Gene Therapy in Neurology

Powered by

All the vital news, analysis, and commentary curated by our industry experts.

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.

Scope

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.

Adynxx
Agilis Biotherapeutics
Amicus Therapeutics
Antisense Therapeutics
Asklepios BioPharmaceutical
AveXis
Axovant Gene Therapies
Bamboo Therapeutics
Biogen
Ionis Pharmaceuticals
Jazz Pharmaceuticals
MeiraGTx Holdings
Novartis
Orchard Therapeutics
Pfizer
PTC Therapeutics
RegenxBio
Roche
SanBio
Sylentis
Takara Bio
UniQure NV
ViroMed
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

Frequently asked questions

Gene Therapy in Neurology thematic reports
Currency USD
$7,995

Can be used by individual purchaser only

$23,985

Can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company


Undecided about purchasing this report?

Enquire Before Buying Request a Free Sample

Get in touch to find out about multi-purchase discounts

reportstore@globaldata.com
Tel +44 20 7947 2745

Every customer’s requirement is unique. With over 220,000 construction projects tracked, we can create a tailored dataset for you based on the types of projects you are looking for. Please get in touch with your specific requirements and we can send you a quote.

Sample Report

Gene Therapy in Neurology was curated by the best experts in the industry and we are confident about its unique quality. However, we want you to make the most beneficial decision for your business, so we offer free sample pages to help you:

  • Assess the relevance of the report
  • Evaluate the quality of the report
  • Justify the cost

Download your copy of the sample report and make an informed decision about whether the full report will provide you with the insights and information you need.

Below is a sample report to understand what you are buying

See what our customers are saying

“The GlobalData platform is our go-to tool for intelligence services. GlobalData provides an easy way to access comprehensive intelligence data around multiple sectors, which essentially makes it a one-for-all intelligence platform, for tendering and approaching customers.

GlobalData is very customer orientated, with a high degree of personalised services, which benefits everyday use. The highly detailed project intelligence and forecast reports can be utilised across multiple departments and workflow scopes, from operational to strategic level, and often support strategic decisions. GlobalData Analytics and visualisation solutions has contributed positively when preparing management presentations and strategic papers.”

Business Intelligence & Marketing Manager, SAL Heavy Lift

“COVID-19 has caused significant interference to our business and the COVID-19 intelligence from GlobalData has helped us reach better decisions around strategy. These two highlights have helped enormously to understand the projections into the future concerning our business units, we also utilise the project database to source new projects for Liebherr-Werk to use as an additional source to pitch for new business.”

Market Analyst & Management, Liebherr-Werk

Your daily news has saved me a lot of time and keeps me up-to-date with what is happening in the market, I like that you almost always have a link to the source origin. We also use your market data in our Strategic Business Process to support our business decisions. By having everything in one place on the Intelligence Center it has saved me a lot of time versus looking on different sources, the alert function also helps with this.

Head of Key Accounts, Saab AB

Having used several other market research companies, I find that GlobalData manages to provide that ‘difficult-to-get’ market data that others can’t, as well as very diverse and complete consumer surveys.

Marketing Intelligence Manager, Portugal Foods

Our experience with GlobalData has been very good, from the platform itself to the people. I find that the analysts and the account team have a high level of customer focus and responsiveness and therefore I can always rely on. The platform is more holistic than other providers. It is convenient and almost like a one stop shop. The pricing suite is highly competitive and value for our organisation.

I like reports that inform new segments such as the analysis on generation Z, millennials, the impact of COVID 19 to our banking customers and their new channel habits. Secondly the specialist insight on affluent sector significantly increases our understanding about this group of customers. The combination of those give us depth and breadth of the evolving market.

I’m in the business of answering and helping people make decisions so with the intelligence center I can do that, effectively and efficiently. I can share quickly key insights that answer and satisfy our country stakeholders by giving them many quality studies and primary research about competitive landscape beyond the outlook of our bank. It helps me be seen as an advisory partner and that makes a big difference. A big benefit of our subscription is that no one holds the whole data and because it allows so many people, so many different parts of our organisation have access, it enables all teams to have the same level of knowledge and decision support.

Head of Customer Insight and Research, Standard Chartered

“I know that I can always rely on Globaldata’s work when I’m searching for the right consumer and market insights. I use Globaldata insights to understand the changing market & consumer landscape and help create better taste & wellbeing solutions for our customers in food, beverage and healthcare industries.

Globaldata has the right data and the reports are of very high quality compared to your competitors. Globaldata not only has overall market sizes & consumer insights on food & beverages but also provides insights at the ingredient & flavour level. That is key for B2B companies like Givaudan. This way we understand our customers’ business and also gain insight to our unique industry”

Head of Consumer Sensory Insights, Givaudan

GlobalData provides a great range of information and reports on various sectors that is highly relevant, timely, easy to access and utilise.  The reports and data dashboards help engagement with clients; they provide valuable industry and market insights that can enrich client conversations and can help in the shaping of value propositions. Moreover, using GlobalData products has helped increase my knowledge of the finance sector, the players within it, and the general threats and opportunities.

I find the consumer surveys that are carried out to be extremely beneficial and not something I have seen anywhere else. They provided an insightful view of why and which consumers take (or don’t) particular financial products. This can help shape conversations with clients to ensure they make the right strategic decisions for their business.

One of the challenges I have found is that data in the payments space is often piecemeal. With GD all of the data I need is in one place, but it also comes with additional market reports that provide useful extra context and information. Having the ability to set-up alerts on relevant movements in the industry, be it competitors or customers, and have them emailed directly to me, ensures I get early sight of industry activity and don’t have to search for news.

Senior Account Manager, TSYS
Go even deeper with GlobalData Intelligence Center

Every Company Report we produce is powered by the GlobalData Intelligence Center.

Subscribing to our intelligence platform means you can monitor developments at Gene Therapy in Neurology in real time.

  • Access a live Gene Therapy in Neurology dashboard for 12 months, with up-to-the-minute insights.
  • Fuel your decision making with real-time deal coverage and media activity.
  • Turn insights on financials, deals, products and pipelines into powerful agents of commercial advantage.