After proving its worth in the sickle cell disease space, what’s the next horizon for gene editing technology? Some drug developers are gunning for the lofty target of Type 1 diabetes. 

Using CRISPR-based techniques, researchers implanted edited donor islet cells that successfully secreted insulin in a Type 1 diabetes patient, according to results published in The New England Journal of Medicine in August. The therapy developed by Sana Biotechnology is just a first step for the company, which aims to move another gene editing Type 1 diabetes treatment into human trials next year. 

Gene editing therapies for T1D come with a distinct edge — they don’t have to combine with immunosuppressive medications, removing a regimen that can be dangerous for patients. 

Vertex Pharmaceuticals, a major name behind the first approved CRISPR-based drug, is unsurprisingly also in the hunt for a gene editing breakthrough in T1D. But to get there, its first-generation T1D treatment will have to succeed in the clinic. Today, we’re looking at Vertex’s far-reaching goals to transform care in the T1D space. 

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