CIRM Awards $29.7 Million to Advance Clinical Projects
FOR IMMEDIATE RELEASE
Contact:
Koren Temple-Perry
Sr. Director, Marketing & Communications
press@cirm.ca.gov
South San Francisco, CA, November 21, 2024 – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has approved awarding $29.7 million to fund two clinical projects aimed at addressing rare pediatric diseases.
The awards will support 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 111.
The successful applicants in CIRM’s Clinical program this month are:
| Application # | Program Title | Principal Investigator/Institution | Amount |
| CLIN2-17078 | Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFSADA lentiviral vector encoding for human ADA gene in ADA-SCID subjects | Kohn, Donald — UCLA | $14,798,337 |
| CLIN2-17127 | Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector | Cowan, Mort — UCSF | $14,999,999 |
Advancing a Stem Cell Clinical Trial for ADA-SCID
A $14 million award to Donald B. Kohn, MD, of UCLA will support a clinical trial evaluating a gene-edited stem cell therapy for the treatment of Adenosine Deaminase-Deficient Severe Combined Immunodeficiency (ADA-SCID), a rare and life-threatening condition in which babies are born without a functioning immune system.
While a stem cell transplant from a matched-related donor is currently the best option for ADA-SCID, it is only available to about 20 percent of patients. For others, mismatched transplants carry a high risk of rejection and long-term complications.
Unlike a transplant, the proposed therapy takes the patient’s own stem cells and genetically modifies them in the lab to produce the missing ADA enzyme. From there, the stem cells are returned to the patient’s body to restore immune function. The one-time therapy eliminates the risk of rejection and significantly reduces the potential for long-term side effects.
“This therapy represents a significant step forward in improving the treatment landscape for ADA-SCID, offering hope for a potentially curative treatment for this life-threatening condition,” said Jonathan Thomas, JD, PhD, President and CEO of CIRM. “We are proud to support therapies that target rare diseases and address the critical unmet needs of these young patients.”
This follow on funding comes after Orchard Therapeutics transferred the FDA license back to UCLA to continue conducting the clinical trial in 2021.
“This funding will help us advance this curative therapy toward FDA approval, bringing us closer to making it widely available to patients nationwide,” said Dr. Kohn. “We’re deeply grateful to CIRM for its continued partnership in working toward our shared goal: ensuring that every child who needs this therapy can access it.”
Supporting Gene Therapy for Rare Pediatric Disease
Another CIRM award also aims to support a potential therapy for a rare pediatric disease. Through a $15 million award to Mort Cowan, MD, of UCSF, CIRM will support a clinical trial for Artemis-deficient severe combined immunodeficiency (Artemis-SCID), a life-threatening genetic disorder that primarily affects infants.
This trial will focus on patients with SCID who have mutations in a gene called DCLRE1C which encodes for Artemis protein. Artemis-SCID is the most difficult form of SCID to treat when using a standard bone marrow transplant from a healthy donor.
Babies born with Artemis-SCID lack a functioning adaptive immune system with absence of T and B lymphocytes, leaving them highly vulnerable to life-threatening infections and unable to survive without definitive treatment. While bone marrow transplants from a healthy donor are the current standard of care, they carry significant risks. Complications can include rejecting the marrow graft, graft-vs.-host disease – in which the donor T cells attack the recipient’s tissues – chronic infections leading to organ damage, stunted growth, and premature death.
The UCSF team will genetically modify the patient’s own blood stem cells with a functional copy of DCLRE1C, with the goal of creating a new adaptive immune system while eliminating the risks associated with a bone marrow transplant.
“CIRM is proud to support this Phase 2 clinical trial,” said Rosa Canet-Avilés, PhD, Chief Science Officer (CSO) at CIRM. “This innovative approach has the potential to make treatment significantly safer for ART-SCID while improving survival rates for babies affected by this life-threatening condition.”
“We are very excited about this definitive therapeutic approach and expect that the new funding from CIRM will enable us to secure a biologics license from the FDA, making this therapy accessible to all affected patients worldwide,” said Dr. Cowan.