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.

The report 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.

Companies mentioned


Agilis Biotherapeutics

Amicus Therapeutics

Antisense Therapeutics

Asklepios BioPharmaceutical


Axovant Gene Therapies

Bamboo Therapeutics


Ionis Pharmaceuticals

Jazz Pharmaceuticals

MeiraGTx Holdings


Orchard Therapeutics


PTC Therapeutics





Takara Bio

UniQure NV


Voyager Therapeutics

WAVE Life Sciences

Xalud Therapeutics

Table of Contents

1 Table of Contents (PowerPoint Deck)

1 Preface

1.1 Table of Contents

1.2 Abbreviations

1.3 Related Reports

2 Executive Summary

2.1 Key Findings

2.2 KOL Insights on Competitive Landscape for Gene Therapy for Neurology Indications

3 Overview – Gene Therapy in Neurology

3.1 Objectives of Gene Therapy

3.2 Gene Therapy Versus Conventional Therapies

3.3 Optimization of Gene Expression

3.4 Gene Transfer Methods and Vectors Used for Gene Therapy

3.5 Classifications of Gene Therapy

3.6 Sources

4 Gene Therapy in the 8MM

4.1 Global Regulatory Agencies’ Definitions of Gene Therapy

4.2 Gene Therapy in the US

4.3 Gene Therapy in the EU

4.4 Gene Therapy in Japan

4.5 Gene Therapy in China

4.6 Currently Marketed Gene Therapies in Neurology

4.7 Sources

5 Pipeline Assessment in the 8MM

5.1 Pipeline Overview

5.2 Pipeline Products – Phase III

5.3 Pipeline Products – Phase II

5.4 Orchard Therapeutics’ OTL-200

5.5 Biogen’s Tofersen sodium

5.6 Roche’s RG-6042

5.7 Sylentis’ Tivanisiran

5.8 ViroMed’s Donaperminogene Seltoplasmid

5.9 Sources

6 Clinical Trials Mapping and Design

6.1 Clinical Trial Mapping for all Pipeline Products by Phase, by Sponsor, and by Location

6.2 Clinical Trial Mapping for all Pipeline Products by Status and by Indication

6.3 Clinical Trial Mapping by Phase and Indication for Phase III Therapies

6.4 Clinical Trial Mapping by Phase for Phase II Therapies

6.5 Clinical Trial Duration by Indication for Phase III Therapies (By Types of Molecules)

6.6 Clinical Trial Duration by Indication for Phase II Therapies (By Types of Molecules)

6.7 Ongoing Clinical Development of Phase III Gene Therapies

7 Unmet Needs, Barriers, and Key Company Strategies

7.1 Unmet Needs Within Gene Therapy for CNS Indications

7.2 Challenges and Other Factors to Consider During Different Stages of Product Development

7.3 Key Company Strategies: Acquisitions

7.4 Key Company Strategies: Strategic Partnerships

7.5 Sources

8 Payer Perspective on Gene Therapies in Neurology

8.1 Current Neurology Space

8.2 Challenges Associated with Reimbursement of Novel Gene Therapies

8.3 Cost of Gene Therapies

8.4 Strategies to Tackle the Cost of Gene Therapies

8.5 Innovative Reimbursement Models and Clinical Comparators

9 Market Outlook

9.1 Phase III Gene Therapy Pipeline for Neurology

9.2 Key Launch Dates for Phase III Gene Therapy Pipeline Products

10 Appendix

10.1 Methodology

10.2 Primary Research

10.3 About the Authors

10.4 About GlobalData

10.5 Contact Us

10.6 Disclaimer


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