How California Built a Global Powerhouse in Stem Cell and Gene Therapy 

Two men engaged in a discussion for a podcast, seated at a table in a modern office setting with natural light.

A decision California voters made more than two decades ago helped build an unparalleled scientific ecosystem that’s now paying dividends for the state, according to Johnathan Thomas, the president and CEO of the California Institute for Regenerative Medicine (CIRM).

“(California) is pretty much now the largest funder of stem cell and gene therapy research in the world,” said Thomas, during an interview on UC San Diego’s Sanford Stem Cell Institute’s reGEN podcast.

Delivering on a promise 

As the field of research has evolved and matured, so too has CIRM’s focus.

Early on, the agency focused on seed grants, training grants, and providing space where California researchers could work with embryonic stem cells. Now, those initial investments in training scientists, basic research, and university and medical center infrastructure have evolved into supporting active early- and late-stage clinical trials across a broad range of conditions in locations throughout the state. CIRM has funded research on more than 85 diseases and has awarded almost 1,500 grants in stem cell and gene therapy.

“The agency began by funding what was primarily basic research and infrastructure throughout the state to house stem cell research,” Thomas said, explaining the evolution of CIRM over two decades. “Since that time as the field has matured so has the programs that CIRM funds.”

After more than 20 years of creating the pipeline, JT pointed to the first approval for a therapy that emerged from CIRM-supported research and clinical trials. In March, the FDA approved KRESLADI™, a one-time therapy developed by Rocket Pharmaceuticals, Inc., for the treatment of severe leukocyte adhesion deficiency-I (LAD-I) in children who have no bone marrow donor match for stem cell transplantation. For context, the FDA approved just 46 new drugs in 2025.

“(W)e are delivering on our commitment to Californians to support the development of new stem cell and gene therapies that save lives,” Thomas said at the time.

A voter-driven origin story 

In 2004, California voters decided that if the federal government wouldn’t support stem cell research, they would. That decision led to the creation of CIRM, with its initial $3 billion of funding.

Voters approved another proposition adding another $5.5 billion and expanding the scope of the scientific research to include gene therapy with an emphasis on neurodegenerative diseases like Parkinson’s disease and Alzheimer’s.

“So now it’s an $8.5 billion (investment),” said Thomas. “That’s allowed us the great privilege of funding science in academic institutions, research institutions, and biotech companies.”

Helping to power a biotech economy

It’s hard to overstate how outsized California has become in the field and in biotechnology more generally. The state’s Business and Economic Development agency estimates that the state’s biotech sector generates more than $414 billion in annual economic output and employs about 466,000 people, numbers that dwarf those seen in many countries.

The focus remains on finding new treatments, but CIRM’s work is also instrumental in creating the unique public-private ecosystem in which those discoveries are made, Thomas said. That means training scientists and future scientists, supporting labs and their researchers, helping support clinical trials, and ensuring that clinical trial centers across the state are accessible to more people.

“This is a rare era we’re in right now,” Thomas said. “I mean, the advances in science and scientific techniques have allowed for a great acceleration in biological and medical discovery. History will look back on this time as very central to the advancement of therapies and cures for many diseases.”

Check out the full podcast episode: