Lead vector design is a critical step in the development of effective AAV gene therapies. AI guided vector optimization enables the selection of constructs with the right balance of transgene expression, tissue specificity, manufacturability, and safety. Thoughtful vector design helps reduce development risk, improves the likelihood of clinical success, and accelerates the path from discovery to the clinic.

DINAMIQS recommends starting each project with a thorough lead vector inspection and offers vector optimization capabilities around:

• AI guided cassette engineering including the gene of interest, regulatory elements, plasmid backbone, and others
• Functional validation and optimization in small scale experiments
• IP landscape screening
• Capsid optimization, including AI approaches via dedicated partnerships

Robust process development with a complete and accurate understanding of all process parameters and product characteristics is essential for translating a promising AAV vector into a scalable, reproducible therapeutic drug product. By optimizing upstream production, downstream purification, and analytical methods early in development, manufacturers can improve product quality, maximize yield, and establish a process that supports regulatory compliance and successful clinical and commercial manufacturing.

• DINAMIQS’ Process development (PD) capabilities start with DoE driven parameter optimization, reproduced on small scale up to process robustness verification in 2L. In-process controls will be applied at all stages to monitor process performance and support development activities.

In detail this includes:

• Upstream process (USP) optimization: The vector feasibility study.

Identify optimal manufacturing conditions with the aim of maximizing both vector yield and product quality. This will be achieved through a structured Design of Experiments (DoE) approach evaluating key critical process parameters (CPPs), including plasmid ratios, total DNA amount, transfection reagents, enhancers and /or other parameters. Best performer conditions will be reproduced on a small scale with robustness verification in 2L bioreactors.

• Downstream process (DSP) optimization:

Advance DSP development, with a focus on key unit operations including filtration, AAV affinity capture, and ion exchange (IEX) polishing. For the capture step, a dynamic binding capacity study will be conducted  to define appropriate column sizing for subsequent scale-up, while elution conditions will be screened using a static binding setup. In addition, ion exchange chromatography conditions will be optimized to enrich full AAV particles, with the goal of achieving significantly reduced residual empty capsids (~10%) for enhanced vector quality.

High-quality preclinical-grade AAV material is essential for generating reliable in vitro and in vivo efficacy, biodistribution, and toxicology data before clinical development. Producing material with a well-characterized, scalable process enables meaningful preclinical studies while providing a strong foundation for a smooth transition into GMP manufacturing and first-in-human clinical trials.

At DINAMIQS, we work in suspension only with single use technology and GMP compatible raw materials early in the process. Our harmonized infrastructure across different scales guarantees a seamless scale-up from small scale via 2L up to 50L. High-end DSP capabilities, including chromatography and ultracentrifugation-based techniques, guarantee supply of AAV material with the highest possible quality and safety standards.

Preclinical manufacturing services at DINAMIQS includes:

• Small scale manufacturing for in vitro/ in vivo proof of concept studies
• Material supply for large animal studies up to 50L
• Material supply for TOX studies

From Phase I/II clinical studies to commercial supply, robust GMP manufacturing is essential for delivering high-quality AAV gene therapies.

DINAMIQS’ integrated capabilities span both drug substance (DS) manufacturing and drug product (DP) manufacturing, including on-site fill and finish, providing a seamless path from bulk vector production to the final sterile product. This end-to-end approach enhances quality, streamlines technology transfer, reduces supply chain complexity, and supports reliable clinical and commercial supply.

DINAMIQS offers manufacturing of drug substance (DS) under cGMP conditions, in accordance with the requirements of regulatory bodies. The filling of the DP will be done in a SKAN isolator with C-background in AT-Closed Vial®.

DINAMIQS’ cGMP capabilities include:

• Manufacturing from 20L – 1000L
• Fully closed, single use systems, chromatography purification
• Annex 1 compliance
• On-site fill and finish with isolator technology
• Density ultracentrifugation for empty/full separation
• Fully portable processes, no IP/ vendor lock-in
• Flexibility in critical raw materials (e.g. plasmids/ cell lines)
• Regulatory support by >30 regulatory experts

DINAMIQS offers a comprehensive suite of assays for the characterization of AAV products, fully aligned with regulatory requirements. Large panel of assays to characterize critical quality attributes (CQAs) identity, purity, potency, strength and safety are performed in-house, ensuring speed, consistency, and quality.

DINAMIQS performs product specific analytical method development, implemented and optimized in DINAMIQS laboratories to support process establishment and to prepare for subsequent GMP qualification activities.

DINAMIQS’ fill-finish solution is a semi- automated SKAN Pure isolator technology equipped with AT Filler Crystal® Pure M1, designed for aseptic vial filling and well suited for small batches of viral vector DP manufacturing. The isolator is installed in class C environment.

Key benefits:

• Dedicated to small batches, designed for novel therapies such as viral vectors
• Minimal line losses
• Semi-automated AT-Closed Vial® filling
• Filling format from 1mL to 50 mL vials, 2 mL format qualified
• AT-Closed Vial® specially designed ready-to-fill COC vial