California Institute for Regenerative Medicine

California Institute for Regenerative Medicine The California Institute for Regenerative Medicine,
California's Cell & Gene Therapy Agency CIRM is California's Stem Cell Agency.
(207)

Our mission is to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world.

CIRM continues to advance research in neuropsychiatric and central nervous system (CNS) disorders through its ReMIND pro...
01/23/2025

CIRM continues to advance research in neuropsychiatric and central nervous system (CNS) disorders through its ReMIND program. This initiative supports large, collaborative teams that utilize stem cell and genetic research to address critical gaps in the field.

CIRM granted $12.9 million to Xin Jin, PhD, of Scripps Research, and her co-investigators to study neuropsychiatric risk genes linked to inherited psychiatric disorders. Their work represents a major step toward understanding how these conditions develop.

This blog is part of a series highlighting awards funded through the ReMIND program, which supports multidisciplinary discovery research at CIRM.

From top left to right: Xin Jin, PhD; Joseph Ecker, PhD; Rusty Gage, PhD Bottom left to right: Eran Mukamel, PhD and Maria Carol Marchetto, PhD As outlined in Proposition 14, one of the goals of th…

CIRM recently sponsored the ALS Network Research Summit, an event that brought together world-renowned researchers, inve...
01/23/2025

CIRM recently sponsored the ALS Network Research Summit, an event that brought together world-renowned researchers, investigators, clinicians, biotech companies, government representatives, and advocates in the ALS and other regenerative medicine fields.

To date, CIRM has funded more than $120 million in research projects that seek to better understand ALS and develop new cell and gene therapies for neurodegenerative diseases.

CIRM is advancing research into central nervous system (CNS) disorders through our ReMIND Program. CIRM awarded $12.2M t...
01/17/2025

CIRM is advancing research into central nervous system (CNS) disorders through our ReMIND Program. CIRM awarded $12.2M to principal investigator Alex Pollen, PhD, and a team of co-investigators from UCSF, UC Berkeley, and UC Santa Cruz. Their work uses patient-derived neural organoids to uncover the genetic foundations of autism spectrum disorder and develop diagnostic tools.

This blog is part of a series highlighting awards funded through the ReMIND program, which supports multidisciplinary discovery research at CIRM.

Principal investigator Alex Pollen, PhD, with his co-investigators and team of researchers. As outlined in Proposition 14, one of the goals of the California Institute for Regenerative Medicine (CI…

"Our message to Californians is simple: be proud. CIRM exemplifies the state’s frontier mentality and willingness to tak...
01/13/2025

"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."

Stephanie Vine from The Medicine Maker wrote a three-part feature of CIRM President and CEO Jonathan Thomas (JT). Check out part one, out now!

We speak with Jon Thomas, President and CEO of CIRM to get the background on the California Institute and how it is funding stem cell and gene therapy.

This week, CIRM Chief Science Officer Rosa Canet-Aviles attended The White House Cell and Gene Therapy Forum, which brou...
01/10/2025

This week, CIRM Chief Science Officer Rosa Canet-Aviles attended The White House Cell and Gene Therapy Forum, which brought together leaders across science, government, industry, and patient advocacy to drive action in accelerating equitable access to cell and gene therapies.

We heard from White House Fellow Kamal Menghrajani, MD, and discussed ways to reduce therapy development complexity, foster sustainable funding, and expand equitable access to cell and gene therapies.

Concrete solutions emerged including supporting Centers for Medicare & Medicaid Services (CMS) pilot programs, public-private manufacturing collaborations, and streamlined pathways for ultra-rare disease approvals.

CIRM is proud to join this collaborative effort to make bold ideas a reality for patients everywhere.

CIRM is advancing research into central nervous system (CNS) disorders through our ReMIND Program. CIRM recently awarded...
01/10/2025

CIRM is advancing research into central nervous system (CNS) disorders through our ReMIND Program. CIRM recently awarded $12.5M to Jonathan Sebat, PhD, and his co-investigators at the UC San Diego Verge Center to address critical gaps in studying neuropsychiatric conditions like schizophrenia and autism.

This blog is the first in a series highlighting awards funded through the ReMIND program, which supports multidisciplinary discovery research at CIRM.

From top left to right: Anne Bang, PhD; Carrie Bearden, PhD; Eugene Yeo, PhD; Jonathan Sebat, PhD Bottom left to right: Alysson Moutri, PhD; Lilia Iakoucheva, PhD; Sébastien Jacquemont; Trey Ideker…

The MIT Technology Review featured a CIRM-funded clinical trial by Neurona Therapeutics, which explores an innovative st...
01/09/2025

The MIT Technology Review featured a CIRM-funded clinical trial by Neurona Therapeutics, which explores an innovative stem-cell-based therapy for epilepsy.

Since undergoing the procedure at UC San Diego in 2023, a clinical trial participant has seen a remarkable improvement, reducing his seizures from once a day to about once a week.

After years of controversy, stem cells are on the cusp of cures for conditions like epilepsy and type 1 diabetes.

CIRM's President and CEO Jonathan Thomas (JT), joined Stem Cell Podcast hosts Daylon James, PhD, and Arun Sharma, PhD, f...
01/08/2025

CIRM's President and CEO Jonathan Thomas (JT), joined Stem Cell Podcast hosts Daylon James, PhD, and Arun Sharma, PhD, for their first episode of the new year.

Tune in as JT discusses what CIRM has been up to with the development of the Strategic Allocation Framework (SAF), some of CIRM's greatest achievements over the last 20 years, and CIRM's vision of the future for regenerative medicine.

https://media.blubrry.com/stemcellpodcast/ins.blubrry.com/stemcellpodcast/SCP_285_JT.mp3Podcast: Play in new windowGuest:Dr. Jonathan Thomas “JT” is the President and CEO of the California Institute for Regenerative Medicine (CIRM), an organization that provides funding for stem cell research wi...

UCLA  researchers have developed a new, clinically guided method to engineer more powerful immune cells called invariant...
01/03/2025

UCLA researchers have developed a new, clinically guided method to engineer more powerful immune cells called invariant natural killer T cells, or iNKT cells, that can be used for an “off-the-shelf” cancer immunotherapy in which immune cells from a single cord-blood donor can be used to treat multiple patients.

The study was published earlier this year in Nature Biotechnology.

Immunotherapies have revolutionized cancer treatment by harnessing the body’s own immune system to attack cancer cells and halt tumor growth. However, these therapies often need to be tailored to each individual patient, slowing down the treatment process and resulting in a hefty price tag that co...

Diana Farmer, MD, from UC Davis Health, is leading a pioneering clinical trial funded by CIRM to treat spina bifida. Thi...
01/02/2025

Diana Farmer, MD, from UC Davis Health, is leading a pioneering clinical trial funded by CIRM to treat spina bifida. This trial is the first in the world to combine fetal surgery with stem cell therapy for treating spina bifida.

One of the first patients, 20-month-old Nixon Metcalf, was diagnosed with the most severe form of spina bifida and underwent in-utero surgery where stem cells were applied directly to his spine. Now, Nixon is taking his first steps.

CIRM has supported innovative stem cell research throughout our 20-year history to treat a wide range of diseases and conditions.

A landmark clinical trial is underway at UC Davis Health: the world's first spina bifida treatment combining fetal surgery with stem cells.

Stem cells have the power to regenerate damaged tissues, and researchers at the University of Southern California are un...
12/30/2024

Stem cells have the power to regenerate damaged tissues, and researchers at the University of Southern California are unlocking their full potential. Charles Murry, PhD, MD, head of USC Stem Cell, leads a team advancing clinical regenerative medicine to tackle diseases like heart failure, ALS, and macular degeneration.

From growing brain organoids to regenerating neurons to developing 3D-printed skull implants seeded with stem cells, these breakthroughs are reshaping what's possible in human health.

If successful, CIRM-funded projects like Mark Humayun, PhD, MD's stem cell-derived eye patch for macular degeneration and Denis Evseenko, PhD, MD's regenerative cartilage pouches could revolutionize treatment options.

USC researchers are revolutionizing how we treat disease by harnessing stem cells as “living medicine.”

December is HIV/AIDS Awareness Month, and to commemorate this important event, CIRM supported the 3rd Annual Timothy Ray...
12/27/2024

December is HIV/AIDS Awareness Month, and to commemorate this important event, CIRM supported the 3rd Annual Timothy Ray Brown Community Cure Symposium, held at the Palm Springs Art Museum on World AIDS Day.

This inspiring event featured CIRM-funded researchers leading groundbreaking efforts to combat HIV/AIDS. Paula Cannon, PhD, of Keck Medicine of USC, discussed gene therapy and its transformative potential in fighting the virus. Angelo Cardoso, MD, PhD, of City of Hope, and Sumit Chanda, PhD, of Scripps Research, shared insights into the promising role of CAR T cell therapies in the search for a cure.

To date, CIRM has invested more than $104 million in advancing regenerative medicine research for HIV/AIDS, from discovery research to clinical trials.

Event recap written by guest writer Jacqueline Hantgan, CIRM Sr. Outreach Advisor for Outreach and Advocacy December is HIV/AIDS Awareness Month. To commemorate this important event, CIRM recently …

Check out our Top 5 most read blogs of 2024! We look forward to bringing you even more inspiring stories in 2025. 🎉
12/18/2024

Check out our Top 5 most read blogs of 2024! We look forward to bringing you even more inspiring stories in 2025. 🎉

2024 was a significant year for the California Institute for Regenerative Medicine (CIRM). We reached many milestones, including celebrating our 20th anniversary and introducing our Strategic Alloc…

With funding support from CIRM, including a recent $14.7 million grant, Donald Kohn, MD, of Broad Stem Cell Research Cen...
12/16/2024

With funding support from CIRM, including a recent $14.7 million grant, Donald Kohn, MD, of Broad Stem Cell Research Center at UCLA's gene therapy for ADA-SCID offers new hope to patients with this rare and life-threatening immune disorder.

To date, 39 of 41 patients at UCLA have seen their immune function restored through this innovative approach, which aims to provide a one-time treatment option. This includes Jakob, little fighter, a young patient whose family’s journey reflects the life-changing impact of this therapy.

Photo Courtesy of Andrea Fernandez de Soto Imagine a life where every time you step outside, everything in your environment poses a life-threatening risk. For children born with ADA-SCID (adenosine…

CIRM spoke with John LeGall, MD, the principal investigator of ALLO-316, an allogeneic CAR-T cell therapy targeting CD70...
12/10/2024

CIRM spoke with John LeGall, MD, the principal investigator of ALLO-316, an allogeneic CAR-T cell therapy targeting CD70—a protein prevalent in clear cell RCC tumors. This groundbreaking therapy, developed by Allogene Therapeutics, received funding support from CIRM.

Recently, the FDA designated ALLO-316 an RMAT designation, highlighting its potential to transform the treatment of metastatic renal cell carcinoma (RCC).

RCC remains a challenging diagnosis, with limited treatment options and a five-year survival rate of less than 17%. Early findings from the Phase 1 TRAVERSE trial suggest promising results, including encouraging response rates and enhanced anti-tumor activity.

Photo Courtesy: Allogene Therapeutics Metastatic renal cell carcinoma (RCC) is an advanced form of kidney cancer with few treatment options and a five-year survival rate of less than 17%. With fund…

Neurona Therapeutics has reported encouraging interim results from its ongoing Phase 1/2 clinical trial of NRTX-1001, an...
12/10/2024

Neurona Therapeutics has reported encouraging interim results from its ongoing Phase 1/2 clinical trial of NRTX-1001, an investigational allogeneic inhibitory neuron cell therapy to treat drug-resistant mesial temporal lobe epilepsy (MTLE).

Data from both low- and high-dose cohorts show reductions in seizure frequency, with some participants experiencing sustained, long-term seizure control. This approach offers a potential alternative to traditional, tissue-destructive treatments like resection and ablation, preserving brain function while reducing seizures.

CIRM is supporting Neurona with an $8 million clinical project grant to test NRTX-1001 and has supported Neurona's research from the initial discovery research stage to the ongoing first-in-human clinical trial. Read the full press release: https://bit.ly/3ONaP7r

CIRM is continuing to support Donald Kohn, MD in developing a gene therapy for children affected by ADA-SCID. With $14.7...
12/02/2024

CIRM is continuing to support Donald Kohn, MD in developing a gene therapy for children affected by ADA-SCID. With $14.7 million in new funding, Dr. Kohn and his team aim to make this treatment accessible to patients in need. Their goal is to obtain the U.S. Food and Drug Administration's approval for the therapy so that insurance companies will cover the costs.

ADA-SCID affects 8 to 10 newborns in the U.S. each year, putting them at risk of life-threatening infections. This innovative therapy, developed by Dr. Kohn's team at the Broad Stem Cell Research Center at UCLA, utilizes the patient's stem cells to correct the genetic mutation responsible for the condition.

Dr. Donald Kohn has been awarded a $14.7 million grant from the California Institute of Regenerative Medicine to develop a commercial manufacturing protocol for a groundbreaking gene therapy targeting ADA-SCID, a rare and life-threatening immune disorder.

HIV/AIDS affects more than 1.2 million people in the U.S., including over 140,000 in California. CIRM has committed over...
12/01/2024

HIV/AIDS affects more than 1.2 million people in the U.S., including over 140,000 in California.

CIRM has committed over $104 million to fund and support projects aimed at advancing stem cell and gene therapies to better understand and treat HIV/AIDS.

By funding innovative research, CIRM supports research that addresses the urgent need for new treatments.

A recent article in Nature shared the news about a 53-year-old man in Germany who was declared free of HIV after receiving virus-resistant cells. The man—referred to as the “Düsseldorf patient”—wa…

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Accelerating Stem Cell Treatments to Patients

Our mission at CIRM is to accelerate stem cell treatments to patients with unmet medical needs by funding promising stem cell research in California.