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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.

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

Program Operating Plan

Stage 1: Baseline Process (Q2 2024 - Q3 2025)

  • Establish a process baseline that can be described publicly in terms of manufacturing unit operations
  • Using this process, manufacture a batch and explain how a batch was made, what the process performance was, and what the product quality outcomes were
  • Build capability, socialize and develop support and enthusiasm for the NIIMBL platform that is to follow

Stage 2: NIIMBL Platform (Q1 2025 - Q2 2026)

  • Using the Baseline Process as a departure point, develop a Platform process and analytical package and demonstrate performance capabilities
  • The Platform will include accessible GMP cell bank options and will investigate applicability to multiple serotypes and transgenes
  • Using this Platform, manufacture a batch and explain how a batch was made, what the process performance was, and what the product quality outcomes were
  • Deploy this platform so it is accessible to rare disease product sponsors

Stage 3: AAV Technology Innovation (Q3 2026 +)

  • Provide funding for new process, analytical, and data technology development

Workstreams

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

Processes Workstream

Stage 1
  • Process definition based on publicly accessible information
  • Identify and contract CDMO for 1-3 batches (e.g., 50L including feedstocks)
  • Tech transfer and support 1st campaign CDMO development runs and share data publicly
Stage 2
  • Develop accessible GMP MCB & WCB options; evaluate multiple serotypes and transgenes
  • Develop and demonstrate upstream and downstream performance capabilities
  • Process lockdown – balancing performance and breadth of applicability
  • Tech transfer and support 2nd campaign CDMO development runs and share data
  • Develop a network of willing and capable manufacturing sites to run this process
Stage 3
  • Explore novel technologies such as (for example): Packaging/producer MCB/WCB, Sf9 access, novel affinity resins, improved full capsid enrichment
  • Explore data tools such as AI/ML-based optimization models

Analytics Workstream

Stage 1
  • Identify the minimum CQA list with corresponding methods
  • Tech transfer and support 1st campaign CDMO development runs and share data publicly. Data may be generated using proprietary technology
Stage 2
  • Determine best method for each product attribute. This may require head-to-head comparison of multiple methods.
  • Develop publicly accessible SOPs for each platform method
  • Develop in-process methods and tools
  • Tech transfer and support 2nd campaign CDMO development runs and share data
Stage 3
  • Design alternative sample-sparing methods
  • Develop robust and repeatable rcAAV method
  • Standardize and simplify Next-Gen Sequencing (NGS)
  • Develop in vitro potency standards

Progress and updates

Program History

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June 2024

Program plan update at the National Meeting (Community Portal Login Required)

April 2024

Joint leadership teams Program Planning meeting

November 2023

Advancing a Platform Approach to Accelerate AAV-based Gene Therapy (webinar)

October 2023

Program launch

August 2023

RFI for Next-Generation Sequencing Method Development Study

2023

Formation of Steering Committee and Analytics and Process Leadership Teams

November 2022

Viral Vector Manufacturing and Analytics (webinar)

Program Participants

NIIMBL Program Leader

Eric Hacheri, Co-Lead Viral Vectors Program

Eric Hacherl

NIIMBL Senior Fellow
Contact

Program Advisor

Tim Charlebois, Senior Fellow

Tim Charlebois

NIIMBL Senior Fellow
Contact

NIIMBL Scientific Program Manager

Angie Snell Bennett

Scientific Project Manager
Contact

Alexion Pharmaceuticals

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Learn more and get involved

NIIMBL members: login for more

To access more program information, including:

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

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