![]() ![]() The NHLBI will then quickly decide if it wants to partner with CIRM on co-funding the project and if the CIRM governing Board approves the project for funding, the two organizations will agree on a cost-sharing partnership for the clinical trial. CIRM will share application information with the NHLBI and CIRM’s Grants Working Group (GWG) – an independent panel of experts which reviews the scientific merits of applications – will review the applications and make recommendations. Programs that are ready to start an IND-enabling or clinical trial project for sickle cell can apply to CIRM for funding from both agencies. Under the agreement CIRM and the NHLBI will coordinate efforts to identify and co-fund promising therapies targeting SCD. This innovative partnership between CIRM and NHLBI is an encouraging sign of progress, and I applaud both organizations for their tireless work to cure Sickle Cell Disease.” “As a member of the House Appropriations Subcommittee on Labor, Health and Human Services, and Education, I know well the importance of this work. “I am deeply grateful for organizations like CIRM and NHLBI that do vital work every day to help people struggling with Sickle Cell Disease,” said Congresswoman Barbara Lee (D-CA 13 th District). Given CIRM’s new partnership with the National Heart, Lung, Blood Institute (NHLBI) to accelerate promising therapies that could help people with Sickle Cell Disease (SCD) the Agency is proposing to set aside $30 million in funding for this program.Ĭongresswoman Barbara Lee (D-CA 13th DIstrict) Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndromeįinally, the Board approved the Agency’s 2019 research budget. Modulation of the Wnt pathway to restore inner ear function Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseasesĭrug Development for Autism Spectrum Disorder Using Human Patient iPSCsĪ screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs Pluripotent stem cell-derived bladder epithelial progenitors for definitive cell replacement therapy of bladder cancer Universal Pluripotent Liver Failure Therapy (UPLiFT) The Quest program promotes the discovery of promising new stem cell-based technologies that will be ready to move to the next level, the translational category, within two years, with an ultimate goal of improving patient care. The Board also approved funding for seven projects in the Discovery Quest Program. Almost $4.24 million to Stanford’s Ted Leng to develop an off-the-shelf treatment for age-related macular degeneration (AMD), the leading cause of vision loss in the elderly.$4.17 million to Karin Gaensler at the University of California at San Francisco to use a leukemia patient’s own cells to develop a vaccine that will stimulate their immune system to attack and destroy leukemia stem cells.$5.068 million to University of California at Los Angeles’ Steven Schwartz to use a patient’s own adult cells to develop a treatment for diseases of the retina that can lead to blindness.The Board also approved three projects under its Translation Research Program, this is promising research that is building on basic scientific studies to hopefully create new therapies. This is a late stage pre-clinical program with a goal of developing the cell therapy and getting the data needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial. Making CAR T therapies safer and more efficient would not only help produce a new HIV treatment but would help with CAR T cancer therapies and could facilitate CAR T therapies for other diseases.” An exciting feature of this approach is the way it is combined with the cytomegalovirus (CMV) vaccine. ![]() “With 37 million people worldwide living with HIV, including one million Americans, a single treatment that cures is desperately needed. Jeff Sheehy, a CIRM Board member and patient advocate for HIV/AIDS, says there is a real need for a new approach. ![]()
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