South San Francisco, CA, Oct. 27, 2023– The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, awarded $42.1 million to fund various translational research — from treating brain cancers and salivary gland conditions to advancing a therapy for a rare metabolic disease.

The goal of CIRM’s Translational program is to support promising stem cell-based or gene projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use. Those can include therapeutic candidates, diagnostic methods or devices and novel tools that address critical bottlenecks in research.

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

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

A $3.9 million award to Nicole Kristen Paulk, PhD, CEO and President of Siren Biotechnology will advance a novel adeno-associated virus (AAV) immuno-gene therapy for treating patients with glioblastoma, the most common primary brain tumor in adults and second in children.

Glioblastomas remain one of the deadliest brain cancers with a 5-year survival rate at 5 percent. There are more than 13,000 diagnoses of this type of cancer each year in the U.S.

This new therapy is designed to deliver a single-dose administration of engineered anti-cancer cytokine proteins directly to glioblastoma tumor cells through an AAV vector, a proven gene therapy vehicle. Following the delivery of cytokine proteins with an AAV, a patient’s immune system is triggered to fight and eliminate the tumor cells.

“Siren Biotechnology is thrilled and deeply honored to receive this support from CIRM. These significant funds will help advance the world’s first AAV gene therapy in oncology and speed our development as we push towards the clinic. We are excited to develop this completely new modality for cancer and grateful to CIRM that they share this vision and excitement with us,” said Paulk.

This award has the potential to bring direly-needed, effective therapies to the 1,400 Californians diagnosed with glioblastomas each year.

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.

For more information go to www.cirm.ca.gov