California Institute for Regenerative Medicine (CIRM)

Treating fetuses with stem cells proves safe in milestone spina bifida trial The study is now scaling up to do more in utero procedures and evaluate effects on disability as children grow

Honored to be part of this trial, led by my amazing colleague Dr. Farmer, with much teamwork. Thank you for funding from @cirmnews.bsky.social (CIRM). 🫶
Treating fetuses with stem cells proves safe in milestone spina bifida trial | Science | AAAS www.science.org/content/arti...

Cory Nicholas is smiling against a blue background. The text on the image reads: "Epilepsy Hope: Neurona's CIRM-backed clinical trial advances. CIRM-funded clinical trials are helping patients with epilepsy, offering them a new hope.

3M Americans have #epilepsy; anti-seizure meds work for only half. Neurona's CIRM-funded #ClinicalTrial shows a new therapy has minimal side effects and fewer seizures.

Read the full story: https://bit.ly/4tWzidS

A young girl with glasses and a hat is smiling outdoors in a park setting. The image text reads: "Gene therapy offers kids with rare immune disorder a new chance. A gene therapy developed by UCLA offers young patients a new chance by editing the gene causing their rare immune disorder, LAD-1." Several people and trees are visible in the blurred background.

A @uclastemcell.bsky.social and Rocket Pharma gene therapy is transforming lives for kids with LAD-1. Marley, diagnosed at 8, is now healthy and enjoying life thanks to a CIRM-funded trial correcting her own stem cells. Read her inspiring story: https://bit.ly/4aBvHZT #RareDisease #GeneTherapy

🙌 A $7.4 million California Institute for Regenerative Medicine grant awarded to SSCI expert Stephanie Cherqui, PhD, and team will ready a revolutionary new treatment — a stem cell-based gene therapy for Friedreich’s ataxia — for clinical trial. 🎉 More: bit.ly/4jw1qiY. H/t @cirmnews.bsky.social

Stem Cell Clinical Trials: Behind the Scenes with Julissa Gonzalez at UC San Diego This episode features our conversation with Julissa Gonzalez, MS, who serves as the clinical research data manager at the UC San Diego Sanford Stem Cell Clinical Center. Her work helps frame and prepare insights into stem cell-related clinical trials. Our discussion explores information the public may not readily know about preparing for and administering therapeutics via clinical trials, including unique perspectives on validation, analyses, and patient care outcomes. Hosted by Bobby Gordon, MA, MBA. This episode was recorded on June 2, 2025, and information is current as of the date of this recording.

Get a behind the scenes look at stem cell and related clinical trials at our @cirmnews.bsky.social Alpha Clinic and Sanford Stem Cell Clinical Center with Julissa Gonzalez, MS, in our latest reGEN Podcast episode. View here or listen where you find your podcasts: bit.ly/49y2EY1

New $100M CIRM Initiative Centers Platform Approaches for Rare Genetic Diseases Over the next two years, the planned funding will be allocated to projects that have received some FDA feedback as well as emerging efforts.

CIRM's new $100M RAPID program accelerates gene therapies for #RareDiseases. Utilizing a platform model, inspired by successes like baby KJ, to bring hope to millions faster. Funding supports projects from early-stage to clinical trials.

Read more by GEN News: https://bit.ly/4clsv6Y

A newborn with medical tubes, wearing a knitted cap. Overlaid text reads: "Accelerating Rare Disease Cures for Californians. A $100 million investment will support platform technologies to bring more cures for more rare disease patients." The California Institute for Regenerative Medicine (CIRM) logo is in the upper right corner. Image credit: Children's Hospital of Philadelphia.

CIRM is adopting a platform approach to rare disease therapies. This program aims for faster development and delivery of genetic therapies for more rare diseases. $100 million allocated to bring treatments to those with limited options. Learn more:: https://bit.ly/4qYLvNd

A healthcare professional wearing a mask interacts playfully with a child in a medical setting. The text reads: "Building Blocks for Rare Disease Cures. CIRM's new program aims to deliver more genetic therapies to more patients faster." Image credit: University of California, San Francisco.

Millions with rare diseases lack therapies. CIRM's new program, leveraging genetic therapy advances and FDA pathways, aims to change that. $100M over 2 yrs will support platform tech for faster, broader treatment delivery. Read more: https://bit.ly/4qYLvNd

Promotional graphic for a California Institute for Regenerative Medicine event titled 'Closer to Cures' featuring Dr. Crystal Mackall. Scheduled for January 29, 2026, at 10:30 AM Pacific. Includes a quote about the potential of cellular therapy.

Learn about advancements in pediatric cancer treatment at CIRM's Closer to Cures Jan 29, 10:30 AM (PST). Dr. Crystal Mackall (@stanforduniversity.bsky.social) discusses CAR-T therapy for aggressive brain tumors. Watch live: youtube.com/cirmtv #CIRM #PediatricCancer #BrainTumors

Promotional poster for a California Institute for Regenerative Medicine (CIRM) event titled "Closer to Cures," featuring Dr. Cory Nicholas from Neurona Therapeutics. The poster includes the event date, January 29 at 10:30 AM, and highlights the importance of CIRM's support in regenerative cell therapy research. The background is blue with a swirling graphic design.

Watch CIRM's Closer to Cures speaker series on Jan 29, 10:30 AM (PST). Hear about progress toward FDA approval from CIRM grantees like Dr. Cory Nicholas (Neurona Therapeutics) on their #epilepsy cell therapy and how this research is already helping patients. #ClinicalTrials

Promotional graphic for a CIRM (California Institute for Regenerative Medicine) webinar titled 'Closer to Cures', featuring Dr. Cory Nicholas from Neurona Therapeutics, focusing on epilepsy, and Dr. Crystal Mackall from Stanford University, focusing on cancer. The event is scheduled for January 29, 2026, at 10:30 AM (PST) with a link to watch live on CIRM's YouTube channel. The background includes abstract blue and yellow design elements.

Join CIRM's Closer to Cures speaker series Jan 29, 10:30 AM (PST). Hear from Dr. Cory Nicholas (Neurona Therapeutics) & Dr. Crystal Mackall (@stanforduniversity.bsky.social) on #Epilepsy & #BrainCancer clinical trials. Streaming on our YouTube Channel: https://www.youtube.com/@CIRMTV #ClinicalTrials

Two healthcare professionals, one younger and one older, are viewing a digital tablet together. In the background is text discussing CIRM's commitment to making clinical trials accessible statewide, mentioning Marisela Bonneville as the chair of the Access & Affordability Working Group.

CIRM's new Access & #Affordability strategy aims to bring cell and gene therapies to all Californians, addressing barriers to care and cost.

With a $15.6 million Patient Assistance Fund, CIRM is committed to equitable access.

Read full release: https://bit.ly/4sgVAWP

Three individuals wearing lab coats focusing on a task in a laboratory, with a text overlay reading 'Supporting the new generation of biomedical rockstars. CIRM is training today's and tomorrow's scientists and broaden opportunities for students in biomedical research.'

CIRM expands training for students (high school, undergrad, grad, and post-doc). SPARK, Bridges, & COMPASS offer enhanced training, research & mentorship to build CA's regenerative medicine workforce. "Training scientists is critical..." says Kelly Shepard, PhD. Full release: https://bit.ly/4sgVAWP

Promotional poster for CIRM (California Institute for Regenerative Medicine) announcing over $160 million in funding for research and education to enhance medicine, decrease diseases, and develop innovative treatments in California. The poster features graphic icons of a DNA strand, a microscope, and a magnifying glass.

CIRM funded $160M+ for 16 research projects to bring discoveries to patients. "A breakthrough is only the beginning," says CSO Rosa Canet-Aviles.

Projects target Parkinson's, Huntington's & more, aiming to offer options where none exist. Full release: https://bit.ly/4sgVAWP

A quote about hoping for a full-blown cure for a condition, attributed to Audrey Davidow, President of Pitt Hopkins Research and a patient advocate. The graphic has a dark blue background with stylized text in white and gold.

Audrey tirelessly advocates for Pitt-Hopkins Syndrome #research, fueled by hope for her son, Calvin. CIRM supports early-stage research, laying the foundation for future cures. Calvin's story shows the need for scientific investment: https://bit.ly/3KKXoq33KKXoq3

From Isolation to Inspiration: Evangelina's Journey with ADA-SCID YouTube video by UCLA Broad Stem Cell Research Center

Stories like Evangelina's highlight why CIRM funds research. Supporting the scientific path to clinical trials helps us achieve life-changing treatments and cures. Watch her inspiring speech: https://youtu.be/SofJVAi19_Y?si=6dU-dHyrbramdtM6

🎥 : @uclastemcell.bsky.social

Game changer for kidney transplant patients: UCLA clinical trial could eliminate need for lifelong immunosuppressive drugs When Karina Ledesma’s kidneys failed in high school, she was told she’d need a transplant — and a lifetime of immunosuppressive drugs to keep it working. But thanks to an experimental UCLA clinical tr...

A $6.7 million grant from @cirmnews.bsky.social will support UCLA's clinical trial of a stem cell-based therapy designed to help kidney transplant patients safely stop taking lifelong immunosuppressive drugs. ➡️ bit.ly/4rkHUcW

Epilepsy affects millions, with 1/3 unresponsive to meds. CIRM funds early- to clinical-stage research for new stem cell therapies at UCSF & Neurona Therapeutics, offering hope for a better life. Learn more: https://bit.ly/3LWmYZz

Collage of four images featuring Loma Linda University: top left shows the Community Health System building, top right displays the facade of The Lundquist Institute, bottom left captures a researcher working in a lab, and bottom right is an aerial view of the university campus.

Access to clinical trials shouldn’t depend on where you live. CIRM is investing $27M to establish three Community Care Centers of Excellence in South LA, the Inland Empire & Desert Region, & the Central Valley—bringing stem cell & gene therapy trials closer to home. For more: https://bit.ly/3Wwl4Rq

Yael Wyte, the newest CIRM Board member and Alzheimer's patient advocate, engaged in a conversation at a table beside a window.

Yael Wyte has spent 20+ years advocating for people with #Alzheimers. Now, as a CIRM Board member, she’s helping advance #RegenerativeMedicine with compassion and purpose.

Read her full story: https://bit.ly/48HhD1B

Exposure to any germs could've killed her. 11 years later, she's living a normal life thanks to a gene therapy treatment. New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of trial participants.

Backed by early CIRM funding, a @uclastemcell.bsky.social-led gene therapy for a deadly childhood immune condition appears successful and has achieved success with 95% of the children in the trial regaining full functioning immune systems.

Read more: nbcnews.to/46Wd81X

CIRM’s 2025 Annual Report, Looking Forward: Advancing California’s Scientific Vision, is now available. It highlights #ScientificProgress, patient stories, and efforts to expand access to #RegenerativeMedicine across California.

Explore it here: https://adobe.ly/42FSVed

Quote about the lengthy process of bringing a successful treatment to the market by Kim Barrett, Vice Dean for Research, UC Davis School of Medicine, and member of the CIRM Board. The background is dark blue with the CIRM logo at the bottom.

Behind every breakthrough is years of science. Kim Barrett, PhD, says @ucdavishealth.bsky.social CuRe Trial for spina bifida was possible thanks to early studies funded by CIRM’s Discovery program. Investing across the pipeline turns discoveries into therapies.

Learn more: https://bit.ly/3W2nUNF

Portrait of Adrienne Shapiro, an advocate known for her work on sickle cell disease, smiling with a headline reading "Transforming sickle cell's future" and text describing her advancement in a clinical trial that gained national recognition.

Adrienne Shapiro, a 4th-generation mother of children with sickle cell disease, has been a tireless advocate for stem cell research and a powerful patient voice at CIRM. Her efforts helped advance a groundbreaking trial at @uclastemcell.bsky.social.

Read her story: https://bit.ly/3VAQfKY

“Discovery research is critical for uncovering novel disease targets and biomarkers that we can translate into therapies for clinical use,” said Kelly Shepard, PhD, Director of Discovery & Education programs at CIRM.

Read the full announcement: https://bit.ly/4gDLREC

Image shows a male crouching and hugging a happy dog, both displaying big smiles, in front of a house.

Evie Junior, born with #SickleCellDisease, participated in a CIRM-funded clinical trial at @uclastemcell.bsky.social, using his own modified stem cells. He’s now free from pain crises and meds.

This #SickleCellAwarenessMonth, we highlight the promise of curative therapies. https://bit.ly/47YO3o6

Illustration of a fetus with a magnifying glass over it held by a hand focused on its spine, showcasing spina bifida.

Babies with spina bifida are growing into walking, healthy toddlers thanks to a CIRM-funded trial at @ucdavishealth.bsky.social. Board member Kim Barrett shares why this work brings real hope.

Read the full story: https://bit.ly/3W2nUNF

“An awesome responsibility”: A conversation with CIRM board member Kim Barrett Written by Holly Alyssa MacCormick It’s not every day that you get to contribute to something that could help save a life, but when Kim Barrett joined the board of the California Institute for Rege…

When Kim Barrett joined CIRM’s board in 2021, she called it “an awesome responsibility.” Now Vice Dean for Research at @ucdavis.bsky.social, she helps guide how California invests in regenerative medicine.

UCLA scientists reprogram stem cells to create renewable cancer-fighting T cells In a first-of-its-kind clinical trial, UCLA scientists have shown it’s possible to reprogram a patient’s blood-forming stem cells to generate a continuous supply of functional T cells, the immune syst...

CIRM-funded @uclastemcell.bsky.social researchers reprogrammed blood #StemCells to produce #cancer-targeting T cells—offering a renewable path for immunotherapy. Early results are promising for treatment-resistant tumors.

#BlueskyBiotech

Quote graphic of CIRM COMPASS work trainee alum, Maya Singh.

Maya Singh, a CIRM COMPASS trainee from @ucberkeleyofficial.bsky.social, helped advance #CancerResearch and gained key skills for med school. Now at Tuoro University California, she credits the COMPASS program for shaping her path.

blog.cirm.ca.gov/2025/08/27/c...