A New Publication Covering The CELL AND GENE THERAPY INDUSTRY
The Best Way to Keep up with the Growing Cell and Gene Therapy Industry
From Science and Medicine Group, the company behind Instrument Business Outlook, Kalorama Information, SDi and other publications, comes a new publication: Cell and Gene Therapy Business Outlook.
With thousands of potential therapies on the market, cell and gene therapy promises future potential for pharmaceutical developers and those serving them.
A new twice-monthly publication dedicated to cell and gene therapy, Cell and Gene Therapy Business Outlook will offer the following:
Market Sizing and Forecasting of CGT Markets in Every Issue
Executive News Summaries – What is Happening in CGT Markets and Why It Matters
Deals Between CGT Companies Tracked in Every Issue
Important Science That Will Shape Tomorrow’s Business
Updates on Pipelines and Important Clinical Trials
Cell and Gene Therapy Tools, CMOs, Manufacturing Developments
Market Analysis of a Cell and Gene Therapy Segment in Every Issue
There are many websites, publications and sources on cell therapy. Cell and Gene Therapy Business Outlook differs from these sources in that it is created by market researchers and editors focused on business opportunity. Each issue will track the market size and potential for a key market segment.
Who Is Dealing with Whom? Tracking of Cell and Gene Company Deals In Every Issue.
There is a never-ending stream of activities in this market. How can you keep up? Each issue of Cell and Gene Therapy Business Outlook will keep track of mergers, investments, licensing, technology transfers and partnerships in the industry. Each issue of Cell and Gene Therapy Business Outlook contains an updated CGT Recent Deals Table with information on these important events.
Future issues will also analyze of the number of deals and increases or decreases in activity as a measure of business. You’ll never miss an important happening with Cell and Gene Therapy Business Outlook. Also, the Recent Deals Table is a great resource for tracking companies in the market.
The News That Matters
Edited by Blake Middleton, a professional CGT researcher and former Staff Research Associate at UCLA Department of Pharmacology, Cell and Gene Therapy Business Outlook is designed to provide the most relevant news. Included is news that could affect business decisions near-term. Cell and Gene Therapy Business Outlook also explains the relevant science.
With a focus on what the recent news of the day means for business, our curated news and news analysis means that you and your organization can be confident you won’t miss an important development in cell and gene therapy.
Convenient and Cost-Effective Seat-Based Pricing: Pricing depends on the number of users. Subscriptions can be as low as $2,200 annually for a limited one-person (single user) subscription.
Open up access: If more than one person will be reading, you can unlock access to other members of your organization. It’s easy to do: team subscription prices are as little as $4,995 annually for up to five readers. Larger team? Other licenses are available. Consult our website. Convenient and Cost-Effective Seat-Based Pricing: Pricing depends on the number of users. Subscriptions can be as low as $2,200 annually for a limited one-person (single user) subscription.
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THE CELL AND GENE THERAPY MARKET IN ONCOLOGY is $1,582M
MARKET SIZE: The global market for cell and gene therapy for oncology reached $1,582 million in 2020 and is expected to climb to $2,744 for 2021.
There are over 100 different types of cancer; some of the more prominent include lung, breast, brain, blood, prostate and colon cancer. The immune system plays a primary role in the body’s defense against malignancy. Although a tumor is derived from the body’s own cells and is expected to possess proteins that are recognized as self and nonantigenic, neoplastic cells can express antigens that are not recognized as self. These cells can often be eliminated by the immune system.
FORECAST: projected to increase to $7,391 in 2025; $17,490 million by 2030.
Treating cancer is difficult because it is not a single disease and because all the cells in a single tumor do not behave in the same way. Although most cancers are thought to be derived from a single abnormal cell, by the time a tumor reaches a clinically detectable size, the cancer may contain a diverse population of cells.
Market Forecast: Strong increases in the CAR-T therapy market, increasing from just $16 million in 2017 to $1,081 million in 2020 and projected to increase to $7,391 in 2025; $17,490 million by 2030. Blood cancers are the leading driver in the segment, representing 68% of total sales. This is expected to be the primary segment through the forecast, representing 80% of sales by 2025 and 80% in 2030. The United States and Europe are the largest markets due to overall product approvals and cost associated with the therapies. The US market represented nearly 77%, while Europe represented 19% in 2020. Gilead and Novartis combined represent 68% of the market for cell and gene therapy in oncology. Industry refocuses on oncology cell and gene therapies in a post-pandemic arena, returning to pre-pandemic growth.
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AAV CAPSID DISCOVERY UPDATE
Adeno-associated viruses (AAV) are small human viruses which provoke only a mild immune response and are not known to cause any human disease. AAVs are quite simple in organization, possessing a small (4.7kb) single-stranded DNA genome with only two open reading frames (ORFs), rep and cap, flanked by short (145 base) inverted terminal repeats (ITRs). The rep ORF encodes multiple overlapping sequences for proteins required for replication, and the cap ROF does the same for capsid proteins, which are the proteins forming the outer viral protein coat. These genes alone are not sufficient for viral replication, and AAVs require co-infection with a second, helper virus (such as an adenovirus or HSV) to supply the remaining gene products for replication (hence the name adeno-associated virus).
Gene therapy AAV vectors are further modified to remove the rep and cap genes from the viral genome (along with their promoters and polyadenylation signal), replacing them with a therapeutic expression cassette. Production of recombinant AAV vectors in cell lines requires the rep and cap genes to be supplied by a plasmid transfected in trans, in addition to the genes supplied by the helper virus. None of these externally supplied viral genes are packaged into the final construct, so the resulting viral delivery vehicle consists only of the therapeutic cassette encased in an AAV capsid, without any viral genes present. The gene therapy vector is therefore incapable of replication, even with co-infection by a suitable helper virus.
In addition to their safety, AAV vectors possess many features which make them attractive gene therapy candidates. They have extremely low immunogenicity, they can infect both dividing and non-dividing cells, and they can persist outside the genome to offer stable, long-term expression without the risks associated with host genome integration.
AAV vectors also suffer from several shortcomings, however:
• Because of their wide distribution, many individuals have already been exposed to naturally occurring AAV serotypes and produce immune responses against them.
• AAV vectors cannot reach most tissues efficiently, and do not spread easily within those tissues if they do.
• Vectors will preferentially target some cell types but not others.
• Transduction efficiency is often extremely low.
Each of these shortcomings can be addressed by innovations in capsid structure. In addition to protecting the DNA payload, the capsid is responsible for binding to specific receptors on the target cell and safely delivering the DNA payload to the cell machinery that so will be transported to the nucleus. Viral packaging efficiency, host immunological response, tissue and cell type specificity, and transduction efficiency are all determined by the capsid serotype. Unfortunately, initial gene therapy experiments were restricted to a handful of natural AAV serotypes which had limited tropism in many human cell types. Common serotypes also present problems with pre-existing immunity (PEI), as up to 90% of the human population have already been exposed to at least one AAV serotype. For these reasons, novel capsid discovery is a current hotbed of gene therapy research.
More information on this topic can be found in the latest issue. SUBSCRIBE TODAY
THE LATEST NEWS FROM CELL AND GENE THERAPY OUTLOOK
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.
https://d3lstfzn07k02o.cloudfront.net/wp-content/uploads/sites/12/2019/01/08092216/logo_ki.gif00Daniel Grandersonhttps://d3lstfzn07k02o.cloudfront.net/wp-content/uploads/sites/12/2019/01/08092216/logo_ki.gifDaniel Granderson2021-04-16 16:05:262021-09-23 09:57:35Watch the Companies That Make Cell and Gene Therapies For Pharma, Says New Report
Watch the Companies That Make Cell and Gene Therapies For Pharma, Says New Report
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/.