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AAV Gene Therapy Analytics & Quality Control

Analytical Development and QC consulting
Analytical Target Profile through BLA

Product Quality & Compliance Excellence

Gene Therapy & AAV Analytical Experience

DES Pharma delivers a comprehensive AAV characterization portfolio designed to navigate the complexities of developing advanced therapies. We leverage deep experience with viral vector-specific testing challenges, utilizing robust analytical methods adapted across serotypes to ensure consistent, reliable data. By prioritizing integration with FDA/EMA gene therapy guidelines from day one, we ensure your products meet rigorous standards for safety, efficacy, and global compliance.

We establish a comprehensive AAV testing portfolio aligned with your product’s structure and regulatory expectations. Our strategic approach develops robust analytical tools to rigorously confirm Identity, demonstrate Potency, characterize Purity, ensure Safety, and accurately determine Quantity.

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AAV Gene Therapy Characterization Methods

Specialized analytical capabilities for adeno-associated virus vector quantification and quality assessment

Specialized AAV characterization methods including empty/full capsid ratio analysis, vector genome titer (ddPCR, qPCR), and residual DNA testing. Our analytical consultants develop fit-for-purpose methods that meet FDA and EMA expectations for gene therapy IND and BLA submissions per ICH Q2(R2) and ICH Q14.

🔬 Empty/Full Capsid Ratio (AUC, AEX)

🔬 Vector Genome Titer (ddPCR / qPCR)

🔬 Potency Assay Development & Validation

🔬 Residual DNA & Host Cell Protein Testing

🔬 Capillary Electrophoresis (CE-SDS)

🔬 DLS / SEC Particle Characterization

🔬 Capsid Amino Acid Analysis (CD-Mass Spec)

🔬 Transduction Efficiency & In-Vitro Potency

🔬 Sterility & Adventitious Agent Testing

Guiding Analytical Strategy Through Data

Our AD & QC Frameworks for Gene Therapy

We build structured frameworks that uphold the core principles of analytical development and quality control, calibrated to the unique complexities of AAV and viral vector products.

Analytical Development (AD)

High Structure, High ROI

Analytical Development provides scientific expertise to support key decision points related to AAV gene therapy product safety and efficacy across all phases of development.

✓ Develop accurate, fit-for-purpose analytical methods to measure critical quality attributes (CQAs) for identity, safety, potency, and purity

✓ Define the Analytical Target Profile (ATP) aligned with the Target Product Profile (TPP) — including gene therapy-specific CQAs

✓ Leverage AAV characterization data to enhance product knowledge and optimize methods with innovative technologies

✓ Establish product degradation profiles and identify degradation pathways for vector stability

✓ Develop specifications aligned with ICH Q6B and gene therapy guidance from FDA/CBER and EMA

✓ Analytical lifecycle management per ICH Q14 and USP framework

Quality Control (QC)

Driving Data-Backed Decisions

Quality Control ensures AAV gene therapy products are consistently safe, effective, and high-quality by establishing a clear testing system aligned with global regulations.

✓ Method validation of test panels for product release, routine testing, and stability studies across the viral vector product lifecycle

✓ Monitor analytical method performance to maintain consistent product quality, patient safety, and potency

✓ Meet regulatory requirements for data integrity (21 CFR Part 11) and ICH Q14 standards

✓ Risk-based approach to confirm testing methods are reliable across all development phases

✓ Continuous monitoring and improvement processes to ensure trustworthy data for regulatory approvals

✓ OOS investigation management and CAPA implementation for analytical deviations

AAV-Specific Analytical Methods Reference

Critical Quality Attributes & Testing Methods

CQA / Attribute	Recommended Method(s)
Vector Genome Titer	ddPCR, qPCR (ITR or transgene-based)
Empty/Full Capsid Ratio	AUC, AEX-HPLC, TEM, ELISA
Capsid Titer (Physical Particles)	ELISA (capsid), MALS, DLS
Potency (Transduction)	In vitro transduction assay, luciferase reporter, FACS
Purity — HCP	Host Cell Protein ELISA (process-specific)
Purity — Residual DNA	qPCR (host cell DNA), ThunderBird assay
Identity	SDS-PAGE / Western Blot, VP1/VP2/VP3 ratio
Sterility & Mycoplasma	USP <71> sterility; PCR mycoplasma
Adventitious Agents	Broad-range PCR, cell-based assays, in-vivo testing

Gene Therapy Analytical Expertise

Deep Expertise in AAV Vector Characterization

Gene therapy analytical development requires more than standard biologic methods. AAV vectors present unique characterization challenges. Quantifying both physical and functional particles, assessing capsid integrity and empty/full ratio, and developing potency assays that are clinically relevant, reproducible, and transfer-ready is the goal.

Our team has built and validated AAV-specific analytical panels across serotypes, supporting programs from early pre-clinical qualification through BLA method validation packages. We align every method with the Analytical Target Profile (ATP) and support regulatory submissions in accordance with current guidelines for gene therapy products.

From selecting the right ddPCR approach for vector genome titer to developing a robust cell-based potency assay that reliably passes acceptance criteria, DES Pharma delivers analytical solutions that hold up through tech transfer and regulatory scrutiny.

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Potency Assay Development & Validation

In-cell western, cell-based transduction assays, and reporter systems designed for robustness, transferability, and compliance

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Empty/Full Capsid Ratio Analytics

AUC, AEX-HPLC, and TEM methods for capsid occupancy critical for reliability determining a CQA for AAV efficacy and safety

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Method Lifecycle Management

ATP-driven approach from development through routine QC method transfer, and post-approval change management

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Specification Development & Justification

Science- and risk-based specification setting for release and stability, with regulatory justification documents aligned

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Analytical Method Tech Transfer

Structured transfer protocols, acceptance criteria, and comparative testing packages for CDMO and QC laboratory transitions

Full-Spectrum Analytical Services

AD & QC Services for Gene Therapy Programs

Comprehensive analytical and quality control consulting across the entire AAV gene therapy development lifecycle.

📐 Analytical Development (AD) Services

→ Laboratory space design and equipment commissioning

→ Selection of fit-for-purpose analytical technologies for AAV

→ Method design for starting/raw materials, drug substance, drug product, and process intermediates

→ Establish product degradation profile and identify degradation pathways

→ Correlative analysis of analytical and stability data with product performance

→ AAV-specific specification development (vector genome titer, capsid titer, potency, purity)

→ Analytical Control Strategy development and deployment

→ Generate data sets and analytical summaries for IND/BLA filings

→ Reference standard program development and qualification

🧪 Quality Control (QC) Services

→ Routine GMP testing: identity, safety, potency, and purity for AAV release

→ Retained Sample Management and Storage Verification

→ Analytical method tech transfer (development → QC → CDMO)

→ Method validation per ICH Q2(R2) and ICH Q14

→ Laboratory operation requirements for GLP and GMP testing

→ Lifecycle Management and performance monitoring

→ Lot Disposition, Deviation Investigation, and Batch Release

→ Laboratory audit support and data integrity assessments

→ QC Microbiology, EM & Facility Control

→ OOS investigation and CAPA management

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Advance Your AAV Analytical Program with Confidence

Build robust, regulatory-ready analytical methods for your gene therapy product today.