Given the potential of gene therapies to permanently cure a diverse array of clinical conditions at the genetic level, the current pipeline is rapidly growing; a number of such therapies are currently being evaluated in late stages of development.
Roots Analysis has announced the addition of “Gene Therapy Market (5th Edition), 2022-2035” report to its list of offerings.
Post the emergence of blockbuster gene therapies, such as ZOLGENSMA® (for spinal muscular atrophy type I) and Libmeldy™ (for metachromatic leukodystrophy), this domain has garnered significant interest in the medical community. Moreover, considering the promising trial results of pipeline candidates, notable players have made investments to support product development and commercialization efforts. An analysis of the investments made at various stages, such as seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings, by gene therapy companies that are engaged. An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.
Key Market Insights
Over 1,150 gene therapies are currently being developed across different stages
Apart from 11 approved products, most of the aforementioned therapies (70%) are in early stages of development (preclinical / discovery), while the rest are being evaluated in clinical trials. It is worth mentioning that more than 40% of the clinical stage candidates target oncological diseases. Further, majority of such therapies (66%) use adenovirus vectors as molecular carriers.
More than 250 companies claim to be engaged in the development of gene therapies, globally
In the past decade, over 160 companies have been established in this domain. Of the total, 60% of the players are small firms. In addition, majority (62%) of these gene therapy developers are based in North America, primarily in the US.
290+ clinical trials evaluating gene therapies have been registered, worldwide
Of the total number of trials registered during the period 2015-2020, close to 10% have already been completed, while 56% are presently recruiting patients. Further, presently, around 55% of the trials are evaluating therapy candidates in phase II studies.
USD 8.5+ billion has been invested by both private and public investors, since 2015
The maximum funding amount was raised through secondary offerings (40%), venture capital (28%) and IPOs (16%), during the period 2015-2022. Interestingly, majority (77%) of the funding instances were reported by players based in North America.
Over 5,800 patents have been filed / published related to gene therapies, since 2017
Around 90% of the total number of patent applications were related to gene therapies, while the remaining were focused on gene editing-based therapies. Further, majority of the patent assignees were industry players. However, it is worth mentioning that the contribution of non-industry players in the overall patent filing activity has increased considerably (CAGR of 19%), over the past few years.
Merger and acquisition activity in this market has grown at a CAGR of 22%, during 2015-2021
It is worth mentioning that, across most of the instances (43%) both the acquirer and the acquired company were based in North America. Moreover, majority of the acquisitions (26%) were focused on providing access to novel platform / technology, followed by acquisitions undertaken for therapeutic area consolidation (24%) and drug class consolidation (24%).
The overall market is anticipated to grow at a CAGR of over 18%, during the period 2022-2035
Gene therapies based on gene augmentation currently represent a significant share of the market, and this trend is unlikely to change in the foreseen future, as several such candidates are being evaluated in late stages. North America (primarily the US) is expected to capture 60% of the market share by 2035, in terms of the sales-based revenues.
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Key Questions Answered
- Who are the key industry players engaged in the development of gene therapies?
- How many gene therapy candidates are currently in the development pipeline? Which key disease indications are targeted by such products?
- Which types of vectors are most commonly used for effective delivery of gene therapies?
- What are the key regulatory requirements for gene therapy approval, across various geographies?
- Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of the development cycle?
- What are the different pricing models and reimbursement strategies currently being adopted for 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 engaged in supplying viral / plasmid vectors for gene therapy development?
- What are the key value drivers of the merger and acquisition activity in the gene therapy industry?
- Who are the key stakeholders that have actively made investments in the gene therapy domain?
- Which are the most active trial sites (in terms of number of clinical studies being conducted) for gene therapies?
- How is the current and future market opportunity likely to be distributed across key market segments?
The financial opportunity within the gene therapy market has been analyzed across the following segments:
- Type of Therapy
- Gene augmentation
- Oncolytic viral therapy
- Type of Gene Delivery Method Used
- Ex vivo
- In vivo
- Type of Vector Used
- Adeno-associated virus
- Herpes simplex virus
- Non-viral vectors
- Target Therapeutic Areas
- Cardiovascular diseases
- Dermatological diseases
- Genetic diseases
- Hematological diseases
- Infectious diseases
- Metabolic diseases
- Muscle-related diseases
- Oncological diseases
- Ophthalmic diseases
- Route of Administration
- Key Geographical Regions
- North America
- Rest of the World
The research includes brief profiles of key players (listed below) engaged in the development of gene therapies; each profile features an overview of the therapy, current development status, clinical trials and its results (if available), target indication, route of administration and recent developments (if available).
- Abeona Therapeutics
- BioMarin Pharmaceutical
- bluebird bio
- Castle Creek Biosciences
- CG Oncology
- GenSight Biologics
- Holostem Terapie Avanzate
- Inovio Pharmaceuticals
- Kolon TissueGene
- Krystal Biotech
- Neurophth Therapeutics
- Orchard Therapeutics
- PTC Therapeutics
- Sangamo Therapeutics
- Spark Therapeutics
- VBL Therapeutics
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