An ever-evolving regulatory environment makes navigating gene therapy products through to clinic much more complicated than a traditional biologic. While manufacturing platforms and regulatory requirements for testing of antibodies has existed for decades, gene therapy platforms and their testing requirements are changing rapidly with the progression of products toward commercialization.

1. Gene
Therapy
Services

2. An ever-evolving regulatory
environment makes navigating
gene therapy products through
to clinic much more complicated
than a traditional biologic.
While manufacturing platforms and
regulatory requirements for testing
of antibodies has existed for decades,
gene therapy platforms and their
testing requirements are changing
rapidly with the progression of
products toward commercialization.
complex & evolving
environment
The regulatory pathway for gene therapies
is complex and time-intensive. As the
global market rapidly expands, the
interpretation of regulations continues
to evolve, which is why careful planning
of your safety testing program will always
be crucial to clinical success.

3. WuXi Advanced Therapies' integrated approach
to gene testing program design
Building the right safety testing program
requires expertise and experience in a broad
range of disciplines, including virology,
molecular biology and cell-based techniques.
meeting your challenges
Testing plan design
Custom assay development
Multiple testing methodologies
Phase-appropriate validation
Integrating data packages
Meeting aggressive milestones
Understand the goal
timelines, deliverables
Understand the product
vectors, cell lines, clinical phase
Customize to unique needs
standard or custom assays
Navigate regulatory environment
phase-specific requirements
Provide trouble-shooting
Communicate timely updates
dedicated project management
Generate high-quality data
Perform thorough data review
Deliver interpretative reports
Provide comprehensive regulatory support

4. Our state-of-the-art facilities provide adaptable
manufacturing and testing solutions utilizing our
integrated services. We have the capacity to support
any program from early to late stage, with the
additional resources needed to support commercial
volumes.
Our viral vector experience is second to none, and
includes the following:
• Adeno-Associated Virus (AAV) • Adenovirus
• Lentivirus • Retrovirus
Gene Therapies Testing & Manufacturing
Discovery Clinical / CommercialPre-Clinical / Development
In-Vivo
Testing
Testing
Cell & Virus
or Plasmid
Assay
Development
Safety
–––––––
Toxicology
–––––––
Biodistribution
Upstream /
Downstream
Process
Development
Custom
Assay
Development
Small-Scale
Manufacturing
Scale-Up /
Process
Optimization
Process & QC
Analytical
Validation
cGMP
Manufacturing
Product
Release /
Stability
Testing
Patient
Monitoring
Analyses
experience
Ever since the first clinical gene therapy
programs were initiated, WuXi Advanced
Therapies has been providing the technical
testing competencies and regulatory
expertise to help navigate novel biologics
from development through to late-stage
clinical trials — supporting hundreds of
successful regulatory submissions.
WuXi Advanced Therapies offers a fully integrated set of services
to support development and manufacturing of your gene therapy
product — from concept to commercialization.
WuXi Advanced Therapies’unmatched preclinical
services for gene therapy products leverage our
unique mix of scientific and regulatory expertise
in virology, bioanalytical assays and other studies.
Our staff experience, state-of-the-art facilities, and
broad understanding of advanced therapeutic
development will support your IND.
Exceptional Preclinical
Expertise
With decades of experience in biotherapeutics
testing, WuXi Advanced Therapies offers
unparalleled capacity and guidance to reduce
time to clinic. We provide the full spectrum of
testing services, including custom assay
development. As a unique single-source, fully
integrated CDMO, we support gene therapy
products from procurement to comprehensive
product testing for safety, stability and release.
Advanced Testing
Capabilities
Flexible, Commercial-Capacity
Facilities
Extensive Viral Vector
Experience
Services for Gene Therapies
Features of our process development services include:
• Dedicated specialists with pharma experience
• Programs from pre-clinical to commercial
• Upstream and downstream optimization
• Form and fill
• Scale up and scale out
Process Development
Our GMP manufacturing program provides speed and
reliability to support the rapid advance of client
timelines. We offer:
• Dedicated suites
• Clinical and commercial package options that scale
• SUS capability
• Cross-trained staff
• Coverage 7 days a week across multiple shifts
GMP Manufacturing

5. Our state-of-the-art facilities provide adaptable
manufacturing and testing solutions utilizing our
integrated services. We have the capacity to support
any program from early to late stage, with the
additional resources needed to support commercial
volumes.
Our viral vector experience is second to none, and
includes the following:
• Adeno-Associated Virus (AAV) • Adenovirus
• Lentivirus • Retrovirus
Gene Therapies Testing & Manufacturing
Discovery Clinical / CommercialPre-Clinical / Development
In-Vivo
Testing
Testing
Cell & Virus
or Plasmid
Assay
Development
Safety
–––––––
Toxicology
–––––––
Biodistribution
Upstream /
Downstream
Process
Development
Custom
Assay
Development
Small-Scale
Manufacturing
Scale-Up /
Process
Optimization
Process & QC
Analytical
Validation
cGMP
Manufacturing
Product
Release /
Stability
Testing
Patient
Monitoring
Analyses
experience
Ever since the first clinical gene therapy
programs were initiated, WuXi Advanced
Therapies has been providing the technical
testing competencies and regulatory
expertise to help navigate novel biologics
from development through to late-stage
clinical trials — supporting hundreds of
successful regulatory submissions.
WuXi Advanced Therapies offers a fully integrated set of services
to support development and manufacturing of your gene therapy
product — from concept to commercialization.
WuXi Advanced Therapies’unmatched preclinical
services for gene therapy products leverage our
unique mix of scientific and regulatory expertise
in virology, bioanalytical assays and other studies.
Our staff experience, state-of-the-art facilities, and
broad understanding of advanced therapeutic
development will support your IND.
Exceptional Preclinical
Expertise
With decades of experience in biotherapeutics
testing, WuXi Advanced Therapies offers
unparalleled capacity and guidance to reduce
time to clinic. We provide the full spectrum of
testing services, including custom assay
development. As a unique single-source, fully
integrated CDMO, we support gene therapy
products from procurement to comprehensive
product testing for safety, stability and release.
Advanced Testing
Capabilities
Flexible, Commercial-Capacity
Facilities
Extensive Viral Vector
Experience
Services for Gene Therapies
Features of our process development services include:
• Dedicated specialists with pharma experience
• Programs from pre-clinical to commercial
• Upstream and downstream optimization
• Form and fill
• Scale up and scale out
Process Development
Our GMP manufacturing program provides speed and
reliability to support the rapid advance of client
timelines. We offer:
• Dedicated suites
• Clinical and commercial package options that scale
• SUS capability
• Cross-trained staff
• Coverage 7 days a week across multiple shifts
GMP Manufacturing

6. WuXi Advanced Therapies offers a
broad selection of routinely performed
assays and the additional expertise to
develop and validate new custom
assays for your unique programs.
As a leader in gene therapy services, WuXi
Advanced Therapies provides complete support
for your gene therapy program at every stage of
development.
We offer testing services to support programs
in pre-clinical preparation for IND, and phase-
appropriate testing for clinical development,
stability and lot release. Our technical and
regulatory experts are available to design the
right testing program for your unique product.
standard
assays
Leveraging our experience in gene therapy testing,
WuXi Advanced Therapies has developed and
validated a broad selection of routinely performed
standard assays to support gene therapy programs.
These assays are effective and ready to use, saving
time and money compared to developing a new
assay from scratch. Our standard assays include a
complete range of residuals tests to monitor
by-products of the manufacturing process during
development and for lot release. These assays cover
everything from specific host cell proteins and DNA
to leached affinity column ligands, as well as empty
to full ratios of capsids.
Preclinical Services
DNA Sequencing
Raw Material Testing
Efficacy and tK/pK Studies
Risk Assessment
Extractables / Leachables Testing
Toxicology
Dose Finding (MTD) | Subacute | Subchronic | Chronic
Biodistribution
Viral vectors | Human cells
Tumorigenicity
Model development | ESC | iPSC | Adult SC | Cell lines
GMP Cell Banking & Characterization
Viral Bank Safety & Characterization
Viral Clearance

7. custom
assays
Because every product is different, development of
custom assays is often still required for critical testing
that addresses unique aspects of your gene therapy.
Our technical staff has the expertise to rapidly design,
develop and validate these technically challenging
assays – such as product-specific potency assays – to
keep your program moving forward. Semi-custom
assays – such as capsid identity or purity and genome
copy number – can also be validated without lengthy
transfer or development times.
Testing and Release of
GMP Vector Lots
General Characteristics
pH | Appearance | Osmolality
Content / Dose
Viral particle concentration
Vector genome titer
Vector Infectious titer by TCID50
Potency
Custom cell-based gene expression (FACS, ELISA, PCR)
Purity and Identity
Capsid protein identity and purity
Detection of Non-Packaged Transgene DNA
Residuals:
HEK293 Protein
Infectious helper virus
Host cell DNA by qPCR
Benzonase
AVB Resin
Leached Camelid Antibody Fragment
Aggregation by Dynamic Light Scattering
Total Protein Concentration
Empty:Full Capsid (OD 260/280)
Aggregation
Particulates
Purity by SDS-PAGE
Safety
Vector Genome Sequencing
Replication-competent virus: rcAAV, RCL, RCR, RCA
In vitro assay for adventitious viral contaminants
Sterility | Mycoplasma | Endotoxin | Bioburden
Patient Monitoring Testing
In vivo transgene expression | In vivo vector persistence
Custom Assays Include:
Genomic Copy by qPCR
Transgene Expression
Potency Assays
Capsid Protein Identity
Capsid Protein Purity
Infectivity & Expression Assays
Viral Clearance Validation for Clearance
of Helper Viruses

8. The Advanced Therapies business unit of WuXi AppTec is a
global Contract Development and Manufacturing Organization
dedicated to accelerating and transforming development, manufacturing,
and commercialization of cell, gene, and other advanced therapies.
Accelerating Progress
and Time to Market
Integrated CDMO and Testing Service Platform
advancedtherapies.com
CONNECT WITH US
WuXiATU-107-1110118