First CRISPR therapy seeks landmark approval
Vertex and CRISPR Therapeutics’s first-in-modality genome-editor exa-cel, for the treatment of two haemoglobinopathies, has entered the regulatory spotlight.
Vertex and CRISPR Therapeutics have submitted their CRISPR-based ex vivo cell therapy exagamglogene autotemcel (exa-cel) for FDA approval, for sickle cell disease (SCD) and beta-thalassemia. A regulatory decision on the gene-editing candidate is expected within 8 to 12 months. The companies have also filed for approval in Europe and the UK.
“This is an incredible validation for a technology that 10 years ago was a paper,” says Bastiano Sanna, chief of cell and genetic therapies at Vertex, which partnered with CRISPR Therapeutics in 2015 to develop exa-cel, formerly called CTX-001. “It was like opening a bottle: when the right tool came, suddenly we could use all the science that the [haemoglobinopathy] field had stored up for so many years,” says Sanna.
“It’s astounding,” agrees Jennifer Doudna, one of the scientists who showed in 2012 that the CRISPR–Cas system could be used to edit genomes. “It was clear that having the ability to edit genomes was a powerful tool, but I don't think any of us could have imagined how fast the field would move.” Doudna co-founded the genome-editing companies Editas and Intellia, while Emmanuelle Charpentier, a co-author of the 2012 paper, co-founded CRISPR Therapeutics. Doudna and Charpentier shared a Nobel Prize in 2020 for their ground-breaking work.
Exa-cel data, from 75 patients in ongoing open-label studies, helps set the bar.
“It’s been spectacular, and beyond anybody's expectations,” says Martin Steinberg, a haematologist at Boston University and a member of Vertex’s steering committee for exa-cel’s development.
Vertex’s gene-editing therapy stopped painful vaso-occlusive crises in SCD, and led to transfusion-independence in 90% of patients with beta-thalassemia over 1.2 to 37.2 months, the company has reported.
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