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New funding and a clinical trial for Wave 2 gene therapy product

The Cystic Fibrosis Trust has announced further funding for the Gene Therapy Consortium (GTC), which could potentially support the first in-human clinical trials of their new gene therapy product. The investment complements additional investment the GTC has secured from the Wellcome Trust and Department of Health to continue their ground-breaking work.

The Cystic Fibrosis Trust is investing £500,000 to support the scientific work required to underpin the development of the Wave 2 product, which is essential to position the GTC to run the first clinical trial of this product.

The Wave 2 product uses a virus as a new way to deliver a healthy copy of the CFTR gene to the lungs.

The additional funding comes as the GTC has announced a substantial investment in the trial from the Wellcome Trust and Department of Health’s Health Innovation Challenge Fund.

The Phase 1/2a trial will assess the safety of the viral mechanism that delivers the gene, and hopefully provide evidence for its effectiveness as a treatment.

The start date will depend on how long it takes to produce the Wave 2 product in a form that can be delivered clinically. The team will look to recruit 24 patients for the trial, which will last around nine months. A further update on timelines will be provided by the GTC once manufacturing data are available.

David Ramsden, Chief Executive of the Trust, said: “We are delighted to continue our association with and investment in the Gene Therapy Consortium, which represents one of the many exciting projects in our portfolio of world-class research in our fight for a life unlimited by cystic fibrosis.

“The CF community has long supported the work of the GTC, and our wide-ranging portfolio of research is only possible thanks to the generosity and dedication of fundraisers throughout the UK.”

The product was developed in parallel with the GTC’s Wave 1 product which uses a liposome (a fat) as the delivery vehicle for the healthy gene.

The GTC commented: “We are focusing our research and development efforts on Wave 2, which has proved to be considerably more efficient than the Wave 1 product. However, we are continuing to discuss the Wave 1 product with potential commercial partners who could help us bring it to market.”

Find out more about the research we are funding

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