Our Work

Developed the first CpG-Free plasmids

Start of the product evaluation study to assess the effectiveness of multiple gene transfer agents (GTAs) in pre-clinical studies.

Completed the Tracking Study

Volunteers helped us with the Tracking Study in 2006

Completed a Run-In Study

In preparation for the multi-dose trial, we designed a study of almost 200 people with cystic fibrosis. The purpose of this study was to help design the multi-dose trial and to choose the patients most likely to be able to demonstrate benefit.

Completed a Single Dose Clinical Trial Pilot Study

The aim of the pilot study was to assess the safety of a single dose of the Wave 1 product.  Over 30 patients took part in the study and received one dose of the gene therapy.

Completed a Multi-Dose Clinical Trial

We recruited ~130 people with CF (aged 12 years and above) into the trial. The trial was a blinded, placebo-controlled study, meaning that each participant had a 50:50 chance of receiving gene therapy or 'dummy' treatment, with neither participants nor investigators knowing which.

Introduction to Gene Therapy

Introduction to CF

Why use Gene Therapy to treat CF?

Treating other diseases in our portfolio

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