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Global collaboration to take Wave 2 gene therapy product forwards

A three-way partnership will bring together expertise in cystic fibrosis (CF) gene therapy, lentiviral development and production and the treatment of respiratory diseases to develop a gene therapy treatment.

The partnership will see the UK Gene Therapy Consortium (GTC) join with pharma company Boehringer Ingelheim on research and development, and Oxford Biomedica on the viral delivery manufacturing for the Wave 2 gene therapy product.

Set up with the support of the Cystic Fibrosis Trust in 2001, the GTC has been developing two approaches to gene therapy, a Wave 1 product using liposomes (a kind of fat) to deliver the healthy copy of the gene into the lungs, and a Wave 2 viral vector as the delivery mechanism.

The Wave 1 product was successful in stabilising lung disease in a large placebo-controlled trial, proved the concept that gene therapy can have a beneficial effect on lung disease, and paved the way for the Wave 2 product.

The Trust has invested over £1 million in the GTC over the last five years, ensuring its work in both products could continue while searching for a major partner with the resources to take one of the products forwards.

If successful, the product would be the first ‘genotype-agnostic’ CF treatment to tackle the underlying cause of CF, meaning it could work for anyone with the condition, regardless of their genetic mutation.

In 2017 the GTC announced it was predominantly concentrating its efforts on Wave 2, which had proved to be more efficient than the Wave 1 product. However, the knowledge gained from taking Wave 1 to trial helped develop the Wave 2 product to the point where it was suitable for a major collaboration with the biopharmaceutical industry.

This collaboration will provide the substantial level of investment needed to take the Wave 2 product towards clinical trials, to establish its safety and effectiveness in humans.

David Ramsden, Chief Executive at the Cystic Fibrosis Trust, said: “We are delighted to hear the news that the Wave 2 gene therapy product will be taken forwards. It is a testament to the unwavering support of the CF community over the past 17 years, and the hard work of every fundraiser and donor. We look forward to seeing the fruit of this partnership between academia and industry.”

Prof Eric Alton, Coordinator of the GTC, said: “The UK CF Gene Therapy Consortium has, for the last 17 years, vigorously sought to establish whether gene therapy can become a clinically viable option for patients with CF. We are grateful to the Trust and the CF community for supporting this research over many years. From the beginning the GTC identified that this goal would require incremental increases in knowledge, and we have built on our experience with the Wave I non-viral gene therapy to develop a new viral vector-based product.

“The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice, relevant to all patients, irrespective of their mutation status, and in due course to both prevent lung disease as well as treat established problems.”

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