A 6-month collaborative project was announced today that will help to accelerate the introduction of regulated advanced gene therapies that use Adenovirus-associated vectors (AAVs).
The government funded project, supported by the department of Business, Energy & Industrial Strategy (BEIS) is a collaboration between the Cell and Gene Therapy Catapult (CGT Catapult) and the National Institute of Biological Standards and Control (NIBSC) and will result in preparing the first reference material of an Adenovirus-associated vector that will be globally available to facilitate harmonisation of modern analytics.
The AAV reference material will be manufactured at the newly setup CGT Catapult’s state-of-the art facility in Braintree and will then be transferred to the NIBSC for additional testing, dispensing and labelling ready for distribution to a selected group of international laboratories for independent measurement. These laboratories comprise a mixture of research and development labs, large scale manufacturers and clinical trials scientists that have extensive experience with AAV-based medicines.
The principle of reference material measurement is to achieve overall industry consensus by providing an agreed benchmark against which all laboratories can compare. Being able to define a reference material for AAV has the result of achieving optimal patient dosing for safe and effective treatment. It is also the basis for QC/QA and regulatory approval that validates successful R&D work, tech transfer and the full GMP compliant scale-up and manufacturing process.
The project with the NIBSC represents the first major project benefitting from the new capabilities developed at the site. CGTC’s Braintree site was originally funded in 2020 with £127M by BEIS through Innovate UK and selected by the government appointed Vaccine Taskforce to provide flexible GMP-qualified manufacturing innovation facilities specifically for powering the cell and gene therapy sector in the UK and for pandemic vaccine manufacturing, if required by the UK Vaccine Taskforce. It boasts 755m2 of clean room manufacturing space and has the capacity to house a variety of innovative processes, including mRNA at large scale.
Business Minister Lord Callanan said:
Good regulation should spur entrepreneurship, not stand in its way, and this project will help keep the UK at the cutting-edge of innovation.
Physical Standards for Adeno-associated virus (AAV) Gene Therapy could pave the way for exciting developments in the sector, bringing benefits to British businesses and consumers alike.
Matthew Durdy, CEO of the CGT Catapult, outlined why this project is so important:
Working together with industry we have developed the CGTC’s Braintree site into one of the most advanced cell and gene therapy manufacturing innovation facilities in the world with access to latest technology for commercial scale manufacturing of viral vectors. It was setup to continue to grow the UK capabilities and support the cell and gene therapy industry. We are all incredibly proud that the NIBSC has chosen to partner with CGTC to manufacture this first AAV reference standard here at Braintree. It is another step towards an industry supplying gene therapies to the world.
Adenovirus-associated vectors are particularly useful across a broad range of diseases that until now have been very challenging or impossible to treat long-term. Typically, this type of gene therapy produces stable results that can last for months or many years. AAVs also have an acceptable safety profile with many serotypes of the virus that can be selected to treat diseases relating to different cell or organ types. One of the most successful examples of AAV use has been in treating haemophilia B, where patients can experience life changing correction of the faulty FIX gene lasting for possibly ten years or more.
Dr Sarah Kempster, Scientist at NIBSC said:
Gene therapy is one of the tools set to transform the clinical management of a number of health issues and the Life Science industry is well placed to contribute. Through this funding award, close collaboration between the NIBSC and the Cell and Gene Therapy Catapult will accelerate the development of reference standards that will underpin the quality of manufacture of AAV based therapies and so work towards patient safety.
This project has been made possible by a grant from the £3.7 million Regulators' Pioneer Fund launched by The Department for Business, Energy and Industrial Strategy (BEIS).
The fund enables UK regulators and local authorities to help create a UK regulatory environment that unleashes innovation and makes the UK the best place to start and grow a business.
For further information about the funding visit:
https://www.gov.uk/government/publications/projects-selected-for-the-regulators-pioneer-fund
About NIBSC
The National Institute for Biological Standards and Control (NIBSC) plays a leading national and international role in assuring the quality of biological medicines and diagnostics. We supply quality control reagents and standards to help laboratories identify technique and process issues for best practice in testing, ensuring patients obtain accurate biological test results.
About Innovate UK
Innovate UK drives productivity and economic growth by supporting businesses to develop and realise the potential of new ideas, including those from the UK’s world-class research base. They connect businesses to the partners, customers and investors that can help them turn these ideas into commercially successful products and services, and business growth.