CIRM celebrates the first taxpayer-funded gene therapy approved by @fda.gov— KRESLADI™ for LAD-1, developed by Rocket Pharmaceuticals, Inc. https://bit.ly/4ta5sRP

A graphic announcing that the CIRM Board approves $111M for discovery and clinical research. It details $80 million for six new discovery grants and $31 million for three clinical trial grants, aimed at developing treatments for both rare and common diseases. The CIRM logo is visible at the bottom right.

CIRM's governing board approved over $111 million to support advances in discovery and clinical research for children's #RareDiseases, #VisionLoss, and treatments for #neurodegenerative conditions. Learn more: bit.ly/4dtnLwY

The FDA granted accelerated approval for KRESLADI, a gene therapy for a rare pediatric immune disorder called LAD-I.

Dr. Donald Kohn at UCLA has spent 30+ years developing therapies like this. All 9 trial patients have restored immune function.

This is what science is for. 🔬
bit.ly/4uVBcfg

A pipette dispensing liquid into laboratory test tubes. Overlay text: "Breaking: First California-taxpayer funded therapy receives FDA approval. After 21 years of funding stem cell and gene therapy research, CIRM hits a major milestone with its first supported therapy receiving FDA approval for patient use.

CIRM celebrates a major milestone—the first therapy funded by California taxpayer dollars is now approved by the @fda.gov. This new gene therapy is a one-time therapy developed by Rocket Pharmaceuticals, Inc. to treat the rare childhood disease LAD-I. Read full story: https://bit.ly/4ta5sRP

Closer to Cures: Stanford researcher Crystal Mackall discusses progress on clinical trials for rare cancers of the brain and spine with no known cure. Written by Holly Alyssa MacCormick In 2023, the California Institute for Regenerative Medicine (CIRM) funded a clinical trial led by Stanford researcher Crystal Mackall, MD, to investigate a possib…

Major progress in rare brain tumor treatment. A CIRM-funded CAR-T trial led by Dr. Crystal Mackall of @stanfordmedicine.bsky.social shows tumor shrinkage & disappearance in H3K27M patients. A new chapter for young lives. Details: https://bit.ly/4b4epWV

A CIRM-funded trial by @RocketPharma offers hope for LAD-1, a rare immune disease. All nine trial participants had their immune function restored by a #GeneTherapy that successfully treated the life-threatening disorder.

Read the story of the Langenhops: https://bit.ly/4uP7EQy

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