Update on California's $72 Million Bet on a Diabetes Cure: Gene Editing Holds Promise

Viacyte video

A San Diego stem cell firm fueled by $72 million from the state of California this week announced an "important step" in its search for a diabetes cure in collaboration with a Massachusetts gene-editing enterprise.

The California business is ViaCyte, Inc., a privately held company that has received more funding from the California stem cell agency than any other business. 

It ranks 9th on the list of all recipients of cash from the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. The ranking places it ahead of such highly regarded research institutions as Salk in La Jolla and Gladstone in San Francisco.

The East Coast firm is CRISPR Therapeutics, AG, a publicly traded firm that aims at "developing transformative gene-based medicines for serious human diseases."

CRISPR and Viacyte announced on Tuesday that their research is now showing that ViaCyte's "CyT49 pluripotent stem cell line, which has been shown to be amenable to efficient scaling and differentiation, can be successfully edited with CRISPR. The CyT49 pluripotent stem cell line is currently being used to generate islet progenitors for clinical trials."

Paul Laikind, CEO of ViaCyte, said in a news release that the latest news brings the firms "potentially one step closer to a transformational therapy for patients with insulin-requiring diabetes through the development of an immune-evasive gene-edited version of our technology."

Laikind described the gene editing result as "an important step" in achieving "yet another first, the development of an immune-evasive cell replacement therapy as a potential cure for type one diabetes."

In 2017, CIRM gave ViaCyte $1.4 million for work on the CyT49 line. Maria Millan, president of CIRM, said at the time,

“Development of an immune-evasive cell therapy would increase the chances of engraftment and durable effect of a cell replacement therapy for diabetes."

Investors were not energized by the CRISPR/ViaCyte announcement. CRISPR's stock price closed at $49.40 today, down from a $49.67 close on Monday, the day prior to the announcement. The 52-week high for the firm is $53.97. The low is $22.22. 

CRISPR and ViaCyte announced their $15 million collaboration a year ago. Here is a 2018 overview of Viacyte by Bradley Fikes of the San Diego Union Tribune.