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This is an exciting time for the cell and gene therapy industry. Its products are transforming the treatment of cancers and genetic diseases, as well as a host of other illnesses including autoimmune diseases, cardiovascular diseases, musculoskeletal disease, dermatological diseases and many others.

As the science rapidly advances, the industry is proceeding to standardize product manufacturing and commercialization. This includes standardized set-ups and closed automated systems. As this occurs, opportunities are arising for contract manufacturers (CMOs) to play leading roles in this process.

 

There’s much talk about cell and gene therapy but a new report from a leading market research firm says that there’s a growing market over nearly two billion dollars for companies that make the cell and gene therapies.

In 2020, the total global cell and gene therapy BCMO market reached $1,842 million. Over the following 5 years, as cell therapy BCMO services continue to expand strongly along with usage of cell therapies.  This is the finding in Kalorama Information’s new report, The Market for Cell and Gene Therapy Contract Manufacturing Organizations (CMOs).

The report said large CMOs include Lonza, Patheon, Catalent, WuXi and FUJIFilm Diosynth among many others.

Cell and gene therapies are groundbreaking treatments with the potential to actually cure disease rather than simply manage symptoms. They are often designed as one-time treatments, that in some cases, can actually reverse the progression of an illness. With cell therapy, cells are cultivated or modified outside the body then injected into the patient, where they become a “living drug.” With gene therapy, genes are replaced, inactivated or introduced into cells, either outside or inside the body, to treat a disease.

Consequently, cell and gene therapy products are transforming the treatment of cancers and genetic diseases. Additionally, cell and gene therapies are expanding into other areas of medicine including autoimmune diseases, cardiovascular diseases, musculoskeletal disease, dermatological diseases and many others. With an estimated 50 million persons suffering from neurodegenerative diseases in the U.S. alone, the unmet need is great.

With this exciting potential, the science is moving ahead and now the industry needs to industrialize and standardize the manufacturing and commercialization of emerging products. Important next steps include standardized set-up and closed automated systems.

Cost is one of the largest issues facing the industry. The high cost of producing these new therapies is staggering, resulting in prohibitively high product costs. For example, after launching Kymriah, Novartis’s price tag was $475,000 and a second product, Yescarta, cost $375,000. A gene therapy for the eye, Luxturna, was priced at $425,000 per eye. Some therapies are even more expensive, with costs for Zolgensma and Zyntegio estimated at more than $2.1 million and $1.8 million, respectively. The high price tag seems to be related to the high cost of goods (CoG).

Advancements in manufacturing are helping to drive costs down but cell and gene therapy remain an expensive process, with researchers at the Department of Pediatrics at Oregon Health & Science University and Doernbecher Children’s Hospital in Portland recently predicting that of the 40 – 50 gene therapies currently undergoing clinical assessment, each will likely to be priced at more than $1 million per regimen. These high costs present major barriers to affordability and usage, particularly in government-funded healthcare plans such as Medicaid and Medicare. Talks have been ongoing as to how to fund these medicines in the short term and long term through capital markets, a risk pool from insurers or other means.

The current goal of industry is to transition from a manual process to a more standardized and automated set-up, as did monoclonal antibody production. This will take the industry to a platform that will serve large patient populations while reducing both costs and risks. In the meantime, some manufacturers are making progress with insurance reimbursement.

When a product is first introduced, a company must ensure that the pace of manufacturing can keep up with demand, as there will initially be a backlog. This is particularly true for new cell and gene therapies, which offer potentially curative solutions to debilitating illnesses. Experienced CDMOs can meet this need by seamlessly supplying these early customers, avoiding delays that often arise in the build-out of entirely new manufacturing facilities.

Although the logistics of cell and gene therapies are more complicated than for most other drugs, these challenges can be met. For example, Dendreon’s Provenge was initially criticized for its complicated logistics. After a rough start, Provenge has become the most sold cell-based medicinal product, with thousands of shipments worldwide within tight delivery windows.

These trends and more are covered in Kalorama’s report, available at https://kaloramainformation.com/product/market-for-cell-and-gene-therapy-contract-manufacturing-organizations/.

Modern developments in cell and gene therapy products are transforming the treatment of cancers and genetic diseases, and in the process they have helped create a multi-billion dollar global industry that is poised to more than double over the next ten years, according to 2020 estimates by leading medical market research firm Kalorama Information as reported in Cell Therapy and Gene Therapy Markets. Therapies for cancer generate almost half of the cell and gene therapy market total, led by oncology drugs including Kymriah, Provenge, and Yescarta.  Now a new entrant into the market aims to join the ranks of these notable cell and gene therapy products for cancer.

In March 2020, the U.S. Food and Drug Administration (FDA) approved a new cell-based gene therapy idecabtagene vicleucel, which will be sold as Abecma. The new therapy is for adult patients with relapsed myeloma (myeloma that returns after completion of treatment) or refractory myeloma (myeloma that does not respond to treatment) who have not responded to at least four different types of therapy. Abecma is the first cell-based gene therapy for the treatment of multiple myeloma that has been approved by the FDA. The safety and effectiveness of Abecma were established in a study of 127 patients with relapsed myeloma and refractory myeloma who received at least three prior antimyeloma lines of therapy.

Multiple myeloma is one of several areas in the oncology sector that Kalorama Information identified as having strong development pipelines, alongside hematologic cancers (leukemia, lymphoma), brain cancer, and ovarian cancer.

Though the exact cause is unknown, multiple myeloma is a cancer that forms in a type of white blood cell called a plasma cell. Plasma cells help people fight infections by making antibodies that recognize and attack germs. Multiple myeloma causes cancer cells to accumulate in the bone marrow, where they crowd out healthy blood cells. Rather than produce helpful antibodies, the cancer cells produce abnormal proteins that can cause complications, including damage to the bones, kidneys, and immune system.

Multiple myeloma is not curable but is treatable and patient outlook is often dependent on age and the stage of the condition when diagnosed. Myeloma accounted for approximately 1.8% (32,000) of all new cancer cases in the U.S. in 2020, according to the National Cancer Institute. For people with multiple myeloma who require treatment, several treatments are available to help control the disease—now including Abecma. Multiple myeloma is one of several areas in the oncology sector that Kalorama Information identifies as having strong development pipelines, alongside hematologic cancers (leukemia, lymphoma), brain cancer, and ovarian cancer.

In the case of Abecma as a treatment option, the therapy is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. Abecma doses are customized treatments created using a patient’s own T-cells, which are a type of white blood cell, to help fight the myeloma. The T-cells are collected and genetically modified to include a new gene that facilitates targeting and killing myeloma cells. Once modified, the cells are infused back into the patient.

 

Cell therapy involves the modification of human cell which are used to replace or repair damaged tissues or cells. Two areas with growing development and commercialization include chimeric antigen receptor (CAR)-T therapies and stem cell therapies.

According to a recent report from Kalorama Information, the global market for cell and gene therapy in 2020 is estimated to reach $3,866 million And most of the market is for the treatment of cancer. Dermatological, Cardiovascular – Blood, Oncology, Ophthalmic, Musculoskeletal and other conditions.

With the COVID-19 crisis, there are some efforts to adapt this promising technology to the treatment and prevention of the disease. There are a number of companies that are responding to the call to develop a therapeutic or vaccine for the coronavirus including:

Vitro Biopharma offers its umbilical cord derived stem cells AlloRx stem cells and has a patent-pending and scalable manufacturing platform to provide stem cell
therapies to COVID-19 patients.

Celularity and Sorrento Therapeutics entered into collaboration for CYNK-001, an allogeneic, off the shelf, placental-derived NK cell therapy. The companies on January 30, 2020, launched a clinical and manufacturing collaboration designed to expand the therapeutic use of Celularity’s CYNK-001 to COVID-19. Sorrento and Celularity agreed to assess CYNK-001 as a potential novel therapy for coronaviruses, specifically SARS-CoV-2

It’s even possible CAR-T, currently used for types of cancer with limited therapeutics, could be used for COVID-19. CAR-T involves using modified versions of the patient’s own cells for treatment. The engineering of specific virus-targeting receptors onto a patient’s own immune cells is now being explored by scientists from Duke-National University of Singapore (Duke-NUS) Medical School (Duke-NUS), as a potential therapy for controlling infectious diseases, including the COVID-19-causing virus, SARS-CoV-2. This therapy that has revolutionized the treatment of patients with cancer has also been used in the treatment of other infectious diseases such as Hepatitis B virus (HBV), as discussed by the School’s researchers in a commentary published in the Journal of Experimental Medicine.

According to the university, this type of immunotherapy requires specialized personnel and equipment, and it needs to be administered indefinitely. This makes it cost-prohibitive for treating most types of viral infections. However, in the case of HBV infections, for example, current anti-viral treatments merely suppress viral replication and cure less than 5% of patients. Treating these patients with a combination of anti-virals and CAR/TCR T cells could be a viable option. The team’s approach using mRNA electroporation to engineer CAR/TCR T cells limits their functional activity to a short period of time, and hence provides enhanced safety features suited for its deployment in patients with

Despite these efforts, the majority of the market currently is and is expected to represent treatments for cancer.  Kalorama’s report breaks down the market by types of cancer including Blood cancers, Prostate cancer, Melanoma, Other cancers. Oncology drugs Kymriah, Provenge, and Yescarta are primarily responsible for generating sales in the segment.