We advance AAV programs within the pharmaceutical value chain under a rigorous CMC mindset, progressing from Pharmaceutical Development & Manufacturing to Drug Substance Development and into specialized Gene Therapy Vector Development. Our focus is phase-appropriate, data-driven, and tightly aligned to downstream manufacturability.
Overview of Adeno-Associated Virus (AAV) Vector Development
AAV vector development centers on designing a stable, manufacturable genome-capsid system, then engineering scalable upstream and purification strategies with analytics that truly reflect product quality. We integrate plasmid set selection, capsid and genome architecture, and packaging efficiency into a unified framework. Emphasis is placed on empty/full separation, impurity control, potency-indicating assays, and stability under realistic handling conditions. By harmonizing construct design with host–process fit and high-fidelity characterization, we create platformable paths that shorten iteration cycles and reduce technical risk.
Our Services
We provide a focused set of AAV development services:
AAV Genome Engineering Service
We design and refine ITR-based genomes, including single-stranded and self-complementary formats, with attention to payload size, polyA performance, and sequence elements that affect packaging and stability. We assess promoter/enhancer options, codon usage, and regulatory cassettes, providing in-silico liabilities screening and bench confirmation.
Capsid Selection & Optimization Service
We support serotype and engineered capsid selection using structured matrices that weigh tropism literature, manufacturing tractability, and impurity trends. Small-scale packaging screens compare AAV2, AAV5, AAV6, AAV8, AAV9 and other commonly used capsids for titer, full/empty ratios, aggregation propensity, and process compatibility. Where appropriate, we iterate on production parameters (e.g., MOI, plasmid ratios) to de-risk the chosen capsid prior to scale exploration.
Upstream Packaging Development Service
Using transient systems typical for development phases, we optimize transfection chemistry, plasmid ratios (rep/cap, helper, GOI), cell density, feed strategies, and harvest timing. We establish critical process parameters, robustness ranges, and DoE-informed set-points that balance packaging efficiency with impurity control. Early impurity tracking (HCP, host DNA, residual plasmid) is embedded to anticipate purification demands.
Purification & Empty/Full Resolution Service
We develop phase-appropriate purification trains such as clarification, capture, intermediate polishing, and enrichment of full capsids via established orthogonal methods (e.g., anion-exchange strategies, density-based enrichment where applicable). We map step yields, define buffer windows, and institute in-process controls for aggregation and vector integrity. Methods are selected for transferability to later scalable formats.
AAV Analytical Method Development Service
We build a stability- and identity-focused analytical portfolio: vector genome titer by qPCR/ddPCR; capsid protein quantitation; empty/full determination; purity and aggregation by SEC-HPLC and orthogonal readouts; residuals (DNA, HCP, detergent) by validated approaches; and potency-indicating assays tailored to the construct.
We deliver end-to-end AAV vector development anchored in manufacturability, analytics, and stability. Our modular services de-risk design choices early, build robust laboratory processes, and prepare programs for efficient progression. Contact us to initiate a tailored development plan.
Frequently Asked Questions
Q1: How do you decide between ssAAV and scAAV for a new construct?
We evaluate payload size, expression kinetics, and packaging constraints. scAAV can support rapid expression but halves effective payload capacity, while ssAAV accommodates larger cassettes. We run bench screens to confirm packaging efficiency, vector integrity, and potency-indicating performance before finalizing the genome format.
Q2: Can you adapt the process if the capsid is changed during development?
Yes. We manage capsid transitions through structured comparability, re-tuning upstream parameters and polishing conditions as needed. Analytical bridging—especially genome titer, empty/full, impurity profiles, and potency-indicating assays—anchors decision-making and maintains continuity of product quality attributes.
Our products and services are for research use only.
