Major CIRM & NHLBI Funding Pushes Sickle Cell’s CRISPR Cure Towards the Clinic

The California Institute for Regenerative Medicine (CIRM) and the National Heart, Lung, and Blood Institute (NHLBI) of the NIH have awarded $4.49 million to advance the IGI’s ongoing effort to cure sickle cell disease (SCD), fueling the final stretch to the clinic.

Researchers have known the molecular cause of sickle cell disease (SCD) for exactly 70 years, yet no universally applicable treatment or cure exists. This genetic disease affects nearly 100,000 Americans, and millions more globally. SCD patients share a single-letter DNA mutation that leads to moon-shaped, “sickled,” red blood cells which can block blood vessels, causing immense pain and poor oxygen transport throughout the body. Current patients anticipate life expectancies in the mid-forties, but a new approach to this old disease is on the way.

Since its inception, the IGI has been developing tools and techniques to safely and effectively treat SCD. This funding builds upon an earlier $4 million investment from CIRM, which supported foundational work by Jacob Corn to refine CRISPR-Cas9 editing and reverse the sickle mutation. Other studies improved the efficiency of inserting a healthy genetic sequence. Synergistic studies across other IGI program areas have simultaneously improved the safety and accuracy of genome editing. The culmination of these efforts is an approach that is able to correct the SCD mutation in a proportion of stem cells that should be high enough to produce a substantial benefit in sickle cell patients. Now with the recent funding from CIRM and NHLBI, the IGI aims to take this novel therapy to patients.

“Sickle cell disease is a major public health challenge with no current options for effective and sustainable treatment. CRISPR is a once-in-a-lifetime opportunity to cure this disease at the molecular level,” says IGI Executive Director Jennifer Doudna. “We are all-in and ready to make this happen for patients.”

The IGI is honored to partner with two of the world’s foremost institutions, UCSF Benioff Children’s Hospital in Oakland and UCLA, where efforts are led by Mark Walters and Don Kohn, respectively. This collaboration has been essential and fruitful since the early days of this project, and the clinical expertise of our partners will be vital in translating laboratory discovery into curative therapies for patients with sickle cell disease and other hereditary blood disorders.

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