Webinar on Biomanufacturing 4.0 – A New Era in Cell and Gene Therapy Development

1 www.bisresearch.com
Biomanufacturing 4.0- New Era in Cell and Gene Therapy Development
Biomanufacturing
4.0- New Era in Cell
and Gene Therapy
Development
Webinar February 2023

2 Biomanufacturing 4.0- New Era in Cell and Gene Therapy Development www.bisresearch.com
Likhesh Sharma
Product Manager
Mario Belman
Sales and Application Specialist
Akash Mhaskar
Principal Analyst
Stuti Kandpal
Sr. Research Associate
Speakers

Agenda
1. To understand the cell and gene therapy ecosystem
2. To analyze the factors promoting the growth of the cell and
gene therapy biomanufacturing market
3. To identify the new trends and key developments in the market
4. To understand biomanufacturing 4.0 and its current as well as
future adoption scenario
5. Conclusion

Industry 4.0 – Digital Revolution
Industry 4.0, also known as the fourth industrial revolution, is a term used to describe the current trend of
automation and data exchange in manufacturing and other industries.
Interconnectivity
• Systems are connected with each other
creating efficient communication and allowing
the automation of processes
Data Transparency
• Development and deployment of sensors and
data collection devices enable real-time data
capture and help in faster decision-making.
Decentralized Decision Making
• Independent decisions can be made by
systems and machines via computerized
algorithms limiting bottlenecks.
Customer Centricity
• Enables to produce customer-specific
products catering to individual needs and
markets.
Reduced
Manufacturing
Cost
Flexibility
Real Time
Decision Making
Customization
Efficiency
Industry
4.0
IoT
Big Data
3D Printing/
Additive
Manufacturing
AR/ VR
Simulation
Automation
Cloud
Computing
Integration

Smart Biomanufacturing Triad – A Key Component in Biomanufacturing 4.0
Production
In silico
Design
Automated
Learning
Data-Driven Modelling

Smart Biomanufacturing
Smart Biomanufacturing is the use of advanced analytical tools and technologies to streamline the entire
biomanufacturing process.
▪ Primarily focused on the development of biological products
(biologics, biopharmaceuticals, and other biological products).
▪ Relies heavily on the information generated by the sensors and
data-gathering equipment within the entire biomanufacturing
process. Introduction of new sensing technologies that provide
high levels of flow rate measurements, temperature, pressure,
and other metrics.
▪ Enables biomanufacturers to make efficient, cost-effective, and
environment-friendly manufacturing processes along with
improved efficacy, productivity, and reliability of finished
products.

Automated Learning in Biomanufacturing
Automated learning is the use of machine learning and other AI tools to analyze data gathered from
biomanufacturing processes, identify data patterns and correlations to optimize processes in real time.
▪ Deep Learning - has enabled the study of complex large
datasets of information gathered from various steps of
bioprocessing.
▪ Reinforcement Learning - machine learning platform
that enables training systems to make decisions based on
environmental feedback.
▪ Transfer Learning – use of machine learning to share
knowledge from a pre-trained system to a new system.
▪ Explainable AI – AI-enabled systems that enable
understanding and help interpret the decisions made by
machine learning algorithms.

In-silico Designing in Biomanufacturing
In-silico designing is the use of computer simulations for designing biological products, bioprocesses and systems.
▪ Generative Models Using AI and Machine
Learning – use of machine learning to simulate new
biological designs, predict system behavior, and bioprocessing
optimization.
▪ Multi-Scale Modeling – a simulation technique that
helps in modeling bioprocessing systems on various scales.
This helps in scaling up/scaling down predictions and making
accurate predictions on system behavior.
▪ In-silico Based Synthetic Biology – use of in-
silico designing for accelerated development of synthetic
biologicals, systems, and components.

Smart Biomanufacturing – In a Nutshell
Digitalization Integration Modelling Management Automation
A
d
d
e
d
V
a
l
u
e
Smart Digital Twin
Risk Assessment
Basics for
Control and
Automation
Decision Basis
Transfer Learning
Knowledge Integration
Connectivity to all info
sources
Smart Digital
Stakeholder
Basics for Control
and Automation
Decision
Basis
Transfer
Learning
Knowledge
Integration
Connectivity to all
info sources
Smart Factory
Smart Robots
Real-time
Optimization
Parallel
Learning
Scheduling
Operations
Digital Platform
Centralizations
IT Infrastructure
Connectivity
Process
Infrastructure
Connectivity
Online
Visualizations
Digital Data
Standardization
Data Sources
Accessibility

Industrializing the
cell therapy
ecosystem
Enabling key pillars of innovation
Likhesh Sharma, PhD
Product Manager, Cytiva

Cytiva 2
Outline
1. Engineering a new therapeutic landscape
2. Addressing roadblocks
3. Detours — finding a new way
4. Building connected and technology-driven
digital highways

Cytiva 3
Science has ushered in a
new world of personalized
medicine
3
Your journey is to bring a new therapy safely and
efficaciously to market, where it can become a
life-changing option for all who need it.
Our journey is to help you do so, enhancing speed to
market, industrializing processes, expanding access, and
reducing costs. We want our journey to be part of yours.
But at the end of the day, both of our journeys are in the
service of another — the patient journey. Like you, we are
deeply committed to improving patient lives.
In this rapidly evolving landscape, we
are all on a journey.

Cytiva 4
4
Our vision is a world
in which access to
life-changing therapies
transforms human
health.

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Things on our mind
How can we speed up time to market?
What is the optimal manufacturing process for
increasingly fragmented markets?
How can the industry manufacture with agility
and efficiency?
Is this overall supply chain robust enough to
support rapid growth?
5

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1
Engineering a new
therapeutic landscape

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Alliance for Regenerative Medicine, Report on Oncology and Regenerative Medicine. https://alliancerm.org/indication-
data/oncology-regenerative-medicine/. Published June 2019. Accessed 14 April 2021.
1001+
22
Oceania*
*Australia, New Zealand, Marshall Islands
1
Africa
13
South America
543
North America
515
Gene therapy
632
Cell therapy
136
Tissue engineering/biomaterials
Active regenerative medicine and
advanced therapy developers
worldwide
1078 Clinical trials currently ongoing worldwide
Gene therapy
359
Cell-based
immuno-oncology
471
Cell therapy
204
Tissue engineering
44
238
Europe and Israel
184
Asia
Sector growth

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The success of cell and gene
therapies depends on an
evolving manufacturing
toolbox
8

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New therapeutic modalities are increasingly complex and synthetic
10
Lipsitz et al. 2017 CELL Stem Cell
Lipsitz YY, Timmins NE, Zandstra PW. Quality cell therapy manufacturing by design.
Nat Biotechnol. 2016;34(4):393-400. doi:10.1038/nbt.3525

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Interdependency of evolving elements
Process focus
• Scalability
• Digital
• Sensor enabled/Process
analytical technologies (PAT)
Capacity and cost
Biology
Supply chain
Manufacturing
Therapeutics focus
• Cell-based
• Synthetic
• Derivatives (e.g., exosomes)
• Personalized
Complexity and correlation
Logistics focus
• Identity/custody/quality
• Banking
• Tissue processing
• Network orchestration
Connectivity and control
Digital

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2
Addressing roadblocks

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3 – 7 Years. $1M – $25M
Research Development Launch
Challenge
It takes years and significant investment to bring new production platforms to market
1 2 3 4 5 6 7 8 9
TRL
Level
Product
Idea
Innovation
loop
Product
loop
Close this gap
- Prototyping, testing, iterating
- Design for manufacturability
- Design for serviceability
- Process development and optimization

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Designing modular and high dynamic range expansion platforms
We can’t pick therapeutic winners and losers, but can aim to support them all
• T-cells
• NK Cells
• Dendritic cells
• iPSCs (aggregate)
• Lentivirus
• AAV
• Chondrocytes
• Neurons
• Cardiomyocytes
• MSCs
• iPSCs
• AAV
• Lentivirus
• Fibroblasts
Control / process
analytics
• DO
• pH
• Cell density
• Feeding
• Temp
Cell growth
format
• Single
• Aggregate
• Hydrogel
• Microcarrier
• Hollow fiber
Bioreactor
mechanism
• Static
• Stirred tank
• Shaker
• Rocking
Scale
Growth
format
Pick your options
NK = natural killer; iPSCs = induced pluripotent stem cells; MSCs = mesenchymal stem cells; AAV = adeno-associated virus; DO = dissolved oxygen

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Challenge
Manufacturing – Capacity, quality, and cost
Indication Relapsed chronic lymphoid
leukemia immunotherapy
Umbilical cord blood
transplantation for acute myeloid
leukemia
Immunomodulatory MSC therapy
for Crohn’s disease
Cardiomyocyte replacement
therapy after chronic heart
failure
Therapeutic cell type Autologous CAR-engineering T cells Allogeneic umbilical cord blood Allogeneic MSC transplant Allogeneic cardiomyocyte derived
from differentiated PSCs
Cell source Autologous CAR-engineering T cells Fresh allogeneic umbilical cord blood Allogeneic transplant from
differentiated PSCs
Allogeneic MSC transplant
Therapy type Patient specific Bulk manufacturing
Patients per year 1.6 × 104 (USA) 8 × 103 (worldwide) 2.8 × 105 (worldwide) 6 × 106 (USA)
Cells per dose 3 × 107 CAR-T cells/kg 2.5 × 107 cells/kg, or
2 × 105 CD34+ cells/kg
1 × 108 MSCs 109 cardiomyocytes
Doses per batch 1 1 > 100 > 1000
Number of annual batches 160 000 (USA) 8000 280 6000 (USA)
Scale out Scale up
Courtesy CCRM Toronto
PSCs = pluripotent stem cells; MSCs = mesenchymal stem cells

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3
Finding our (new) way

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Plasmid
mAb
Protein
Virus
Bioinformatics and
payload design
Plasmid production/fermentation
Transduction/biomolecular delivery
Synthesis | In silico Bioprocesses
mAb, protein, virus production
Cell therapy, transplant, and banking
RNA production
Cell therapies
(ex vivo gene
therapies)
Biologics
Vaccines
Gene therapies
Payload production
Cell line development
+
Cell therapies
GATCATCGTACCCTGACTGACT
GATCCTAATTGACTAGTCACA
TACGTTACTAGCTTAACGACG
ATCATCGTACCCTGACTGACTG
ATCCTAATTGACTAGTCACATA
CGTTACTAGCTTAACGACGATC
ATCGTACCCTGACTGACTGATC
CTAATTGAC
CONTENT
(Gene of Interest or GOI):
CARs, T cell receptors,
CRISPR, TALENS, transposons,
or any expressible protein
- mAb
- Viral packaging elements
GOI
cGMP Bulk
cGMP Bulk
Drug product or
ancillary material
Cell line development Therapeutic modality
Stage 1: New therapies should conserve modalities

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Harvest and
formulation
Viral
production
Administration
Cryogenics
and logistics
Analytics
and release
Cell
expansion
Cell
engineering
Stage 2: Platforms built from modular subroutines
Selection/
enrichment
Allogeneic
Autologous
Banking and
storage
Connectivity
HSC, cord blood
Allogeneic, iPSC, MSCs
Immunotherapy Gene therapy
Patient
iPSCs = induced pluripotent stem cells; MSCs = mesenchymal stem cells

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• 95 employees
• Global network of partners
• Helped partners raise >$750M
• Established in 2011
• $15M seed funding
• $100M raised to-date
• 400+ opportunities assessed
• 25 technology projects ongoing
• 8 portfolio companies to-date
• 6 business units, unique facilities
• 200+ projects with 38 clients
• $15M in contract services
Key numbers:
Closing the gap
between innovation
and production
CCRM and Cytiva Center of Excellence

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1
2
3
4
5
6
7
8
9
10
11
CCRM
Toronto is home to
40% of Canadian
biotech industry
30+ specialized
medical and science
research centers
Over 140 000
students and
researchers
Toronto Biotechnology
sector ranks fourth in
North America
CCRM and Cytiva Center of Excellence
Toronto:
Toronto is home to
40% of Canadian
biotech industry
30+ specialized
medical and science
research centers
Over 140,000
students and
researchers
sector ranks 4th in
North America
Toronto is home to
40% of Canadian
biotech industry
30+ specialized
medical and science
research centers
Over 140,000
students and
researchers
sector ranks 4th in
North America
1
2
3
4
5
6
7
8
9
10
11

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Advanced manufacturing — Critical CMC gaps
A. Patient-specific cell manufacturing Technology gap B. Bulk-cell manufacturing
Aseptic coupling
Process closure
Automation
Scale out/parallelization
Small scale solutions
Product characterization
Controlled freezing/thawing
Waste stream extraction/thawing
Improved filtration and centrifugation
High-capacity culture
Reduce media cost/utilization
Improved selection technology
Intensification
Metabolomics/proteomics
Vialing technologies
Chromatography and sedimentation
Dynamic range
Transduction technology
Automation, parallelization, and real-time analysis
Cost and post
production
processing
Common
challenges
Source: CCRM
Gap impact score
High
Low
CMC = chemistry,
manufacturing, and
controls

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BRIDGE 1.0 Focus: Fast Trak™ process development services
Culture intensification
Workflow simplification
Media development
Process closure and automation
Cost analysis and risk reduction

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Example 1: Reprogramming, gene editing, and PSC scale-up
Delivered >150 iPSC lines, representing 15 different
disease models to academic investigators
PSC scale-up: >35 billion cells
7.7E+09
1.5E+10
2.5E+10
3.6E+10
1.E+07
1.E+08
1.E+09
1.E+10
8 10 12 14 16 18 20 22 24 26 28
Total
Viable
Cells
Day
ESI-017 - black
NCRM1 - grey
0.1-0.2 L 1 L 10 L
0.1 – 0.2 L 0.1 L 10 L
Total
viable
cells
1.0 × 107
1.0 × 108
1.0 × 109
1.0 × 1010
3.6 × 1010
2.5 × 1010
1.5 × 1010
7.7 × 109
iPSCs = induced pluripotent stem cells

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Example 2: Viral vector production processes
• cGMP suspension producer cell lines
• Higher titer productivity
• Plasmid design and manufacturing for induction
• Improved scalability
• Reduced labor and space
• Improved sterile filtration
• Better affinity reagents
• Higher recoveries
50 L lentiviral vector (LV)
Industry gaps
Seed Production bioreactor Harvest Purification and conditioning operations
ReadyToProcess WAVE™ 25
rocker
Xcellerex™ XDR 50 bioreactor
• Transfection
• Benzonase treatment
WM 620
Filter capsule
Tangential flow
filtration (TFF)
ÄKTA™ brand
readyflux™ column
Hollow fiber
Scavenger
ÄKTA™ ready
ReadyToProcess™ column
Capto™ Core 700 column
Polishing
ÄKTA™ ready
ReadyToProcess™ column
Sepharose™ 4 Fast Flow
column
Sterile filtration
WM 620
Filter capsule

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Example 3: Multivariate culture media optimization
Discovery metabolomics
▼
Discovery proteomics
▼
Literature mining
▼
DoE design
▼
1st HTS iteration
▼
2nd HTS iteration
▼
3rd HTS iteration
▼
In-house built formulation large scale validation
▼
HyClone™ built formulation large scale validation*
▼
Top 1 formulation handover to HyClone™ for production
Media depletion
28 factor, 2 level fractional factorial
15 factor, 2 level fractional factorial
6 factor, 3 and 2 level fractional factorial
Top 3 formulations
Rapid response production
Hundreds of candidates
Tens of candidates
Fractional factorial
200 conditions in parallel
128 conditions × 8 replicates in parallel
144 conditions × 6 replicates in parallel
Commercial medium
*Ongoing

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Example 3: Multivariate culture media optimization
- Robotics
- Analytics
- Modeling
Chemically defined, reduced formulation complexity, animal-component
free, cost optimized in less than 9 months
Small scale media comparison (day 5) Large scale media comparison (day 9)

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4
Building connected and
technology-driven
digital highways

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Collecting and applying insights will
lead to better patient outcomes
Process insights
Product insights
Disease insights
Patient insights
Improved productivity
and effectiveness
Digital
Smarter R&D More effective
clinical trials
Optimized
manufacturing
Better patient
experience
Analytics In R&D In CMC In the market
CMC = chemistry, manufacturing, and controls

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Digitally connecting elements of workflow for cellular therapies
Chronicle™ automation software
VIA Freeze™
range
VIA Thaw™
series
VIA Capsule™
solution
VIA Capsule™
solution
VIA Freeze™
range
VIA Thaw™
series
Highest value product
Least defined science

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The finish line
• Pace of clinical advancement is unprecedented
• Systems biology will underpin advanced
therapies for years to come
• Partnerships can drive progress
• Analytics will improve manufacturing and
clinical outcomes
• There are few shortcuts on the road to success

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What is the next step in your cell
and gene therapy journey?
• Cytiva has the foundational knowledge to help
commercialize your therapy
• We have deep expertise in the critical areas of the
cell and gene therapy ecosystem
• We have the integrated solutions to help bring
your therapy safely and efficaciously to market
31
Our mission is to
advance and
accelerate
therapeutics.
Cytiva

cytiva.com
Cytiva and the Drop logo are trademarks of Life Sciences IP Holdings Corporation or an affiliate.
ÄKTA, Chronicle, HyClone, readyflux, ReadyToProcess, ReadyToProcess WAVE™, Sepharose, VIA Capsule, VIA Freeze and VIA Thaw are trademarks of Global Life Sciences Solutions USA LLC or an affiliate.
All third-party trademarks are the property of their respective owners.
© 2020-2021 Cytiva
For local office contact information, visit cytiva.com/contact
CY20657-DDMMMYY-PP

11
BIOMANUFACTURING
4.0
A New Era in Gene & Cell Therapy Development
Cell Culture Technologies

12
Table of Contents
01
Introduction 03
Challenges
02
Nowadays
What is Cell Therapy?
How is it done?
Production
Global Harmonization
Quality
Applications
Cell Culture Manufacturing

Introduction
How is it done?

14
Repair and/or Replace cells
The main objective of this precision medicine is to
take ill cells, repair them in vitro and introduce
them in the diseased organ to replace ill cells with
healthier ones.

15
How Is it done?
Cell Therapy Action Mechanism
There are two main types of diseases;
● Caused by external factors,
● Caused by internal factors.
There are two main principles by which cells
facilitate therapeutic action:
● Replacement of damaged tissue
● Release soluble factors such as
● Cytokines,
● Chemokines, and
● Growth factors
Cell therapy strategies
Where cells came from?
The origin of cells is diverse, so far there are only
models:
● Allogeneic
● Autologous
● Xenogeneic

16
Type of Cells Involved
So many countries have no regulation or specific law about the use of cells as a medicine or treatment, but Scientifics
and developers have been defining cell types about their capacity and specific function:
● Neural Stem Cell (NSCs)
● Neurological disorders such as Parkinson’s disease and Huntington’s disease.
● Mesenchymal Stem Cell (MSCs)
● They can be used for a wide range of treatments including immune-modulatory therapy, bone and cartilage
regeneration, myocardium regeneration, and the treatment of Hurler syndrome, a skeletal and neurological disorder.
● Hematopoietic stem cells (HSCs)
● Derived from bone marrow or blood,
● CAR-T
● Cancer cell death
● Differentiated or mature cell transplantation
● Investigations are exploring the transplantation of differentiated cells that only possess low or no proliferation ability.
● Human Embryonic Stem Cells
● These cells are being investigated as the basis for a number of therapeutic applications, Not allowed in some countryes

Nowadays
Applications

18
Cell Therapy Manufacturing
Cell culture technology
Cells are the product; They are not modified so we
what to keep their native properties and
capabilities
The first step of production is based on cell culture
technologies.
It is essential to optimize the combination between
cell, culture medium and support. To this screen
work at small volume and well know conditions is
the key

19
Culture Optimization:
The public enemy number one is Time and
resources are not so fast.
Many times, for wanting to obtain results in a short
time, stages are skipped, or studies are omitted,
which can drastically impact the result in
production.
Single-use development could help to reduce
process time but invest in delivery time.
Big data tools simplify processes and give you extra
information to optimize with the lest try and failure.

20
Scale up
Bioreactor technology provides all tools and
benefits that cell need.
Upstream
If media, cell, and carrier are properly selected and
optimised, upstream scale up keeps they
conditions and results. Work in bioreactor could
help us to maintain the quality attributes without
altering the nature of the cells
Downstream
Separation and purification technologies are also
optimized but still at the pilot or low-scale level.

21
Applications
Cell therapy use and application are very varied,
but most of them are in the clinical and medical
fields:
● Meat in Vitro production
● Cosmetic (Skin and Hair)
● Reagents and inductors.
● Cancer
● Autoimmune Diseases (Vaccines)
● Bio degenerative diseases
● Bone marrow transplant
● Fertility therapies

Challenges
Production
Global Harmonization
Quality

23
Production
Main Challenges
For cell therapy manufacture has the same
challenges that every cell culture bioprocess.
● Property conservation
● Cell density (Micro Carriers)
● Viability
● Harvest (Scale up)
● Product Sterility
● “Purification”
● Q-A

24
Downstream Scale-Up
Yield Harvest Scale up
Harvesting and separating cells from
the microcarrier must achieve high
yield and viability greater than 85%
to be a cost-effective process..
Upstream and Downstream yield
are top priorities.
Most of the therapies need the
highest cell densities to be a
success.

25
Clinical Application
Fill and Finish Facilities
Cell production area and clinical
application most be at the same
facility but very different
qualification areas.
Increase maintenance investment.
End product most be applied just
few minutes or not more that
couple of hors at the end user.
Just for fertility app could have
cryopreservation or stored.

26
Cell Therapy Manufacturing
In conclusion:
Cell culture techniques are the basis of cell therapy, but they present a greater challenge and avoid
differentiation as much as possible and keep the properties of the cells intact.
Cell therapy is a good alternative for diseases that require greater precision to be eradicated.
I believe that in a short time the technologies of microcarriers, culture media and separation and harvest
systems will make cell therapy more and more used.

Cell and Gene Therapy
Biomanufacturing Market: Ecosystem,
Market Dynamics, Trends, and Current
& Future Market Outlook

• Thermo Fisher Scientific Inc.,
• Merck KGaA, and
• Sartorius AG, among others
Biomanufacturers
Contract Research Organizations
(CROs)
Contract Manufacturing Organizations
(CMOs)
Life Sciences Companies
Market Ecosystem

Factors Promoting the Growth of Cell and Gene Therapy Biomanufacturing Market
Rising Numbers of Approved Cell and
Gene Therapies
6
11
10
0
2
4
6
8
10
12
2011-2015 2016-2020 2021-2022
Number
of
Approved
Cell
and
Gene
Therapies
Currently, there are more than 25 cell and gene
therapies approved by the FDA in the last 10
years.
Increasing Investments and Funding
$22.7 billion was raised by the developers in
2021
Continuous Entry of New Industry
Participants
Increase within six months
~85% ~15% ~10%
20.8
29.7
35.3
0.0
5.0
10.0
15.0
20.0
25.0
30.0
35.0
40.0
2017-2018 2019-2020 2021-2022
Investment
($Billion)
651
230
453
35
686
244
492
35
0
100
200
300
400
500
600
700
800
North America Europe Asia-Pacific Others

Growing Single-Use Technology Market Penetration:
▪ Construction of stainless-steel-based facilities takes longer time and is more complex than single-use facilities
▪ Therefore, investment in stainless-steel facilities must start in the early clinical phase II
▪ The construction of single-use facilities may start in clinical phase III, reducing investment risks
Probability that the candidate
advances to the next stage
Probability of approval
~60%
~10%
~30% ~50%
Clinical Phase I Clinical Phase II Clinical Phase III
~15% ~50%
Key Trends

Shift from Open Processing to Closed Processing for Cell and Gene Therapy Biomanufacturing:
▪ A closed system provides the protection of a cleanroom against contamination without the costs coupled with maintaining it.
▪ Single- Use Technologies can be utilized to their maximum potential with the integration of closed biomanufacturing in cell and gene
therapy to provide effective therapies.
Unmet Needs What They Did Impact
Need for second-generation
automated systems
Launched CliniMACS
Prodigy system
Made the manufacturing of
various therapies possible using
a single platform, from cell
activation to the final
harvesting step
Need for automated cell
culture system
Launched CompacT
SelecT
Made large-scale expansion of
stem cell cells possible with
high reproducibility and eliminated
the risk of contamination
Key Trends

55%
22%
23%
Gene Therapies Cell Therapies RNA Therapies
Process Intensification Unmet Needs Yields Robust Manufacturing
Robust clinical pipeline of new modalities offers growth
opportunities
▪ Currently, there are 3,726 therapies under development, ranging from the
preclinical through the pre-registration phase.
▪ 2,053 gene therapies (including genetically modified cell therapies such as
CAR-T cell therapies) capture 55% of the total cell, gene, and RNA therapies.
▪ The non-genetically modified cell therapies account for 22% of the cell, gene,
and RNA therapies with 827 entities in the clinical pipeline.
▪ Lastly, RNA therapies make up 23% of the clinical trials with 846 therapies
under development.
▪ Introduction of viral-based therapies and advanced cell and gene therapies are
expected to boost the cell and gene therapy biomanufacturing market.
Opportunities

Current Market Scenario and Future Outlook
Upstream Processing Downstream Processing Harvesting
Biomanufacturing
~20%
~50%
~20%
~30%

4.04
5.82
1.79
5.75
9.02
2.70
9.26
15.92
4.59
0.00 5.00 10.00 15.00 20.00
Equipment
Consumables
Software
Solutions
Market Size ($Billion), by Product Type
2031 2026 2021
49.97%
34.67%
15.36%
Market Share, (by Product Type), 2021
Consumables Equipment Software Solutions
Global Cell and Gene Therapy
Biomanufacturing Market
2021: $11.65 Billion
2031: $29.76 Billion
CAGR: 10.31% (2022-2031)

Discovery
4-5 years
Preclinical
Testing
1 year
Clinical Trials
4-7 years
Approval
1-2 years
2021 2031
CAGR (2022-2031)
~12% Commercial Stage
Manufacturing
~9% Research Stage
Manufacturing
Commercial Stage Manufacturing:
~38%
Research Stage Manufacturing:
~62%
Research Stage Manufacturing:
~54%
Commercial Stage Manufacturing:
~45%

Cell and Gene Therapy Related Research Titles
Published Titles
• Cell and Gene Therapy Market
• Regenerative Medicine Market
• CRISPR Gene Editing Market
• Cell and Gene Therapy Manufacturing QC
Market
• Cell and Gene Therapy Drug Delivery
Devices Market
• Nucleic Acid CDMO Market
• CAR-T cell Therapy Market
• Natural Killer Cell Therapeutics Market
• Stem Cell Therapy Market
• Gene Cloning Services Market
• Oncolytic Virus Therapies
• Next Generation Biomanufacturing Market
Titles in Progress
• Cell and Gene Therapy Biomanufacturing
Market
• Biopharma CMO & CRO Services Market
• Global NGS Liquid Handling Market
• Global Rare Disease Diagnostics Market
• Point-of-Care Cell and Gene
Manufacturing Market
• Exosomes Market
• Allogeneic Cell Therapies Market
• Cell Banking Services Market
• Gene Therapies for Rare Diseases
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