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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 45%, till 2030
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.
Chapter 6 includes information on over 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.
Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.
Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the company’s patent portfolio and collaborations focused on its gene therapy product / technology.
Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.
Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any).
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