The California Institute for Regenerative Medicine awarded $42.1 million for translational research projects. These projects target brain cancers, salivary gland conditions, and a rare metabolic disease.

CIRM’s Translational program supports promising stem cell and gene projects. It helps teams complete key activities needed for clinical studies or broad use. These activities can involve therapeutic candidates, diagnostic methods, devices, or new research tools that remove critical bottlenecks.

CIRM-funded translational research

Application	Program Title	Principal Investigator / Institution	Amount
TRAN1-15227	Development of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting	Lipshutz, Gerald – UCLA	$5,266,504
TRAN1-15257	Adenine Base Editing for Autologous Hematopoietic Stem Cell Gene Therapy of CD3δ SCID	Romero Garcia, Zulema – UCLA	$5,966,928
TRAN1-15230	Ex Vivo Modified Hematopoietic Stem Cells to Treat Danon Disease	Adler, Eric – UCSD	$5,180,389
TRAN1-15252	Hematopoietic Stem Cell Gene Editing for X-linked Agammaglobulinemia (XLA)	Kohn, Donald – UCLA	$4,822,284
TRAN4-15222	T-Pure: Peripheral Blood Processing Tool for Point of Care CAR-T Manufacturing	Nolta, Jan – UC Davis	$1,302,837
TRAN4-15298	Development of a low-cost, clinical-grade iPS maintenance medium for enabling stem cell therapy manufacturing	Rees, Steven – Defined Bioscience, Inc.	$999,848
TRAN1-15317	Noncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosis	Ibrahim, Ahmed – Cedars Sinai	$2,590,224
TRAN1-15330	Neurogenic hydrogel stimulation of stem cells to regenerate radiation-damaged salivary glands	Knox, Sarah – UCSF	$2,312,021
TRAN4-15253	Generation of human universal donor iPS cells	Hosoya, Tomonori – HEALIOS NA, Inc.	$999,989
TRAN1-15325	Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma	Paulk, Nicole – Siren Biotechnology, Inc.	$3,997,919
TRAN1-15341	Optogenetic Therapy for Treatment of Geographic Atrophy	Bresge, Paul – Ray Therapeutics, Inc.	$3,998,930
TRAN1-15291	Pro-regenerative infusible ECM biomaterial for treating acute myocardial infarction	Christman, Karen – UCSD	$4,624,192

An experimental AAV gene therapy for treating glioblastoma

CIRM awarded $3.9 million to Nicole Kristen Paulk, PhD, CEO of Siren Biotechnology. The funding advances a new AAV immuno‑gene therapy for glioblastoma, the most common adult brain tumor.

Glioblastoma remains one of the deadliest brain cancers. Its five‑year survival rate is 5 percent. More than 13,000 people in the U.S. receive this diagnosis each year.

The therapy delivers a single dose of engineered anti‑cancer cytokine proteins to tumor cells. An AAV vector carries these proteins, a proven approach in gene therapy. After delivery, the patient’s immune system activates and attacks the tumor.

Paulk said the award will accelerate development of the first AAV gene therapy in oncology. She noted the support will help Siren Biotechnology move toward clinical testing.

This award could lead to effective therapies for the 1,400 Californians diagnosed with glioblastoma each year.