CIRM awards $26 million to advance clinical research across cancer, heart conditions, and rare diseases


Koren Temple-Perry  
Sr. Director, Marketing & Communications

South San Francisco, CA, June 28, 2024 –
The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, awarded $26 million to fund clinical research that aims to address a variety of cancers, heart conditions, and rare diseases.

The awards will support five projects in the Agency’s clinical program, which provides funding for eligible stem cell and gene therapy-based projects through all stages of clinical trial development. These awards bring the number of CIRM-funded clinical trials to 108.

The successful applicants in CIRM’s Clinical program this month are:

Application #  Program Title  Principal Investigator/Institution  Amount 
CLIN1-14792  Superior forward-oriented b-globin vector for treating Sickle Cell Disease  Abedi, Mehrdad — UC Davis  $4,619,455 
CLIN1-14825  Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE)  Weiss, Yael — Mahzi Therapeutics  $4,000,000 
CLIN1-16244  Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy  Ascheim, Deborah D. — Rejuvenate Bio  $4,000,000 
CLIN2-15218  A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV Gene Therapy in Male Patients with Danon Disease  Schwartz, Jonathan — Rocket Pharmaceuticals, Inc.  $5,808,735 
CLIN2-16156  Selective, Off-the-Shelf Logic Gated CAR NK Cell Therapy Targeting CD33 and/or FLT3 Expressing Hematologic Malignancies  Emery, Rochelle – Senti Bio  $8,000,000 

Supporting Gene Therapy for Desmoplakin-Related Heart Disorders 

Through a $4 million preclinical award, Deborah D. Ascheim, MD, and the team at Rejuvenate Bio will advance the development of a one-time gene therapy (RJB-0402) for the treatment of desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM), a genetic disorder where a mutation in the DSP gene causes problems in the connections between heart muscle cells. This can lead to a rare, life-threatening, and debilitating condition that causes the heart to beat irregularly and potentially fail over time.

RJB-0402 is a new investigational gene therapy that uses AAV8, a type of virus used to deliver genes, to help the liver make the FGF21 protein. This therapy targets several key problems of DSP ACM. FGF21 is known to help heart function and reduce problems like irregular heartbeats, fat buildup, inflammation, and scarring, which are all common in DSP ACM.

“Our goal is to support the most promising research forward,” said Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM. “A one-time gene therapy treatment for patients with this rare cardiac disease DSP ACM, could have a significant impact on patients with this degenerative disorder. We look forward to supporting Rejuvenate Bio in bringing this exciting therapy to patients with this degenerative disease.”

“The funding and strategic support from CIRM will accelerate the development of our gene therapy candidate RJB-0402 into clinical trials for DSP ACM patients in desperate need of new therapies,” said Noah Davidsohn, PhD, Chief Scientific Officer & Co-Founder, Rejuvenate Bio. “We are confident that RJB-0402, a one-time gene therapy, could be a transformative regenerative medicine and the first disease modifying therapy to address the unmet medical need of DSP ACM patients, and the recent funding from CIRM will enable us to initiate our first in human clinical trial.”


Advancing Therapies for Acute Myeloid Leukemia and Myelodysplastic Syndromes

An $8 million award to Rochelle Emery, MD, at Senti Bio, will advance a clinical trial for a novel therapy targeting acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), conditions that affect approximately 20,000 Americans annually.

AML and MDS are both blood cancers that affect the bone marrow and blood cells. Both cancers disrupt the production of healthy blood cells and have a 5-year survival rate of about 32 percent. Patients with recurring and relapsing AML a face median survival of only 3-6 months despite various chemotherapy approaches or clinical trial treatments.

CIRM’s award will support the development of a new therapy that utilizes gene-modified logic-gated natural killer cells (NK cells) to help the body fight these cancers. This unique, and more targeted approach offers a potentially more durable and effective solution, with the aim of increasing the median life expectancy for patients with recurring/relapsing AML.

“We are excited to advance clinical research for AML and MDS for which limited treatment options exist,” added Dr. Creasey. “This innovative therapy holds great promise for improving treatment outcomes and providing new hope to patients facing these challenging conditions.”


About the California Institute for Regenerative Medicine (CIRM)

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.

With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.