The Size of The Cell and Gene Therapy Market

The global market for cell and gene therapy in 2020 is estimated to reach $3,866 million with therapies for cancer generating about 47% of the total market.  This is according to Kalorama Information’s latest report.  Oncology drugs — Kymriah, Provenge, and Yescarta — are primarily responsible for generating sales in the segment. Genetic disorder treatments Givlaari, Onpattro and Zolgensma are generating the majority of sales for other conditions.

The market for 2025 is estimated at $12,954 million and will more than double five years after to $29,960 million for 2030. Sales from oncology, cardiovascular-blood and musculoskeletal conditions will contribute strongly to market performance. Genetic conditions will also provide steady growth.

It is an exciting and interesting time to be involved in the cell and gene therapy industry. The science is moving ahead and now the industry needs to industrialize and standardize the manufacturing and commercialization of products. The industry is moving in the direction of a standardized set-up and a closed automated system.

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.

 

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.

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

_____________________

SUBSCIBE TODAY:
www.cellandgenetherapybusinessoutlook.com

_____________________

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.

_____________________

SUBSCRIBE TODAY:
www.cellandgenetherapybusinessoutlook.com

_____________________

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.


___________________________________________

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.

___________________________________________

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.

Early efforts at capsid discovery focused on discovering previously unknown, naturally occurring capsids, and today 11 main serotypes with over one hundred variants have been discovered. Most of these capsids are still less than ideal for gene therapy applications, so recent attention has turned to the creation of synthetic capsid sequences, entirely unknown to the natural world. A strategy called rational design can generate novel capsids by carefully tailoring key regions within the capsid protein sequences, altering the capsids to avoid antibodies to common serotypes and selectively target and efficiently transduce specific cell types. Rational design requires extensive knowledge of capsid structure and function, however, and that field of study is still in its infancy. Poorly informed capsid design is generally doomed to failure: Out of the staggering number of possible capsid sequences, the overwhelming majority will not even produce functional capsids, and random modifications to existing capsid sequences are likely to either have no effect or to be detrimental to the desired outcome.

THE LATEST NEWS FROM CELL AND GENE THERAPY 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.

 

In this post, we update on and analyze some of the recent stories in the news regarding Cell and Gene Therapy.  For detailed updates, additional news analysis, scientific innovations and market opportunity updates, subscribe to Cell and Gene Therapy Business Outlook from Science and Medicine Group.

  • Avantor, based in Radnor, PA, has agreed to purchase the Vernon Hills, IL-based Masterflex bioprocessing business and related assets of Antylia Scientific in an all-cash deal valued at $2.9 billion ($2.7 billion with anticipated tax benefits). Masterflex manufactures peristaltic pumps and aseptic single-use fluid-transfer technologies, and its 2022 revenues are estimated to top approximately $300 million.  The purchase will strengthen Avantor’s offerings across all bioproduction platforms, including monoclonal antibodies, cell and gene therapy, and mRNA, according to the company. It will also support manufacturing of both therapies and vaccines, including COVID-19.  Earlier this year Avantor acquired German-based Ritter GmbH, a manufacturer of robotic and liquid handling consumables, for more than $1 billion.
  • The FDA has placed a clinical hold on BioMarin’s BMN 307, an investigational gene therapy designed to permanently maintain normal blood phenylalanine levels in phenylketonuria (PKU) patients. The treatment uses AAV5, an adeno-associated virus (AAV) viral vector to target cells in the liver and deliver a functional copy the PAH gene encoding the enzyme phenylalanine hydroxylase.  The hold came in response to liver tumors, with evidence of AAV integration into the genome, reported in mouse studies.  BioMarin has emphasized that cancers caused by AAV integration have not been reported in larger animals or humans.
  • BioLife Solutions, based in Bothell, WA, has closed on its acquisition of Indianapolis, IN-based Sexton Biotechnologies (which spun off from Cook Regentec in 2019). This is their sixth acquisition in the last 2.5 years. The acquisition is part of BioLife’s strategy to assemble a portfolio of class-defining bioproduction tools and services for the cell and gene therapy and broader biopharma markets, company representatives said. Sexton’s bioproduction portfolio includes proprietary closed vials for cell therapy final-dose packaging, human platelet lysate (HPL) media, a bio-defined replacement for fetal bovine serum or human serum used in cell manufacturing, and automated cell processing machines. The company’s products are currently embedded in more than 50 ongoing clinical trials of new cell and gene therapies, BioLife said.
  • Yokohama, Japan-based Reprocell and Osaka-based Texcell Japan (a subsidiary of French-based Texcell Group) have signed a Strategic Collaboration Agreement for induced pluripotent stem cell (iPSC) master cell bank (MCB) manufacturing. Under the terms of the agreement, Reprocell can outsource part of the MCB manufacturing process at Texcell’s GMP facility to meet the increasing demand for GMP iPSC MCBs. The agreement supplements a Master Service Agreement the two companies previously signed.
  • Tokyo-based Astellas Pharma is pausing screening and dosing of more participants in a clinical trial for its AT132 gene therapy to treat X-linked myotubular myopathy (XLMTM). Last year three children died of liver failure after receiving the highest dose, and the FDA placed the study on clinical hold. The FDA lifted the hold in December of last year to continue the study at a lower dose, but Astellas has placed the program on a voluntary hold after a report that a low-dose patient experienced a serious adverse event in the form of unusual liver function.
  • STEMCELL Technologies (based in Vancouver, Canada) and Madison, WI-based WiCell have partnered to launch human pluripotent stem cell (hPSC) characterization and banking services. These services aim to reduce variability and improve reproducibility for various downstream applications, including disease modeling, drug discovery, toxicology screening, and regenerative medicine research. The collaboration will make it easier for pluripotent stem cell researchers to characterize new or existing cell lines and create fully characterized banks of hPSCs, according to both organizations.  Initially, the services only will be offered in the U.S.

For the latest news SUBSCRIBE to CELL AND GENE THERAPY
BUSINESS OUTLOOK: Business Outlook https://kaloramainformation.com/product/cell-and-gene-therapy-business-outlook/

  • Natick, MA-based HebeCell has announced that it has raised $53 million in a Series A financing round led by Beijing-based Jacobio Pharmaceuticals, who will collaborate with HebeCell to advance off-the-shelf pluripotent stem cell-derived chimeric antigen receptor-expressing natural killer (PSC-CAR-NK) cell products to clinics. The companies plan to develop PSC-CAR-NK therapies for cancer, viral infection, and autoimmune diseases. Funds will be used to speed up HebeCell’s R&D program, investigational new drug application, clinical application, and marketing as the company grows its operations both in the US and internationally.
  • NeuExcell Therapeutics and Spark Therapeutics, a Roche subsidiary based in Philadelphia, PA, have announced a collaboration to develop a gene therapy for Huntington’s disease. NeuExcell Therapeutics is a gene therapy company focused on neurodegenerative diseases, with headquarters in State College, PA, and Shanghai, China. Under the terms of the agreement, Spark will gain access to NeuExcell’s neuroregenerative gene therapy platform and will collaborate with NeuExcell’s research team.  Spark has the option to license the exclusive worldwide rights to NeuExcell’s Huntington’s disease program, and NeuExcell is eligible to receive upfront, license fee, R&D, and sales milestone payments worth up to approximately $190 million, plus product royalties. Defying conventional wisdom that mammalian adult neurons cannot be replaced, NeuExcell’s neuroregenerative gene therapy platform performs in vivo astrocyte-to-neuron conversion by introducing neural transcription factor(s) with an AAV vector.  While neurons cannot regenerate by themselves, surrounding glial cells like astrocytes can divide and supply a renewable source for generating new neurons through trans-differentiation.  NeuExcell’s development pipeline includes major neurodegenerative diseases such as stroke, Huntington’s disease, amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, Parkinson’s disease, traumatic brain injury, spinal cord injury, and glioma.  The company recently raised more than $10 million in a series pre-A funding round led by Co-Win Ventures, and has announced that it has added two members to its board of directors: Dr. Xin Huang and Jonathan Sun.
  • NovaRock Biotherapeutics, based in Ewing Township, NJ, has granted an exclusive license to New York-based Flame Biosciences to develop, manufacture, and commercialize NovaRock’s NBL-015, a fully human anti-Claudin 18.2 monoclonal antibody to treat pancreatic and gastric cancer. NBL-015 has been optimized to achieve enhanced antibody-dependent cell-mediated cytotoxicity (ADCC), complement-dependent cytotoxicity (CDC), and antibody-dependent cellular phagocytosis (ADCP) effects.  Its Investigational New Drug Application was approved by the FDA in May 2021. Flame Biosciences will have exclusive rights to NBL-015 outside of greater China (including mainland China, Hong Kong, Macau, and Taiwan), and will be responsible for the development, regulatory approval, and commercialization of NBL-015, according to the companies. NovaRock will also collaborate with Flame on the discovery and preclinical development of two new candidates based on NovaRock’s NovaTE bispecific antibody technology platform,  designed to selectively activate the antigen-experienced T cells in the tumor microenvironment. NovaRock will subsequently grant Flame exclusive rights to further develop, manufacture, and commercialize licensed products.  NovaRock will receive an upfront payment of $7.5 million, with up to $172.5 million in development milestone payments and $460 million in sales milestone payments, plus royalties.  Lead candidates from the collaboration are expected to enter clinical development in late 2023.
  • The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee met September 2nd-3rd to discuss toxicity risks associated with adeno-associated virus (AAV) vector.  Topics included cancer risks caused by vector genome integration, liver toxicity, thrombotic microangiopathy, and neurotoxicity. While the committee did not advise that the FDA should slow down clinical trials, they did make recommendations for more extensive testing in animals, improved screening of clinical trial participants, and more thorough follow-up of treated patients.
  • Vertex Pharmaceuticals, based in Boston, MA, and Arbor Biotechnologies, based in Cambridge, MA, have announced a new agreement to develop ex vivo engineered cell therapies based on Arbor’s CRISPR gene editing technologies. The agreement expands upon a previous partnership the two companies established in 2018 and grants Vertex rights to use Arbor’s technology to research and develop ex vivo engineered cell therapy programs, including programs for generating fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes, for developing next-generation approaches in sickle cell disease and beta thalassemia, and for the treatment of other diseases, according to the companies.  In addition to an upfront cash payment, Arbor will be eligible to receive up to $1.2 billion in potential milestone payments across up to seven potential programs. Vertex will also invest in Arbor in the form of a convertible note and will pay tiered royalties on future net sales of products resulting from the collaboration.