Gene Therapy in Neurology

Gene Therapy in Neurology


Gene therapy is an evolving area in healthcare that promises to revolutionize the treatment landscapes across various therapy areas. In this report, the focus will be on neurology indications. Recently approved gene therapies for spinal muscular atrophy have reinvigorated the potential of such therapies to transform patient care. While various methodologies can be adopted in order to deliver therapeutic benefits of gene therapy including gene augmentation, gene suppression, and gene editing, an important component of gene therapy is whether to use viral or non-viral vectors in order to deliver such therapies to the point of care.

Ongoing collaborations between different industry players and a buildup of real-world evidence establishing safety and efficacy are expected to drive the growth of gene therapies for neurology indications. Of the 38 pipeline products that are currently in development, 45% are adeno-associated virus (AAV) based delivery platforms. Other types include Lentiviral, which accounts for 13%.

A majority of the current pipeline products are in Phase II development and the most common neurology indications - for which gene therapies are currently being evaluated - include Parkinson’s disease, pain and amyotrophic lateral sclerosis. The dominance of viral vectors is expected to continue as such platforms account for the bulk of these pipeline products, with adeno-associated virus being the most common among the viral vectors.

In terms of completed, ongoing and planned clinical trials, academic institutes account for 21% of these trials, despite industry sponsors being most dominant. A deeper analysis of these clinical trials also suggest that across most indications, the average trial duration for a viral based product is longer compared to a non-viral based product such as oligonucleotides or plasmid DNA.

There are also challenges associated with the development of gene therapies, most prominent being their high price points. Key opinion leaders (KOLs) interviewed by GlobalData highlighted the need to create sustainable funding solutions so that such therapies become accessible to patients everywhere irrespective of where patients are located. In terms of unmet needs, KOLs highlighted the need for a favorable route of administration that is both sustainable in terms of usage of healthcare resources and favorable from a patient perspective.

While development of gene therapies are expected to pick up pace, the next wave of such therapies are expected to be ones that target diseases that are more frequent. While monogenic rare diseases are the obvious first-to-go choice for which gene therapies can be developed, targeting more frequent diseases will need a holistic approach in order to address a wider mechanism of action. If gene therapies for frequent diseases do become available, then that will result in a more pronounced effect on healthcare not only in terms of providing better treatment options for patients but also test the ability of healthcare organizations to adapt with high price points of these therapies.

The report “Gene Therapy in Neurology” provides an analysis of the overall gene therapy pipeline that is being developed for various neurology indications with an emphasis on late-stage pipeline products. In addition to pipeline analysis, the report also focuses on trends observed in clinical trials in this area, unmet needs and challenges, as well as partnership strategies adopted by pharmaceutical companies to keep up with developments in the field of gene therapies.


  • GlobalData’s Gene Therapy in Neurology report combines primary research from a cross-specialty panel of neurology experts with in-house analyst expertise to provide an assessment of the development landscape.
  • Components of the slide deck include primary and secondary research:
  • Quotes from 7 key opinion leaders (2 US, 3 EU, 1 Japan, 1 China) and 2 payer key opinion leaders (1 US, 1 EU)
  • Overview of common vectors and indications among current pipeline drugs, with focus on products in Phase II and III
  • Trends in clinical trials (completed, ongoing, planned) in gene therapies based on sponsor type, location, and phase of development
  • Insight from GlobalData’s specialist neurology analysts
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 global gene therapies market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global gene therapies market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies 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.

  • Executive Summary
    • Key Findings
    • KOL Insights on Competitive Landscape for Gene Therapy for Neurology Indications
  • Overview - Gene Therapy in Neurology
    • Objectives of Gene Therapy
    • Gene Therapy Versus Conventional Therapies
    • Gene Therapy Versus Conventional Therapy
    • Practicalities and Expectations for Gene Therapies
    • Optimization of Gene Expression
    • Gene Transfer Methods and Vectors Used for Gene Therapy
    • Classifications of Gene Therapy
    • Viral Vectors - Adenoviruses
    • Viral Vectors - Adeno-Associated Viruses
    • Viral Vectors - Retrovirus/Lentivirus
    • Viral Vectors - Herpes Simplex Virus
    • Non-Viral Vectors
    • On Viral And Non-Viral Gene Therapy
    • On Neutralizing Antibody Formation
    • Sources
  • Gene Therapy in the 8MM
    • Global Regulatory Agencies' Definitions of Gene Therapy
    • Oversight of Gene Therapy in the US
    • Oversight of Gene Therapy in the EU
    • Oversight of Gene Therapy in Japan
    • Oversight of Regenerative Medicine in Japan
    • Oversight of Gene Therapy in China
    • Currently Marketed Gene Therapies in Neurology
    • Sources
  • Pipeline Assessment in the 8MM
    • Pipeline Overview
    • Pipeline Products - Phase III Pipeline
    • Pipeline Products - Phase II Pipeline
    • Competitive Assessment - Phase III Pipeline Therapies
    • On Phase III Products for Gene Therapies in Neurology
    • Sources
  • Clinical Trials Mapping and Design
    • Clinical Trial Mapping
    • Clinical Trial Duration by Indication
    • Clinical Trial Duration by Indication for Phase III Products
    • Clinical Trial Duration by Indication
    • Clinical Trial Duration by Indication for Phase II Products
    • Clinical Trial Mapping
  • Unmet Needs, Barriers, and Key Company Strategies
    • Unmet Needs Within Gene Therapy for CNS Indications
    • Unmet Needs in Gene Therapies in Neurology
    • Challenges and Other Factors to Consider During Different Stages of Product Development
    • Key Company Strategies - Acquisitions
    • Key Company Strategies - Strategic Partnerships
    • Sources
  • Payer Perspective on Gene Therapies in Neurology
    • Pricing and Reimbursement
    • Payer Opinion on Gene Therapies in Neurology
  • Market Outlook
    • Market Outlook for Orchard Therapeutics' OTL-200
    • Market Outlook for Biogen's Tofersen sodium
    • Market Outlook for Roche's RG-6042
    • Market Outlook for Sylentis' Tivanisiran
    • Market Outlook for ViroMed's Donaperminogene Seltoplasmid
    • Anticipated Key Events in Neurology Indications
  • Appendix
    • Methodology
    • Primary Research

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