Cell and Gene Therapy Business Outlook

 

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.

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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

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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.

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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

 

Over 54 BN across funding mechanisms.  Private, VC, SPAC and and IPO Over 20Billion

The enormous amount of funding presages confidence in discoveries of future marketable products as well as platforms in cell and gene therapy.  With an aggressive third quarter 2021 for funding, it is worthwhile to total and assess where we are.  In this section we have gathered and anyalized the deals that have been made this year that have been reported in our issues.

Readers of Cell and Gene Therapy Business Outlook will note there’s been no slowdown in deals activity.    There has been an enormous amount of funding for cell and gene therapies in 2021.  When M&A is included, this funding has reached $54 BN dollars, according to Cell and Gene Therapy Business Outlook’s analysis.  (Even without considering mergers and acquisitions – IPOs, VC, SPAC and private deals exceed $20 billion).

 

Be Sure to Subscribe to Cell and Gene Therapy Business Outlook for more information.

Cell and Gene Therapy Business Outlook

Cell and gene therapies are trending as groundbreaking treatments with the potential to actually cure disease rather than simply manage symptoms. As a result, through 2030, the global market for cell and gene therapy is forecast to expand strongly to $29,960 million, up from $3,866 million in 2020, according to a 2021 report by Kalorama Information and editors at the medical research firm’s new sister publication, Cell and Gene Therapy Business Outlook.

Globally, 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.

Oncology a Key Cell and Gene Therapy Segment

Among the expanding areas of focus for cell and gene therapies, oncology remains one of the most intriguing and notable. Treating cancer is difficult. Not just because there are over 100 different types of cancer, but 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.

Opportunities Amidst Challenges

Despite the challenges, there are opportunities. Cell therapy has been used for years for blood transfusions and hematopoietic stem cell transplants. Advancements in technology has allowed new options for treatment.

The initial development of CAR-T cell therapies focused on treatment for ALL (acute lymphoblastic leukemia), NHL (non-Hodgkin lymphomas), multiple myeloma, and GBM (glioblastoma multiforme). Now in development are products for solid tumors such as pancreatic cancer, ovarian cancer and colorectal cancer. The FDA has approved three CAR-T cell therapies including Kymriah, Yescarta, and Tecartus.

Growth Factors in 2021

Kalorama Information estimates the global market for cell and gene therapy for oncology reached $1,582 million in 2020 and is expected to climb to $2,744 million in 2021. Several factors contributed to strong growth including:

  • 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 and ultimately reach $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.

Growth Factors Beyond 2021

Pricing Trends

In oncology cell and more particularly, gene therapy, the cost of therapies is exceptionally high.  From development to manufacturing processes, these therapies require more complex evaluations to determine eligibility for payment. It is anticipated that as more products become available, prices will fall somewhat but it is an expensive process for all participants.

Expected Market Penetration

It is anticipated that CAR-T oncology products will continue to produce the lion share of revenues for the oncology cell and gene therapy market. Compound annual growth rate for CAR-T is forecast to be 43% for 2020-2025. Market penetration was slowed in 2020 due to the COVID-19 virus but is expected to gain steadily in 2021-2025. The CAR-T market will overshadow the cell immunotherapy, gene therapy and other cell therapy markets.