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Retinitis Pigmentosa Therapeutics – Pipeline Analysis

Retinitis Pigmentosa Therapeutics – Pipeline Analysis, Epidemiology and Drug Forecasting

Report Code: PP10249 Published: April 2020 Pages: 340 Available format: 
Therapeutic Area(s): Ophthalmology Report Type: Indication Pipeline Reports
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Chapter 1. Research Background

1.1 Research Objectives

1.2 Definition

1.3 Research Scope

1.3.1 Pipeline Drugs by Phase

1.3.2 Pipeline and Marketed Drugs by Treatment

1.3.3 Pipeline and Marketed Drugs by Therapy Type

1.4 Key Stakeholders

Chapter 2. Research Methodology

2.1 Secondary Research

2.2 Primary Research

2.2.1 Breakdown of Primary Research Respondents

2.2.1.1 By industry participant

2.2.1.2 By geography

2.2.1.3 By designation

2.2.2 Key Opinion Leaders (KOLs) Validation

2.3 Notes and Caveats

Chapter 3. Executive Summary

Chapter 4. Introduction

4.1 Diseases Overview

4.2 Signs and Symptoms of RP

4.3 Classification of RP

4.4 Pathophysiology of RP

4.4.1 Genetic Basis of RP

4.4.2 Types of RP inheritance

4.5 Diagnosis of RP

4.5.1 Recommended Diagnostic Tests

4.5.2 Differential Diagnosis of RP

4.6 Treatment of RP

4.6.1 General Recommendations

4.6.2 Current Treatment Modalities

4.6.3 Treatment Guidelines by American Association for Ophthalmology

4.6.4 Future Treatment Modalities

4.7 Unmet Medical Needs

4.7.1 Lack of Established Curative Treatment Regimen

4.7.2 Need of More Eloquent Diagnostic Measures

4.7.3 Awareness and Education of the Disease

4.7.4 Need of Rehabilitative Management

4.7.5 Lack of Epidemiology Data

4.8 Drug Designations

4.9 Pipeline Drugs Snapshot by Company

4.10 Key Drivers

4.10.1 Extensive Pipeline of RP Therapeutics

4.10.2 Market Share Potential for Upcoming Therapies

4.10.3 Novel Therapies for Various Targets

4.10.4 Impact Analysis of Drivers on Market Forecast

4.11 Key Barriers

4.11.1 Late Delivery of Genes

4.11.2 Cost Ineffectiveness of Gene Therapy

4.11.3 Genetic Heterogeneity in RP Patients

4.11.4 Variation in Clinical Presentation of RP

4.11.5 Impact Analysis of Barriers on Market Forecast

Chapter 5. Epidemiology of RP

5.1 Epidemiology of RP in the 7MM

5.1.1 Total Prevalence of RP

5.1.2 Prevalence of RP, By Gender

5.1.3 Prevalence of RP, By Age-Group

5.1.4 Prevalence of RP, By Type

5.1.4.1 Prevalence of syndromic RP, by subtype

5.1.5 Prevalence of RP, by Genetic Inheritance Pattern

5.1.6 Prevalence of RP, by Gene Mutation

5.1.7 Prevalence of RP, by Region

5.2 Epidemiology of RP in the U.S.

5.2.1 Rationale

5.2.2 Total Prevalence of RP

5.2.3 Prevalence of RP, By Gender

5.2.4 Prevalence of RP, By Age-Group

5.2.5 Prevalence of RP, By Type

5.2.5.1 Prevalence of syndromic RP, by subtype

5.2.6 Prevalence of RP, by Genetic Inheritance Pattern

5.2.7 Prevalence of RP, by Gene Mutation

5.3 EU-5

5.3.1 Total Prevalence of RP

5.3.2 Total Prevalence of RP, by Country

5.3.2.1 Germany

5.3.2.1.1 Rationale

5.3.2.1.2 Total Prevalence of RP

5.3.2.1.3 Prevalence of RP, by gender

5.3.2.1.4 Prevalence of RP, by age-group

5.3.2.1.5 Prevalence of RP, by type

5.3.2.1.5.1 Prevalence of syndromic RP, by subtype

5.3.2.1.6 Prevalence of RP, by genetic inheritance pattern

5.3.2.1.7 Prevalence of RP, by gene mutation

5.3.2.2 France

5.3.2.2.1 Rationale

5.3.2.2.2 Total prevalence of RP

5.3.2.2.3 Prevalence of RP, by gender

5.3.2.2.4 Prevalence of RP, by age-group

5.3.2.2.5 Prevalence of RP, by type

5.3.2.2.5.1 Prevalence of syndromic RP, by subtype

5.3.2.2.6 Prevalence of RP, by genetic inheritance pattern

5.3.2.2.7 Prevalence of RP, by gene mutation

5.3.2.3 Italy

5.3.2.3.1 Rationale

5.3.2.3.2 Total prevalence of RP

5.3.2.3.3 Prevalence of RP, by gender

5.3.2.3.4 Prevalence of RP, by age-group

5.3.2.3.5 Prevalence of RP, by type

5.3.2.3.5.1 Prevalence of syndromic RP, by subtype

5.3.2.3.6 Prevalence of RP, by genetic inheritance pattern

5.3.2.3.7 Prevalence of RP, by gene mutation

5.3.2.4 Spain

5.3.2.4.1 Rationale

5.3.2.4.2 Total prevalence of RP

5.3.2.4.3 Prevalence of RP, by gender

5.3.2.4.4 Prevalence of RP, by age-group

5.3.2.4.5 Prevalence of RP, by type

5.3.2.4.5.1 Prevalence of syndromic RP, by subtype

5.3.2.4.6 Prevalence of RP, by genetic inheritance pattern

5.3.2.4.7 Prevalence of RP, by gene mutation

5.3.2.5 U.K.

5.3.2.5.1 Rationale

5.3.2.5.2 Total prevalence of RP

5.3.2.5.3 Prevalence of RP, by gender

5.3.2.5.4 Prevalence of RP, by age-group

5.3.2.5.5 Prevalence of RP, by type

5.3.2.5.5.1 Prevalence of syndromic RP, by subtype

5.3.2.5.6 Prevalence of RP, by genetic inheritance pattern

5.3.2.5.7 Prevalence of RP, by gene mutation

5.4 Japan

5.4.1 Rationale

5.4.2 Total Prevalence of RP

5.4.3 Prevalence of RP, By Gender

5.4.4 Prevalence of RP, By Age-Group

5.4.5 Prevalence of RP, By Type

5.4.5.1 Prevalence of syndromic RP, by subtype

5.4.6 Prevalence of RP, by Genetic Inheritance Pattern

5.4.7 Prevalence of RP, by Gene Mutation

Chapter 6. RP Marketed Drugs Analysis

6.1 Marketed Drugs

6.1.1 Luxturna

6.1.1.1 Dosage

6.1.1.2 Regulatory milestones

6.1.1.3 Clinical trials and results

6.1.1.4 Approval

6.1.1.5 Designation

6.1.1.6 NDA filling

6.1.1.7 Strategic development activities

6.1.1.8 Patent

6.1.1.9 Other developments

Chapter 7. RP Pipeline Analysis

7.1 Phase III

7.1.1 HORA-RPE65

7.1.1.1 Clinical trial and results

7.1.1.2 Technology

7.1.1.3 Designation

7.1.1.4 Name change

7.1.1.5 Spin-off

7.2 Phase II/III

7.2.1 AAV8-RPGR

7.2.1.1 Clinical trials

7.2.1.2 Clinical trial results

7.2.1.3 Designation

7.2.1.4 Acquisition

7.2.1.5 Spin-off

7.2.1.6 Financing

7.2.1.7 Collaboration

7.2.1.8 Patent

7.3 Phase II

7.3.1 jCell

7.3.1.1 Clinical trials

7.3.1.2 Clinical trial results

7.3.1.3 Designation

7.3.1.4 Financing

7.3.2 Xxxx

7.3.2.1 Clinical trials

7.3.2.2 Clinical trial results

7.3.2.3 Strategic development activities

7.3.2.4 Designation

7.3.3 Xxxx

7.3.3.1 Clinical trials

7.3.3.2 Clinical trial results

7.3.3.3 Designation

7.3.3.4 Acquisition

7.4 Phase I/II

7.4.1 ReN003

7.4.1.1 Clinical trials

7.4.1.2 Clinical trial results

7.4.1.3 Designation

7.4.1.4 Grant

7.4.1.5 Strategic development activities

7.4.1.6 Technology

7.4.1.7 Patent

7.4.2 Xxxx

7.4.2.1 Clinical trials

7.4.2.2 Designations

7.4.2.3 IND application

7.4.2.4 Financing

7.4.2.5 Strategic development activities

7.4.2.6 Technology

7.4.3 Xxxx

7.4.3.1 Clinical trials

7.4.3.2 Designation

7.4.3.3 Strategic development activities

7.4.3.4 Financing

7.4.3.5 Patent

7.4.4 Xxxx

7.4.4.1 Clinical trials

7.4.4.2 Clinical trial results

7.4.4.3 Designation

7.4.4.4 Name change

7.4.4.5 Financing

7.4.5 Xxxx

7.4.5.1 Pre-clinical studies

7.4.5.2 Clinical trials

7.4.6 Xxxx

7.4.6.1 Pre-clinical studies

7.4.6.2 Clinical trials

7.4.6.3 Clinical trial results

7.4.6.4 Designation

7.4.6.5 Financing

7.4.6.6 Strategic development activities

7.4.7 Xxxx

7.4.7.1 Pre-clinical studies

7.4.7.2 Clinical trials

7.4.7.3 Clinical trial results

7.4.7.4 Strategic development activities

7.4.7.5 Designations

7.4.8 Xxxx

7.4.8.1 Clinical trials

7.4.8.2 Strategic development activities

7.4.8.3 Designations

7.4.9 Xxxx

7.4.9.1 Clinical trials

7.4.9.2 Designation

7.4.9.3 Strategic development activities

7.4.10 Xxxx

7.4.10.1 Clinical trials

7.4.10.2 Name change

7.4.10.3 Strategic development activities

7.4.11 Xxxx

7.4.11.1 Clinical trials

7.4.11.2 Strategic development activities

7.4.11.3 Designations

7.4.12 Xxxx

7.4.12.1 Clinical studies

7.4.12.2 Clinical trial results

7.4.12.3 Designations

7.4.13 Xxxx

7.4.13.1 Strategic Development

Chapter 8. Regulatory Framework for Drug Approval

8.1 U.S.

8.2 Europe

8.2.1 Centralized Procedure

8.2.2 Mutual Recognition Procedure

8.2.3 Decentralized Procedure

8.2.4 Nationalized Procedure

8.3 Japan

Chapter 9. Competitive Landscape

9.1 Key Cross Competition of Pipeline Drugs

9.2 Market Share of Potential Therapies in 2030

9.3 Expected Launch Time of Late Clinical Stage Products

9.4 SWOT Analysis

Chapter 10. RP Market Size Analysis

10.1 Market Revenue of RP in the 7MM

10.1.1 Total Market Revenue of RP

10.1.2 Market Revenue of RP in the 7MM, By Treatment

10.1.3 Market Revenue of RP in the 7MM, by Therapy Type

10.1.4 Market Revenue of RP in the 7MM, by Region

10.2 Market Revenue of RP in the U.S.

10.2.1 Market Outlook

10.2.2 Total Market Revenue of RP

10.2.3 Market Revenue of RP, By Treatment

10.2.4 Market Revenue of RP, by Therapy Type

10.3 Market Revenue of RP in EU-5 Countries

10.3.1 Market Outlook

10.3.2 Total Market Revenue of RP

10.3.3 Market Revenue of RP, by Country

10.3.3.1 Germany

10.3.3.1.1 Total Market Revenue of RP

10.3.3.1.2 Market revenue of RP, by treatment

10.3.3.1.3 Market revenue of RP, by therapy type

10.3.3.2 France

10.3.3.2.1 Total market revenue of RP

10.3.3.2.2 Market revenue of RP, by treatment

10.3.3.2.3 Market revenue of RP, by therapy type

10.3.3.3 Italy

10.3.3.3.1 Total Market Revenue of RP

10.3.3.3.2 Market revenue of RP, by treatment

10.3.3.3.3 Market revenue of RP, by therapy type

10.3.3.4 Spain

10.3.3.4.1 Total market revenue of RP

10.3.3.4.2 Market revenue of RP, by treatment

10.3.3.4.3 Market revenue of RP, by therapy type

10.3.3.5 U.K.

10.3.3.5.1 Total market revenue of RP

10.3.3.5.2 Market revenue of RP, by treatment

10.3.3.5.3 Market revenue of RP, by therapy type

10.4 Market Revenue of RP in Japan

10.4.1 Market Outlook

10.4.2 Total Market Revenue of RP

10.4.3 Market Revenue of RP, By Treatment

10.4.4 Market Revenue of RP, by Therapy Type

Chapter 11 COMPANY PROFILES

11.1 Spark Therapeutics Inc.

11.1.1 Business Overview

11.1.2 Product and Service Offerings

11.2 HORAMA S.A.

11.2.1 Business Overview

11.2.2 Product and Service Offerings

11.3 Xxxx

11.3.1 Business Overview

11.3.2 Product and Service Offerings

11.4 Xxxx

11.4.1 Business Overview

11.4.2 Product and Service Offerings

11.4.3 Key Financial Summary

11.5 Xxxx

11.5.1 Business Overview

11.5.2 Product and Service Offerings

11.5.3 Key Financial Summary

11.6 Xxxx.

11.6.1 Business Overview

11.6.2 Product and Service Offerings

11.6.3 Key Financial Summary

11.7 Xxxx

11.7.1 Business Overview

11.7.2 Product and Service Offerings

11.8 Xxxx

11.8.1 Business Overview

11.8.2 Product and Service Offerings

11.8.3 Key Financial Summary

11.9 Xxxx

11.9.1 Business Overview

11.9.2 Product and Service Offerings

11.9.3 Key Financial Summary

Chapter 12. Appendix

12.1 Sources & References

12.2 Abbreviations

12.3 Related Reports

 

LIST OF TABLES

 

TABLE 1 SUMMARY OF GENES IMPLICATED IN RP

TABLE 2 DRIVERS FOR THE MARKET: IMPACT ANALYSIS

TABLE 3 BARRIERS FOR THE MARKET: IMPACT ANALYSIS

TABLE 4 TOTAL PREVALENT CASES OF RP IN THE 7MM (2014–2019)

TABLE 5 TOTAL PREVALENT CASES OF RP IN THE 7MM (2020–2030)

TABLE 6 PREVALENCE OF RP IN THE 7MM, BY GENDER (2014–2019)

TABLE 7 PREVALENCE OF RP IN THE 7MM, BY GENDER (2020–2030)

TABLE 8 PREVALENCE OF RP IN THE 7MM, BY AGE-GROUP (2014–2019)

TABLE 9 PREVALENCE OF RP IN THE 7MM, BY AGE-GROUP (2020–2030)

TABLE 10 PREVALENCE OF RP IN THE 7MM, BY TYPE (2014–2019)

TABLE 11 PREVALENCE OF RP IN THE 7MM, BY TYPE (2020–2030)

TABLE 12 PREVALENCE OF SYNDROMIC RP IN THE 7MM, BY SUBTYPE (2014–2019)

TABLE 13 PREVALENCE OF SYNDROMIC RP IN THE 7MM, BY SUBTYPE (2020–2030)

TABLE 14 PREVALENCE OF RP IN THE 7MM, BY GENETIC INHERITANCE PATTERN (2014–2019)

TABLE 15 PREVALENCE OF RP IN THE 7MM, BY GENETIC INHERITANCE PATTERN (2020–2030)

TABLE 16 PREVALENCE OF RP IN 7MM, BY GENE MUTATION (2014–2019)

TABLE 17 PREVALENCE OF RP IN 7MM, BY GENE MUTATION (2020–2030)

TABLE 18 PREVALENCE OF RP IN THE 7MM, BY REGION (2014–2019)

TABLE 19 PREVALENCE OF RP IN THE 7MM, BY REGION (2020–2030)

TABLE 20 TOTAL PREVALENT CASES OF RP IN THE U.S. (2014–2019)

TABLE 21 TOTAL PREVALENT CASES OF RP IN THE U.S. (2020–2030)

TABLE 22 PREVALENCE OF RP IN THE U.S., BY GENDER (2014–2019)

TABLE 23 PREVALENCE OF RP IN THE U.S., BY GENDER (2020–2030)

TABLE 24 PREVALENCE OF RP IN THE U.S., BY AGE-GROUP (2014–2019)

TABLE 25 PREVALENCE OF RP IN THE U.S., BY AGE-GROUP (2020–2030)

TABLE 26 PREVALENCE OF RP IN THE U.S., BY TYPE (2014–2019)

TABLE 27 PREVALENCE OF RP IN THE U.S., BY TYPE (2020–2030)

TABLE 28 PREVALENCE OF SYNDROMIC RP IN THE U.S., BY SUBTYPE (2014–2019)

TABLE 29 PREVALENCE OF SYNDROMIC RP IN THE U.S., BY SUBTYPE (2020–2030)

TABLE 30 PREVALENCE OF RP IN THE U.S., BY GENETIC INHERITANCE PATTERN (2014–2019)

TABLE 31 PREVALENCE OF RP IN THE U.S., BY GENETIC INHERITANCE PATTERN (2020–2030)

TABLE 32 PREVALENCE OF RP IN U.S., BY GENE MUTATION (2014–2019)

TABLE 33 PREVALENCE OF RP IN U.S., BY GENE MUTATION (2020–2030)

TABLE 34 TOTAL PREVALENT CASES OF RP IN THE EU-5 COUNTRIES (2014–2019)

TABLE 35 TOTAL PREVALENT CASES OF RP IN THE EU-5 COUNTRIES (2020–2030)

TABLE 36 PREVALENCE OF RP IN EU-5 (2014–2019)

TABLE 37 PREVALENCE OF RP IN EU-5 (2020–2030)

TABLE 38 TOTAL PREVALENT CASES OF RP IN GERMANY (2014–2019)

TABLE 39 TOTAL PREVALENT CASES OF RP IN GERMANY (2020–2030)

TABLE 40 PREVALENCE OF RP IN GERMANY, BY GENDER (2014–2019)

TABLE 41 PREVALENCE OF RP IN GERMANY, BY GENDER (2020–2030)

TABLE 42 PREVALENCE OF RP IN GERMANY, BY AGE-GROUP (2014–2019)

TABLE 43 PREVALENCE OF RP IN GERMANY, BY AGE-GROUP (2020–2030)

TABLE 44 PREVALENCE OF RP IN GERMANY, BY TYPE (2014–2019)

TABLE 45 PREVALENCE OF RP IN GERMANY, BY TYPE (2020–2030)

TABLE 46 PREVALENCE OF SYNDROMIC RP IN GERMANY, BY SUBTYPE (2014–2019)

TABLE 47 PREVALENCE OF SYNDROMIC RP IN GERMANY, BY SUBTYPE (2020–2030)

TABLE 48 PREVALENCE OF RP IN GERMANY, BY GENETIC INHERITANCE PATTERN (2014–2019)

TABLE 49 PREVALENCE OF RP IN GERMANY, BY GENETIC INHERITANCE PATTERN (2020–2030)

TABLE 50 PREVALENCE OF RP IN GERMANY, BY GENE MUTATION (2014–2019)

TABLE 51 PREVALENCE OF RP IN GERMANY, BY GENE MUTATION (2020–2030)

TABLE 52 TOTAL PREVALENT CASES OF RP IN FRANCE (2014–2019)

TABLE 53 TOTAL PREVALENT CASES OF RP IN FRANCE (2020–2030)

TABLE 54 PREVALENCE OF RP IN FRANCE, BY GENDER (2014–2019)

TABLE 55 PREVALENCE OF RP IN FRANCE, BY GENDER (2020–2030)

TABLE 56 PREVALENCE OF RP IN FRANCE, BY AGE-GROUP (2014–2019)

TABLE 57 PREVALENCE OF RP IN FRANCE, BY AGE-GROUP (2020–2030)

TABLE 58 PREVALENCE OF RP IN FRANCE, BY TYPE (2014–2019)

TABLE 59 PREVALENCE OF RP IN FRANCE, BY TYPE (2020–2030)

TABLE 60 PREVALENCE OF SYNDROMIC RP IN FRANCE, BY SUBTYPE (2014–2019)

TABLE 61 PREVALENCE OF SYNDROMIC RP IN FRANCE, BY SUBTYPE (2020–2030)

TABLE 62 PREVALENCE OF RP IN FRANCE, BY GENETIC INHERITANCE PATTERN (2014–2019)

TABLE 63 PREVALENCE OF RP IN FRANCE, BY GENETIC INHERITANCE PATTERN (2020–2030)

TABLE 64 PREVALENCE OF RP IN FRANCE, BY GENE MUTATION (2014–2019)

TABLE 65 PREVALENCE OF RP IN FRANCE, BY GENE MUTATION (2020–2030)

TABLE 66 TOTAL PREVALENT CASES OF RP IN ITALY (2014–2019)

TABLE 67 TOTAL PREVALENT CASES OF RP IN ITALY (2020–2030)

TABLE 68 PREVALENCE OF RP IN ITALY, BY GENDER (2014–2019)

TABLE 69 PREVALENCE OF RP IN ITALY, BY GENDER (2020–2030)

TABLE 70 PREVALENCE OF RP IN ITALY, BY AGE-GROUP (2014–2019)

TABLE 71 PREVALENCE OF RP IN ITALY, BY AGE-GROUP (2020–2030)

TABLE 72 PREVALENCE OF RP IN ITALY, BY TYPE (2014–2019)

TABLE 73 PREVALENCE OF RP IN ITALY, BY TYPE (2020–2030)

TABLE 74 PREVALENCE OF SYNDROMIC RP IN ITALY, BY SUBTYPE (2014–2019)

TABLE 75 PREVALENCE OF SYNDROMIC RP IN ITALY, BY SUBTYPE (2020–2030)

TABLE 76 PREVALENCE OF RP IN ITALY, BY GENETIC INHERITANCE PATTERN (2014–2019)

TABLE 77 PREVALENCE OF RP IN ITALY, BY GENETIC INHERITANCE PATTERN (2020–2030)

TABLE 78 PREVALENCE OF RP IN ITALY, BY GENE MUTATION (2014–2019)

TABLE 79 PREVALENCE OF RP IN ITALY, BY GENE MUTATION (2020–2030)

TABLE 80 TOTAL PREVALENT CASES OF RP IN SPAIN (2014–2019)

TABLE 81 TOTAL PREVALENT CASES OF RP IN SPAIN (2020–2030)

TABLE 82 PREVALENCE OF RP IN SPAIN, BY GENDER (2014–2019)

TABLE 83 PREVALENCE OF RP IN SPAIN, BY GENDER (2020–2030)

TABLE 84 PREVALENCE OF RP IN SPAIN, BY AGE-GROUP (2014–2019)

TABLE 85 PREVALENCE OF RP IN SPAIN, BY AGE-GROUP (2020–2030)

TABLE 86 PREVALENCE OF RP IN SPAIN, BY TYPE (2014–2019)

TABLE 87 PREVALENCE OF RP IN SPAIN, BY TYPE (2020–2030)

TABLE 88 PREVALENCE OF SYNDROMIC RP IN SPAIN, BY SUBTYPE (2014–2019)

TABLE 89 PREVALENCE OF SYNDROMIC RP IN SPAIN, BY SUBTYPE (2020–2030)

TABLE 90 PREVALENCE OF RP IN SPAIN, BY GENETIC INHERITANCE PATTERN (2014–2019)

TABLE 91 PREVALENCE OF RP IN SPAIN, BY GENETIC INHERITANCE PATTERN (2020–2030)

TABLE 92 PREVALENCE OF RP IN SPAIN, BY GENE MUTATION (2014–2019)

TABLE 93 PREVALENCE OF RP IN SPAIN, BY GENE MUTATION (2020–2030)

TABLE 94 TOTAL PREVALENT CASES OF RP IN THE U.K. (2014–2019)

TABLE 95 TOTAL PREVALENT CASES OF RP IN THE U.K. (2020–2030)

TABLE 96 PREVALENCE OF RP IN THE U.K., BY GENDER (2014–2019)

TABLE 97 PREVALENCE OF RP IN THE U.K., BY GENDER (2020–2030)

TABLE 98 PREVALENCE OF RP IN THE U.K., BY AGE-GROUP (2014–2019)

TABLE 99 PREVALENCE OF RP IN THE U.K., BY AGE-GROUP (2020–2030)

TABLE 100 PREVALENCE OF RP IN THE U.K., BY TYPE (2014–2019)

TABLE 101 PREVALENCE OF RP IN THE U.K., BY TYPE (2020–2030)

TABLE 102 PREVALENCE OF SYNDROMIC RP IN THE U.K., BY SUBTYPE (2014–2019)

TABLE 103 PREVALENCE OF SYNDROMIC RP IN THE U.K., BY SUBTYPE (2020–2030)

TABLE 104 PREVALENCE OF RP IN THE U.K., BY GENETIC INHERITANCE PATTERN (2014–2019)

TABLE 105 PREVALENCE OF RP IN THE U.K., BY GENETIC INHERITANCE PATTERN (2020–2030)

TABLE 106 PREVALENCE OF RP IN THE U.K., BY GENE MUTATION (2014–2019)

TABLE 107 PREVALENCE OF RP IN THE U.K., BY GENE MUTATION (2020–2030)

TABLE 108 TOTAL PREVALENT CASES OF RP IN JAPAN (2014–2019)

TABLE 109 TOTAL PREVALENT CASES OF RP IN JAPAN (2020–2030)

TABLE 110 PREVALENCE OF RP IN JAPAN, BY GENDER (2014–2019)

TABLE 111 PREVALENCE OF RP IN JAPAN, BY GENDER (2020–2030)

TABLE 112 PREVALENCE OF RP IN JAPAN, BY AGE-GROUP (2014–2019)

TABLE 113 PREVALENCE OF RP IN JAPAN, BY AGE-GROUP (2020–2030)

TABLE 114 PREVALENCE OF RP IN JAPAN, BY TYPE (2014–2019)

TABLE 115 PREVALENCE OF RP IN JAPAN, BY TYPE (2020–2030)

TABLE 116 PREVALENCE OF SYNDROMIC RP IN JAPAN, BY SUBTYPE (2014–2019)

TABLE 117 PREVALENCE OF SYNDROMIC RP IN JAPAN, BY SUBTYPE (2020–2030)

TABLE 118 PREVALENCE OF RP IN JAPAN, BY GENETIC INHERITANCE PATTERN (2014–2019)

TABLE 119 PREVALENCE OF RP IN JAPAN, BY GENETIC INHERITANCE PATTERN (2020–2030)

TABLE 120 PREVALENCE OF RP IN JAPAN, BY GENE MUTATION (2014–2019)

TABLE 121 PREVALENCE OF RP IN JAPAN, BY GENE MUTATION (2020–2030)

TABLE 122 DESCRIPTION OF LUXTURNA

TABLE 123 CLINICAL TRIALS OF LUXTURNA

TABLE 124 DESCRIPTION OF HORA-RPE65

TABLE 125 DESCRIPTION OF AAV8-RPGR

TABLE 126 CLINICAL TRIALS OF AAV8-RPGR

TABLE 127 DESCRIPTION OF JCELL

TABLE 128 CLINICAL TRIALS OF JCELL

TABLE 129 DESCRIPTION OF XXXX

TABLE 130 CLINICAL TRIALS OF XXXX

TABLE 131 DESCRIPTION OF XXXX

TABLE 132 CLINICAL TRIALS OF XXXX

TABLE 133 DESCRIPTION OF REN003

TABLE 134 CLINICAL TRIALS OF REN003

TABLE 135 DESCRIPTION OF XXXX

TABLE 136 CLINICAL TRIALS OF XXXX

TABLE 137 DESCRIPTION OF XXXX

TABLE 138 CLINICAL TRIALS OF XXXX

TABLE 139 DESCRIPTION OF XXXX

TABLE 140 CLINICAL TRIALS OF XXXX

TABLE 141 DESCRIPTION OF XXXX

TABLE 142 CLINICAL TRIALS OF XXXX)

TABLE 143 DESCRIPTION OF XXXX

TABLE 144 CLINICAL TRIALS OF XXXX

TABLE 145 DESCRIPTION OF XXXX

TABLE 146 CLINICAL TRIALS OF XXXX

TABLE 147 DESCRIPTION OF XXXX

TABLE 148 CLINICAL TRIALS OF XXXX

TABLE 149 DESCRIPTION OF XXXX

TABLE 150 CLINICAL TRIALS OF XXXX

TABLE 151 DESCRIPTION OF XXXX

TABLE 152 CLINICAL TRIALS OF XXXX

TABLE 153 DESCRIPTION OF XXXX

TABLE 154 CLINICAL TRIALS OF XXXX

TABLE 155 DESCRIPTION OF XXXX

TABLE 156 CLINICAL TRIALS OF XXXX

TABLE 157 DESCRIPTION OF XXXX

TABLE 158 KEY CROSS COMPETITION OF PIPELINE DRUGS

TABLE 159 KEY CROSS COMPETITION OF PIPELINE DRUGS (CONTD.)

TABLE 160 KEY CROSS COMPETITION OF PIPELINE DRUGS (CONTD.)

TABLE 161 KEY CROSS COMPETITION OF PIPELINE DRUGS (CONTD.)

TABLE 162 KEY CROSS COMPETITION OF PIPELINE DRUGS (CONTD.)

TABLE 163 KEY CROSS COMPETITION OF PIPELINE DRUGS (CONTD.)

TABLE 164 TOTAL MARKET REVENUE OF RP IN THE 7MM, IN $M (2014–2019)

TABLE 165 TOTAL MARKET REVENUE OF RP IN THE 7MM, IN $M (2020–2030)

TABLE 166 MARKET REVENUE OF RP IN THE 7MM, BY TREATMENT, IN $M (2019–2030)

TABLE 167 MARKET REVENUE OF RP IN THE 7MM, BY THERAPY TYPE, IN $M (2019–2030)

TABLE 168 MARKET REVENUE OF RP IN THE 7MM, BY REGION, IN $M (2014–2019)

TABLE 169 MARKET REVENUE OF RP IN THE 7MM, BY REGION, IN $M (2020–2030)

TABLE 170 POTENTIAL EMERGING THERAPIES FOR RP IN THE U.S.

TABLE 171 TOTAL MARKET REVENUE OF RP IN THE U.S., IN $M (2014–2019)

TABLE 172 TOTAL MARKET REVENUE OF RP IN THE U.S., IN $M (2020–2030)

TABLE 173 MARKET REVENUE OF RP IN THE U.S., BY TREATMENT, IN $M (2019–2030)

TABLE 174 MARKET REVENUE OF RP IN THE U.S., BY THERAPY TYPE, IN $M (2019–2030)

TABLE 175 POTENTIAL EMERGING THERAPIES FOR RP IN EU-5

TABLE 176 TOTAL MARKET REVENUE OF RP IN EU-5, IN $M (2014–2019)

TABLE 177 TOTAL MARKET REVENUE OF RP IN EU-5, IN $M (2020–2030)

TABLE 178 MARKET REVENUE OF RP IN THE EU-5 COUNTRIES, BY COUNTRY, IN $M (2014–2019)

TABLE 179 MARKET REVENUE OF RP IN THE EU-5 COUNTRIES, BY COUNTRY, IN $M (2020–2030)

TABLE 180 TOTAL MARKET REVENUE OF RP IN GERMANY, IN $M (2014–2019)

TABLE 181 TOTAL MARKET REVENUE OF RP IN GERMANY, IN $M (2020–2030)

TABLE 182 MARKET REVENUE OF RP IN GERMANY, BY TREATMENT, IN $M (2019–2030)

TABLE 183 MARKET REVENUE OF RP IN GERMANY, BY THERAPY TYPE, IN $M (2019–2030)

TABLE 184 TOTAL MARKET REVENUE OF RP IN FRANCE, IN $M (2014–2019)

TABLE 185 TOTAL MARKET REVENUE OF RP IN FRANCE, IN $M (2020–2030)

TABLE 186 MARKET REVENUE OF RP IN FRANCE, BY TREATMENT, IN $M (2019–2030)

TABLE 187 MARKET REVENUE OF RP IN FRANCE, BY THERAPY TYPE, IN $M (2019–2030)

TABLE 188 TOTAL MARKET REVENUE OF RP IN ITALY, IN $M (2014–2019)

TABLE 189 TOTAL MARKET REVENUE OF RP IN ITALY, IN $M (2020–2030)

TABLE 190 MARKET REVENUE OF RP IN ITALY, BY TREATMENT, IN $M (2019–2030)

TABLE 191 MARKET REVENUE OF RP IN ITALY, BY THERAPY TYPE, IN $M (2019–2030)

TABLE 192 TOTAL MARKET REVENUE OF RP IN SPAIN, IN $M (2014–2019)

TABLE 193 TOTAL MARKET REVENUE OF RP IN SPAIN, IN $M (2020–2030)

TABLE 194 MARKET REVENUE OF RP IN SPAIN, BY TREATMENT, IN $M (2019–2030)

TABLE 195 MARKET REVENUE OF RP IN SPAIN, BY THERAPY TYPE, IN $M (2019–2030)

TABLE 196 TOTAL MARKET REVENUE OF RP IN THE U.K., IN $M (2014–2019)

TABLE 197 TOTAL MARKET REVENUE OF RP IN THE U.K., IN $M (2020–2030)

TABLE 198 MARKET REVENUE OF RP IN THE U.K., BY TREATMENT, IN $M (2019–2030)

TABLE 199 MARKET REVENUE OF RP IN THE U.K., BY THERAPY TYPE, IN $M (2019–2030)

TABLE 200 POTENTIAL EMERGING THERAPIES FOR RP IN JAPAN

TABLE 201 TOTAL MARKET REVENUE OF RP IN JAPAN, IN $M (2014–2019)

TABLE 202 TOTAL MARKET REVENUE OF RP IN JAPAN, IN $M (2020–2030)

TABLE 203 MARKET REVENUE OF RP IN JAPAN, BY TREATMENT, IN $M (2019–2030)

TABLE 204 MARKET REVENUE OF RP IN JAPAN, BY THERAPY TYPE, IN $M (2019–2030)

TABLE 205 SPARK THERAPEUTICS INC. – AT A GLANCE

TABLE 206 HORAMA S.A. – AT A GLANCE

TABLE 207 XXXX AT A GLANCE

TABLE 208 XXXX – AT A GLANCE

TABLE 209 XXXX – KEY FINANCIAL SUMMARY

TABLE 210 XXXX. – AT A GLANCE

TABLE 211 XXXX. – KEY FINANCIAL SUMMARY

TABLE 212 XXXX. – AT A GLANCE

TABLE 213 XXXX. – KEY FINANCIAL SUMMARY

TABLE 214 XXXX. – AT A GLANCE

TABLE 215 XXXX– AT A GLANCE

TABLE 216 XXXX – KEY FINANCIAL SUMMARY

TABLE 217 XXXX – AT A GLANCE

TABLE 218 XXXX – KEY FINANCIAL SUMMARY

 

LIST OF FIGURES

 

FIG 1 RESEARCH METHODOLOGY

FIG 2 BREAKDOWN OF PRIMARY RESEARCH RESPONDENTS, BY INDUSTRY PARTICIPANT

FIG 3 BREAKDOWN OF PRIMARY RESEARCH, BY GEOGRAPHY

FIG 4 BREAKDOWN OF PRIMARY RESEARCH, BY DESIGNATION

FIG 5 TYPICAL SYMPTOMS OF RP

FIG 6 CLASSIFICATION OF RP

FIG 7 PATHOPHYSIOLOGY OF RP

FIG 8 PATTERN OF ADRP INHERITANCE

FIG 9 PATTERN OF ARRP INHERITANCE

FIG 10 PATTERN OF XLRP INHERITANCE

FIG 11 ALTERED RETINOID CYCLE METABOLIC PATHWAY

FIG 12 FUTURE TREATMENT MODALITIES FOR RP

FIG 13 UNMET MEDICAL NEEDS IN RP

FIG 14 COMPANY BENCHMARKING BASED ON DRUG CANDIDATES

FIG 15 EPIDEMIOLOGY OF RP IN THE 7MM KEY INSIGHTS SNAPSHOT

FIG 16 TOTAL PREVALENT CASES OF RP IN THE 7MM (2014-2030)

FIG 17 PREVALENCE OF RP IN THE 7MM, BY GENDER (2014–2030)

FIG 18 PREVALENCE OF RP IN THE 7MM, BY AGE-GROUP (2014–2030)

FIG 19 PREVALENCE OF RP IN THE 7MM, BY TYPE (2014–2030)

FIG 20 PREVALENCE OF SYNDROMIC RP IN THE 7MM, BY SUBTYPE (2014–2030)

FIG 21 PREVALENCE OF RP IN THE 7MM, BY GENETIC INHERITANCE PATTERN (2014–2030)

FIG 22 PREVALENCE OF RP IN 7MM, BY GENE MUTATION (2014–2030)

FIG 23 PREVALENCE OF RP IN THE 7MM, BY REGION (2014–2030)

FIG 24 TOTAL PREVALENT CASES OF RP IN THE U.S. (2014-2030)

FIG 25 PREVALENCE OF RP IN THE U.S., BY GENDER (2014–2030)

FIG 26 PREVALENCE OF RP IN THE U.S., BY AGE-GROUP (2014–2030)

FIG 27 PREVALENCE OF RP IN THE U.S., BY TYPE (2014–2030)

FIG 28 PREVALENCE OF SYNDROMIC RP IN THE U.S., BY SUBTYPE (2014–2030)

FIG 29 PREVALENCE OF RP IN THE U.S., BY GENETIC INHERITANCE PATTERN (2014–2030)

FIG 30 PREVALENCE OF RP IN U.S., BY GENE MUTATION (2014–2030)

FIG 31 TOTAL PREVALENT CASES OF RP IN THE EU-5 COUNTRIES (2014-2030)

FIG 32 PREVALENCE OF RP IN EU-5 (2014–2030)

FIG 33 TOTAL PREVALENT CASES OF RP IN GERMANY (2014-2030)

FIG 34 PREVALENCE OF RP IN GERMANY, BY GENDER (2014–2030)

FIG 35 PREVALENCE OF RP IN GERMANY, BY AGE-GROUP (2014–2030)

FIG 36 PREVALENCE OF RP IN GERMANY, BY TYPE (2014–2030)

FIG 37 PREVALENCE OF SYNDROMIC RP IN GERMANY, BY SUBTYPE (2014–2030)

FIG 38 PREVALENCE OF RP IN GERMANY, BY GENETIC INHERITANCE PATTERN (2014–2030)

FIG 39 PREVALENCE OF RP IN GERMANY, BY GENE MUTATION (2014–2030)

FIG 40 TOTAL PREVALENT CASES OF RP IN FRANCE (2014-2030)

FIG 41 PREVALENCE OF RP IN FRANCE, BY GENDER (2014–2030)

FIG 42 PREVALENCE OF RP IN FRANCE, BY AGE-GROUP (2014–2030)

FIG 43 PREVALENCE OF RP IN FRANCE, BY TYPE (2014–2030)

FIG 44 PREVALENCE OF SYNDROMIC RP IN FRANCE, BY SUBTYPE (2014–2030)

FIG 45 PREVALENCE OF RP IN FRANCE, BY GENETIC INHERITANCE PATTERN (2014–2030)

FIG 46 PREVALENCE OF RP IN FRANCE, BY GENE MUTATION (2014–2030)

FIG 47 TOTAL PREVALENT CASES OF RP IN ITALY (2014-2030)

FIG 48 PREVALENCE OF RP IN ITALY, BY GENDER (2014–2030)

FIG 49 PREVALENCE OF RP IN ITALY, BY AGE-GROUP (2014–2030)

FIG 50 PREVALENCE OF RP IN ITALY, BY TYPE (2014–2030)

FIG 51 PREVALENCE OF SYNDROMIC RP IN ITALY, BY SUBTYPE (2014–2030)

FIG 52 PREVALENCE OF RP IN ITALY, BY GENETIC INHERITANCE PATTERN (2014–2030)

FIG 53 PREVALENCE OF RP IN ITALY, BY GENE MUTATION (2014–2030)

FIG 54 TOTAL PREVALENT CASES OF RP IN SPAIN (2014-2030)

FIG 55 PREVALENCE OF RP IN SPAIN, BY GENDER (2014–2030)

FIG 56 PREVALENCE OF RP IN SPAIN, BY AGE-GROUP (2014–2030)

FIG 57 PREVALENCE OF RP IN SPAIN, BY TYPE (2014–2030)

FIG 58 PREVALENCE OF SYNDROMIC RP IN SPAIN, BY SUBTYPE (2014–2030)

FIG 59 PREVALENCE OF RP IN SPAIN, BY GENETIC INHERITANCE PATTERN (2014–2030)

FIG 60 PREVALENCE OF RP IN SPAIN, BY GENE MUTATION (2014–2030)

FIG 61 TOTAL PREVALENT CASES OF RP IN THE U.K. (2014-2030)

FIG 62 PREVALENCE OF RP IN THE U.K., BY GENDER (2014–2030)

FIG 63 PREVALENCE OF RP IN THE U.K., BY AGE-GROUP (2014–2030)

FIG 64 PREVALENCE OF RP IN THE U.K., BY TYPE (2014–2030)

FIG 65 PREVALENCE OF SYNDROMIC RP IN THE U.K., BY SUBTYPE (2014–2030)

FIG 66 PREVALENCE OF RP IN THE U.K., BY GENETIC INHERITANCE PATTERN (2014–2030)

FIG 67 PREVALENCE OF RP IN THE U.K., BY GENE MUTATION (2014–2030)

FIG 68 TOTAL PREVALENT CASES OF RP IN JAPAN (2014-2030)

FIG 69 PREVALENCE OF RP IN JAPAN, BY GENDER (2014–2030)

FIG 70 PREVALENCE OF RP IN JAPAN, BY AGE-GROUP (2014–2030)

FIG 71 PREVALENCE OF RP IN JAPAN, BY TYPE (2014–2030)

FIG 72 PREVALENCE OF SYNDROMIC RP IN JAPAN, BY SUBTYPE (2014–2030)

FIG 73 PREVALENCE OF RP IN JAPAN, BY GENETIC INHERITANCE PATTERN (2014–2030)

FIG 74 PREVALENCE OF RP IN JAPAN, BY GENE MUTATION (2014–2030)

FIG 75 DRUG DISCOVERY AND APPROVAL PROCESS (PRE-CLINICAL AND CLINICAL TRIALS STAGES)

FIG 76 DRUG APPROVAL PROCESS (NDA REVIEW AND POST-MARKETING STAGES)

FIG 77 NEW DRUG DEVELOPMENT AND APPROVAL PROCESS IN EU

FIG 78 MARKETING AUTHORIZATION PROCEDURES IN EU

FIG 79 CENTRALIZED PROCEDURE FOR DRUG APPROVAL

FIG 80 MUTUAL RECOGNITION PROCEDURE FOR DRUG APPROVAL

FIG 81 DECENTRALIZED PROCEDURE FOR DRUG APPROVAL

FIG 82 NEW DRUG DEVELOPMENT AND APPROVAL PROCESS IN JAPAN

FIG 83 MARKET SHARE OF POTENTIAL THERAPIES IN 2030

FIG 84 EXPECTED LAUNCH TIME OF LATE CLINICAL STAGE PRODUCTS

FIG 85 MARKET OF RP IN THE 7MM – KEY INSIGHTS

FIG 86 TOTAL MARKET REVENUE OF RP IN THE 7MM, IN $M (2014–2030)

FIG 87 MARKET REVENUE OF RP IN THE 7MM, BY TREATMENT, IN $M (2014–2030)

FIG 88 MARKET REVENUE OF RP IN THE 7MM, BY THERAPY TYPE, IN $M (2019–2030)

FIG 89 MARKET REVENUE OF RP IN THE 7MM, BY REGION, IN $M (2019 AND 2030)

FIG 90 TOTAL MARKET REVENUE OF RP IN THE U.S., IN $M (2014–2030)

FIG 91 MARKET REVENUE OF RP IN THE U.S., BY TREATMENT, IN $M (2014–2030)

FIG 92 MARKET REVENUE OF RP IN THE U.S., BY THERAPY TYPE, IN $M (2014–2030)

FIG 93 TOTAL MARKET REVENUE OF RP IN EU-5, IN $M (2014–2030)

FIG 94 MARKET REVENUE OF RP IN EU-5, BY COUNTRY, IN $M (2014–2030)

FIG 95 TOTAL MARKET REVENUE OF RP IN GERMANY, IN $M (2014–2030)

FIG 96 MARKET REVENUE OF RP IN GERMANY, BY TREATMENT, IN $M (2014–2030)

FIG 97 MARKET REVENUE OF RP IN GERMANY, BY THERAPY TYPE, IN $M (2014–2030)

FIG 98 TOTAL MARKET REVENUE OF RP IN FRANCE, IN $M (2014–2030)

FIG 99 MARKET REVENUE OF RP IN FRANCE, BY TREATMENT, IN $M (2014–2030)

FIG 100 MARKET REVENUE OF RP IN FRANCE, BY THERAPY TYPE, IN $M (2019–2030)

FIG 101 TOTAL MARKET REVENUE OF RP IN ITALY, IN $M (2014–2030)

FIG 102 MARKET REVENUE OF RP IN ITALY, BY TREATMENT, IN $M (2014–2030)

FIG 103 MARKET REVENUE OF RP IN ITALY, BY THERAPY TYPE, IN $M (2019–2030)

FIG 104 TOTAL MARKET REVENUE OF RP IN SPAIN, IN $M (2014–2030)

FIG 105 MARKET REVENUE OF RP IN SPAIN, BY TREATMENT, IN $M (2019–2030)

FIG 106 MARKET REVENUE OF RP IN SPAIN, BY THERAPY TYPE, IN $M (2019–2030)

FIG 107 TOTAL MARKET REVENUE OF RP IN THE U.K., IN $M (2014–2030)

FIG 108 MARKET REVENUE OF RP IN THE U.K., BY TREATMENT, IN $M (2014–2030)

FIG 109 MARKET REVENUE OF RP IN THE U.K., BY THERAPY TYPE, IN $M (2019–2030)

FIG 110 TOTAL MARKET REVENUE OF RP IN JAPAN, IN $M (2014–2030)

FIG 111 MARKET REVENUE OF RP IN JAPAN, BY TREATMENT, IN $M (2014–2030)

FIG 112 MARKET REVENUE OF RP IN JAPAN, BY THERAPY TYPE, IN $M (2019–2030)

FIG 113 XXXX – REVENUE SPLIT BY CATEGORY AND GEOGRAPHY (2019)

FIG 114 XXXX – REVENUE SPLIT BY SEGMENT AND GEOGRAPHY (2019)

FIG 115 XXXX – REVENUE SPLIT BY BUSINESS DIVISION AND GEOGRAPHY (2019)