PharmaSphere: Emerging Biotechnologies – Gene Therapy Market Analysis

Pages: 101 Published: June 01, 2015 Report Code: GDHC014PSR

GlobalData’s PharmaSphere: Emerging Biotechnologies – Gene Therapy Market Analysis report is an essential source of information and analysis on the gene therapy field. Using detailed company data, deal analysis, corporate strategy, and market trends analysis, GlobalData provides an in-depth analysis of the current and future growth drivers of the gene therapy market. The report discusses the key factors shaping and driving the gene therapy business, and provides insights on the competitive landscape and emerging strategies expected to significantly influence the market positions of companies currently involved in the development and commercialization of gene therapies.


This report provides market developments across the gene therapy field, including clinical trials, deals, and pipeline investments

Investment examined on both the industry and financial communities

Broad discussion on the science behind gene therapies, viral vectors, and gene delivery

Clinical trials activity examined across phase of development, therapy area, and indication.

This report offers a deep dive into the synergies behind partnering and acquisition activity

Deals activity examined across geography, entity type, company, financial advisor, phase of development, therapy area, indication, and mechanism of action

Pipeline profiles of 20 leading biotech companies developing gene therapies

Reasons to Buy


Key Players

ALS Therapy Development Institute
Amarna Therapeutics
American Gene Technologies
AnGes MG
Applied Genetic Technologies International
Asklepios BioPharmaceutical
Avalanche Biotechnologies
Benitec BioPharma
Bluebird bio
CFR Pharmaceuticals
Chiesi Farmaceutici
Dimension Therapeutics
GeneCure Biotechnologies
GenSight Biologics
Oxford BioMedica
ReGenX Biosciences
Sangamo BioSciences
Spark Therapeutics
Tacere Therapeutics
and Voyager Therapeutics.

Table of Contents

1Table of Contents

1.1List of Tables

1.2List of Figures


2.1Report Scope

2.2Companies Mentioned

2.3Upcoming Reports

2.4Recently Published Reports

3Gene Therapy Overview

3.1What is Gene Therapy?

3.2Gene Delivery

3.2.1Viral Vectors

3.2.2Non-Viral Vectors and Naked DNA

4Market Developments

4.1Turning Point: Glybera Approval

4.2GSK Files Application to the EMA for Gene Therapy for ADA-SCID

4.3Celladon’s Reports Negative Results for Mydicar in Advanced Heart Failure

4.4Spark Initiates Phase I/II Clinical Trial of SPK-CHM for Choroideremia

4.5FDA Accepts Sangamo’s IND to Begin Trial of Gene Therapy for Beta-Thalassemia

4.6Paying for Gene Therapies: Annuity Model

5Clinical Trials



5.3Phase of Development

5.4Therapy Area





6Company Analysis


6.1.1Entity Type

6.1.2Regional Split

6.2Financial Highlights


6.2.2R&D Spending

6.2.3Market Capitalization

6.2.4Stock Performance

6.3Pipeline Profiles


6.3.2ALS Therapy Development Institute

6.3.3Amarna Therapeutics

6.3.4American Gene Technologies International

6.3.5AnGes MG

6.3.6Applied Genetic Technologies Corporation

6.3.7Asklepios BioPharmaceutical

6.3.8Avalanche Biotechnologies

6.3.9Bluebird bio


6.3.11Dimension Therapeutics

6.3.12GeneCure Biotechnologies


6.3.14GenSight Biologics

6.3.15Oxford BioMedica

6.3.16ReGenX Biosciences

6.3.17Sangamo BioSciences

6.3.18Spark Therapeutics


6.3.20Voyager Therapeutics

7Deals Landscape



7.1.2Entity Type



7.1.5Therapy Area


7.1.7Mechanism of Action

7.2Deal Type

7.2.1Mergers and Acquisitions

7.2.2Licensing and Partnerships

7.2.3Capital Raisings

8Strategic Outlook



9.2Research Methodology


9.2.2Secondary Research

9.3About the Author

9.3.1Adam Dion, Senior Industry Analyst

9.4Global Head of Healthcare

9.5About the Industry Dynamics Team

9.6About GlobalData

9.7Disclosure Information

9.8Contact Us


List of Tables

Table 1: Common Diseases for Gene Therapy, Defects and Target Cells

Table 2: Main Groups of Viral Vectors and Their Characteristics

Table 3: Advantagene

Table 4: ALS Therapy Development Institute

Table 5: Amarna Therapeutics

Table 6: American Gene Technologies International

Table 7: AnGes MG

Table 8: Applied Genetic Technologies Corporation

Table 9: Asklepios BioPharmaceutical

Table 10: Avalanche Biotechnologies

Table 11: Bluebird bio

Table 12: Celladon

Table 13: Dimension Therapeutics

Table 14: GeneCure Biotechnologies

Table 15: Genethon

Table 16: GenSight Biologics

Table 17: Oxford BioMedica

Table 18: ReGenX Biosciences

Table 19: Sangamo BioSciences

Table 20: Spark Therapeutics

Table 21: UniQure

Table 22: Voyager Therapeutics

Table 23: Gene Therapy, M&A Deals Table Summary, 2010–2014

Table 24: Gene Therapy, Licensing Deals Table Summary, 2009–2015

Table 25: Gene Therapy, Capital Raisings Table Summary, 2013–2015

List of Figures

Figure 1: Number of Gene Therapy Clinical Trials, 2009–2014

Figure 2: Number of Gene Therapy Clinical Trials by Indication and Stage

Figure 3:Gene Therapy Clinical Trials by Company

Figure 4: Gene Therapy Clinical Trials by Phase of Development

Figure 5: Gene Therapy Clinical Trials by Therapy Area

Figure 6: Gene Therapy Clinical Trials, Percentage by Therapy Area

Figure 7: Gene Therapy Clinical Trials, Top Indications

Figure 8: Investigators by Specialty, Gene Therapy

Figure 9: Investigators by Country, Gene Therapy

Figure 10: Gene Therapy Companies, Entity Type

Figure 11: Gene Therapy Companies, Regional Split

Figure 12: Gene Therapy Companies by Revenue ($m), 2013

Figure 13:Gene Therapy Companies by R&D Spending ($m), 2013

Figure 14: Gene Therapy Companies by Market Capitalization ($m)

Figure 15: Leading Gene Therapy Companies, Stock Performance ($USD)

Figure 16: Gene Therapy Deals, Total Number of Deals and Deal Values ($m), 2009–2015

Figure 17: Gene Therapy Deals, Deal Value ($m) by DealType, 2014

Figure 18: Gene Therapy Deals, Number of Deals and Deal Values ($m) by Region, 2009–2015

Figure 19: Gene Therapy Deals by Region, 2009–2015

Figure 20: Gene Therapy Deals by Region, Deal Value ($m), 2009–2015

Figure 21: Gene Therapy Deals by Entity Type, Private vs. Public, 2009–2015

Figure 22: Gene Therapy Deals, Number of Deals and Deal Values ($m) by Company, 2009–2015

Figure 23: Gene Therapy Deals, Number of Deals and Deal Values ($m), by Product Stage, 2009–2015

Figure 24: Gene Therapy Deals, Number of Deals and Deal Values ($m), by Therapy Area, 2009–2015

Figure 25: Gene Therapy Deals by Indication, (>$500m), 2009–2015

Figure 26: Gene Therapy Deals by Indication, (<$500m), 2009–2015

Figure 27: Gene Therapy Deals, Number of Deals and Deal Values ($m) by Mechanism of Action, 2009–2015

Figure 28: Gene Therapy Deals, Number of M&A Deals and Deal Values ($m), 2010–2015

Figure 29: Gene Therapy, Number of Licensing Deals and Deal Values ($m), 2009–2015

Figure 30: Gene Therapy, Number of Capital Raisings and Deal Values ($m), 2009–2015

Figure 31: Gene Therapy, Number of Capital Raisings by Financial Advisor, 2009–2015


Can be used by individual purchaser only


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

Get in touch to find out about our multi-purchase discounts
Tel +44 (0) 20 7947 2960

Every customer’s requirement is unique. We understand that and can customize the report basis your exact research requirements pertaining to market insights, innovation insights, strategy and planning, and competitive intelligence. You can also avail the option of purchasing stand-alone sections of the report or request for a country specific report.

GDPR + CCPA Compliant

Personal and transaction information are kept safe from unauthorised use.

Recently Viewed Reports


“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
Looking to stay on top of industry & market trends?

Sign up to receive regular alerts for our latest analysis and reports. No matter your industry focus, you can keep your finger on the pulse with our timely updates.

Net Present Value Model: (borneol + edaravone)
$500 | November 2022
Net Present Value Model: (Aspirin + Dipyridamole) ER
$500 | November 2022