Wave 1 Trial Results For Cystic Fibrosis Therapy Delayed

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by Patricia Silva PhD |

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wave 1 CFThe British Gene Therapy Consortium announced recently that it has decided not to attend the upcoming North American Cystic Fibrosis Conference (NACFC), where they were planning on presenting the latest data about their Multidose Gene Therapy (Wave 1) Trial, which focuses on developing a genetic treatment for cystic fibrosis (CF). The consortium announced the cancellation of the presentation, since they had only less than half of the required data available to present.

“We had hoped to announce the outcome of the Multidose Gene Therapy Trial at the upcoming North American CF Conference (NACFC). The UK CF Gene Therapy Consortium (GTC) only received the first batch of quality-controlled raw data a few days ago, representing perhaps less than half the total information that we will need to look through,” announced Professor Eric Alton from the Gene Therapy Consortium. “We now have thousands of pieces of data to analyse and interpret, something that is just not plausible to do in a credible way in a few days.”

Wave 1 is the largest gene therapy trial being conducted worldwide, and its results are widely anticipated. However, the researchers believe it is better to explore further data in order to publish the results of the analysis in the near future. Professor Alton stated that “the CF community, and the GTC, have spent a considerable time reaching this stage, and it would be unthinkable to rush through this to try and produce a conclusion that may not stand up to later in-depth scrutiny.”

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“We will, therefore, not be presenting at the NACFC and I, with several members of the GTC, will remain in the UK to push forward the data analysis as rapidly as possible so that we can publish the data ASAP. We are obviously disappointed by the delay, and will update the website as soon as we have further information,” he announced.

The director of the Research & Clinical Care at the Cystic Fibrosis Trust, Janet Allen already responded to the news explaining that the organization appreciates “the complexity of this data, and fully understands the need for delaying publication,” as well as that they and “the whole cystic fibrosis community are eagerly looking forward to the results when they are ready.”


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