California Institute for Regenerative Medicine (CIRM)

For 20 years, CIRM has been at the forefront of regenerative medicine—funding critical research, building infrastructure, and championing patient access to clinical trials. Our 2024 annual report, "Our Journey Forward: How CIRM Catalyzes Regenerative Medicine Today and Beyond," showcases the stories, research, and partnerships that drive our mission. We invite you to explore our annual report here: https://lnkd.in/gbE2M5yJ

CIRM leadership was honored to meet with Controller Malia Cohen and the Citizens Financial Accountability Oversight Committee (CFAOC) board to discuss the impact of our work in the stem cell and gene therapy field and our commitment to transparency and accountability. We had an engaging conversation about CIRM's significant achievements and our future. We also saw some familiar faces, including CFAOC board member Al Rowlett, a former CIRM board member.

2024 was a significant year for CIRM. We reached many milestones, including celebrating our 20th anniversary and introducing our Strategic Allocation Framework (SAF) impact goals, all while continuing to fund innovative research and advancements in regenerative medicine. Here is a collection of this year's most read stories from our blog, The Stem Cellar. Thank you for reading, and we look forward to bringing you even more inspiring stories in 2025!

With funding support from CIRM, including a recent $14.7 million grant, Donald B. Kohn, MD, of UCLA Broad Stem Cell Research Center'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, a young patient whose family’s journey reflects the life-changing impact of this therapy. #ADASCID #PatientStory Paola Andrea Fernández de Soto Abdul-Rahim #StemCellResearch #StemCellTherapy #StoriesOfHope #RegenerativeMedicine #ImmunodeficiencyDisorders

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.

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

Congratulations to CIRM Board Member Daniel Enrique Bernal for receiving the Lifetime of Commitment Award from the National AIDS Memorial. Dan has passionately advocated for health equity and HIV/AIDS activism for over 30 years, enhancing lives through policy, community service, and education. His work includes shaping key legislation, such as the Affordable Care Act, with Speaker Emerita Nancy Pelosi and representing the voices of HIV+ patients, showcasing a legacy of resilience and compassion. This well-deserved recognition celebrates Dan's profound impact as a leader, activist, and advocate. We are honored to have him on our Board, advocating for advancing stem cell research and transformative treatments for patients.

CIRM is continuing to support Donald B. 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 FDA 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 UCLA Broad Stem Cell Research Center, utilizes the patient's stem cells to correct the genetic mutation responsible for the condition.

Congratulations to Poseida Therapeutics, Inc. in reaching an agreement to be acquired by Roche. CIRM previously supported Poseida's late-stage preclinical research for the treatment of castrate-resistant metastatic prostate cancer, as well as a clinical project utilizing CAR-T cells in patients with multiple myeloma.

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. #WorldAIDsDay