Month: December 2023

CIRM Announces Interim CEO

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment
Vito Imbasciani, PhD, MD, and Jonathan Thomas, PhD, JD
Vito Imbasciani, PhD, MD, Board Chair and Jonathan Thomas, PhD, JD, Interim President and CEO

South San Francisco, CA, Dec. 15, 2023 – The Independent Citizens Oversight Committee (ICOC), the governing board of the California Institute for Regenerative Medicine (CIRM), named Jonathan Thomas, PhD, JD as CIRM’s Interim President and CEO at the Dec. 14 meeting. Dr. Thomas previously served as the ICOC Board Chair, a position he held for 12 years before retiring in early 2023.

The ICOC formed a presidential search committee to identity a new CEO and President, following the departure of Dr. Maria Millan in November of this year. Dr. Thomas will serve as Interim CEO throughout the executive search process. 

“We are pleased to confirm JT (Dr. Thomas) as the interim President and CEO for CIRM, an appointment that brings valuable support to the agency’s momentum during the ongoing search for a new permanent President and CEO,” said Vito Imbasciani, PhD, MD, Board Chair of CIRM. “The board is dedicated to recruiting a permanent leader with strong strategic leadership experience who will help the organization in its next phase of growth.”

JT was elected as Chair of the Board in 2011 after he was nominated by then-Governor Jerry Brown, Treasurer Bill Lockyer, and Lieutenant Governor Gavin Newsom. In that time, he led CIRM in granting $2.5 billion to support groundbreaking research to accelerate stem cell treatments to patients with unmet medical needs.

He chaired the agency as CIRM expanded its work with industry, revamped its award processes, prepared for the expiration of bond funding, supported the drafting of Proposition 14, and planned for the next phase of CIRM’s programs after the voters approved $5.5 billion in additional funding.

Before joining CIRM, he was a Co-Founding Partner at Saybrook Capital, an investment banking and private equity firm, where he led an early round of financing for Advanced Cell Technology (now Astellas Pharma). JT’s legal experience includes clerking for White House Counsel Lloyd Cutler in the last year of the Carter Administration and clerking for the Honorable George Mackinnon of the United States Circuit Court of Appeals for the District of Columbia Circuit. JT holds a bachelor’s degree from Yale University, a JD from Yale, and a PhD from Oxford University, where he was a George C. Marshall Scholar.

 “I’m excited and honored to rejoin CIRM’s team and Board in this new capacity. My goal during this interim period is to work with CIRM’s incredible leadership team to continue to deliver on our mission and to provide steady guidance throughout this transitional period,” said Dr. Thomas. 

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

Statement from CIRM on the FDA Approval of Gene Therapies for Sickle Cell Disease

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment

The California Institute for Regenerative Medicine (CIRM) applauds and acknowledges the historic significance of the Food and Drug Administration (FDA) approval of Vertex and CRISPR Therapeutics’ CRISPR-based gene therapy (Casgevy) for sickle cell disease.  

As the first regulatory authorization of a CRISPR-based gene editing therapy in the world, this brings forth a new era of treatment options for people living with sickle cell disease, a condition that affects more than 100,000 Americans and 20 million people worldwide.  

“As the first regulatory authorization for a CRISPR-based gene-editing therapy in the world, this approval not only marks a milestone in the advancement of cell and gene therapies, but it also marks a transformative step in the treatment landscape for the millions of people impacted by sickle cell disease,” said CIRM Vice President of Therapeutics Development, Abla Creasey, PhD. 

By leveraging CRISPR gene editing, this therapy has the potential to provide a more effective and potentially curative solution compared to traditional treatments. The FDA also approved another gene therapy for sickle cell disease (Lyfgenia) from bluebird bio based on long-standing lentivirus vector technology. 

Through the Cure Sickle Cell Initiative, CIRM, with funding from the National Heart, Lung, and Blood Institute, remains committed to funding cell and gene therapy programs that will accelerate the development of gene therapies to treat sickle cell disease, as well as create a collaborative, patient-focused research environment. 

CIRM recognizes that long-term innovation is needed to lower the costs of gene therapies, reduce the burden of the treatment on the patient, and make these therapies more accessible. 

CIRM and NHLBI are co-funding two clinical trials testing different types of gene therapy approaches for patients with severe sickle cell disease. Those include a pivotal Phase 2 trial led by David A. Williams, MD of Boston Children’s Hospital and a Phase 1 trial led by Mark Walters, MD of UCSF Benioff Children’s Hospital Oakland. 

In the Phase 2 clinical trial led by Williams, investigators will take a patient’s own blood stem cells and insert a novel engineered gene to silence abnormal hemoglobin and induce normal fetal hemoglobin expression. The modified blood stem cells are then reintroduced back into the patient. The goal of this therapy is the production of normal shaped red blood cells that are robust and able to transport normal amounts of oxygen, thereby reducing the severity of the disease.  

“As a researcher involved in development of new therapies to treat sickle cell disease and as a clinician caring for children with this terrible disease, I am elated to see FDA approval of two different effective genetic therapies. I anxiously await access of these therapies by patients with severe disease as well as the continued development of additional options for curative therapies for our patients,” said Dr. Williams, who has consulted for Vertex Pharmaceuticals and bluebird bio. 

In the Phase 1 trial led by Walters, patients will be transplanted with their own blood stem cells that have been edited with CRISPR-Cas9 to correct the disease-causing mutation in the beta globin gene. This trial will begin enrolling patients in California in 2024.   

Please direct any inquiries to Koren Temple-Perry, Senior Director of Marketing & Communications at press@cirm.ca.gov.

CIRM invests $17.5 million in clinical-stage research, including Phase 2 spina bifida trial

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment
Diana Farmer, MD and Aijun Wang, PhD. Photo courtesy UC Davis Health
Diana Farmer, MD and Aijun Wang, PhD. Photo courtesy UC Davis Health

South San Francisco, CA – The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, has awarded $17.5 million to fund clinical-stage research projects aimed at advancing stem cell and gene therapy treatments for a variety of conditions ranging from neurodegenerative diseases to blood cancers.  

The awards will support three 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 96.

The awards include: 

ApplicationProgram TitlePrincipal Investigator / InstitutionAmount
CLIN1-14945 Pre-Clinical to Clinical Gene Therapy Development for CMT4J Pirovolakis, Terry – Elpida Therapeutics$3,930,964 
CLIN1-15060 A 1XX-enhanced and fully non-viral BCMA chimeric antigen receptor (CAR) T cell therapy for Relapsed and Refractory Multiple Myeloma Martin, Thomas – UCSF$4,585,501 
CLIN2-15115 The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele Farmer, Diana L. – UC Davis $8,996,477

 This month’s clinical awards include two preclinical projects and one clinical-stage project. Among the awards is a $9 million grant to Diana Farmer, MD at UC Davis and chief of pediatric surgery at Shriners Children’s Northern California to support the completion of a Phase 2 clinical trial to improve the outcomes for children born with a severe form of spina bifida called myelomeningocele (MMC).  

MMC is a birth defect that occurs when the spine and spinal cord aren’t formed properly during fetal development, usually resulting in life-long lower body paralysis and bowel and bladder dysfunction.  

The CIRM award will support the advancement of a one-of-a-kind treatment delivered while a fetus is still developing in the mother’s womb via a patch coated with placenta derived stem cells. The treatment is meant to prevent paralysis and preserve normal bodily function, and ultimately improve quality of life of these affected children and their families. Learn more about the procedure here.

Image credit: UC Davis Health

Image credit: UC Davis Health 

“This award supplies critical funding to support a Phase 2 clinical trial to achieve our goal of using stem cells before birth to improve the ability to walk and have bowel and bladder control in patients born with spina bifida,” said Farmer, distinguished professor of surgery at UC Davis, chief of pediatric surgery at Shriners Children’s Northern California and principal investigator for the project, also known as the CuRe Trial. UC Davis Children’s Hospital and Shriners Children’s Northern California have one of the largest and most well-known spina bifida programs in the nation. 

CIRM has funded several stages of Farmer’s research, including preclinical research and the completion of a Phase 1 clinical safety study which indicated no safety concerns in six patients. 

“We are very grateful and proud to have CIRM and Shriners Children’s as partners for Phase 2 of the CuRe trial,” said Aijun Wang, PhD, co-principal investigator with Farmer on the project. Shriners Children’s is co-funding the trial.  

“It is this important partnership that is making this groundbreaking work possible. This is also a testimony to the team science approach that our team has always been taking to conduct innovative research and solve unmet medical needs.” 

In addition to the clinical-stage project, CIRM is supporting two preclinical stage projects. These include a $4 million grant to advance a gene therapy for Charcot-Marie-Tooth disease type 4J (CMT4J), a rare, debilitating, progressive hereditary motor and sensory neurological disease. Also included is a $4.6 million grant to produce a CAR-T cell therapy for multiple myeloma, the second most common malignancy among blood cancers.  

For more information on CIRM’s clinical stage program, please visit CIRM’s funding opportunities page. 

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

CIRM Joins World AIDS Day Campaign, Commits $104 Million for HIV Research

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment
John A. Zaia, MD, of Beckman Research Institute of City of Hope

This year marks the 35th commemoration of World AIDS Day, a global event that aims to end human immunodeficiency virus (HIV) related stigma, honor people lost to the disease, and reaffirm the commitment to ensuring HIV is no longer a public health threat.  

This year, the theme of the global event is World AIDS Day 35: Remember and Commit. 

The California Institute for Regenerative Medicine (CIRM) has joined this global awareness event and remains committed to funding research to find therapies and treatments to fight HIV. 

About HIV & AIDS 

HIV is a virus that attacks the body’s immune system. If HIV is not treated, it can lead to acquired immunodeficiency syndrome (AIDS). There is currently no effective cure, though much progress has been made to reduce HIV transmissions and make the virus undetectable in people. 

Globally, an estimated 38 million people live with HIV. More than 35 million people have died of HIV or AIDS-related illnesses over the past 40 years, making it one of the most devastating pandemics in history.  

In California, there are more than 141,000 people living with HIV as of 2021. That same year, there were 4,444 new HIV diagnoses.  

While the rate of new diagnoses is on the decline in the state, the number of people living with HIV continues to grow. It is estimated that Californians living with HIV will face an average lifetime cost of $510,000 to treat their infection. 

Investing in HIV Research and Therapies at CIRM 

CIRM has invested more than $104 million in the search for stem cell and gene therapy treatments for HIV, ranging from discovery research to clinical trials. 

Recent CIRM investments include a study at UC Davis health, in which researchers take a patient’s own white blood cells, also called T cells, and modify them so they can identify and target HIV cells to control the virus without medication. 

CIRM also funded a clinical trial at UCSF to develop a treatment for HIV. In the study, the UCSF team lead by Steven Deeks, MD will modify a patient’s own immune cells to treat HIV.  

The goal of this one-time therapy is to act as a long-term control of HIV so patients no longer need to take antiretroviral therapy (ART), which involves taking a combination of HIV medicines every day. 

Recent HIV Research Awards 

Other recent CIRM awards include an $11.3 million grant to John A. Zaia, MD, of Beckman Research Institute of City of Hope for a clinical trial that will enroll up to 12 healthy people with HIV to be treated in a first-in-human study.   

Zaia’s City of Hope team, in partnership with the University of San Diego, will test a novel approach developed by City of Hope scientists in which a patient’s T cells are engineered to target both the HIV antigen gp120 and a common virus that infects nearly all persons living with HIV called cytomegalovirus.   

The goal is for these CAR-T cells to persist and eliminate HIV-infected cells so that antiviral drugs are no longer needed. 

World AIDS Day Events

CIRM will be co-sponsoring the upcoming World AIDS Day event in Palm Springs, hosted by HIV+ Aging Research Project and RID HIV. The event will be held at the Camelot Theater on December 1st, with a reception to follow.  

To learn more about CIRM-funded research to find treatments for HIV, visit this web page.