Successful gene therapies start with intelligent design for better AAV performance, with rational genome design being one key component besides capsid optimization to do so.

Our AI-enabled genome engineering approach combines advanced computational tools with extensive AAV development expertise to optimize vector genomes for expression, stability, safety, and manufacturability.

By addressing critical design elements early in development, we help reduce risk, improve product performance, and accelerate the path toward clinical success.

We optimize the lead vector including expression cassette and plasmid backbone to maximize performance and predictability while maintaining development flexibility, leading to more stable, efficient, and predictable transgene expression.

Capabilities include:

• Promoter selection and optimization
• Integration of enhancers and regulatory elements
• Identification and mitigation of cryptic splice sites
• Detection and removal of unwanted polyadenylation signals
• Inverted terminal repeats (ITR) sequence verification and optimization
• Identification and reduction of potentially immunogenic sequence elements
• Strategic intron incorporation to improve mRNA stability
• Codon optimization for efficient protein expression
• CpG content optimization

Our design strategies extend beyond the expression cassette to capsid architecture, helping generate safer and more effective AAV products from the earliest stages of development.

Besides offering the full array of natural AAV capsids, DINAMIQS, has partnered with leading capsid optimization companies to provide access to proprietary, engineered capsids with demonstrated advantages for organ/ cell type specific tropism.

Better genome and capsid design can accelerate development timelines, improve product quality, increase the likelihood of clinical success, and facilitate regulatory review. By combining AI-powered analytics with deep expertise in AAV biology and development, we help clients build optimized vectors designed for performance from day one.

Capabilities include:

• Capsid engineering to enhance tissue targeting and specificity
• Design strategies to improve biological activity and therapeutic performance
• Demonstrated regulatory pathway

Selecting the right cell line is a critical component of successful viral vector manufacturing. We provide licensed, commercially available HEK293 and HEK293T suspension cell lines supported by established GMP manufacturing processes and master cell bank infrastructure. At the same time, we fully support client-owned or third-party licensed cell lines, enabling seamless technology transfer and process migration without unnecessary restrictions.

Unlike traditional licensing models, our approach is designed to eliminate vendor lock-in. When manufacturing with DINAMIQS, no additional cell line licensing fees apply, sublicensing options are available, and clients retain the flexibility to transfer manufacturing as their programs evolve.

Whether you leverage our licensed manufacturing platforms or your own preferred cell line, we provide the expertise, infrastructure, and transparent licensing model needed to support efficient development, scale-up, and commercial manufacturing.

Capabilities include:

• Access to licensed, high-producing suspension HEK293 and HEK293T cell lines
• Established GMP-ready manufacturing processes and Master Cell Bank infrastructure
• Support for client-owned and third-party licensed cell lines
• Technology transfer and process migration support
• Transparent licensing with no additional cell line fees when manufacturing with DINAMIQS
• Sublicensing options and straightforward transfer rights
• No vendor lock-in or exclusive manufacturing commitments
• Scalable manufacturing from process development through commercial production

DINAPEAKS® is our proprietary technology platform for viral vector process development and manufacturing. Unlike fixed platform approaches, DINAPEAKS® is designed to evolve continuously, integrating new technologies, equipment, and process improvements as they emerge.

Rather than forcing every project into a predefined process, DINAPEAKS® provides a flexible, modular framework with proven solutions for common development and manufacturing challenges. We actively evaluate and implement innovations in upstream processing, downstream purification, process intensification, automation, and analytics, ensuring our clients benefit from the latest advances in the field.

Our ongoing internal development activities continuously strengthen the DINAPEAKS® platform, enabling faster process development, improved process performance, and more efficient manufacturing. By combining innovation, flexibility, and practical manufacturing expertise, DINAPEAKS® supports every stage of your program, from early development through commercial manufacturing.

Capabilities include:

• Modular and adaptable process design
• Rapid implementation of new technologies
• Continuous process intensification and optimization
• Innovations in upstream processing and downstream purification
• Advanced automation and analytical solutions
• Proven solutions for common manufacturing challenges
• Flexibility to pivot as project needs evolve

Specialists from our QA team, in collaboration with Siegfried’s global network of more than 30 regulatory affairs experts and selected external partners, provide comprehensive regulatory support for client submissions. This includes IND filings as well as the preparation, review, and lifecycle management of Module 3 (CMC) documentation.

Digitalization is integrated throughout our development and manufacturing workflow. The digital documentation and data management established during process and analytical development in Benchling seamlessly continues into manufacturing and quality operations through a fully paperless electronic Quality Management System (QMS).

Our end-to-end digital infrastructure supports documentation, manufacturing, quality, analytical, and business processes, with all electronic systems validated in accordance with GAMP 5. This ensures data integrity, regulatory compliance, efficient collaboration, and complete traceability throughout the product lifecycle.

Capabilities include:

• Benchling – digital documentation and data management during process and analytical development
• eSOP – electronic documentation and compliance management
• Cornerstone OnDemand – electronic training records with global and local training requirements
• TrackWise – management of Change Controls, Deviations, Complaints, and CAPAs
• MasterControl – electronic Manufacturing Batch Records (eMBRs)
• LabWare LIMS – analytical methods, specifications, and laboratory data management
• SAP S/4HANA – materials management, vendor qualification, and business processes
• End-to-end paperless digital manufacturing and quality operations with GAMP 5-validated systems