Moderna and Biogen have won the backing of five scientists on a panel convened by the regulator to evaluate the safety and efficacy of their CRISPR gene-editing system.
Moderna and Biogen were called to an independent panel by the company, and appeared before it on 14 October. The panel was chaired by Professor Glyn Davis of Queen Mary University of London. He described the CRISPR technology as “remarkable.”
“It’s the most amazing new advance I’ve ever seen in genetics,” he said.
Moderna and Biogen were asked to explain how their technology would be used to potentially prevent or correct defective versions of genes.
They told the scientists that they do not currently plan to develop CF therapies, as their gene-editing system is not yet designed to correct a faulty gene in a living organism.
However, they both said they do plan to commercialise this “extremely exciting technology,” arguing that it could provide an alternative to traditional gene therapy.
Professor Jörg Tiedemann, of the Medical Research Council Division of Primary Care and Paediatrics, said it was positive to see CRISPR improved.
“The technology has the potential to have a fundamental, profound impact on many diseases,” he said.
But the panel also expressed concern that this new technology could be a step too far for the limited number of people who would benefit from it.
“Moderna and Biogen have used the universal gene approach and this hasn’t been tested on large numbers of patients,” said Professor Rosalind (Robyn) Szewczyk, director of the Biomedical Ethics and Regulation Group at the MRC.
“This is a really interesting technology, but it needs to go through a lot of testing.”
The panel also pointed out that it might not be suitable for use in the short term.
“It’s not something that you just take out and throw away – it’s a permanent disruption to the function of the protein,” Professor Charles van Praag, of University College London, said.
He explained that if a patient needed more of a particular protein, they could use a CRISPR tool that breaks the existing healthy gene code, cutting it out of the population, but making the presence of that protein very common.
“The application of these techniques is limited to patients where the alteration would benefit them,” Professor Davies said.
While this is a different kind of treatment from what the panel previously approved in 2016 – when it gave the go-ahead for Kite to develop a CTX and Kite was approved to launch – it is still a significant step for CRISPR.