The California Institute for Regenerative Medicine (CIRM), established by the voters of California, is accelerating research and awareness of Sickle Cell Disease.
The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, is proud to announce the launch of “Championing Change for Sickle Cell in California,” a pioneering campaign aimed at increasing awareness of Sickle Cell Disease (SCD) and research aimed at improving the treatment landscape for the condition.
With more than 100,000 Americans and 20 million people worldwide affected by this debilitating condition, CIRM is committed to funding cutting-edge cell and gene therapy programs that have the potential to transform the lives of SCD patients.
Sickle cell disease is a genetic disorder that causes red blood cells to assume a sickle shape under stress, leading to clogged blood vessels, excruciating pain crises, and organ damage.
CIRM 2021-2022 Annual Report
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This disease disproportionately affects people of African American and Latinx heritage. It is estimated that SCD occurs among about 1 out of every 365 Black or African-American births. Latinx Californians make up 7.3 percent of sickle cell disease cases—and Latinx newborns make up 11 percent of new cases.
In collaboration with key organizations such as sickle cell patient advocacy group Axis Advocacy, the National Heart, Lung, and Blood Institute’s (NHLBI) Cure Sickle Cell Initiative, and the UCSF Sickle Cell Center of Excellence, CIRM is working to champion change and drive progress for sickle cell treatments.
Community partners and patient advocates play a crucial role in the development and success of these treatments, and CIRM recognizes their importance. Among the key calls to action of the campaign include becoming a sickle cell disease advocate, spreading awareness of clinical trial options for sickle cell, downloading a fact sheet and learning more about the NHLBI Cure Sickle Cell Initiative.
In addition to spreading awareness of this condition, the campaign highlights how CIRM is funding research into stem cell, gene therapy, and other regenerative medicine procedures for sickle cell disease. To date, CIRM has invested over $65 million towards research for sickle cell, ranging from basic discovery to clinical trials.
CIRM and the NHLBI are co-funding two clinical trials that are investigating different gene therapy approaches for patients with severe sickle cell disease. These trials include a Phase 1/2 trial led by Dr. Mark Walters of UCSF Benioff Children’s Hospital Oakland and a Phase 2 trial led by Dr. David A. Williams of Boston Children’s Hospital. These clinical trials are essential in the search for new therapies to treat sickle cell disease.
CIRM’s new campaign comes at the heels of two recently FDA-approved gene therapy treatments for sickle cell disease. Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia have brought forth a new era of treatment options for people living with the condition.
“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.
For more information on CIRM’s Sickle Cell Disease campaign, ongoing research projects, and ways to champion change, please visit cirm.ca.gov/sickle-cell-disease. To get involved in the campaign, please contact CIRM’s Community Outreach Manager Aditi Desai at adesai@cirm.ca.gov.
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- Source: https://blog.cirm.ca.gov/2024/04/15/state-agency-launches-championing-change-for-sickle-cell-in-california-campaign/