CIRM awards boost clinical trials for cancer, heart, and rare diseases

The California Institute for Regenerative Medicine (CIRM) has allocated $26 million to support clinical research targeting cancers, heart conditions, and rare diseases. This investment reinforces CIRM’s commitment to advancing innovative stem cell and gene therapy treatments through all stages of clinical development.

This round of awards funds five projects within CIRM’s Clinical Program, which provides essential support for eligible projects at various stages of development. With these awards, CIRM now supports 108 clinical trials, reflecting our continued dedication to groundbreaking research and transformative therapies.

This month’s Clinical Program Awards

Grant Title and Application Number Principal Investigator/Institution Award 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  

These projects represent a crucial step in improving patient outcomes and offering new hope for individuals affected by these severe health challenges.

CIRM remains committed to supporting the most promising regenerative medicine research, intending to accelerate the development of treatments and cures. We are excited to see the progress that will be made possible through these new grants and look forward to their positive impact on patients’ lives. 

Rare disease are not so rare

A rare chance to help those in need

CIRM Top Awards

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