The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions
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.
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Key Market Insights
Around 470 gene therapies are currently under development
Nearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.
More than 30% of clinical stage pipeline therapies are being designed for treating oncological disorders
Considering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.
Over 60% of gene therapy developers are based in North America
Of the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.
More than 31,000 patents have been filed / published related to gene therapies, since 2016
Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.
USD 16.5 billion has been invested by both private and public investors, since 2014
Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.
30+ mergers / acquisitions have been established between 2014 and 2019
Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).
North America and Europe are anticipated to capture over 85% of market share by 2030
With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.
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Key Questions Answered
- Who are the leading industry players in this market?
- How big is the development pipeline and which new indications are coming in focus? Which vectors are being used for effective delivery of the therapeutic agents?
- Who are the key investors in the gene therapy market?
- How is the current and future market opportunity likely to be distributed across key market segments?
- What kind of commercialization strategies are being adopted by gene therapy developers?
- What are the different pricing models and reimbursement strategies used for gene therapies?
- What are the prevalent R&D trends related to gene therapies?
- What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?
- Who are the key CMOs / CDMOs that claim to supply viral / plasmid vectors for gene therapy development?
The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:
- Key therapeutic areas
- Cardiovascular disorders
- Muscle-related disorders
- Nervous system disorders
- Ophthalmic disorders
- Oncological disorders
- Type of vector
- Adeno associated virus
- Plasmid DNA
- Type of therapy
- Ex vivo
- In vivo
- Type of gene modification
- Gene augmentation
- Oncolytic viral therapy
- Route of administration
- Key geographical regions
- Rest of the World
The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:
- Adam Rogers (CEO, Hemera Biosciences)
- Al Hawkins (CEO, Milo Biotechnology)
- Buel Dan Rodgers (Founder & CEO, AAVogen)
- Cedric Szpirer (Executive & Scientific Director, Delphi Genetics)
- Christopher Reinhard (CEO and Chairman, Cardium Therapeutics)
- Ryo Kubota (Chairman, President and Chief Executive Officer, Acucela)
- Jeffrey Hung (CCO, Vigene Biosciences)
- Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory)
- Michael Triplett (CEO, Myonexus Therapeutics, acquired by Sarepta Therapeutics)
- Robert Jan Lamers (CEO, Arthrogen)
- Tom Wilton (Chief Business Officer, LogicBio Therapeutics)
The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).
- bluebird bio
- FKD Therapies
- Freeline Therapeutics
- GenSight Biologics
- Inovio Pharmaceuticals
- Marsala Biotech
- Orchard Therapeutics
- Sarepta Therapeutics
- Spark Therapeutics
- uniQure Biopharma
- VBL Therapeutics
For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html or email email@example.com
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SOURCE Roots Analysis