眼科における細胞治療および遺伝子治療

現在、眼科を適応症とする細胞治療および遺伝子治療薬（CGT）が8種類上市されています。このうちSpark TherapeuticsのLuxturna（voretigene neparvovec）は、網膜色素変性症（網膜炎）とレーバー先天性黒内障（LCA）を適応症とし、世界的に販売されており、眼科領域で上市されている唯一の遺伝子治療薬です。他のすべてのCGTは細胞療法です。Holostem Terapie AvanzateのHoloclarは辺縁幹細胞欠損症（LSCD）を適応症とし、欧州で販売されています。富士フイルムのNepic、J.TecのOcural、Hirosaki Lifescience InnovationのSakracyはいずれもLSCDを適応症とし、日本で販売されています。Aurion BiotechnologiesのVyznovaは角膜浮腫を適応症とし、日本で販売されています。Reliance Life SciencesのReliNethraはLSCDを、ReliNethra Cはアレルギー性結膜炎、眼瞼結膜炎、翼状片を適応症とし、いずれもインドで販売されています。

一般に、市販されているCGTは、代替治療がない重症で希少な疾患を対象としています。関連する研究開発コストや患者数が比較的少ないため、1つあたりの治療費はかなり高額になります。価格設定と償還は、CGTの潜在的な売上高を制限する可能性があります。

CGTは38の眼科適応症で開発中であり、発見から登録前段階まで約500の資産に及んでいます。CGTのパイプライン資産が多い眼科適応症の、トップ５は、加齢黄斑変性（AMD）、網膜炎、緑内障、LCA、網膜症です。パイプラインは、治験薬（IND）/臨床試験申請（CTA）済み、前臨床、探索といった医薬品開発の段階に集中しています。

世界の眼科における細胞治療および遺伝子治療市場は緩やかな成長を遂げると予測されており、2023年の4,600万米ドルから2029年には30億米ドルに増加し、CAGRは50.8%になるとみられています。遺伝子治療は眼科領域のCGT市場を独占すると予想されます。これは、パイプラインにある遺伝子治療資産の数が多いこと（総資産の74.6%）と、販売製品Luxturnaを擁するSpark Therapeuticsのような強力な参入企業がいることによる。

当レポートでは、眼科における細胞治療および遺伝子治療市場について調査し、眼科適応症上位5つに焦点を当てています。眼科領域における主要なCGT薬剤の競合情勢、市場における課題と機会、今後の見通しなどをまとめています。

目次

第1章 序文

第2章 エグゼクティブサマリー

第3章 現在の治療オプション

• 細胞治療および遺伝子治療とは何か
• 上市済み製品プロファイル- 遺伝子治療
• 上市済み製品プロファイル- 細胞療法
• 眼科におけるCGTの発展の歴史
• 眼科における上市済みCGTの商業活動
• 眼科におけるCGTの課題と機会

第4章 価格設定と償還評価

• 遺伝子治療と細胞治療の価格
• 価格設定と償還のケーススタディ：Luxturna

第5章 規則

• 8MMにおけるCGTの規制
• FDA業界向けガイダンス- 網膜疾患に対するヒト遺伝子治療

第6章 将来の市場評価

• CGT開発による眼科適応症、トップ２０
• CGTパイプライン資産が最も多い眼科適応症、トップ５
• 分子タイプ別眼科適応症、トップ５
• 眼科CGTパイプライン資産
• 眼科におけるCGT-第III相および第II/III相
• 5年以内にさらなる参入企業が競合に加わると予想される
• 眼科におけるCGTの応用に関する業界動向
• CGT市場促進要因、2024年

第7章 承認の可能性とフェーズ移行の成功率分析

第8章 売上予測

• 売上高は2029年までに30億米ドルに達すると予測

第9章 付録

第10章 お問い合わせ

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.

Scope

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.

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.

Table of Contents

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