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Gene Therapy Quality Assurance & GMP Compliance Consulting

Comprehensive quality assurance for AAV gene therapy and viral vector programs — from QMS design and GMP compliance through adventitious agent testing strategies, vector shedding protocols, and BLA-ready quality systems.

Support Across All Stages

Ensuring Compliance & Product Integrity for Gene Therapy

Dynamic Engineering Solutions (DES) provides integrated quality assurance support to ensure patient safety and regulatory compliance throughout the drug development lifecycle. Our team works alongside development, manufacturing, and regulatory groups to implement practical quality systems that maintain product integrity while meeting evolving global regulatory expectations.

DES supports advanced therapy programs—including gene and cell therapies—by strengthening quality infrastructure across development, technology transfer, manufacturing, and clinical operations. Our approach focuses on building sustainable quality systems, preparing organizations for regulatory inspections, and ensuring consistent oversight of complex biologic products such as viral vectors.

Because advanced therapies present unique quality challenges, DES brings specialized expertise in areas that extend beyond traditional biologic QA frameworks. This includes support for viral vector quality strategies, regulatory inspection readiness, risk-based quality oversight, and alignment with FDA/CBER expectations for emerging therapeutic platforms.

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Advanced Therapy Quality & Compliance Services

Quality systems and regulatory compliance support for gene therapy, biologics, and advanced therapy programs

DES provides specialized quality assurance consulting to support advanced therapy and biologic development programs. Our team helps organizations build and strengthen GMP quality systems, prepare for regulatory inspections, and ensure that manufacturing and CMC documentation meet FDA and global regulatory expectations.

We support sponsors, biotech companies, and manufacturing organizations in establishing scalable quality frameworks for viral vector and advanced therapy products—bridging the gap between early-stage development and fully compliant commercial GMP operations.

✅ GMP Quality System Development & Remediation

✅ FDA & CBER Inspection Readiness

✅ Quality Oversight for Viral Vector Manufacturing

✅ CAPA & Deviation System Implementation

✅ Gene ICH Q5A(R2) Compliance & Viral Safety Strategy

✅ IND / BLA CMC Quality Section Review

Our Quality Approach

Smart, Efficient QA Operations for Viral Vector Programs

We deliver value through operational efficiency — a streamlined approach to gene therapy quality assurance that prioritizes critical quality activities directly impacting patient safety and robust product supply.

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Supplier Management & Qualification

→ GMP compliance evaluation of viral vector CDMOs and raw material suppliers

→ Quality management system (QMS) design for gene therapy programs

→ Vendor qualification for plasmid manufacturers, CDMO, and analytical labs

→ Quality agreement (QA) development with risk categorization for AAV supply chain

→ Adventitious agent testing laboratory qualification

→ Biosafety laboratory qualification and classification support

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Auditing & Assessment Systems

→ ICH Q9 risk management framework implementation for gene therapy

→ Comprehensive GMP audit programs for AAV manufacturing facilities

→ Audit finding resolution and CAPA oversight systems

→ FDA (CBER) response support and inspection preparation

→ Mock FDA Pre-Approval Inspection (PAI) readiness assessments

→ EMA GMP inspection readiness support for gene therapy sites

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Material Quality Management

→ Incoming material inspection and release for viral vector raw materials

→ Raw material identification and approval systems (plasmids, resins, media)

→ Release documentation management for GMP viral vector components

→ Adventitious agent control strategy for raw materials (ICH Q5A(R2))

→ Cell substrate qualification and testing program oversight

→ Master Cell Bank (MCB) / Working Cell Bank (WCB) release support

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Manufacturing Operations QA

→ GMP document review, batch record approval for viral vector manufacturing

→ Deviation investigation management for gene therapy manufacturing events

→ Change control management (manufacturing process, equipment, site)

→ Personnel training oversight and GMP qualification programs

→ Contamination control strategy development and review

→ Environmental monitoring program design for viral vector suites

Supporting Reliable Outcomes

Robust QA Processes Across All Operations

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Technical Operations

CMC technical documentation review, analytical method lifecycle management, and technical operations qualification

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QC Operations

Laboratory document review, deviation investigation, OOS investigation management, and data integrity assessments (21 CFR Part 11)

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Product Release

Batch release approval, specification review, COA review, Product Quality Review (PQR), and Annual Product Review (APR)

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Gene Therapy-Specific

Adventitious agent testing, vector shedding protocols, genotoxicity risk, and CBER-specific compliance oversight

Gene Therapy QA Expertise

Specialized Compliance for Viral Vector Programs

Gene therapy quality assurance is categorically distinct from conventional biologic QA. AAV programs face unique compliance requirements — from the design of adventitious agent testing panels aligned with ICH Q5A(R2) to the development of vector shedding study protocols that meet FDA and EMA post-marketing expectations, to the genotoxicity risk frameworks required by CBER.

DES Pharma’s QA consultants have built quality systems specifically for gene therapy programs, understanding the intersection of viral vector biology, manufacturing complexity, and regulatory expectations. We help gene therapy companies build quality cultures and systems that hold up under CBER pre-approval inspections — avoiding the delays that poorly designed gene therapy QA programs consistently produce.

Whether you need to design your first GMP-compliant QMS, prepare for a CBER audit, or develop a vector shedding risk assessment for your clinical program, DES Pharma delivers the specialized quality expertise to keep your gene therapy program compliant and on track.

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Adventitious Agent Testing Program Design

ICH Q5A(R2) compliant testing strategy including in-vitro and in-vivo assays, PCR-based screening, and cell-based detection methods for AAV manufacturing

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Vector Shedding Protocol Development

FDA and EMA-aligned shedding study designs for clinical trials and post-marketing commitments — sample collection strategy, assay selection, and data interpretation frameworks

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Genotoxicity & Insertional Mutagenesis Risk

Risk-based framework development for integration site analysis, long-term follow-up (LTFU) study design, and regulatory justification for AAV genotoxicity risk assessment

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FDA CBER Inspection Readiness

Mock PAI assessments, CBER-specific inspection preparation, prior approval supplement (PAS) strategy, and FDA response support for gene therapy manufacturing

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IND / BLA Quality Section Authorship

CMC quality narrative development, quality overall summary (QOS), and CBER-aligned QA section documentation for gene therapy IND and BLA submissions

Key Regulatory Framework

Gene Therapy QA Regulatory Reference Guide

Critical regulatory requirements that define the compliance framework for AAV gene therapy quality systems — each requiring specialized expertise beyond standard biologic QA.

Regulatory Requirement	Guideline / Reference	Gene Therapy Significance	Type
Adventitious Agent Testing	ICH Q5A(R2); 21 CFR 610; USP <71>	Mandatory for all viral vector cell substrates and production processes — more extensive than standard biologics	Required
Vector Shedding Studies	FDA CBER Guidance 2015; EMA Guideline 2009	Clinical and post-marketing studies to assess patient-to-patient transmission risk via body fluids	Required
Long-Term Follow-Up (LTFU)	FDA CBER Guidance 2020 (GT LTFU)	15-year patient monitoring program required for integrating gene therapies; AAV risk stratification may reduce duration	Required
Genotoxicity / Insertional Risk	FDA 2006 GTGTF; EMA Guideline EMEA/236981	Risk-based assessment required; AAV generally considered low integration risk but documentation required	GT-Specific
Replication Competent Virus (RCV)	FDA CBER Guidance; ICH Q5A	RCV assays required for every AAV lot to confirm absence of replication-competent particles	Required
Cell Substrate Qualification	ICH Q5B; ICH Q5D; USP <1010>	MCB/WCB characterization and qualification required for HEK293, Sf9, and other cell substrates used in AAV manufacturing	Required
Data Integrity (GMP)	21 CFR Part 11; FDA DI Guidance 2018; EMA ANNEX 11	Electronic records and signatures requirements for all GMP data generated during viral vector manufacturing and testing	Required

Our Quality Methodology

How We Build Gene Therapy Quality Systems That Last

A four-step approach to building quality systems that are both compliant and operationally sustainable for gene therapy programs.

Quality Risk Assessment

Begin with ICH Q9-based risk assessment of your specific gene therapy product, manufacturing platform, and regulatory pathway to identify the highest-priority quality system gaps.

Phase-Appropriate QMS Design

Build quality systems calibrated to your development stage — IND-enabling QA has different requirements than PPQ-stage or BLA commercial quality systems.

Gene Therapy-Specific Protocol Development

Design and execute adventitious agent testing, vector shedding, and genotoxicity risk protocols that meet FDA CBER and EMA expectations for viral vector products.

Inspection Readiness & BLA Support

Prepare for CBER pre-approval inspections, author quality sections for regulatory submissions, and ensure your QA documentation package supports BLA approval.

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Build a CBER-Ready Gene Therapy Quality System

Ensure your AAV program meets FDA and EMA compliance expectations at every stage.