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California Institute for Regenerative Medicine (CIRM)

@cirmnews.bsky.social
175 followers 71 following 86 posts

CIRM's mission is to accelerate world-class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world. Visit our website at cirm.ca.gov. The official CIRM account.

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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 30/09/2026
Christelle, cured of sickle cell after a bone marrow transplant at 28, now helps bridge gaps between patients, providers, and scientists at the UCSF Sickle Cell Center. She empowers warriors with clear info and reminds us: patients don’t just join innovation, they drive it: bit.ly/3Vm5rie
A person stands at a podium giving a speech. Behind them is a dark curtain. The text on the image reads: "Patients are the drivers of innovation. Without a patient, there is no problem, and without a problem, there is nothing to solve or innovate." Below, the name "Christelle Salomon," with the title "Health Policy and Education Analyst, UCSF Benioff Children's Hospitals and Sickle Cell Warrior," is displayed.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 29/09/2026
Clinical trials offer hope, but travel and lodging costs shouldn't block access. CIRM's Patient Support Program helps California families afford travel, food and lodging during trials, allowing them to focus entirely on healing: bit.ly/4hDubKp
A healthcare professional wearing a stethoscope holds the hand of another person. Text on the image reads: "The CIRM Patient Support Program offers financial assistance to eligible Californians enrolled in CIRM-funded clinical trials. Up to 30% of clinical trial participants drop out due to travel costs and logistical barriers. CIRM helps ease these burdens, making treatments more accessible and supporting trial success."
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 25/09/2026
“Every person in California should be able to access the most promising medical advances, including cell and gene therapies that are changing what is possible for serious and life-threatening diseases.” - Shyam Patel, Associate Vice President of Patient Access. Press release: bit.ly/46Feeyb
The image shows a multi-story medical building with a red-tiled roof, set against a blue sky. Text overlaid on the image states that CIRM invests $9M for a Community Care Center in Northern California to expand access to cell and gene therapies. The text also mentions that the center in Chico aims to serve nearby communities with therapies, clinical trials, patient support, and workforce training. The image is credited to Enloe Health.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 22/09/2026
Join us for a Closer to Cures event with Dr. Maria Grazia Roncarolo, Tr1x Inc. co-founder, as she shares updates on CIRM-funded clinical research for multiple sclerosis. Watch live on September 24 at *2:45 PM (PST): www.youtube.com/@CIRMTV *Time of presentation may be changed without notice.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 16/09/2026
Can "virtual stress" halt brain cancer? @ucsantabarbara.bsky.social researchers used light-responsive plant proteins to trigger virtual stress in glioblastoma cells, completely stopping their invasive growth. A major step toward safer brain tumor treatments: bit.ly/4h42OtF
news.ucsb.edu
Light-triggered stress response suppresses glioblastoma spread in 3D models
Researchers find that triggering a stress response in glioblastoma cells with light stops them from spreading — a development that could point the way for treatments for the hard-to-reach brain tumors
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 14/09/2026
For CIRM Bridges trainee Michael Zepeda, scientific research is a powerful tool for community advocacy. After completing his Master's at Cal State San Bernardino, Michael is heading to UC Irvine for his PhD to study the real-world toxicology of nicotine & e-cigarettes: bit.ly/3VnHlDy
csusb.edu
Preparing for a Ph.D., CSUSB alumnus focused on communication and advocacy | CSUSB News | CSUSB
Through CSUSB's CIRM Bridges Program, two-time alumnus Michael Zepeda discovered a passion for research that improves lives and serves communities.
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Reposted by California Institute for Regenerative Medicine (CIRM)
Sanford Stem Cell Institute UC San Diego @ucsdstemcells.bsky.social · 09/09/2026
Our latest reGEN Podcast 🎙️ features Mark Walters, MD, program director of the UCSF @cirmnews.bsky.social Alpha Clinic - & his work in blood and bone marrow transplants for children with blood disorders and cancer. Watch here bit.ly/4wYfA1t, or listen 🎧where you find your podcasts. #podcast #cancer
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Blood Cells and Gene Therapy | Mark Walters, MD
Mark Walters, MD, is the Jordan Family Director of Bone Marrow Transplantation at the UC San Francisco Benioff Children’s Hospital Oakland and serves as the program director of the UCSF CIRM Alpha Clinic. He has been the director of stem cell therapy and a division leader since 1999. Walters is a hematologist-oncologist who cares for children with blood disorders and cancer and specializes in blood and bone marrow transplants. Walters' research focuses on transplant and gene therapy for sickle cell disease and thalassemia – which are both red blood cell disorders that involve abnormalities of hemoglobin; the protein in red blood cells that carries oxygen. His goal is to offer a curative therapy to every child he treats. Because finding a healthy donor for sickle cell disease can be difficult, he also investigates methods that aim to correct the patient's own blood system through gene therapy and return the edited blood cells to the patient's circulation. Hosted by Bobby Gordon, MA, MBA This episode was recorded on Oct. 17, 2025, and all information presented is current as of this date.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 27/08/2026
CAR T-cell therapy works by extracting a patient's T-cells, genetically engineering them to target disease, and reinfusing them to destroy harmful cells. CIRM-funded research is expanding this "living drug" from blood cancers to HIV and autoimmune diseases. Learn more: bit.ly/4zKELra
Image of a T-cell with protrusions on a blue background. Text across the image reads, "What is CAR T-cell therapy?"A microscopic image of a T-cell, depicted with vibrant colors and textures highlighting its complex structure. Overlaid text reads, "First thing you need to know: T-cells are defense cells that look for threats in the body."An individual donates blood for CAR T-cell therapy, with a medical professional handling the process. The text on the image explains CAR T-cell therapy as collecting a patient’s T-cells.A person wearing a lab coat and gloves operates a centrifuge machine. A test tube rack is nearby, holding several test tubes with orange caps. Text overlay reads, "then genetically reprograms the T-cells to recognize and attack the harmful cells."
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 20/08/2026
“Family groups and patient organizations are absolutely central to research,” said Matthew State, MD, PhD, chief scientific advisor for Aligning Research to Impact Autism (ARIA) initiative and a scientific advisor for CIRM-funded projects: bit.ly/4xVXVZu
A network structure made of blue rods and multicolored nodes resembles a human brain. Below, text reads: "CIRM funds collaborative projects that bring patient voices directly into the lab. Families, patient advocates, and scientists are teaming up to accelerate breakthroughs in profound autism spectrum disorder research through the CIRM ReMIND initiative."
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 18/08/2026
CIRM launched a $10M program to fund open-source software that helps researchers integrate, analyze, and share regenerative medicine data. The goal: turn complex datasets into faster discoveries. Learn more: bit.ly/4g4MZ5A
Two individuals wearing lab coats are working at a desk with multiple computer screens, a microscope, and scientific equipment. They are reviewing printed images and data displayed on the monitors. The setting suggests a laboratory environment focused on research and analysis.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 14/08/2026
Former Broadway dancer Tara Ghiglieri joined a @ucsdstemcells.bsky.social trial testing GNSC-001, an investigational gene therapy for knee #osteoarthritis funded in part by CIRM. The therapy aims to target inflammation and cartilage damage in the joint. Read Tara's story: bit.ly/3TTpXpK
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Experimental Treatment for Knee Osteoarthritis Explained
Explore an innovative experimental treatment for knee osteoarthritis to alleviate pain and improve mobility after injuries.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 04/08/2026
CIRM-funded early trial results, presented by Dr. Steven Deeks of @ucsfstemcell.bsky.social, suggest a single infusion of modified CAR-T cells may help control HIV in some participants after stopping standard treatment, pointing to a possible future beyond lifelong therapy. bit.ly/3UdghpZ
A quote on a blue background reads: "A so-called one-shot cure [for HIV] is no longer aspirational... I think it's feasible." Below is the name Steven Deeks, MD, with an affiliation to the University of California, San Francisco. The logo for CIRM (California Institute for Regenerative Medicine) is at the bottom right.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 31/07/2026
At the 2026 #CIRMTraineeConference, nearly 500 trainees gathered to share research, explore careers, and hear from patient advocates. Programs like Bridges, COMPASS, and CIRM Scholars reflect #California’s investment in the next generation of scientists. #regenerativemedicine
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 21/07/2026
A Phase 1 trial at @stanfordmedicine.bsky.social suggests a safer stem cell transplant approach for children with Fanconi anemia, using a targeted antibody instead of radiation or high-risk chemo. CIRM invested $10M+ in this research. stan.md/4yByCwY
stanmed.stanford.edu
A gentler approach to pediatric stem cell transplants
Researchers are pioneering a safer stem cell transplant for children with Fanconi anemia, reducing toxicity and improving outcomes.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 08/07/2026
On the @ucsdstemcells.bsky.social podcast, Dr. Sheldon Morris discussed how collaboration with @ucsdhealth.bsky.social, industry, and researchers drives discoveries in stem cells, regenerative medicine, and gene therapies. youtu.be/5E_YA0-PKm8?si=zDf50hZGKRl…
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Sanford Stem Cell Clinical Center | Sheldon Morris, MD, MPH
This episode features Sheldon Morris, MD, MPH, and an overview of the Sanford Stem Cell Clinical Center where Morris serves as director. The Clinical Center at UC San Diego encompasses a wide scope of services and outreach, including the California Inst...
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 06/07/2026
3M+ Californians live with knee osteoarthritis. With $12M from CIRM, Genascence is advancing investigational gene therapy GNSC-001, designed to reduce inflammation, pain, and cartilage damage. Early data are promising; a larger trial is next. Read more: bit.ly/3QA4aCf
Person holding their knee, possibly in discomfort, with the text "1 in 5 Californians suffer from symptomatic knee OA." The background shows blurred stairs.Person sitting on a couch holding their knee, with text stating knee osteoarthritis is a leading cause of disability and pain among Californians.The image features text with a dark blue background. It states the annual financial burden of knee osteoarthritis (OA) is over $8 billion. Additional text below explains that Californians with knee OA face challenges like high costs for steroid injections and surgeries, and experience wage loss.A slide titled "California funds clinical research" describes a $12 million grant awarded by CIRM to Genasence for gene therapy research targeting knee osteoarthritis. Early trials show reduced pain and improved function. CIRM logo is at the bottom.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 03/07/2026
USC Roski Eye Institute and @keckschoolusc.bsky.social are launching a CIRM-funded Phase 2b trial of a stem cell-derived retinal implant for advanced dry AMD. The study will test whether it can safely replace damaged retinal cells and improve vision. bit.ly/4p59fPE
news.keckmedicine.org
Can a retinal implant reverse macular degeneration?
LOS ANGELES — Age-related macular degeneration, the leading cause of vision loss and blindness for Americans 65 and older, is a progressive disease affecting central vision. Over time, faces, book pag...
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 26/06/2026
CIRM’s largest research budget to date is an investment in Californians and the power of regenerative medicine to improve lives. In 20 years, CIRM funding has supported 100+ clinical trials for diseases without cures and expanded access. Full press release: bit.ly/4oORKTv
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 23/06/2026
New @ucsfstemcell.bsky.social research suggests CAR-T cell therapy may offer a new approach to HIV treatment. In a CIRM-funded trial led by Dr. Steve Deeks, engineered immune cells suppressed HIV in two patients. Hear more June 25 at 10:30 AM (PT): www.youtube.com/CIRMTV
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 16/06/2026
Hear from Genascence's founder and CEO, Tom Chalberg, PhD, on June 25 at 10:30 AM (PT), who will share an update on GNSC-001, a potential first-in-class gene therapy for knee osteoarthritis, and the progress of the CIRM-funded DONATELLO clinical trial. Watch live at www.youtube.com/CIRMTV
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 15/06/2026
Join us for Closer to Cures on June 25 at 10:30 AM PT. Hear CIRM awardees Dr. Tom Chalberg and Dr. Steve Deeks share updates on clinical trials in knee osteoarthritis and HIV. Watch live on CIRM’s YouTube: www.youtube.com/user/CIRMTV
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 12/06/2026
CIRM invested $2M to support @uclastemcell.bsky.social’s new Center for Advanced Biotherapies, helping expand cell and gene therapy manufacturing, speed early clinical trials, and build California’s regenerative medicine future. Read more: bit.ly/4fsfXMZ
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 11/06/2026
Finding a #ClinicalTrial in California is now easier. CIRM’s new dashboard helps patients quickly search active cell and gene therapy trials at nine CIRM-funded Alpha Clinics across the state. Learn more: bit.ly/4ojqyfx
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 03/06/2026
CIRM’s RAPID funding opportunity aims to speed development of platform-based genetic therapies for rare diseases. The RFA is now available for view and the mandatory consultation period is open. Interested applicants must schedule a consultation. Learn more at bit.ly/4uGKUli
Two people are sitting and smiling at each other. The person on the right is wearing a pink beanie and a purple jacket. The text reads: "New Funding Opportunity. CIRM’s RAPID funding program’s Request for Applications is now available, and potential applicants can schedule their mandatory consultations." The image credit is Stanford University.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 01/06/2026
California voters helped create a stem cell research ecosystem supporting research in 85+ diseases and nearly 1.5K grants. On the @ucsdstemcells.bsky.social reGEN Podcast, CIRM's President & CEO explains how that vote made California a global leader in regenerative medicine: bit.ly/3QaOHbl
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 29/05/2026
At @uclahealth.org's California Center for Rare Diseases, progress is accelerating for patients with complex genetic conditions. From coordinated specialty care to promising gene therapy research, including work led by Dr. Donald Kohn, hope is growing. bit.ly/4tUh4Z5
stemcell.ucla.edu
UCLA research prioritizes hard-to-treat patients through rare disease innovation
At the UCLA California Center for Rare Diseases (CCRD), “rare” is a word that includes over 7,000 conditions and affects 3 million Californians. With hundreds of thousands of patients and families tou...
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 28/05/2026
On the @ucsdstemcells.bsky.social reGEN Podcast, @cityofhope.bsky.social's Dr. Leo Wang highlights how CIRM Alpha Clinics is helping bring cutting-edge therapies to more patients across California through collaboration, access, and innovation. youtu.be/qCNBwbcc8-4?si=Uq7KtpPKSa_…
youtu.be
Novel Therapeutics and Discoveries | Leo D. Wang, MD, PhD
YouTube video by Sanford Stem Cell Institute at UC San Diego
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 26/05/2026
Dr. Stephanie Cherqui and her team at @ucsdhealth.bsky.social developed a CIRM-supported stem-cell-based genetic therapy that changed Jordan Janz’s life. Now, CIRM’s RAPID initiative aims to build on breakthroughs like this to help more people with rare diseases. Learn more: bit.ly/3Rydc2D
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 22/05/2026
CIRM Vice Chair Maria Gonzalez Bonneville joined after her father’s Parkinson’s diagnosis. Now she chairs CIRM’s #AccessibilityAndAffordability Working Group to help ensure CIRM-backed therapies reach all Californians. bit.ly/3QZj052
The image features a quote in white text on a dark blue background: "I wanted to be part of an organization whose mission [and commitment to the people of California] was just so clear, it's unwavering." Below the quote is a small circular photo and the text: "Maria Gonzalez Bonneville, CIRM Independent Citizens' Oversight Committee Vice Chair." The CIRM logo, standing for California Institute for Regenerative Medicine, is at the bottom right.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 20/05/2026
Clinical trials don’t move forward without people. For #ClinicalTrialsDay, we’re honoring the patients who make progress possible. CIRM supports 100+ clinical trials and a statewide Alpha Clinics network to expand access across California. Read more: bit.ly/4wHEbss
blog.cirm.ca.gov
Clinical Trials: A Vital Step in Medicine
Join us in celebrating clinical trials and the brave participants who help advance medical research and treatment options.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 19/05/2026
Solano Community College’s CIRM-funded COMPASS program trains students to spot and help build life-saving therapies. Alum Joel Saenz now helps make CAR-T products at UC Davis: “If it wasn’t for CIRM…” Full recap: bit.ly/4uivoeW
A group of five people stand in front of a stone wall. One individual is wearing a sweatshirt with the word "Chicago" on it. The others are dressed in business casual attire. They are all smiling and standing in a line.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 14/05/2026
CIRM President & CEO Jonathan Thomas joined the @ucsdstemcells.bsky.social reGEN Podcast to discuss CIRM’s impact, why research funding matters, CA’s leadership in stem cell & gene therapy, and the #CIRMAlphaClinics Network for clinical trials access. bit.ly/4u84YfQ
youtu.be
A Conversation with CIRM's President and CEO | Jonathan Thomas, PhD, JD
This episode features our conversation with Jonathan Thomas, PhD, JD, President and CEO of the California Institute for Regenerative Medicine (CIRM). Thomas was previously the chair of the CIRM governing board from June 2011 to March 2023 and has played...
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Reposted by California Institute for Regenerative Medicine (CIRM)
Rimas Orentas @cgtgaithersburg.bsky.social · 12/05/2026
So our anti-HIV CAR makes it to the NYT. LV from Miltenyi, funded by CIRM @cirmnews.bsky.social - article should also highlight Dr Abedi’s team @ucdavismedschool.bsky.social @ucdavis.bsky.social where patients are being treated. #HIVcure #HIV #ASGCT26 www.nytimes.com/2026/05/11/h...
nytimes.com
A Single Infusion Could Suppress H.I.V. for Years, Study Suggests
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 11/05/2026
Maria Gonzalez Bonneville, vice-chair of CIRM’s governing board and chair of CIRM’s Accessibility and Affordability Working Group discusses her path to CIRM, her work, and her commitment to help Californians live healthier lives. Read more of Maria's story: bit.ly/3QZj052
The image shows a person standing in front of the United States Capitol building. They are smiling and wearing sunglasses. Text on the image reads: "From poli-sci to science. Maria Gonzalez Bonneville, Vice Chair of the CIRM Board, started her career in state politics. Now, the mission is to give Californians access to advanced gene and stem cell cures to those who need them.
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Reposted by California Institute for Regenerative Medicine (CIRM)
Sanford Stem Cell Institute UC San Diego @ucsdstemcells.bsky.social · 11/05/2026
Our reGEN Podcast 🎙️ with Jonathan Thomas, PhD, JD, President and CEO of @cirmnews.bsky.social reviews the Alpha Clinic network and patient-focused novel therapeutics, clinical trials and gene therapies. Watch here bit.ly/4ffhe9K or listen 🎧 where you find your podcasts. #stemcells #genetherapy
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A Conversation with CIRM’s President and CEO | Jonathan Thomas, PhD, JD
This episode features our conversation with Jonathan Thomas, PhD, JD, President and CEO of the California Institute for Regenerative Medicine (CIRM). Thomas was previously the chair of the CIRM governing board from June 2011 to March 2023 and has played an integral role in supporting patient-centric research, strategies and outcomes within the Alpha Clinic network and UC San Diego’s Sanford Stem Cell Institute. We discuss his history with CIRM and the impact the organization has had across California. Thomas has a long-standing commitment to patient advocacy. He spent more than 15 years on the board of the Crippled Children’s Society of Southern California and served as chair for four years. The organization, now called AbilityFirst, assists children with spinal cord injuries and mental disabilities that could be targets of stem cell therapies. Hosted by Bobby Gordon, MA, MBA This episode was recorded on Oct. 17, 2025, and information presented herein is current at the time of recording.
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Reposted by California Institute for Regenerative Medicine (CIRM)
Sanford Stem Cell Institute UC San Diego @ucsdstemcells.bsky.social · 27/04/2026
A $13M award from @cirmnews.bsky.social is funding groundbreaking #regenerativemedicine research led by @geneyeo.bsky.social and team to tackle “RNA pollution”🧬 as a root cause of neurodegeneration to protect the aging brain 🧠. Learn more @: bit.ly/41VgKOv. H/t @salkinstitute.bsky.social
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 23/04/2026
Recently, Diana Farmer, MD, surgeon-in-chief at @ucdavishealth.bsky.social Children’s Hospital and chief of pediatric surgery at Shriners Children's NorCal, shared an update on a CIRM-funded clinical trial of a stem cell treatment for a paralyzing form of spina bifida. bit.ly/496XIIG
Healthcare professionals in a medical setting conduct a procedure. They wear protective gowns, masks, and caps. The text on the image reads: "CuRe Trial Update: Stem cell therapy to improve mobility in Spina Bifida. Clinical trials show early promise for in-utero stem cell treatments for children diagnosed with spina bifida, enabling them to walk and run unassisted as they grow older." Image credit: UC Davis Health.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 15/04/2026
CIRM celebrates the first taxpayer-funded gene therapy approved by @fda.gov— KRESLADI™ for LAD-1, developed by Rocket Pharmaceuticals, Inc. bit.ly/4ta5sRP
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 03/04/2026
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
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.
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Reposted by California Institute for Regenerative Medicine (CIRM)
UCLA Broad Stem Cell Research Center @uclastemcell.bsky.social · 27/03/2026
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
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 27/03/2026
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: bit.ly/4ta5sRP
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.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 19/03/2026
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: bit.ly/4b4epWV
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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…
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 17/03/2026
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: bit.ly/4uP7EQy
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Reposted by California Institute for Regenerative Medicine (CIRM)
Jan Nolta, PhD @jannolta.bsky.social · 03/03/2026
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...
science.org
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
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 26/02/2026
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: bit.ly/4tWzidS
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.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 18/02/2026
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: bit.ly/4aBvHZT #RareDisease #GeneTherapy
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.
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Reposted by California Institute for Regenerative Medicine (CIRM)
Sanford Stem Cell Institute UC San Diego @ucsdstemcells.bsky.social · 08/01/2026
🙌 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
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Reposted by California Institute for Regenerative Medicine (CIRM)
Sanford Stem Cell Institute UC San Diego @ucsdstemcells.bsky.social · 15/01/2026
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
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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.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 10/02/2026
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: bit.ly/4clsv6Y
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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.
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California Institute for Regenerative Medicine (CIRM) @cirmnews.bsky.social · 06/02/2026
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:: bit.ly/4qYLvNd
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.
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