Report cover image

Cell and Gene Therapies in CNS Disorders

Publisher GlobalData
Published Mar 18, 2024
Length 44 Pages
SKU # GBDT18714171

Description

Cell and Gene Therapies in CNS Disorders


Summary

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.

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

44 Pages
    • Global CGT Sales Are Forecast to Reach $66.0 Billion by 2029
    • What Is Cell & Gene Therapy?
    • Marketed Product Profiles – Gene Therapy + Gene-Modified Cell Therapy
    • Marketed Product Profiles – Cell Therapy
    • History of the Development of CGT in CNS Disorders
    • Commercial Activities of Marketed CGTs in CNS Disorders
    • Challenges and Opportunities in CGT in CNS Disorders
    • Price of Gene Therapy and Gene-Modified Cell Therapy
    • Pricing and Reimbursement Case Study: Zolgensma
    • Regulation of CGTs in the 8MM
    • FDA Guidance for Industry - Human Gene Therapy for Neurodegenerative Diseases
    • Top 20 CNS Disorders with CGT Development
    • Top Five CNS Disorders with the Most CGT Pipeline Assets
    • Top Five CNS Disorders Stratified by Molecule Type
    • Top Five CNS Disorders with the Most CGT Pipeline Assets
    • CGT in CNS Disorders – Phase III and II/III
    • Additional Players Expected to Join the Competition Within Five Years
    • Industry Trends in the Application of CGTs in CNS Disorders
    • Most of CGT Market Catalyst Is Concentrated in 2024
    • CGT Candidates Have Lower-Than-Average LoA and PTSR • Looking at the top five CNS disorders with
    • Sales Are Forecast to Reach $12.2 Billion by 2029
    • Methodology
    • Methodology – Sales Forecasts
    • Methodology – Phase Transition Success Rate and Likelihood of Approval Analysis
    • Primary Research: KOL Information

Pricing

Currency Rates
How Do Licenses Work?

How Do Licenses Work?

The primary function of a report license is to define how many people within a company are authorized to use the purchased report. This is separate to how a report might be delivered. Unless specifically identified otherwise, the purchase option is Single User. Below is a helpful description of that license, along with a sampling of others most commonly offered.

  • This license allows only one user to have access to and to use/read the report. It is not one user at a time or one user group; it is an individual who will be reading and utilizing the report.

  • This license allows for the entire purchasing company to read and use the report. This is the license level that would allow for a report to be placed on an internal company shared drive for access by all employees of that direct company. This license does not extend to parent or sister company use.

  • This license is designed to allow an entire department within a company full access to a report. The departmental license is a great option for companies that have more than one location and the department is spread out all over the country/world

  • This license is for companies that have one location where they want the report to be accessible by all employees within that physical location.

  • This is the most flexible of the licensing options. Some publishers will allow you to get a license for a preset number of people (typically 5). These licenses are ideal for small work groups.

License options are at the discretion of each publisher. As such, not all licenses indicated above are available for every product. If you are uncertain of the best license for your needs or would like a license that is not proactively available, please contact us and we will be happy to assist.

Head shot

Questions or Comments?

Our team has the ability to search within reports to verify it suits your needs. We can also help maximize your budget by finding sections of reports you can purchase.
Chat Now
Contact Us