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Cell & Gene Therapies in CNS Disorders - Update

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    Report

  • 46 Pages
  • March 2025
  • Region: Global
  • GlobalData
  • ID: 6059095
Currently, there are nine cell and gene therapies (CGTs) on the market for various central nervous system (CNS) disorders. This includes three gene therapies, three 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).

The analyst anticipates that the global CGT market value in CNS disorders will experience significant growth-sales are expected to increase from $2.3 billion in 2024 to $12.6 billion in 2030, at a compound annual growth rate (CAGR) of 32.8%. Gene therapies will dominate the CGT market in CNS disorders. This is attributed to strong players such as Sarepta Therapeutics and Novartis with their marketed products Elevidys and Zolgensma, respectively.

Scope

  • 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

1. Preface
1.1. Contents
1.2. Abbreviations
1.3. Related Reports
2. Executive Summary3. Introduction and Scope
4. Current Treatment Options
4.1. What is Cell & Gene Therapy?
4.2. Marketed Product Profiles - Gene Therapy + Gene-Modified Cell Therapy
4.3. Marketed Product Profiles - Cell Therapy
4.4. History of the Development of CGT in CNS Disorders
4.5. Commercial Activities of Marketed CGTs in CNS Disorders
4.6. Challenges and Opportunities in CGT in CNS Disorders
5. Pricing and Reimbursement Assessment
5.1. Price of Gene Therapy and Gene-Modified Cell Therapy
5.2. Pricing and Reimbursement Case Study: Zolgensma
6. Regulations
6.1. Regulation of CGTs in the 8MM
6.2. FDA Guidance for Industry - Human Gene Therapy for Neurodegenerative Diseases
7. Future Market Assessment
7.1. Top 20 CNS Disorders with CGT Development
7.2. Top Five CNS Disorders with the Most CGT Pipeline Assets
7.3. Top Five CNS Disorders Stratified by Molecule Type
7.4. CGT in CNS Disorders - Phase III and II/III
7.5. Additional Players Expected to Join the Competition Within Five Years
7.6. Industry Trends in the Application of CGTs in CNS Disorders
7.7. Most of CGT Market Catalyst is Concentrated in 2025
8. Likelihood of Approval and Phase - Transition Success Rate Analysis
8.1. CGT Candidates Have Lower Than Average LoA and PTSR
9. Sales Forecast
9.1. Sales are forecast to reach $12.6 billion by 2030
10. Appendix
10.1. Methodology
10.2. Primary Research: Key Opinion Leader Information
10.3. About the Authors
11. Contact the Publisher

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • Kyowa Kirin Co Ltd
  • Novartis AG
  • Nipro Corp
  • PTC Therapeutics Inc
  • bluebird bio Inc
  • Sarepta Therapeutics Inc
  • Corestemchemon Inc
  • Biostar Stem Cell Research Institute
  • SanBio Co Ltd