The genetics market size is expected to grow from 79 in 2022$400 million grows to $44.5 in 2029$100 million, growing at a CAGR of 279%。The Spinal Muscular Atrophy (SMA) indication segment will lead the global genetic** market by 2029.
Over the past decade, significant progress has been made in genetically and gene-based new**. For certain diseases, clinically effective genetic approaches have been developed, and many other potential drugs have been discovered, a trend that is particularly pronounced in the field of blood disorders. Delivery vectors, genetic engineering methods, and applications in chimeric antigen receptor (CAR) T cells** are just some of the recent advances in gene** and gene-based methods**.
Advances in the field of advanced and gene delivery technologies have created a competitive environment in which a large number of market companies are trying to commercialize their gene products. *Developers are considering collaborations, mergers and acquisitions as potential options to enhance internal knowledge and strengthen product pipelines. These factors are expected to contribute to the growth of the market. While the currently available grids encourage the development of methods, they also limit availability and can lead to market exits due to the lack of economic sustainability of the commodity.
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Figure 1Cell & Gene Manufacturing Services Market Size; The Cell and Gene Manufacturing Services Market is segmented by Type by: Cells (Allogeneic, Autologous, and Viral Vectors) and Gene (Non-Viral Vectors and Viral Vectors), Application (Clinical Manufacturing and Commercial Manufacturing), Indications (Oncological Diseases, Cardiovascular Diseases, Orthopedic Diseases, Ophthalmic Diseases, Central Nervous System Diseases, Infectious Diseases, and Other Indications), End Users (Pharmaceutical and Biotechnology Companies, Academic and Research Institutions, and Other End Users).
The factors driving the growth of the cell and gene therapy market include the high prevalence of cancer and other target diseases and the increasing R&D investments in pharmaceutical companies. For instance, according to an article published in the Chinese Medical Journal in February 2022, it is estimated that there were about 4,820,000 and 2,370,000 new cancer cases in China and the United States in 2022, respectively. The most common cancers are lung cancer in China and breast cancer in the United States. As the number of cancer cases increases, the dependence on genetics** will also increase, thereby driving the market growth.
In addition, according to data released by the National Cancer Center of Japan in June 2022, 158,200 new cases of cancer are expected in Japan this year, with an estimated 89,500 cases in men and 68,700 cases in women. The increasing incidence of colorectal cancer is expected to drive the demand for gene and cell** products, thereby driving the segment growth during the period.
Moreover, according to a report published in October 2021 in the Journal of Orthopaedic Surgery and Research, the combined prevalence of osteoporosis worldwide has been reported to be 183%。Stem cells*** osteoporosis may reduce susceptibility to fractures. Therefore, as the burden of orthopedic diseases increases, the demand for cell and gene manufacturing services is also expected to grow.
Moreover, the growing cell** facilities are also expected to drive the market growth. For instance, in June 2022, Organabio, LLC launched Good Manufacturing Practice (GMP) manufacturing operations at its new cell** manufacturing facility in the United States. The new facility provides contract and development manufacturing of customized solutions for drug developers.
Thus, the above-mentioned factors, such as the rising prevalence of various diseases such as cancer and osteoporosis, are expected to drive the growth of the market studied during the ** period. However, the high operating costs associated with cell and gene manufacturing** are expected to restrain the market growth.
Figure 2R&D services for Ginkgo biological products; Design Biology: Biology is the most advanced manufacturing technology on the planet. Ginkgo Bioworks programs cells to make everything from food to materials to medicines.
Ginkgo Bioworks has end-to-end capabilities and unmatched scale in biology; The biomanufacturing services that Ginkgo Bioworks is able to provide include:
Industrial Enzyme Services: Enzymes power biology, driving virtually all of the active chemicals that make life possible. They also power a range of bio-based industries, with applications ranging from industrial to pharmaceutical. Ginkgo can support your enzyme R&D project, covering all the ways enzymes are designed, manufactured, tested, and manufactured at scale.
Working with Ginkgo means running your project at the Ginkgo Foundry, an expansive facility at the forefront of synthetic biology. The foundry combines the latest AI and computational design tools with high-throughput automation, allowing us to efficiently explore the enzyme space to find the perfect design for your application.
Ginkgo provides end-to-end support for your enzyme engineering projects. Partner with us from early discovery to developing production and purification processes to transfer technology to your manufacturer.
AI-guided enzyme engineering powered by large biological datasets, AI opens up new design spaces for enzyme engineers and new opportunities for enzyme-driven applications. Ginkgo's enzyme R&D program deploys AI tools at all stages: discovery of novel enzymes, structure-guided engineering, manufacturing, and process optimization.
Identifying a new enzyme or enhancing the performance of a known enzyme, the enzyme discovery project starts with the reaction chemistry you want. Utilizing AI-guided search tools and our vast in-house database of DNA sequences, Ginkgo can identify unique candidate enzymes from different natural** sources. Enzymes discovered by Ginkgo often have new sequences that are different from those previously described as candidate enzymes, opening up new intellectual property options for Ginkgo's partners.
Protein Expression Services: help reduce risk and accelerate protein production time; Whether you're looking to get a head start or take advantage of Ginkgo's top-of-the-line custom fungal and yeast strains, optimize strain performance for protein production, achieve higher titers with classic strain modification using non-GMO methods, or increase production efficiency, Ginkgo's best-in-class capabilities can enhance your protein R&D capabilities.
Leverage Ginkgo's end-to-end capabilities to overcome bottlenecks in protein expression; Protein plays a key role in influencing taste, texture, and health benefits. These functions are controlled by protein sequences and cell-mediated modifications during production, which often pose challenges for precision fermentation and cell engineering. For example, the hydrophilic nature of milk proteins is essential for cheese function, but makes it susceptible to intracellular degradation.
Optimizing the production host is essential to enable the fermentation of these proteins. This includes the elimination of proteases, the introduction of chaperones, the creation of promoters, the expression system, and the customization of each protein to the unique requirements. The process also requires design, downstream processing, and scale-up work.
Bringing protein to market successfully is a complex process. Leverage Ginkgo's expertise for fast, scalable solutions. From host selection to optimizing host strain development and amplification, Ginkgo biloba services can help you overcome bottlenecks, cut capital expenditures, accelerate development, and reduce R&D risk.
Ginkgo has invested heavily in the development of our in-house host strain suite, designed for the production of glycoproteins, iron-binding proteins, and structural proteins with the goal of improving titer and function, including protein targets in sweeteners, alternative dairy products, alternative eggs, thickeners, and preservatives.
Figure 3building a global bioradar system with Ginkgo; Strain Optimization Services: Ginkgo can optimize the performance and stability of strains for any application; Whether you're looking to increase your titer, improve the robustness of your strain under harsh industrial conditions, or evolve your strain to grow with more cost-effective inputs, Ginkgo's best-in-class strain optimization services can help you achieve your goals.
Diverse strain optimization capabilities supported by scale, from computational tools to in-house DNA design and sequencing services to ultra-high-throughput screening methods, Ginkgo's capabilities enable strain optimization for a wide range of applications. Take advantage of Ginkgo's extensive strain modification technology, from random mutagenesis and adaptive lab evolution to a rational design approach to achieve your KPIs. Partner with Ginkgo to design strains that meet your technical specifications, regulatory constraints, and R&D timelines, while increasing your probability of success.
Process Design & Scale-up Services: Scale up and deploy optimized strains on a commercial scale-up, leveraging Ginkgo's best-in-class scale-up, processing, and production services at benchtop, pilot, and commercial scales. Ginkgo's capabilities enable and improve the production of your products at scale, whether it's cells or genes**, agricultural products, food proteins, industrial commodities, or anything in between.
Regardless of size, Ginkgo offers end-to-end capabilities, from proof-of-concept for plates, shake flasks, and small bioreactor systems, to modeling, analysis, and process development, characterization, and commercial launch, Ginkgo's services can help you move from R&D to large-scale manufacturing.
Leverage Ginkgo's in-house laboratories and capabilities, including quality control, upstream production, downstream purification, pilot plant facilities, technology transfer to a partnering Ginkgo manufacturing site, or a customer's preferred CDMO. Ginkgo's team of experts will provide process modeling, development and optimization, analytical support for your project, including method development and validation, and titer or purity determination, and transfer the technology to your team or your preferred production partner.
Biosecurity services: Build an end-to-end biosecurity program, Ginkgo's platform provides the capabilities needed to collect, detect, and analyze pathogens, providing end-to-end capabilities to quickly respond to biological threats.
Because biology knows no borders, Ginkgo provides a rapid response pathogen early warning system;
While biology may not be possible, a robust biosafety infrastructure can provide accessible and scalable solutions to reduce risk. Ginkgo's pathogen early warning system is installing key collection points in the global pathogen surveillance network. These nodes enable decision-makers to identify biological threats before they become emergencies. In addition, Ginkgo's bioinformatics services provide insights into pathogens of interest and provide the ability to detect variant evolution and change.
The evolution of Ginkgo Bioworks.
Figure 4The business model of Ginkgo Bioworks.
Ginkgo Biotech focuses on its foundry and library assets to provide R&D services to customers and reduce costs. Ginkgo Bioworks has two main assets: Foundry and Library. Foundry operates as an automated laboratory and is able to transform R&D investments from fixed to variable costs and make them available as a service. On the other hand, the library acts as a repository of experimental data, helping to increase the success rate of the project while reducing the cost to the customer. It can be seen that Ginkgo adopts a service-based business model. In order to simplify sales, the company has been actively working to standardize all aspects of its products. This includes establishing clear milestones, cost structures, IP provisions, and project timelines. This standardization is aimed at increasing the transparency and accessibility of Ginkgo's services. In order to expand its business, Ginkgo has adopted the following strategic initiatives:
Ginkgo partners with Pfizer for drug discovery.
Ensuring the success of a drug discovery project is much more challenging than manufacturing R&D contracts. The main reason is that pharmaceutical companies often view drug discovery as a critical proprietary function and often prefer to do it in-house rather than outsource.
Ginkgo Bioworks' recent deal with Pfizer falls into the drug discovery category, with a particular focus on mRNA**. This area of drug development, which has received a lot of attention for its application in COVID-19 vaccines, represents a modern and technologically advanced approach. The biotech company will partner with Pfizer to provide value to three projects$3.1 billion drug discovery agreement for RNA drugs. The two companies did not elaborate on the details of the transaction, but 3The $3.1 billion includes upfront payments, research expenses, development and commercial milestone payments, and future royalties.
Ginkgo struck a deal with Google to integrate artificial intelligence.
Ginkgo's partnership with Google Cloud aims to enhance its development capabilities. The scalable computing resources provided by Google Cloud are particularly important for training a large number of underlying AI models, which are presumably offered at competitive rates. Google has also pledged to provide financial support to Ginkgo's model development program after certain milestones are reached.
In the 3Q23 earnings report, Ginkgo's chief executive officer Jason Kelly revealed that Ginkgo has achieved the initial cash milestone under the agreement and is on track to achieve subsequent milestones in the near future. This development should be carefully evaluated, particularly in terms of its impact on Ginkgo's operational efficiency, AI model development and overall financial trajectory, taking into account the highly competitive and fast-moving biotech landscape.
The impact of artificial intelligence on Ginkgo Bioworks.
Figure 5Artificial intelligence (AI) technology will have a significant impact on biomedical technology.
Located in Boston, Ginkgo Bioworks' foundry facility is the center of the automation lab. Foundry's core goal is to scale up data generation at a lower cost, essentially being a factory for testing genetic designs. This highlights the strategic focus on efficiency and scalability in genetic experiments and data production.
The data generated in the foundry is systematically compiled into what is known as the Ginkgo "** library". A key feature of this library is its broad applicability; It is not limited to a single project, but is leveraged in a variety of customer plans. This process accelerates the development of new projects, even if they are for different clients, by leveraging the insights of one project to inform and accelerate the progress of other projects.
This accumulated data is critical for training large AI models. These models, in turn, guide the direction of future experiments, which further contributes to the refinement of the models. This creates a collaborative feedback loop where AI capabilities and data generation continuously enhance each other, increasing the accuracy and operational efficiency of the model over time.
To measure the operational efficiency of Ginkgo Biologics, it is essential to have a comprehensive understanding of the company's cost structure. This involves dissecting various aspects of its process, namely: the cost per unit operation, the number of unit operations per cycle, and the number of cycles per project.
In this context, Ginkgo's use of AI and machine learning models is crucial. These models have shown remarkable ability to identify promising enzyme sequences, narrowing down countless possibilities to the variant most likely to succeed. A related example at the Q3'23 earnings conference** involved a library of 500 enzyme variants from both known enzymes from the Ginkgo** library and from novel custom-engineered enzymes. During the initial test cycle, we found an enzyme that performed 21 times better than the original enzyme provided by the customer.
The important aspect here is not only the discovery itself, but also its speed, which is facilitated by a streamlined workflow and a relatively small library size (500 variants versus the typical thousands). The precision of Ginkgo's AI ML model in terms of the most effective enzyme sequences contributed to this rapid success.
Patent filed by Ginkgo Bioworks.
Figure 6Patent filed by Ginkgo Bioworks.
Ginkgo Bioworks: There's a long way to go
While Ginkgo's cell engineering business has great potential, the data suggests that the business is still in its infancy. Investors who are expecting a lot of downstream value need to be patient.
While the progress of Ginkgo's pharmaceutical and biotech customers is an important development, most of these projects are yet to be completed.
Patent filed by Ginkgo Bioworks
1.Synthetic Methanol Inducible Promoters and Uses Thereof
Publication number: 20240002847
Abstract: This application describes a synthetic promoter capable of promoting the synthesis of proteins and molecules in high yields.
2.Chimeric terpene synthases
Publication number: 20210147880
Abstract: This article describes chimeric terpene synthases, methods for preparing chimeric terpene synthases, and methods for using them to prepare terpenes.
3.Production of oligosaccharide
Publication number: 20220372501
Abstract: The present disclosure relates to a method and composition for the production of fructan using sucrose:sucrose 1-fructosyltransferase (1-SST), fructan:fructosan-1-fructosyltransferase (1-fft), or sucrose-6-fructosyltransferase (6-SFT) enzymes.
4.Methods for Preparative in Vitro cloning
Patent No.: 9752176
Abstract: Methods and devices involve the isolation of nucleic acids of interest from nucleic acid populations (e.g., nucleic acid sequence libraries).
Ginkgo Bioworks is involved in the field of cell engineering and biomedical research.
Figure 7Ginkgo Bioworks is involved in the field of cell engineering and biomedical research.
Ginkgo Bioworks is involved in the field of cell engineering and biomedical research.
biology.
biochemistry - biochemistry.
chemistry.
genetics- genetics.
gene-gene.
enzyme-enzyme.
Escherichia coli - Escherichia coli.
Computational Biology - Computational Biology.
Biosynthesis.
yeast - yeast.
bacteria - bacteria.
stereochemistry - stereochemistry.
Molecular Biology.
genome-genome.
peptide sequence.
Saccharomyces cerevisiae - Saccharomyces cerevisiae.
Metabolic Engineering.
plasmid-plasmid.
Polyketide - polyketone compounds.
mutant-mutant.
Yarrowia - Saccharomyces jirovecii.
amino acid - amino acids.
ATP synthase-ATP synthase.
Cell Biology - Cell Biology.
fatty acid - fatty acids.
metabolism.
Microbiology.
protein structure.
gene expression.
DNA - deoxyribonucleic acid.
Organic Chemistry.
biotechnology.
heterologous expression - heterologous expression.
chromatography-chromatography.
fermentation - fermentation.
binding site.
botany - botany.
Cannabinoid-** hormone.
operon- operon.
Polyketide synthase-polyketide synthase.
Bioinformatics.
cofactor-cofactor.
Metabolic pathway - metabolic pathway.
biophysics.
Computer Science.
promoter - the initiator.
cyanuric acid.
exosporium - exosporium wall.
Mutagenesis (Molecular Biology Technique).
Sequence Analysis.
Ginkgo Bioworks' entire business is built on the belief that scale and data will create a sustainable competitive advantage. The data created by the cell engineering program should improve strain engineering capabilities over time. Gingko's database has grown rapidly in recent years, but the company does not currently have financial performance that supports the expected economies of scale. That's not to say that improvements haven't occurred, though.
Project-level data shared by Ginkgo Bioworks shows that in some cases, the company was able to create significant improvements early in the iterative design-build testing cycle.
There are also potential economies of scope between Ginkgo Bioworks' foundry foundry and biosafety operations. Metagenomic data from the biosafety business provides training data for the cell engineering business, and tools developed for the cell engineering business can be used for the biosafety business.
Figure 8Ginkgo Bioworks is a research institute involved in the field of cell engineering and biomedicine.
Major research institutes in these areas include:
Massachusetts Institute of Technology - Massachusetts Institute of Technology.
University of California, Berkeley.
Centre National de la Recherche Scientifique - National Centre for Scientific Research.
INRAE - National Aeronautics and Space Administration.
University of Minnesota.
University of Tokyo.
Kyoto University.
Kyushu University - Kyushu University.
University of Freiburg.
Stanford University.
Biotechnology Institute.
Max Planck Society.
Agricultural Research Service.
Chinese Academy of Sciences-Chinese Academy of Sciences.
j.Craig Venter Institute-J. Craig Venter Institute.
Reference study books related to the research topic "Cell Engineering and Biomedicine".
Figure 9The Handbook of Cells and Genes: From Proof of Concept to Manufacturing to Commercialization
1.Handbook of Cell and Gene Therapy: from Proof-of-Concept through Manufacturing to Commercialization
This handbook provides an in-depth review of information on the entire development spectrum of Gene and Cell** products. From introductory information to state-of-the-art techniques and concepts, this book provides insight into upstream processes such as vector design and construction, purification, formulation and filling completion, and delivery options. Planning steps from complying with current Good Manufacturing Practices (cGMP) to preparing for Chemistry, Manufacturing, and Controls (CMC) are also discussed. This book summarizes examples of successes and pitfalls addressed by experts who have made all the innovative efforts and overcome these challenges.
Features of the book:
This book is intended to serve as a one-stop resource for researchers, scientists, administrators, and other academic and research institutions with the latest information related to cell and gene products.
This book provides the latest information on the development of genetics, from the genetic correction techniques involved to genome editing technologies.
Discuss siRNA, mRNA, and plasmid manufacturing.
The importance of business-sponsor synergies on the road to commercialization of cells and genes is described.
The book has a diverse audience and is written by individuals with a wealth of core technical and supporting practices.
Cell & Gene** products represent a dramatic shift in the field of medical interventions. Unlike traditional organisms, this technology aims to modify or manipulate the expression of genes or alter the biology of living cells for use. By replacing, manipulating, or modifying cells and/or genetic material, they are expected to provide an opportunity to improve outcomes for patients by injecting genetically modified cells for at least certain diseases and conditions for life.
Traditional biologics (monoclonal antibodies, recombinant products) have reached a mature stage where platform manufacturing technologies can be applied, and regulatory and compliance issues are clear. For cell and gene products, there is no established manufacturing manual to refer to, and regulations are constantly evolving. Personalization** has transformed from a primary clinical phase in a hospital setting to a full-fledged commercial enterprise, with the routine manufacturing of these products following established regulatory and industry best practices.
The biggest blind spot in the development lifecycle is being able to transition and realistically understand the time, resources, and costs required to develop such a complex biologic. Decisions made at an early stage are critical to a product's future viability to an unprecedented degree. There are a lot of moving parts in product development and cGMP production. In addition, the specifics related to clinical study planning, logistics, and execution, as well as meeting specific regulatory expectations, must be addressed. Early planning is an important strategic foundation for cells and genes. Having a business vision must be a goal from the outset and help form a critical path of understanding.
The goal of the authors of this handbook in this handbook is to provide an overview of development, manufacturing, testing, and regulatory expectations related to cells and genes. The up-to-date information provided here is intended for researchers, scientists, administrators, and other academic and research institutions in the field. Readers should be able to follow key principles and concepts, including but not limited to process design, facility design and construction; technology transfer, analytical method development and transfer; Validate the life cycle; and regulatory frameworks around the world. If this manual saves you a little bit of time, the authors have achieved their goal.
Technology and knowledge will continue to evolve, but the basic principles described in this handbook should serve as a benchmark for colleagues across the industry. Progress is built on history and the lessons of the past; The future is shaped by these lessons and history.
To quote the American writer and playwright Alfred Sheinwald: "Learn everything you can from someone else's mistakes, you don't have time to make them yourself." ”
2.Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective
Figure 10Regulatory Aspects of Gene and Cellular** Products: A Global Perspective
Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective discusses the different regulatory pathways for genetically (GT) and cell** (CT) medicines implemented by national and international agencies around the world, such as North and South America, Europe, and Asia. Each chapter of the book is written by experts from various regulatory agencies in the international community, guiding readers through the application of non-clinical to translational clinical research, as well as the licensing of these innovative products. More specifically, each chapter of the book provides insights into the fundamental considerations that are critical for CT and GT product developers in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as relevant "must-know" guidelines and regulations.
Regulatory Aspects of Gene and Cell Products: A Global Perspective is part of a highly successful subseries of the American Society for Gene and Cell's highly successful Advances in Experimental Medicine and Biology series. This is a must-read for graduate students, clinicians, and researchers interested in genes and cells** and drug regulation.
The advanced ** has matured all over the world. Many genetically and cell-based medicines are now available in Europe, North America, South America, and Asia, and can cause many serious diseases that have not been seen before. The American Society of Genes and Cells** and Springer Nature contributed to the publication of the first edition of this book and the second edition of this book to update the regulatory environment for advanced** pharmaceutical products (ATMPs). Believing that this updated version of the book will be especially useful for those who are interested in or working in the field of ATMP, since the first edition of the book has proven to be the most important technique for patients and ...
This book provides an up-to-date overview of the regulatory policies and requirements used to develop ATMPs into market-licensed medicines in different parts of the world. The book contains chapters on the regulatory bodies already available in the first edition, as well as chapters from other regions (i.e., India, Malaysia, Spain, Thailand) and contributions to international harmonization. Each chapter of this book provides up-to-date information on the regulatory procedures and requirements implemented by specific regulatory agencies to achieve clinical development of ATMPs, as well as some useful information on the health technology assessment applied to the market of products. Since the first edition, the basic regulations applicable to ATMPs have not changed, while all players in the field (regulators and developers) have gained considerable experience, thus refining the regulatory and development processes, allowing many products to be available in most countries. The similarities are now more pronounced than the differences between the regions, which is also a positive result of the efforts of the international community to harmonize procedures and requirements.
In conclusion, this book provides current perspectives from regulators around the world and hopefully serves as a resource for academia, industry, and institutions on ATMP regulation.
We believe that the outstanding contributions made in this book will continue to promote the global development of safe and effective ATMPs for the benefit of all patients."
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