.1.2. Retinitis Pigmentosa Collaboration Deals Company-Company Collaborations (Licensing / Partnering) Analysis Company-University Collaborations (Licensing / Partnering) Analysis Retinitis Pigmentosa Acquisition Analysis

5. Therapeutic Assessment

5.1. Clinical Assessment of Pipeline Drugs 

5.1.1. Assessment by Phase of Development

5.1.2. Assessment by Product Type (Mono / Combination) Assessment by Stage and Product Type

5.1.3. Assessment by Route of Administration Assessment by Stage and Route of Administration

5.1.4. Assessment by Molecule Type Assessment by Stage and Molecule Type

5.1.5. Assessment by MOA Assessment by Stage and MOA

5.1.6. Assessment by Target Assessment by Stage and Target

6. Retinitis Pigmentosa Late Stage Products (Phase-III)

7. Retinitis Pigmentosa Mid Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Retinitis Pigmentosa Discontinued Products

13. Retinitis Pigmentosa Product Profiles

13.1. Drug Name: Company 

13.1.1. Product Description Product Overview Mechanism of action

13.1.2. Research and Development Clinical Studies

13.1.3. Product Development Activities Collaboration Agreements Acquisition Patent Detail

13.1.4. Tabulated Product Summary General Description Table

Detailed information in the report? 

14. Retinitis Pigmentosa Key Companies

15. Retinitis Pigmentosa Key Products

16. Dormant and Discontinued Products

16.1. Dormant Products

16.1.1. Reasons for being dormant

16.2. Discontinued Products 

16.2.1. Reasons for the discontinuation

17. Retinitis Pigmentosa Unmet Needs

18. Retinitis Pigmentosa Future Perspectives

19. Retinitis Pigmentosa Analyst Review  

20. Appendix

21. Report Methodology

21.1. Secondary Research

21.2. Expert Panel Validation 



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