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Viral Vector Manufacturing and Analytics Program

Mission

Develop and make broadly available a robust, economically viable, shared-access platform for the technical development, manufacturing, and characterization of AAV-based gene therapy vectors. Ensure that workforce training for this platform can integrate into existing curriculum or training models to encourage adoption, and integrate improvements into the platform based on feedback from these training programs.

Vision

A gene-based therapeutics industry capable of serving patients across the full spectrum of unmet needs—from prevalent indications to ultra-rare diseases—that has access to high-quality viral vectors without cost or speed limitations.

A magnified view of cells as seen through a microscope

What are viral vectors and how will they benefit the industry?

One of the most important vector platforms in use in the biopharmaceutical industry today is adeno-associated virus (AAV) vectors. Our Program focuses on advancing the manufacturing process and analytical capabilities for AAV vectors. The advances are critical for broad and rapid expansion of the emerging industry of gene-based therapeutics and ultimately the many patients and families who can benefit from access to these novel therapies.


NIIMBL is well-positioned to bring together the right resources to accelerate development of core manufacturing process platforms and essential analytical capabilities for viral vector product and process characterization, which can

  • Ensure manufacturing platforms are flexible and robust to accelerate business objectives
  • Improve therapy yield of known manufacturing platforms
  • Reduce individual investment and resources needed to help manufacture life-improving and life-saving gene therapies 

Viral Vectors Program Structure

Workstreams

The work of the program is organized around workstreams, topical focus areas.

Processes Workstream

Shared-Access AAV Process Platform for Rare Diseases

Objective: Develop broadly available, robust, replicable, well-understood, and documented AAV manufacturing process.

Technical Needs
  • HEK Expression System: Transfection-based upstream processing and protocols; key starting materials with documentation for freedom of operation
  • Media & Upstream Processing: Chemically defined media and protocols for serum-free suspension; cell density balance; process performance capability demonstrations
  • Downstream Toolkit: Purification processes for AAV serotypes; affinity resins and full capside enrichment; process applications across serotypes and transgenes; validation studies
  • Fill/Finish/Storage/Stability: Platform formulation(s); sterility operations; flexible container closures; quality impact assessments; platform validation studies
Advancing AAV Process Performance

Objective: Drive and demonstrate process capabilities enabling broad expansion of AAV gene therapy applications

Technical Needs
  • SF9: Understanding scalability advantages and perception of lower vector quality and potency in a scalable way. Standardization of titer and BV seed stocks.
  • Scalable Transient HEK 293: Address and identify gaps in HEK knowledge. Improve productivity and scalability for product capacity.
  • Improved Expression Systems: Engineering ‘producer cells’ to reduce and eliminate limitations of transient transfection.
  • Downstream Improvements: Identifying more cost-effective and improved affinity ligands. Improve overall product yield and scalable full capsid enrichment.

Analytics Workstream

Enhancing Access & Utility of AAV Analytical Toolkit

Objective: Develop and disseminate AAV analytical capabilities for process and product characterization, batch release, and stability monitoring

Technical Needs
  • Batch Release and Stability: Platform methods to harmonize, disseminate, apply, and build dataset; enabling methods to develop and determine feasibility and applicability
  • Product and Process Characterization: Tools to evaluate and define full/empty/partial capsids; conduct in-depth characterization of AAV vectors; assess potential process- and product-related impurities; and support rapid-turnaround, high-throughput process development
AAV Reference Resources

Objective: Support availability and utilization of physical and documentary standards, reference materials, and data.

Technical Needs
  • AAV Reference Material Development: Assess reference material needs and attribute target profile; generate/acquire prototype materials; innovate community characterization and analytics; facilitate adoption by standards development organizations
  • Documentary Standards and Reference Data: Evaluate and support documentary standards landscape; convene experts to discuss phase appropriate data for AAV submissions; design and deliver community data resource

Progress and updates

High-level program timeline

Program Participants

NIIMBL Co-Leads

Tim Charlebois, Senior Fellow

Tim Charlebois

NIIMBL Senior Fellow
Eric Hacheri, Co-Lead Viral Vectors Program

Eric Hacherl

NIIMBL Senior Fellow

NIIMBL Scientific Program Manager

Angie Snell Bennett

Scientific Project Manager

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  • Program charters
  • Roadmaps and webinars
  • Roster of leadership and participants
  • Program progress reports and workshop results

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