BREAKING: Canada OK’s First T1D Gene Editing Trial

Yesterday the Canadian health regulatory agency gave a landmark approval for a type 1 diabetes human trial that combines stem-cell therapy with gene editing.
The trial will be conducted through a partnership between ViaCyte and CRISPR Therapeutics. ViaCyte, which we have covered many times, has long offered hope that a surgical implant of stem cells could replace the body’s ability to produce insulin. CRISPR Therapeutics is a relative newcomer to T1D, but this partnership offers the possibility that insulin-producing cells could be hidden from the immune attack via gene editing. This will be the first human trial that attempts to use CRISPR Cas-9 technology to treat or cure T1D.
Unlike the cadaver-sourced cells commonly used in islet transplantation surgery today, ViaCyte’s stem-derived cells can theoretically be reproduced infinitely. However, the company has struggled to find a way to stop the immune system from rejecting the cells without the use of immunosuppressive drugs. CRISPR Therapeutics, co-founded by 2020 Nobel Prize winner Emmanuelle Charpentier, partnered with ViaCyte in 2018 to develop a solution to this problem. The new product, called VCTX210, is derived from a stem cell line that has been genetically edited – using CRISPR Cas-9 technology – to evade detection by the immune system. In theory, these insulin-producing cells would be safe from immune rejection without the need for full-body immunosuppression or physical encapsulation of the cells.
Now that the companies have the green light to begin a human trial of this product in Canada, they say they will start recruiting patients “by the end of the year.” However, at this time, the companies are not disclosing the inclusion criteria of the patients they will recruit or the specific clinical objectives of the trial. As a result, we cannot say if the trial will be open to all people with T1D, or just a small subset of the population. We will provide updates about this trial as the companies make them available.

To read yesterday’s press release, click here.

For further JDCA coverage of gene editing in T1D, check out our roundup from March.