Vertex Pharmaceuticals’ CRISPR-based therapy made history as the first drug of its type, transforming care options for sickle cell disease. Now, the company aims to make its mark in Type 1 diabetes with another potentially transformative therapy that could untether patients from insulin, the condition’s primary treatment for more than a century.
So far, signs for its Type 1 diabetes treatment zimislecel have been encouraging.
The company released results from a small, phase 1/2 trial earlier this year demonstrating that a single infusion of the off-the-shelf islet cell therapy hit blood sugar targets during one year of follow-up. And 10 of the study’s 12 participants no longer needed insulin.
The treatment uses stem cells to create islets, clusters of hormone-producing cells in the pancreas that regulate blood sugar and are damaged by a misguided immune system…