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

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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.

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 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.

Novartis AG
Sarepta Therapeutics Inc
PTC Therapeutics
Kyowa Kirin Co Ltd
bluebird bio Inc
Corestemchemon Inc
Biostar Stem Cell Research Institute
Nipro Corp
Pfizer Inc
Capricor Therapeutics Inc
Rapa Therapeutics LLC
BrainStorm Cell Therapeutics Inc

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

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