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Advanced Therapy Medicinal Products CDMO Industry is Rising Rapidly – BioSpace
According to latest study, the global advanced therapy medicinal products CDMO Market size was valued at USD 6.10 billion in 2023 and is projected to reach USD 34.53 billion by 2033, growing at a CAGR of 18.93% from 2024 to 2033.
Key Takeaways:
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owing to risingclinical trialsfor advanced therapy medicinal products and the increasing awareness among researchers about the benefits of advanced therapies, driving the advanced therapy medicinal products (ATMP) CDMO market growth. Tissue engineering has greatly benefited in recent years from technological development. The damaged tissues and organ function are replaced or restored using this technique. Similarly, gene and cell therapy are attracting a lot of patients for the treatment of rare diseases, whose incidence is rising globally.
With rising demand for robust disease treatment therapies, key players have focused their efforts to ramp up research and development for effective gene therapies that target the cause of disorder at a genomic level. According to ASGCT, the number of cell and gene therapies in the U.S. pipeline programs (phase I-III trials) increased from 483 in 2021 to 529 in 2022. Furthermore, the FDA delivers constant support for innovations in the gene therapy field via a number of policies with regard to product manufacturing. In January 2020, the agency released six final guidelines on the manufacturing and clinical development of safe & efficient gene therapy products.
Moreover, awareness about ATMP treatment options is being driven by initiatives aimed at informing the public about the benefits of these products, which, in turn, is leading to increased adoption of advanced therapies and fueling market growth for CDMOs. For instance, Alliance for Regenerative Medicine Foundation for Cell and Gene Medicine prioritizes activities for increasing public awareness through educational programs, underlining the clinical & societal benefits of regenerative medicine.
Increasing clinical trial activity along with new product launches generates growth opportunities for the market. As of 2022, there are 1451 ATMPs in preclinical stages and 535 are being studied in Phase 1 to 3 studies. Since August 2020, EMA has approved six of these additional ATMPs, and five more will be approved by 2023. In the UK, there were approximately 168 advanced therapy medicinal product trials underway in 2021, up from the 154 studies reported the year before, which is a 9% increase. 2021 saw a 32% increase in phase 1 trials, indicating a significant shift from experimental medicines to first-in-human studies.
On the other hand, key players are undertaking various strategic initiatives to introduce novel products, which is expected to propel market growth. For instance, in March 2021, CureVac N.V. signed a partnership agreement with Celonic Group, engaged in the manufacture of CVnCoV, CureVacs mRNA-based COVID-19 vaccine candidate. CureVac's COVID-19 vaccine candidate is manufactured at Celonic's commercial manufacturing unit for ATMPs and biologics in Heidelberg, Germany. Under the terms of the commercial supply agreement, the Celonic facility could produce over 100 million doses of CVnCoV.
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Advanced Therapy Medicinal Products CDMO Market Trends
Segments Insights:
Product Insights
The gene therapy segment held the largest share of over 49.11% in 2023. Increase in financial support and rise in number of clinical trials for gene therapies are driving demand for gene therapy segment. In 2020, in the first three quarters, gene therapies attracted financing of over USD 12 billion globally, with around 370 clinical trials underway. Additionally, in mid-2022, approximately 2,000 gene therapies were in development, targeting several therapeutic areas, such as neurological, cancer, cardiovascular, blood, and infectious diseases.
The cell therapy segment is expected to show lucrative growth over the forecast period. The field of cellular therapeutics is constantly advancing with inclusion of new cell types, which, in turn, provides ample opportunities for companies to enhance their market positions. Furthermore, the market is attracting new entrants due to high unmet demand for cell therapy manufacturing, the recent approval of advanced therapies, and proven effectiveness of these products.
Indication Insights
The oncology segment accounted for the largest revenue share in 2023. The segments dominance is attributed to disease burden, strategic initiatives undertaken by key players, and availability of advanced therapies used for treating various cancer indications. In January 2021, around 18,000 to 19,000 patients and 124,000 patients were estimated to be potential patients for treating cancer using cell & gene therapy products Kymriah (Novartis AG) and Yescarta (Gilead Sciences, Inc.), respectively. Furthermore, a publication on PubMed reports that as of the conclusion of the first quarter of 2023, there have been over 100 distinct gene, cell, and RNA therapies approved globally, along with an additional 3,700-plus in various stages of clinical and preclinical development.
The cardiology segment is estimated to register the fastest CAGR over the forecast period. This is attributed to the increasing prevalence of cardiovascular diseases and research collaboration for development of advanced therapies. For instance, in October 2023, Cleveland Clinic administered a novel gene therapy to the first patient globally as part of a clinical trial, aiming to deliver a functional gene to combat the primary cause of hypertrophic cardiomyopathy (HCM). Similarly, in February 2021, Trizell GmbH entered into partnership with Catalent, Inc. for development of phase 1 cell therapy to treat micro- and macroangiopathy. Trizell's medication is an Advanced Therapy Medicinal Product (ATMP) that employs regulatory macrophagesa platform technology developed in Germany.
Phase Insights
The phase I segment dominated the market in 2023 due to growing R&D activities and increasing number of human trials for advanced therapies. Phase 1 helps ensure the safety levels of a drug at different doses and dosage forms administered to a small number of patients. This phase is mainly conducted to determine the highest dose a patient can take without any adverse effects. Around 70% of drugs in phase 1 move to the next phase.
The phase II segment has been anticipated to show lucrative growth over the forecast period. Phase II clinical studies comprise the largest number of developing ATMPs, due to the high clearance rate of phase I clinical studies. According to data published by Alliance for Regenerative Medicine, as of June 2022, more than 2,093 clinical trials are ongoing globally, out of which 1,117 are under phase II clinical trials accounting for 53%. Thus, the increase in number of products in phase II is driving the segment.
Regional Insights
North America dominated the overall market share of 49.11% in 2023. This can be attributed to increasing outsourcing activities and rising awareness about advanced therapy. North America has consistently been a leader in R&D for advanced treatments, and it is anticipated that it will keep this position during the forecast period. Recent approvals of products such as Kymriah and Yescarta have propelled investments in the regional market. Moreover, in March 2021, the U.S. FDA approved Abecma, the first approval of CAR-T cells to fight against cancer. Similarly, in December 2023, Casgevy and Lyfgenia, the initial cell-based gene therapies for sickle cell disease (SCD) in patients aged 12 and above, received approval from the U.S. Food and Drug Administration, marking a significant milestone.
The U.S. accounted for the largest share of the global market in the North America region in 2023. The U.S. maintains dominance in this sector due to the presence of a robust and highly advanced biopharmaceutical industry with a considerable focus on research and development. Additionally, the continuous presence of numerous pharmaceutical and biotechnology companies, along with academic and research institutions, generates a sustained demand for rigorous safety testing, further reinforcing the country's leadership in the field.
The Asia Pacific region is expected to grow at the fastest CAGR over the forecast period due to the increasing demand for novel ATMPs and rising R&D activities to develop novel therapies. Moreover, the market growth is driven by continuously expanding CDMO Cell Therapy in the country, a number of domestic players have collaborated with biotech companies from other countries involved in mesenchymal stem cell research and therapy development. In addition, in September 2022 Takara Bio, Inc. launched CDMO Cell Therapy for gene therapy products using siTCR technology for its genetically modified T-cell therapy products.
China accounted for the largest share of the global market in the Asia Pacific region in 2023 due to its strategic focus on advancing research and development capabilities, particularly in the pharmaceutical and biotechnology sectors. Additionally, with a rapidly growing biopharmaceutical industry and supportive government initiatives, China has become a key market for advanced therapy medicinal products (CDMO) market.
Recent Developments
Key Companies & Market Share Insights
Some of the key players operating in the market include AGC Biologics,WuXi Advanced Therapies and Celonic
Minaris Regenerative Medicine and BlueReg are some of the emerging market players in the global market.
Key Advanced Therapy Medicinal Products CDMO Companies:
Segments Covered in the Report
This report forecasts revenue growth at country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Nova one advisor, Inc. has segmented the Advanced Therapy Medicinal Products CDMO market.
By Product
By Phase
By Indication
By Region
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Advanced Therapy Medicinal Products CDMO Industry is Rising Rapidly - BioSpace
What Is CRISPR Gene Editing and How Does It Work?
In 2013, two biochemists published a paper proclaiming theyd discovered a potentially game-changing method of manipulating genes. CRISPR which sounds like a veggie-forward gastro pub won them each a Nobel Prize.
In the years since, CRISPR (or Clustered Regularly Interspaced Short Palindromic Repeats) has lived up to the hype. Its altered the global scientific landscape and raised questions about what kinds of revolutionary changes scientists and healthcare providers could and should pursue.
What if we could make foods allergy-free and crops drought-resistant? What if we could eliminate invasive species and protect against infectious diseases like malaria? What if we could revive extinct species? What if we could remove or repair mutations that cause inherited conditions? Or create custom immunotherapies to treat an individuals cancer?
The prospects are that exciting.
If your understanding of genetics starts and ends with high school biology or the (very fictional) Jurassic Park movies youre not alone. This stuff is complicated. Thats why we asked genomics and immunotherapy expert Timothy Chan, MD, PhD, to break CRISPR down for us, so we can better understand why, over a decade later, its still got researchers so excited.
Before we jump into CRISPR, lets start with the concept of gene editing.
Gene editing is the process of altering genetic material (DNA). That could mean changing a few individual genes or an entire sequence. Research has been ongoing for more than a decade thats looking at using gene editing on mutations that cause serious health conditions in people. The goal of this gene editing research is to eliminate or correct the mutation thats causing the health condition, or has the potential to cause one, such as certain cancers. In other research studies, gene editing is being explored so a mutation isnt passed down to children at birth.
For example, the U.S. Food and Drug Administration (FDA) approved a gene therapy in late 2022 that introduces a gene needed for blood clotting into people with hemophilia B. Its one of several cellular and gene therapy products currently in use today.
There are many different techniques and applications for gene editing. CRISPR is one approach to gene editing thats showing promise in ongoing clinical trials.
Now that were clear on what gene editing is, lets focus on a specific approach: CRISPR.
Clustered Regularly Interspaced Short Palindromic Repeats, otherwise known as CRISPR, was originally identified in bacteria, as a bacterial defense system, says Dr. Chan.
Thats right. Bacteria have immune systems, too.
CRISPR contains spacers sequences of DNA left over from unfriendly viruses or other entities as well as repeating sections of genetic material. Those sequences provide acquired immunity, and form the building blocks of the gene editing system or process. It creates a sort of blueprint that allows enzymes in genetic material to make changes to sequences of DNA in living cells. One of the best-known enzymes used for this purpose is called Cas9, which is why youll sometimes hear people talk about CRISPR-Cas9.
Over the years, people have discovered that specific enzymes that allow CRISPR to work Cas9 is one of them.But there are other ones, and they can be tailored to target sequences of interest in the DNA for specific cuts to be made, Dr. Chan explains.
You can think of the underlying mechanism of CRISPR gene editing as being similar to the way magnetic shapes are drawn to each other or the way Lego blocks fit together.
The segments in CRISPR are transcribed into RNA. This RNA includes a guide sequence, which is a match to existing DNA in a persons body.
That guide sequence can be tailored to whatever you want, Dr. Chan says. And as a result, you can make specific alterations or mutations in a part of the genome that you are targeting with a high degree of accuracy.
Along for the ride with this guide sequence is an enzyme like Cas9.
When the guide sequence and enzyme find the desired DNA to edit, the enzyme can then get down to business. It attaches itself to this DNA and makes changes, whether thats a cut or alteration.
CRISPR technology has come a long way, Dr. Chan says. The first generation of CRISPR was a great way to inactivate genes. It only made a break in genes. Then, the DNA would get filled up with natural repair enzymes.
But new versions of CRISPR like CRISPR prime or CRISPR HD are more advanced.
These can allow actual replacements to occur, Dr. Chan continues. You can even very accurately replace one sequence one of the letters in the genome with another letter. And you can make specific mutations.
CRISPRs ability to make very specific, very small cuts has the potential to transform how healthcare providers can address certain genetic diseases.
Dr. Chan is optimistic about the future of CRISPR based on the success of ongoing clinical trials in human subjects. For any type of genetic diseases caused by a single mutated gene, you can use CRISPR to mutate it and make it normal. Thats why its useful. Its a way for us to change errors in the genome.
Right now, CRISPR is geared toward correcting a single change in genes, he adds. While combinations may be possible in the future, were just not there yet.
While gene editing is already in use, CRISPR is still in the clinical trials phase, Dr. Chan says. Its used all the time in research laboratories and industries, he notes. Many clinical trials are testing CRISPR in the setting of genetic diseases and cancer.
Interestingly, CRISPR can be used to detect certain diseases. The best-known example is the Sherlock CRISPR SARS-CoV-2 Kit: A COVID-19 test that received emergency use authorization (EAU) from the FDA in 2020.
But theres no FDA-approved CRISPR therapy right now. The clinical trials are ongoing, he says.
These include trials looking at CRISPR to correct genetic diseases such as cystic fibrosis, Huntingtons disease and muscular dystrophy.
Dr. Chan adds that there are also major clinical trials in process for blood disorders, where CRISPR is being used to correct the gene alteration that causes the condition. As one example, he cites a promising trial looking at CRISPR-Cas9 gene editing for sickle cell disease and -thalassemia, written about in an early 2021 issue of the New England Journal of Medicine. -thalassemia is an inherited blood disorder that impacts the bodys ability to create hemoglobin an iron-dense protein that serves as the primary ingredient in red blood cells.
There are also clinical trials looking to see if CRISPR can be used to treat certain cancers. Dr. Chan notes that chimeric antigen receptor (CAR)T-cell therapy is one of the first gene therapies approved for leukemias. Current research is looking at whether CRISPR technology can make this treatment even more effective.
In CAR T-cell therapy, you take out T-cells from someone and put in a receptor a new way for these cells to target something on cancer cells and then put these cells back in the patient, he explains. Researchers are running trials now where they use CRISPR to alter those T-cells to make them even more active.
CRISPR therapies can take on many different forms. CRISPR has been inserted directly into the body before. It was famously injected into the eyes of seven people with a rare hereditary blindness disorder in 2020, two of whom later told NPR that they regained some ability to see colors. There are human trials in process right now that deliver CRISPR through gels and creams, through food or drink, skin grafts or injections. Ex-vivo delivery is also common: Thats when CRISPR is used to modify a cell outside the body. The cells are then re-inserted into the body using a harmless virus.
The results have been promising so far. I do believe in the next three to five years possibly even sooner were going to see approval to treat some diseases, Dr. Chan states.
With any type of CRISPR therapy, Dr. Chan says theres a risk of getting off-target effects or unexpected side effects.
Whenever youre altering something as fundamental as DNA, you just dont know what might happen he explains. Theres always a chance for the unexpected. You can potentially have effects on your DNA that were not intended.
At the moment, he doesnt have any specific examples of what these effects might be and he notes that existing research suggests the risk is pretty low. Still, data from future research might tell a different story.
Dr. Chan nevertheless sees a lot of potential for CRISPR in the coming years.
The field is moving very quickly, he says. Were seeing continual improvement of the actual CRISPR tools being used.
Its getting more accurate and more flexible in terms of what you can do. There are various engineered modified variants of CRISPR now that are allowing very specific, very accurate changes with fewer off-target effects. So, I think the future is very bright.
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What Is CRISPR Gene Editing and How Does It Work?