Cell and Gene Therapies in Ophthalmology – Disease Overview, Treatment Options and Future Market Assessment

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Currently, there are eight cell and gene therapies (CGTs) on the market for various ophthalmology indications. These comprise Spark Therapeutics' Luxturna (voretigene neparvovec), which is indicated for retinitis pigmentosa (retinitis) and Leber congenital amaurosis (LCA), is marketed globally, and is the only gene therapy on the market within the ophthalmology space. All other marketed CGTs are cell therapies. Holostem Terapie Avanzate’s Holoclar is indicated for limbal stem cell deficiency (LSCD) and marketed in Europe. Fujifilm’s Nepic, J.Tec’s Ocural, and Hirosaki Lifescience Innovation’s Sakracy are all indicated for LSCD and marketed in Japan. Aurion Biotechnologies' Vyznova is indicated for corneal edema and marketed in Japan. Reliance Life Sciences' ReliNethra is indicated for LSCD and ReliNethra C is indicated for allergic conjunctivitis, blepharoconjunctivitis, and pterygium, and both are marketed in India.

Generally, marketed CGTs target severe and rare diseases for which there are a lack of alternative treatments. The course per therapy is considerably high due to the associated R&D costs and the relatively small patient populations. Pricing and reimbursement could limit the potential sales revenue of CGTs.

Throughout the eight major markets (8MM: US, France, Germany, Italy, Spain, UK, Japan, and India), CGT guidelines have been published covering: Chemistry, Manufacturing, and Controls (CMC), preclinical assessment, and clinical trials. The FDA provides a guidance specifically for the application of gene therapy for retinal disorders.

CGTs are in development for 38 ophthalmology indications and span almost 500 assets from discovery to pre-registration phases. The top five ophthalmology indications with the most CGT pipeline assets are age-related macular degeneration (AMD), retinitis, glaucoma, LCA, and retinopathy. The pipeline is concentrated towards the stages of drug development, such as investigational new drug (IND)/clinical trial application (CTA) filed, preclinical, and discovery.

GlobalData anticipates that the global CGT market value in the ophthalmology space will experience moderate growth. Sales are expected to increase from $46 million in 2023 to $3.0 billion in 2029, at a compound annual growth rate (CAGR) of 50.8%. Gene therapies are expected to dominate the CGT market in ophthalmology. This is attributed to the high number of gene therapy assets (74.6% of total assets) in the pipeline, and strong players such as Spark Therapeutics, with its marketed product Luxturna.


Overview of cell and gene therapies (CGT) in ophthalmology. The report focuses on the marketed CGT assets addressing ophthalmology indications as well as the top five ophthalmology indications with the most CGT pipeline assets. Clients will gain insight into the competitive landscape of leading CGT agents in ophthalmology including launch date projections, analyst consensus forecasts, likelihood of approval analysis, and commentary on current and future players. The report also includes outlook from three key opinion leaders in the 5EU, and US, and analyses key challenges and opportunities in the application of CGTs in ophthalmology. Additionally, the report includes commentary on the regulatory landscape of CGTs and on the reimbursement environment.

Key Highlights

Report deliverable includes a PowerPoint report.

Reasons to Buy

• Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.

• Develop business strategies by understanding the trends shaping and driving the CGT in ophthalmology market.

• Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the opthalmology CGT therapeutics market in the future.

• Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.

• Identify emerging players with potentially strong product portfolios and create effective counterstrategies to gain a competitive advantage.

• Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Spark Therapeutics
Johnson & Johnson
Fujifilm Holdings Co
Aurion Biotech
Japan Tissue Engineering Co

Table of Contents

1. Preface

1.1. Contents

1.2. Abbreviations

1.3. Related Reports

2. Executive Summary

3. Current Treatment Options

3.1. What is Cell & Gene Therapy?

3.2. Marketed Product Profiles – Gene Therapy

3.3. Marketed Product Profiles – Cell Therapy

3.4. History of the Development of CGT in Ophthalmology

3.5. Commercial Activities of Marketed CGTs in Ophthalmology

3.6. Challenges and Opportunities in CGT in Ophthalmology

4. Pricing and Reimbursement Assessment

4.1. Price of Gene Therapy and Cell Therapy

4.2. Pricing and Reimbursement Case Study: Luxturna

5. Regulations

5.1. Regulation of CGTs in the 8MM

5.2. FDA Guidance for Industry – Human Gene Therapy for Retinal Disorders

6. Future Market Assessment

6.1. Top 20 Ophthalmology Indications with CGT Development

6.2. Top Five Ophthalmology Indications with the Most CGT Pipeline Assets

6.3. Top Five Ophthalmology Indications Stratified by Molecule Type

6.4. Ophthalmology CGT Pipeline Assets

6.5. CGT in Ophthalmology – Phase III and II/III

6.6. Additional Players Expected to Join the Competition Within Five Years

6.7. Industry Trends in the Application of CGTs in Ophthalmology

6.8. CGT Market Catalysts in 2024

7. Likelihood of Approval and Phase – Transition Success Rate Analysis

7.1. CGT Candidates Typically Have Lower Than Average LoA and PTSR

8. Sales Forecast

8.1. Sales are forecast to reach $3.0 billion by 2029

9. Appendix

9.1. Methodology

9.2. Primary Research: Key Opinion Leader Information

9.3. About the Authors

10. Contact Us

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