Cell and Gene Therapy Market (By Therapy Type: Gene Therapy, Cell Therapy; By Application: Oncology, Genetic Disorders, Dermatology Disorders, Musculoskeletal Disorders, Others; End-Users: Hospitals, Cancer Care Centers, Wound Care Centers, Others) - Global Industry Analysis, Size, Share, Growth, Trends, Regional Outlook, and Forecast 2022-2030

The cell and gene therapy market size was valued at USD 5.9 billion in 2021 and is expected to hit around USD 42.56 billion by 2030, growing at a CAGR of 39.42 % during the forecast period 2022 to 2030.

Cell and gene therapies (C&GT) continue to be at the very center of healthcare innovation and are the fastest-growing areas of therapeutics, having already contributed to some of the most significant disruptions in the pharmaceutical and biotech indus­tries. Continuous advancements in C&GT are transforming how certain diseases are treated and potentially cured, and signifi­cantly changing healthcare outcomes. So, what’s next? Where is C&GT headed? The following explores the industry dynamics that will shape the C&GT space throughout the next decade.

COST VERSUS VALUE

The US Food and Drug Administration has approved seven cell and gene therapy drugs, but the new product pipeline has approximately 1,200 experimental therapies, with more than half of these in Phase 2 clinical trials. CAGR estimates are about 15% for cell therapies and nearly 30% for gene therapies, and the Al­liance for Regenerative Medicine points to a rapid rise in cell and gene therapy developers, with 1,100 such ventures in 2020, an increase of about 10% from 2019.1-3 The industry is still transi­tioning out of the early development phase and sits at the very start of the rapid growth phase with a lot of opportunity ahead.

Showing the therapies are providing a lifelong cure and not just another type of ongoing treatment will be key to justifying the pricing and gaining acceptance from patients, providers, and payors, be they in­surance or governmental. Unfortunately, when new cell and gene products hit the market, the discussion in media often fo­cuses on the price tag instead of highlight­ing the availability of a new therapy with the potential to cure instead of simply managing what may have been a previ­ously untreatable disease.

This is creating increased pressure to drive down manufacturing costs – a com­mon challenge across the pharma and biotech industries. In C&GT, this effort will be focused on several fronts, such as in­creasing batch sizes, standardizing and streamlining processes, and reducing the cost of the materials used in manufactur­ing. This will go hand in hand with devel­oping processes that provide high yields of consistent product.

The markets will also have an influ­ence. Where there is a broader market upon which to spread the development costs, such as cancers, where there is usu­ally a larger patient population, therapies can hit a lower price point. For the rare or orphan diseases, it will be harder to spread out costs. But there is potential to achieve lower costs in the long run, as in these cases, it may be fruitful to have a platform, such as a particular viral vector, upon which multiple therapeutics can be more easily developed, thereby reducing some of R&D costs.

The following factors are likely to contribute to the growth of the cell and gene therapy market

• Merger & Acquisition
• Expansion of Manufacturing Plants & Technological Advancement
• Expanding Application for Cell & Gene Therapies
• Growing Demand for CAR T- Cell Therapies
• New Products Approvals & Increasing Pipeline Products

In the cell and gene therapy field, gene therapy gathered the pace last from 2 decades because of the discovery of several genes responsible for mutation in various diseases. The advancement in the cell & gene therapy field and innovative technologies give the new era for biological therapeutics. Also, PRIME Designation and marketing authorization for products provide a new opportunity for the manufacturer’s financing and revenue generation.

CHALLENGES AROUND CAPACITY, TECHNICAL CAPABILITIES & EXPERTISE

Physical capacity to produce gene and cell therapy products continues to be a bottleneck. Even as more capacity is added, the industry seems to be growing even faster. Some of the expansion is being hampered by the lack of availability of materials – the supply of items, such as plasmids, media, and the numerous dis­posables from pipettes to bioreactors, has continued to be tight.

The solution will, in part, come from increasing the production of these materi­als. There also has to be a greater focus on learning how to better utilize the supply so more product can be manufactured with the same amount of material.

The ability to scale up the manufac­ture of therapies in a cost-effective manner is also essential. The industry must find systems that can be run as a “small” ver­sion for the preclinical and early trial work but then be scaled up into larger equip­ment for commercial use, rather than sim­ply scaling volumes by duplicating small systems that inherently uses more re­sources and physical space within facilities.

The availability of a workforce with appropriate skills is also going to continue to be a challenge. Currently, the industry is largely clustered in a few locations – in the US, there are a few key metropolitan areas on the east and west coasts that have very high concentrations of C&GT companies. As a result, the companies in these locations are running into problems finding the workers they need. This com­petition for the limited number of workers is driving up wages and that is anathema to the idea of reducing costs. It also means projects are delayed because the expertise and workers needed to do the work are not available. There may be several ap­proaches to solving this. The industry may expand into locations other than the east and west coasts; a longer-term solution however would involve federal, state, and local governments supporting and funding dedicated life science workforces. The cities, states, and countries that invest in educating and training a workforce will at­tract the companies and the jobs and tax dollars they bring. Any country that wants to stay in the forefront of this industry must be able to supply the workers, otherwise they will see the industry and jobs move elsewhere.

REGULATORY APPROACHES

A decade ago, the new treatments in the G&CT space advancing to the clinic sparked excitement about the eventual path to commercialization. This spurred regulatory agencies to work with re­searchers and companies to move these treatments forward, but given the un­charted waters, even the questions to be asked were evolving. Now, some of these treatments have been approved, and par­ties on both sides of the regulatory equa­tion have learned how to evaluate the development phase and the important regulatory factors and questions. For ex­ample, progress in regulatory perspectives has led to a preference for a single pro­ducer cell line clone for use through all de­velopment phases.

Regulators, such as the FDA and the European Medicines Agency (EMA), have published formal and informal guidelines to help viral vector companies produce products. Due to the evolving nature of the C&GT industry, regulations surrounding these products are updated to keep up with the rapidly growing field and its development – for example, the FDA has provided or updated nine guidance documents within the last 2 years.4

This will continue to evolve, and each new type of therapy will bring its own learning curve to the regulatory process. Moreover, it will continue to be important for gene therapy developers and manu­facturers to work hand in hand with regu­latory agencies as more open discussions around issues, challenges, and best prac­tice will put the industry in a better position to collaborate on any future regulations and guidance.

DRIVING THE INDUSTRY FORWARD

If the industry wants to keep moving up the steep growth curve, then capacity and raw materials must be readily avail­able. The innovations in therapies, plat­forms, and processes will all come with time, money and increasing availability of resources.

Investment into basic research at the university level and into small start-ups coming from venture capital, private eq­uity, industry, and the capital markets will be essential – university labs and small start-ups are well-positioned for the needed innovation. Invariably, these com­panies start out with a small team that is willing to take risks and then grow into larger companies or go through a series of acquisitions that bring those innovations into larger biotechs and big pharma. These larger entities can use their resources and expertise to commercialize the product and get it to market.

Outsourcing to niche CDMOs with the smaller scale capabilities and expertise required to develop scalable processes at this stage will be essential to expediting a C&GT’s path to market.

KEY HIGHLIGHTS

• As per the American Society of Cell + Gene Therapy report in 2021, increasing the number of cellular and gene therapy products, application rate and products in clinical trials drive the market growth.
• As per the Dive Biopharma report 2021, biotech companies who actively engaged in regenerative medicines and therapies reported USD 14 billion funding only in six months of 2021 which was reported to be USD 19.9 billion for the overall year.

WHAT’S NEXT?

Throughout the past 5 or 6 years, a lot of the excitement, especially for in­vestors, has been around cancer thera­pies. That work has largely been on bloodborne cancers, but there are now approaches being applied to solid tumor therapies. There is likely to be a continued focus on LV and it being used in other CAR-T and Natural Killer cell therapies both for bloodborne cancers as well as solid tumors.

As previously noted, most LV vectors currently use the VSVG envelope, and one area of innovation will involve developing other envelopes to see if there are ways to use LV vectors in vivo and targeting specific organs or used in combination with some immune suppression to reduce the im­mune response to the virus.

Other platforms will likely develop over time. There has been interest in foamy viruses – a RV belonging to the Spumavirus genus – but they proved ex­tremely difficult to make. Recently, there has been a great deal of interest in using bocavirus as a vector platform, it shows promise in systemic applications like an AAV, but with the added benefit of being capable of carrying a transgene almost as large as that of an LV vector. However, in­novation in the space must be facilitated through investment and addressing sev­eral challenges that are currently ham­stringing progress.

CELL AND GENE THERAPY MARKET SEGMENTATION

• Increasing application of gene therapies in diseases diagnosis and rapidly growing new drugs applications will give new market space in upcoming years.
• In 2020, around USD 2.3 billion funding was reported only from private companies for gene therapies. 
• By 2025, the FDA is expected to approve 10?20 products each year, driving the global cell and gene therapy manufacturing market.
• In 2020, Medicine in Development Report 2020, around 176 products were reported in cancer therapies in development procedures.

GEOGRAPHICAL OUTLOOK

North America: High economic status and high expenditure on healthcare services drive the cell and gene therapy market in North America. National Health Institutes, industries, academic institutes, and hospitals are the significant contributors of sponsorship and financial funding for cell and gene therapy products.

Europe: The increasing funding for cell and gene therapy drives the cell and gene therapy market growth consistently in Europe. Around USD 2.6 billion financings were reported in Europe for CGTs in 2020, which increased by 103% compared to previous years. In the cell therapy segment, USD 1.8 billion and in gene therapy, USD 2.3 billion funding accounted in 2020, which increased by 196% and 111% growth respectively

VENDOR LANDSCAPE

• The key players in the cell and gene therapy market are Gilead Sciences, Novartis, Smith Nephew, Amgen, Organogenesis, Roche (Spark Therapeutics), Dendreon, Vericel, and Bristol-Myers Squibb Company.
• An increasing number of mergers and acquisitions gives new potential to market growth. Gilead Sciences acquired Kite Pharma in 2020. Also, Novartis acquired Avexis in 2018, and Smith & Nephew acquired Osiris Therapeutics.

KEY QUESTIONS ANSWERED:

1. How big is the global cell & gene therapy market?
2. What are the latest trends in the cell & gene therapy market?
3. Who are the key players in the cell & gene therapy market?
4. Which region has the highest share in the cell & gene therapy market?
5. Who are the end-users of the cell & gene therapy market?

Market Segmentation

By Therapy Type

• Gene Therapy
• Cell Therapy

By Application

• Oncology
• Genetic Disorders
• Dermatology Disorders
• Musculoskeletal Disorders
• Others

By End-Users

• Hospitals
• Cancer Care Centers
• Wound Care Centers
• Others

Key Vendors

• Gilead sciences
• Novartis
• Smith Nephew (Osiris Therapeutic)
• Amgen
• Organogenesis
• Roche (Spark Therapeutics)
• Dendreon
• Vericel
• Bristol-Myers Squibb

Other Prominent Vendors

• Abeona Therapeutics
• APAC Biotech Pvt Ltd
• Alnylam
• Allovir
• Tego Sciences
• Avita Medical
• Anterogen
• AnGes Inc.
• BioSolution Co. Ltd.
• Cheisi Farmaceutici
• CollPlant
• CO.DON
• Corestem
• Bluebird Bio Inc
• Biosolution
• Stempeutics Research
• GC Cell
• Sanofi
• Gensight biologics
• Human Stem Cells Institute
• JW CreaGene Co. Ltd
• JCR Pharmaceuticals
• Japan Tissue Engineering (J-TEC)
• Kolon TissueGene
• Medipost
• MolMed
• Nuvasive Inc.
• Nipro Corporation
• Orchard Therapeutics
• Orthocell
• Pfizer
• Pharmicell
• Sibiono Genetech
• Shanghai Sunway Biotech
• RMS Regenerative Medical System
• Takeda Pharmaceuticals Company
• Terumo

Upcoming Vendors

• Biomarin Pharmaceutical
• Bellicum Pharmaceutical
• Castle Creek Biosciences Inc
• Libella Gene Therapeutics
• CARsgen Therapeutics
• Hrain Biotechnology Co. Ltd
• Helixmith
• Krystal Biotech Inc.
• JW Therapeutic
• Poseida Therapeutics