Cell and Gene Therapies in CNS Disorders

出版日: | 発行: GlobalData | ページ情報: 英文 44 Pages | 納期: 即納可能 即納可能とは

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出版日: 2024年03月18日
発行: GlobalData
ページ情報: 英文 44 Pages
納期: 即納可能 即納可能とは
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世界の中枢神経系疾患における細胞および遺伝子治療(CGT)の市場規模は大きく成長すると予測され、売上は2023年の15億米ドルから2029年に122億米ドルに増加し、CAGRで42.4%の成長が見込まれます。中枢神経系障害においては遺伝子治療がCGT市場を独占します。これはSarepta TherapeuticsやNovartisのような有力企業がそれぞれElevidysとZolgensmaを販売しているためです。



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・遺伝子治療とは
  • 上市済み製品のプロファイル - 遺伝子治療+遺伝子組み換え細胞治療
  • 上市済み製品のプロファイル - 細胞治療
  • 中枢神経系障害におけるCGTの開発の歴史
  • 中枢神経系障害における上市済みのCGTの商業活動
  • 中枢神経系障害におけるCGTの課題と機会

第4章 価格設定と償還の評価

  • 遺伝子治療と遺伝子組み換え細胞治療の価格
  • 価格設定と償還のケーススタディ:Zolgensma

第5章 規制

  • 主要8市場におけるCGTの規制
  • 業界向けFDAガイダンス - 神経変性疾患向けヒト遺伝子治療

第6章 将来の市場の評価

  • CGT発症を伴う上位20のCNS障害
  • CGTパイプラインアセットがもっとも多い上位5つのCNS障害
  • 上位5つのCNS障害:分子タイプ別
  • CNS障害向けCGT - フェーズIII、II/III
  • 5年以内に追加の企業が競合に加わると予測される
  • CNS障害におけるCGTの応用に関する業界動向
  • CGT市場のカタリストのほとんどは2024年に集中している

第7章 承認可能性とフェーズ移行成功率の分析

  • CGT候補のLoAとPTSRは平均より低い

第8章 売上予測

  • 売上は2029年までに122億米ドルに達すると予測される

第9章 付録

第10章 お問い合わせ

Product Code: GDHCHT469

Currently, there are eight cell and gene therapies (CGT) on the market for various central nervous system (CNS) disorders. This includes three gene therapies, two gene-modified cell therapies, and three conditionally approved cell therapies. Novartis's Zolgensma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA) was the first CGT asset to receive traditional approval in the CNS market in 2019. Over the past five years, there has been an increase in the number of traditionally approved CGTs for genetic disorders such as metachromatic leukodystrophy (MLD), cerebral adrenoleukodystrophy (CALD), aromatic L-amino acid decarboxylase deficiency (AADCD), and Duchenne muscular dystrophy (DMD).

GlobalData anticipates that the global CGT market value in CNS disorders will experience significant growth-sales are expected to increase from $1.5 billion in 2023 to $12.2 billion in 2029, at a compound annual growth rate (CAGR) of 42.4%. Gene therapies will dominate the CGT market in CNS disorders. This is attributed to strong players such as Sarepta Therapeutics and Novartis with marketed products Elevidys and Zolgensma, respectively.


Overview of cell and gene therapies (CGT) in CNS Disorders. The report focuses on the marketed CGT assets addressing CNS disorders as well as the top five CNS indications with the most CGT pipeline assets. Clients will gain insight into the competitive landscape of leading CGT agents in CNS disorders 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, US, and Japan, and analyses key challenges and opportunities in the application of CGTs in CNS disorders. 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 CNS market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the CNS disorder targeting 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 + Gene-Modified Cell Therapy
  • 3.3. Marketed Product Profiles - Cell Therapy
  • 3.4. History of the Development of CGT in CNS Disorders
  • 3.5. Commercial Activities of Marketed CGTs in CNS Disorders
  • 3.6. Challenges and Opportunities in CGT in CNS Disorders

4. Pricing and Reimbursement Assessment

  • 4.1. Price of Gene Therapy and Gene-Modified Cell Therapy
  • 4.2. Pricing and Reimbursement Case Study: Zolgensma

5. Regulations

  • 5.1. Regulation of CGTs in the 8MM
  • 5.2. FDA Guidance for Industry - Human Gene Therapy for Neurodegenerative Diseases

6. Future Market Assessment

  • 6.1. Top 20 CNS Disorders with CGT Development
  • 6.2. Top Five CNS Disorders with the Most CGT Pipeline Assets
  • 6.3. Top Five CNS Disorders Stratified by Molecule Type
  • 6.4. CGT in CNS Disorders - Phase III and II/III
  • 6.5. Additional Players Expected to Join the Competition Within Five Years
  • 6.6. Industry Trends in the Application of CGTs in CNS Disorders
  • 6.7. Most of CGT Market Catalyst is Concentrated in 2024

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

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

8. Sales Forecast

  • 8.1. Sales are forecast to reach $12.2 billion by 2029

9. Appendix

  • 9.1. Methodology
  • 9.2. Primary Research: Key Opinion Leader Information
  • 9.3. About the Authors

10. Contact Us