The California Institute for Regenerative Medicine (CIRM) is pleased to announce the next round of funding opportunities for Translational (TRAN) research projects. Click here to access the Program Announcement.
The objective of this program is to fund promising stem cell-based and gene therapy projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use. These may include:
TRAN 1: Stem cell-based or gene therapy therapeutic candidate
TRAN 2:Diagnostic based on stem cells or critical for stem cell-based or genetic therapy development or use
TRAN 3:Medical device (non-diagnostic) for a stem cell-based therapy or critical for stem cell-based or genetic therapy development or use
TRAN 4: Novel tool that addresses a critical bottleneck to the discovery or development of stem cell-based or genetic therapy
Successful CIRM translational research projects that meet the program objective will be in position to progress rapidly to our Clinical Stage Programs.
Applications are due June 13th, 2023 at 2pm PDT.
Visit our websitefor more details on how to apply. We look forward to your applications!
For her first year of life, Seersha Sulack stayed mostly in her bedroom because something as simple as a common cold could have killed her. The five-year-old was born with ADA-SCID, a condition so rare that only eight babies a year are born with it in the United States.
Seersha’s journey was recently featured in CNN and is a true testament to the power of partnering to advancing medical research, including for rare diseases.
“I don’t like the word ‘normal’, but I’m ready to have something normal for her,” her mom told CNN.
According to the National Institutes of Health (NIH), approximately 7,000 rare diseases affect between 25 and 30 million Americans like Seersha. That equates to 1 in 10 Americans suffering from conditions for which limited treatment options exist.
“At CIRM we have funded several projects using gene therapy to help treat, and even cure, people with rare diseases such as SCID,” says Dr. Maria T. Millan, the President and CEO of CIRM. “But even an agency with our resources can only do so much.”
That’s why last year,CIRM signed a Memorandum of Understanding (MOU) with the Foundation for the National Institutes of Health (FNIH) to join the Bespoke Gene Therapy Consortium (BGTC), a public-private partnership managed by FNIH that brings together multiple public and private sector organizations to streamline the development and delivery of gene therapies for rare diseases.
As part of the Consortium, CIRM will identify specific rare disease gene therapy research programs in California that are eligible to be part of the AMP BGTC. CIRM funding can then support the IND-enabling research, manufacturing and clinical trial activities of these programs.
“This agreement with the Bespoke Gene Therapy Consortium will enable us to be part of a bigger partnership, one that can advance the field, overcome obstacles and lead to breakthroughs for many rare diseases,” Millan added.
Investing in Rare Disease Research
In addition to funding research and clinical trials for prevalent diseases, CIRM recognizes the importance of finding treatments for rare condition such as SCID, retinitis pigmentosa, sickle cell disease, Huntington’s disease, and Duchenne muscular dystrophy.
Part of our investment in rare diseases includes developing infrastructure programs to promote their access and affordability to diverse communities. That includes launching a Patient Support Program, which will provide resources and financial support to California patients being evaluated or enrolled in CIRM-funded clinical trials, with a particular emphasis on helping underserved populations.
These efforts will ensure that more patients like Seersha will have access to lifesaving treatments through clinical trials.
CIRM is pleased to welcome two new members to its leadership team, including Rafael Aguirre-Sacasa as General Counsel and Koren Temple-Perry as Senior Director of Marketing & Communications.
Rafael has over 25 years of experience in corporate law and joins CIRM after seven years at Standard BioTools (formerly Fluidigm Corp). There, he provided worldwide commercial, strategic and transactional legal support for all functions of the NASDAQ-listed life science company.
Prior to this role, he held a variety of management positions and provided legal support for a wide range of commercial, intellectual property and corporate matters at Teradici Corporation, PMC-Sierra, Inc., Autodesk, Hyperion solutions, Grupo Financiero and Xilinx Inc.
Rafael received his bachelor’s degree in history and government from Dartmouth College and law degree from the University of California-Hastings. As CIRM’s new General Counsel, Rafael will support the President, Board (ICOC), management and working groups on all legal matters affecting the agency.
“Joining the Institute as General Counsel is not just a job to me,” says Raphael. “It’s an opportunity to be part of an organization that is changing lives. I am enthusiastic about helping to implement CIRM’s strategic plan and contributing to their mission of accelerating stem cell treatments to patients in need, especially those in underserved and underrepresented communities.”
The agency also welcomes Koren Temple-Perry as Senior Director of Marketing Communications.
Koren joins CIRM after spending the last 15 years translating complex scientific information into compelling content for diverse audiences.
As a former journalist, she has worked extensively in the areas of patient and community health education, health advocacy, and scientific communications for hospitals, research institutes, and healthcare organizations.
Prior to joining CIRM, she founded Temple Communications LLC, which specializes in scientific copywriting and marketing communications for public health organizations.
She served as consulting communications director for March for Science, where she led the media and communications campaign for the global marches. She spent many years as a communications writer and digital marketing manager at academic medical centers throughout New York.
Koren earned her master’s degree in journalism from the S.I. Newhouse School of Public Communications at Syracuse University and a bachelor’s degree in communications from Santa Clara University. Her passion for improving health equity led to her appointment as District 5 Commissioner to the Alameda County Public Health Commission and to the Board of Directors for Jamal’s Helping Hands, a non-profit dedicated to helping families of color navigate rare diseases.
“I am truly honored to lead CIRM’s marketing communications team as the Agency aims to bring promising stem cell and gene therapy science to diverse communities,” says Koren. “I see incredible potential in building meaningful partnerships and elevating our outreach strategies to amplify cutting-edge treatments so that all Californians have equitable access to them. I am eager to make a positive impact in my new role and contribute to CIRM’s overall success.”
“We are delighted to have these talented individuals join the CIRM team,” says CIRM President and CEO Dr. Maria Millan. “They all bring unique qualifications and critical skills to support our strategic plan and advancement of CIRM’s mission to accelerate world class science for California and the world. We look forward to working with all of them.”
The purpose of the Quest Awards is to promote discovery of promising new stem cell-based and/or gene therapy technologies that could be translated to enable broad use and ultimately, improve patient care. The expected outcome, at the end of the award, is a candidate therapeutic or technology that can immediately progress to translational stage activities.
Applications are due May 2nd, 2023 at 2:00 PM PDT. Please visit this page on CIRM’s website for application instructions.
CIRM encourages the submission of proposals to advance the treatment and/or understanding of central nervous system (CNS) disorders.
Important Update: Please note that the DISC2 Program Announcement (PA) has been updated since the last round of applications.In addition, CIRM has implemented new guidelines and requirements for data sharing in our Discovery programs.
Have questions about this funding opportunity? Join the CIRM Scientific Programs team for an informational webinar.
This webinar provides an opportunity for prospective applicants to learn more about CIRM’s DISC2 Quest Awards and how to assemble a competitive application. Click here to register for the webinar.
The CIRM team will elaborate on DISC2 eligibility requirements, the application and review process, and how this program fits into CIRM’s R&D Pillar initiatives as well as our Strategic Plan and Mission.
The webinar includes a half-hour presentation by CIRM staff followed by a half-hour for Q&A.
Imbasciani (pictured above at left) was elected to the six-year term at CIRM’s January Board meeting. He will replace outgoing chair Jonathan Thomas, who has served in the position since 2011.
“My experience has positioned me to champion the aims of CIRM, advocate for it cogently, and represent it responsibly before the public and their state and federal elected representatives,” Imbasciani said. “I look forward to the challenge of advancing the groundbreaking work of this Agency, at the same time nourishing the hopes for medical advances held by the citizens of our great State.”
Imbasciani has served as the Secretary of the California Department of Veterans Affairs (CalVet) since 2015. As Secretary, he created several new programs within the department, including forging eight independent California veteran homes into a unified system, establishing programs for veterans in state prisons, and supporting the 58 county veteran service offices.
Current CIRM Vice Chair Maria Bonneville, Fmr. Vice Chair Sen. Art Torres (ret). and Fmr. Chair Jonathan Thomas
CIRM also bid farewell to Sen. Art Torres (ret) and Jonathan Thomas (also known as JT) for their service on our Board.
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.
He served as chair of the Governing Board for more than 12 years. In that time, he led CIRM in granting $2.5 billion in grants to support groundbreaking research and discovery to advance stem cell research and accelerate stem cell treatments to patients with unmet medical needs.
He led the agency as it 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.
Senator Torres was nominated in 2009 as CIRM Vice Chair by then-Lieutenant Governor Gavin Newsom and Treasurer Bill Lockyer, and shortly after was elected by the CIRM Governing Board.
Senator Torres served on the Grants and Standards working groups, and served as the inaugural Chair of the Accessibility and Affordability Working Group. He served on numerous subcommittees, led CIRM’s government relations efforts, and played a pivotal role in launching CIRM’s SPARK high school internship program.
CIRM Vice Chair Maria Bonneville, Fmr. Chair Jonathan Thomas, Chair Vito Imbasciani, and President & CEO Maria Millan
CIRM expresses its deepest gratitude to Senator Torres and JT for their service on its Governing Board and for their dedication to the advancement of stem cell research and our mission to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world.
Her list of achievements continues to grow, as President Biden has appointed Duron to National Cancer Advisory Board (NCAB), which plays an important role in setting the course for the national cancer research program.
The National Cancer Advisory Board will complement the Cancer Moonshot, which President Biden reignited a year ago to invest in research and development that will help advance breakthroughs to prevent, detect and treat diseases like cancer.
“As a Latina, and a long-time patient and community advocate, it humbles me to join this roster of stellar new appointees,” Duron said. “I look forward to the challenge of amplifying the voices of racial and ethnic communities and other vulnerable populations.”
Duron came into the cancer space after her own bout with Hodgkins Lymphoma in 1999. She covered her own cancer battle using her reporting skills to raise awareness about the disease.
Over time, she turned a spotlight on the many disparities—lack of access, income inequality, language barriers, among other social determinants on health—that has exacerbated the disproportionate burden of cancer in Latino communities.
In 2017, Ms. Duron founded The Latino Cancer Institute (TLCI), a nationwide network dedicated to developing and sharing best practice programs to enhance the work of Latino community service agencies, to provide collaboration with the global cancer research community, and drive policy to solve the issues and burden of Latinx/Hispanic cancer.
PD is characterized by a loss of dopamine producing neurons that result in motor symptoms, such as dyskinesias (involuntary, erratic, writhing movements of the face, arms, legs or trunk) and non-motor effects such as dementia, depression and sleep disorders.
PD is the second-most common neurodegenerative disease after Alzheimer’s disease affecting approximately 1 million people in the U.S. In California, it is estimated that 116,900 people live with PD, representing the highest number of people with the disease in the country.
At its early stages, PD can be treated with medication such as Levodopa to treat symptoms but these become less effective as the disease progresses.
The proposed stem cell therapy in this project offers the potential to restore dopamine neurons, which play a role in many important body functions, including movement and memory.
Investigators at Ryne Bio are aiming to deliver dopamine producing cells to replace the lost neurons to the brain of Parkinson’s disease patients to restore/improve motor function.
The current grant is being funded to conduct Investigational New Drug (IND) enabling, nonclinical safety studies per the US Food and Drug Administration (FDA) Guidance. The IND is the authorization needed to begin a clinical trial in Parkinson’s patients.
CIRM has a vested interest in seeing this therapy succeed. To date, CIRM has invested more than $59 million in helping research for Parkinson’s disease progress from a basic or Discovery level through clinical trials.
Dr. Vito Imbasciani will be the new Chair of the California Institute for Regenerative Medicine (CIRM), the state agency created by voters in 2004 and funded again in 2020 to invest in stem cell and regenerative medicine research and treatments.
At January’s Board meeting, the agency’s 35-member Governing Board elected Imbasciani to the six-year term, replacing outgoing chair Jonathan Thomas, who has served in the position since 2011.
“Dr. Imbasciani’s experience across many relevant fronts will help him hit the ground running in guiding the Agency as it continues to grow its programs to bring treatments to patients with unmet medical needs,” Thomas said in welcoming Imbasciani to the role. “The agency, as well as the people of California and the world, will be well served by Imbasciani’s appointment as Chair of the CIRM Governing Board.”
Imbasciani expressed excitement in taking on the role, citing his extensive career in academia, government, military service and medicine.
“My experience has positioned me to champion the aims of CIRM, advocate for it cogently, and represent it responsibly before the public and their state and federal elected representatives,” Imbasciani said. “I look forward to the challenge of advancing the groundbreaking work of this Agency, at the same time nourishing the hopes for medical advances held by the citizens of our great State.”
Imbasciani has served as the Secretary of the California Department of Veterans Affairs (CalVet) since 2015. As Secretary, he created several new programs within the department, including forging eight independent California veteran homes into a unified system, establishing programs for veterans in state prisons, and supporting the 58 county veteran service offices.
In addition, Dr. Imbasciani has been a practicing urologic surgeon for 30 years, treating a mostly older population suffering from congenital and acquired conditions.
Dr. Imbasciani completed medical school at the University of Vermont College of Medicine, and his surgical and urologic residencies at Yale-New Haven Hospital and the West Haven VA Hospital in Connecticut. At the University of Vermont, he worked in the laboratory assisting in studies of neurodegenerative diseases.
He earned MA and PhD degrees from Cornell University, and was a Fulbright Scholar to Rome, Italy in 1973. He held academic teaching positions at the University of Florida, Cornell University and Middlebury College in Vermont.
He also served for 27 years as a surgeon in the United States Army Medical Corps, with four wartime deployments that exposed him to battlefield medicine and post-acute care.
Dr. Imbasciani also has a documented history in successful stem cell research advocacy. As an elected member of the Board of Directors of both the California Medical Association and the Los Angeles County Medical Association, he advocated for investments in basic stem cell research, and for the passage of Proposition 71, the ballot initiative that created CIRM. This included participating in activities aimed at educating the wider medical community in the long-term benefits of stem cell research.
CIRM President and CEO Dr. Maria T. Millan applauded Imbasciani’s appointment as Chair.
“Dr. Imbasciani’s experience as a state secretary, surgeon, professor, stem cell research advocate, and board member of various medical agencies and organizations makes him exceptionally well-suited to fill the role of ICOC Chair and to lead CIRM in accelerating world class science and treatments for a diverse California and the world. I look forward to working with him in his new role.”
Imbasciani will be sworn in and start on March 28, 2023.
Dr. Karin Gaensler. Photo credit: Steve Babuljak/UCSF
Adult acute myelogenous leukemia—also known as acute myeloid leukemia (AML)—is a blood cancer in which the bone marrow makes a large number of abnormal blood cells.
About 20,000 new cases of AML are diagnosed each year in the US with a 5-year survival rate of around 29%. In 2022, there were nearly 12,000 deathsfrom AML. Many AML patients—a majority of which are over 60 years old—relapse after treatment. Blood stem cell transplant can be curative, but many older patients do not qualify, showing that there is a significant unmet medical need in treating AML.
To develop the cancer vaccine, Dr. Gaensler and her team will engineer the patient’s blood stem cells to maximize stimulation of leukemia-specific killing activity and reintroduce engineered cells back to the patient to target and kill residual leukemia stem cells.
This approach holds the potential for long-term effectiveness as it targets both AML blasts and leukemic stem cells that are often the source of relapse.
This award is a continuation of a previous CIRM grantthat will support the manufacture of the vaccine and the completion of late-stage testing and preparation needed to apply to the US Food and Drug Administration (FDA) for permission to begin a clinical trial.
Blood stem cell transplantation following high dose chemotherapy is standard of care and potentially curative for aggressive forms of lymphoma. However, this treatment regimen is limited by severe toxicity and life-threatening complications due to delayed recovery of the blood system and vascular related damage of multiple organs.
This brings the number of clinical trials funded by CIRM to 86.
The Board awarded $15,000,000 to Dr. Paul Finnegan and Angiocrine Bioscience to test AB-205, human endothelial cells engineered to express a pro-survival factor.
Prior data suggest that, in the setting of chemotherapy and stem cell transplantation, AB-205 cell therapy can accelerate the recovery of the blood system and protects from toxicity by enhancing the recovery from vascular damage. AB-205 is being studied in a Phase 3 trial in adults with lymphoma undergoing high-dose chemotherapy and autologous blood stem cell transplant.
“If successful, this approach can overcome hurdles to the success of chemotherapy and blood stem cell transplantation for the treatment of advanced blood cancer,” says Dr. Maria T. Millan, President and CEO of CIRM. “This Phase 3 trial is the culmination of preclinical research and the initial clinical trial previously funded by CIRM.”
Lymphoma is the most common blood cancer and one of the most common cancers in the United States, accounting for about 4% of all cancers according to the American Cancer Society and the 6th most commonly diagnosed cancer among men and women in California. It is estimated that there will be 89,010 new cases of lymphoma and 21,170 lymphoma related deaths in the US in 2022 alone. In California, it is estimated that there will be over 9,250 new cases of lymphoma with over 2,100 deaths.
“Angiocrine Bioscience is honored to be awarded this grant from CIRM to support our AB-205 Phase 3 trial,” commented Angiocrine CEO Dr. Paul Finnegan. “CIRM has been an instrumental partner in our development of AB-205, a novel therapeutic that acts on the patients’ endogenous stem cell niches. The grant award will considerably aid in our effort to bring forth a solution to the unmet need of transplant-related complications.”
The Stem Cellar 