California Institute for Regenerative Medicine

Newborn Screening Awareness Month is celebrated each September to recognize the life-saving power of early detection.

Newborn screening tests identify over 12,000 infants each year with conditions that could lead to severe disability or death if left untreated. These vital screenings ensure every baby has the best chance for a healthy start. Learn more at https://rarediseases.org/es/newborn-screening-history-future-and-awareness-month/

Each year, around 20,000 Americans are diagnosed with acute myeloid leukemia (AML), a type of cancer that originates in the bone marrow and rapidly spreads to the bloodstream. Many others are affected by myelodysplastic syndromes (MDS), which disrupt the formation or functioning of blood cells.

CIRM has awarded $8 million to Rochelle Emery, MD, at Senti Bio to advance a clinical trial for a novel therapy targeting AML and MDS.

This new therapy utilizes gene-modified natural killer (NK) cells to help fight these cancers, offering a durable and effective solution to increase life expectancy for patients with recurring or relapsing AML.

CIRM awards $8 million to Supports Groundbreaking Gene Therapy for AML and MDS Image source: Canva  Around 20,000 Americans are affected by acute myeloid leukemia (AML) each year. AML is a type of cancer that originates in the bone marrow and rapidly enters the bloodstre…

The Cell & Gene Therapy Workshop: CMC Development and Patient Access, hosted by California Life Sciences - CLS and CIRM, was a day of learning and brainstorming about the bright future of cell and gene therapies.

Dr. Deepak Srivastava, president of the Gladstone Institutes, started the day by discussing the opportunities and challenges of translating scientific insights on cardiac diseases into cell and gene therapies for patients.

Dr. Paolo Gargini, Chairman of IRDS and former director of technology at Intel, provided insights from the semiconductor industry on the value of linking forward-thinking with sustained collaborative problem-solving to advance manufacturing.

Throughout the event, expert speakers provided practical tactics for navigating FDA requirements, raising funds, and generating evidence to overcome patient and market access barriers for cell and gene therapies.

In honor of Sickle Cell Disease Awareness Month, learn more about this patient’s journey to receiving a newly approved gene therapy for sickle cell. Despite the breakthrough, this story sheds light on the significant barriers patients face when accessing life-changing treatments.

First Day of a ‘New Life’ for a Boy With Sickle Cell Kendric Cromer, 12, is among the first patients to be treated with gene therapy just approved by the F.D.A. that many other patients face obstacles to receiving.

CIRM awarded $4 million to support the development of a one-time gene therapy for desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM).

This funding supports Deborah Ascheim, MD, and her team at Rejuvenate Bio in advancing RJB-0402, a therapy designed to address the root causes of DSP ACM.

DSP ACM arises from a mutation in the DSP gene, leading to irregular heartbeats and potential heart failure over time. RJB-0402 uses AAV8 to deliver the gene responsible for producing the FGF21 protein, which supports heart function and mitigates issues common in DSP ACM. This award will help transition the therapy into clinical trials, providing a new treatment avenue for patients in need.

CIRM $4 million awarded to support innovative Gene Therapy for Rare Heart Disorder Image Source: American Heart Association Journals The California Institute for Regenerative Medicine (CIRM) recently awarded $4 million to support the development of a one-time gene therapy for de…

CIRM would like to honor the contributions of Fred Fisher, MSW, LCSW, a dedicated member of our Independent Citizens’ Oversight Committee (ICOC) governing board and an enthusiastic patient advocate. Fred passed away this week.

Fred joined the CIRM ICOC as the ALS patient advocate in June 2021 and held various roles during his tenure, including co-chair of the Standards Working Group and member of the Application Review Subcommittee, Task Force on Neuroscience and Medicine, Presidential Search Subcommittee, Facilities Working Group, and Grants Working Group.

In addition to his enthusiasm for CIRM’s mission, he had an unwavering commitment to advancing research to find treatments for patients and people living with neurodegenerative diseases.

For over 20 years, Fred served as President Emeritus of the ALS Network, an organization dedicated to finding an effective treatment and cure for amyotrophic lateral sclerosis (ALS).

Additionally, he had over 35 years of experience leading non-profit social service and health organizations, including those serving children, families, seniors, high-risk youth, and individuals with mental health issues.

A tribute in memory of Fred Fisher has been posted on the ALS Network website: https://bit.ly/47sWchZ

For everyone in the Bay Area, join us this Saturday, September 14, at Oakland Tech for the 4th annual KitsCubed STEM Fair presented by California Life Sciences - CLS—it's free!

CIRM is thrilled to be part of this fantastic event again, celebrating the next generation of scientists. Bring the whole family for fun and interactive science activities. Register to attend at: https://www.eventbrite.com/e/kits-cubed-stem-fair-2024-tickets-954455972507

CIRM has allocated $26 million to support clinical research targeting cancers, heart conditions, and rare diseases.

Our commitment to advancing innovative stem cell and gene therapy-based treatments continues to grow.

CIRM awards $26 million to boost clinical trials for cancer, heart, and rare diseases The California Institute for Regenerative Medicine (CIRM) is proud to announce the allocation of $26 million to support clinical research targeting a range of cancers, heart conditions, and rare di…

Diagnosed with a rare SCN2A gene mutation, Connor has experienced hundreds of seizures daily as an infant. After years of searching, whole genome sequencing has finally provided answers.

Thanks to N-Lorem Foundation and funding from CIRM, Connor is now receiving personalized therapy that is bringing new hope and progress to his life.

Turning Hope into Reality: How a Genetic Therapy Helped Connor Photo credit: n-Lorem Connor Dalby is a 15-year-old teenager who loves music and the beach, but his journey has been far from ordinary. Diagnosed with a rare mutation in the SCN2A gene, his life ha…

Over 400 students and postdoctoral fellows from across California gathered early this month at the University of Southern California for CIRM's first-ever Trainee Networking Conference.

At the conference, attendees were connected with top scientists, patient advocates, and industry professionals.

The event provided invaluable learning, networking, and career development opportunities in regenerative medicine.

Next Generation of Regenerative Medicine Scientists and the Golden Era of Biomedical Innovation CIRM CEO and President, Jonathan ‘JT’ Thomas speaking in front of conference attendees. During the first-ever California Institute for Regenerative Medicine (CIRM) Trainee Networking Co…

At the 2024 CIRM Trainee Network Conference, patient advocate Jake Javier shared his incredible journey from a life-altering spinal cord injury to being one of the first to participate in a CIRM-funded clinical trial. His story is a powerful testament to medical innovation and personal resilience.

Where Are They Now: Jake Javier The California Institute for Regenerative Medicine (CIRM) in partnership with the University of Southern California (USC) recently hosted the 2024 CIRM Trainee Network Conference, which provided tr…

We had the pleasure of chatting with Cory Nicholas, PhD, the CEO and Co-Founder of Neurona Therapeutics. We discussed the newest milestone for the clinical-stage company and the importance of funding support from CIRM.

A Conversation with CEO Cory Nicholas on Neurona Therapeutics’ Recent RMAT Designation At the California Institute for Regenerative Medicine (CIRM), we enjoy highlighting achievements that come out of the research we support.   We recently sat down with Cory Nicholas, PhD, …

CIRM hosted a webinar to highlight advances in eye diseases and regenerative medicine with a lovely panel of clinicians and clinical trial patients.

If you missed it, you can watch the webinar now on our YouTube channel here: https://www.youtube.com/watch?v=ToQY6uXogmQ

Thank you to our panelists Kristin McDonald (patient advocate), Veronica McDougall (patient advocate), Dr. Pablo Avalos (Associate Director-Translational Medicine at Cedars-Sinai Medical Center), and Dr. David Liao (board-certified ophthalmologist with Retina Vitreous Medical Group) for making the time to share valuable information and their stories.

Let us (re)introduce you to our new CEO, Jonathan "JT" Thomas!

Check out our interview with JT and learn more about what excites him about leading CIRM and his vision for the future of gene and cell therapy.

Meet Our New CEO: Jonathan Thomas We are thrilled to (re)introduce Jonathan Thomas (JT) as the new President and CEO of the California Institute for Regenerative Medicine (CIRM). You can check out the original press release here. W…

Since 2012, CIRM has funded more than 800 internships for high school students across California to provide hands-on opportunities in and research.

Check out some of the photo submissions from the 2024 SPARK interns. 📸

We look forward to seeing these bright students at this year’s SPARK conference at !

If you’ll be at the SPARK conference this week, be sure to tag and use the hashtag ✨

From the theater to the lab, Josh Nelson’s career transformation is a story of dedication and adaptability.

With the support of the CIRM Bridges Program through Cal Poly Humboldt, Josh transitioned from entertainment to stem cell research and contributed groundbreaking work at the Gladstone Institutes.

A CIRM Bridges trainee’s unconventional path into regenerative medicine Image Credit: Gladstone Institutes Josh Nelson, a CIRM Bridges trainee, didn’t experience a straightforward path into regenerative medicine. For 20 years, Josh worked in entertainment as a te…

Sacramento State's Fatima Miranda Chavez and Amber Maifeld are at the forefront of stem cell research in the UC Davis Health lab.

With the support of a $2.9 million CIRM education and training grant, they are exploring the potential of stem cells to treat osteoporosis and other bone disorders.

Sac State students have been participating in the program since 2011, and the University is among 14 CSUs that receive funding from the Institute.

Sac State Biology students tap the potential of stem cells to cure illnesses In a state-of-the-art laboratory at UC Davis Health, Sac State science students are studying the power of stem cells to treat diseases including bone disorders.

Ground-breaking progress in stem cell and gene therapies for vision loss are being made! Hear from clinicians and patients in CIRM-funded trials at our "Exploring Advances in Eye Disease and Regenerative Medicine" webinar on August 8, 12:30-1:30 PM (PDT).

Register now to learn about current advancements, future directions, and what's next for vision loss therapies: https://cirm-ca.zoom.us/webinar/register/WN_aLXpJR8lQ6Ga8hqEGptbCg

Exciting update from the world of stem cell research!

A seventh individual has achieved HIV remission following a stem cell transplant nearly a decade ago. This 60-year-old German man, who also battled acute myeloid leukemia, has been off anti-retroviral drugs since 2018 and shows no signs of HIV or cancer..

This breakthrough highlights stem cell research's transformative potential. To date, CIRM has funded more than $104 million for HIV research and projects.

A 7th person with HIV is probably cured after stem cell transplant for leukemia, scientists say A German man has probably been cured of HIV, a medical milestone achieved by only six other people in the more than 40 years since the AIDS epidemic began.

Dr. Noriyuki Kasahara from UCSF has received an $11.8 million award from CIRM to advance a groundbreaking clinical trial targeting newly diagnosed high-grade gliomas.

This innovative gene therapy, DB107, aims to convert a harmless compound into a potent cancer-fighting drug directly within tumor cells, potentially transforming brain cancer treatment.

Gene therapy trial tests cancer-killing virus in brain tumors Dr. Noriyuki Kasahara from UCSF has been awarded $11.8 million by the California Institute for Regenerative Medicine (CIRM) to target newly diagnosed high-grade gliomas. This groundbreaking clinica…

With the support of a $6 million CIRM grant, Dr. Arjun Deb and his team at UCLA are advancing a pioneering treatment for heart attack and heart failure.

Their therapeutic antibody targets ENPP1 to reduce post-heart attack inflammation and fibrosis, aiming to prevent heart failure and improve patient outcomes.

CIRM awards $6 million to advance research for heart attack and heart failure treatments In a promising development for cardiovascular health, Arjun Deb, MD, a professor of medicine and molecular, cell, and developmental biology at UCLA, has received a $6 million award from the Califor…