Presently, there are more than 10 approved gene therapies; over 465 product candidates are being evaluated for the treatment of a variety of disease indications

Gene Therapy Market is projected to grow at an annualized rate of 4...

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA® (AveXis) and ZYNTEGLO™ (bluebird bio).

Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

The USD 11.6 billion (by 2030) financial opportunity within the Gene Therapy Market has been analyzed across the following segments:

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Key therapeutic areas
• Autoimmune disorders
• Cardiovascular diseases
• Genetic disorders
• Hematological disorders
• Metabolic disorders
• Ophthalmic disorders
• Oncological disorders
• Others

Type of vector
• Adeno associated virus
• Adenovirus
• Herpes simplex virus type 1
• Lentivirus
• Plasmid DNA
• Retrovirus
• Vaccinia Virus

Type of therapy
• Ex vivo
• In vivo

Type of gene modification
• Gene augmentation
• Immunotherapy
• Oncolytic therapy
• Others

Route of administration
• Intraarticular
• Intracerebellar
• Intramuscular
• Intradermal
• Intravenous
• Intravitreal
• Intravesical
• Subretinal
• Others

Key geographical regions
• North America
• Europe
• Asia-Pacific

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The Gene Therapy Market (3rd Edition), 2019-2030 report features the following companies, which we identified to be key players in this domain:
• Advantagene
• Advaxis
• BioMarin
• bluebird bio
• FKD Therapies
• Freeline Therapeutics
• GenSight Biologics
• Gradalis
• Inovio Pharmaceuticals
• Marsala Biotech
• Orchard Therapeutics
• Pfizer
• Sarepta Therapeutics
• Spark Therapeutics
• Tocagen
• Transgene
• uniQure Biopharma
• VBL Therapeutics
• ViroMed

Chapter Outline

Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

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