Meet the California Institute Pushing Stem Cell and Gene Therapy Research: Part 1

In the first ever issue of The Medicine Maker (published in September 2014), an expert from the California Institute for Regenerative Medicine (CIRM) discussed the potential of stem cell therapy and the challenges of manufacturing. Ten years on, although there is a lack of approved stem cell therapies, incredible progress has still been made. Regenerative medicine is one of the hottest topics in the industry, with advances in gene therapy and the broader cell therapy field showing that commercialization of such advanced medicines is possible.

Here, we speak with Jon Thomas, President and CEO of CIRM. In the first of our three-part interview, we get the background behind CIRM and its impact to date.

What’s the story behind CIRM?
 

CIRM is truly a unique entity. Many companies and institutes like to describe themselves as “unique”, but it is genuinely appropriate in the case of CIRM. CIRM came into existence through a ballot measure around 20 years ago.

The visionary behind CIRM’s founding was Bob Klein. His son has type I diabetes, which prompted his interest in stem cell research. In the early 2000s, however, there were major roadblocks in regenerative medicine because President Bush had restricted federal funding through the NIH for creating new embryonic stem cell lines.

Bob was a real estate financier familiar with bond financing and he wanted to establish a state agency that could fund stem cell research outside of federal restrictions. He wrote a proposed bond measure that qualified for the November 2004 ballot that would create an agency to fund this research. The measure, if passed, would also authorize the state of California to issue $3 billion in general obligation bonds. These bonds, funded by Californian taxpayers, would primarily support grants (and some loans) to academic institutions, research institutions, and eventually biotech companies – though at the time, biotech’s involvement was minimal since the field was just getting going.

Bob gathered over one million signatures to qualify the initiative. It needed a simple majority of 50 percent plus one vote, but ended up passing with 59 percent. This is how the agency was officially established. There was some litigation afterwards that delayed things for a couple of years, but the agency won across the board. Funding began in late 2006.

In the early days, CIRM focused heavily on grants for basic research, training, and infrastructure – providing essential funding to build standalone stem cell institutes and repurposed existing lab spaces across California. As the field evolved and research advanced, our funding shifted more towards translational research, and eventually, clinical trials. Over the years, we’ve funded more than 1,400 grants, ranging from amounts in the tens of thousands up to awards as large as $20 million. For context, the average NIH R01 grant is around $380,000.

How has CIRM’s focus expanded over time?
 

Initially, our focus was purely on stem cell therapies, but as gene editing and gene therapies emerged, we began funding projects in those areas too. By 2020, we had allocated the full $3 billion from our initial funding, so Bob led a new initiative that authorized an additional $5.5 billion.

Consider CIRM as a bank. We don’t conduct research ourselves: we fund it – and have become the largest dedicated funder of stem cell and gene therapy research in the world. This has drawn substantial talent to California, from researchers and their teams to companies who are all keen to access the funding opportunities.

As of December 2024, we’ve supported 111 clinical trials spanning a wide spectrum of diseases, from rare conditions to more prevalent ones. In short, CIRM has made significant impact in the field. No other state has any entity like it. Our relations with the state government are very important. We have direct oversight from the State Controller to ensure we’re good stewards of taxpayer dollars, and have built strong relationships with other elected officials including the other constitutional officers and the state legislature. Our message to Californians is simple: be proud. CIRM exemplifies the state’s frontier mentality and willingness to take risks in the name of progress – very much in line with Silicon Valley's spirit.

Do you also get involved in inspiring and training the future generation of scientists?
 

Absolutely. We place a huge emphasis on educating the future workforce in regenerative medicine. We have different programs, starting with a high school summer internship (which is one of my personal favorites). These students come in with only a basic understanding of stem cells from their biology classes, but after an eight-week crash course they end up giving presentations and sound like mini-PhDs. It really blows you away!

We also have programs for undergraduates, graduate and Masters’ students, postdocs, and those in advanced training. These programs are generously funded, and many of the students are so inspired by what they learn that they choose to stay in the field.

What was your path into the stem cell industry?
 

I double-majored in biology and history at Yale. After that, I received a Marshall Scholarship, which allowed me to study at Oxford in the UK. There, I earned a doctorate in a department that didn’t formally exist at the time: the impact of disease and medicine on history. I was one of the early people to explore this area, which, as it turns out, has become a highly relevant topic in recent years.

After Oxford, I returned to Yale for law school and spent some time practicing law, including a fascinating stint in the White House Counsel’s Office. After a few years, I shifted into finance, working in investment banking and private equity for over two decades – mostly in areas unrelated to life sciences. However, after raising a one-off early venture round for a company called Advanced Cell Technology in Worcester, Massachusetts, I ended up with a real interest in regenerative medicine.

As I continued along with my career in private equity, I would periodically look at the CIRM website to see what was going on. Among other things, after the 2004 election, Bob Klein was named the Agency’s first Chair of the board. In 2010, Bob announced he was stepping down. I’d been looking for a way to get into life sciences, so I reviewed the criteria for the open position and I seemed to check all the boxes. I succeeded Bob as Chair in 2011 and had two six-year terms, which ended in March 2023.

In November 2023, when the previous President and CEO of CIRM stepped down, the board asked if I would consider stepping in on an interim basis while they conducted a search. I agreed, but as I got into the role I realized how much I enjoyed it, so I decided to formally apply for the position! I was chosen by the Board for the job and officially started at the end of July. My journey has definitely been an unorthodox path, but my experiences in biology, law, investment banking, and other fields have been useful. I’ve been privileged to be a part of this remarkable effort for more than 13 years.