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.
The world’s first regulatory authorization for a CRISPR-based gene-editing therapy begins a new era for sickle cell disease treatment.
Sickle cell disease affects more than 100,000 Americans and 20 million people worldwide.
“This approval marks a milestone for cell and gene therapies,” said Abla Creasey, PhD, CIRM’s vice president of therapeutics development.
“It also transforms treatment options for millions of people with sickle cell disease.”
CRISPR gene editing could offer a more effective, potentially curative option than traditional treatments.
The FDA also approved Lyfgenia, another sickle cell gene therapy from Bluebird Bio. Lyfgenia uses established lentiviral vector technology.
Through the Cure Sickle Cell Initiative, CIRM continues funding cell and gene therapy programs.
The National Heart, Lung, and Blood Institute funds the initiative.
These programs aim to accelerate sickle cell disease treatments and create a collaborative, patient-focused research environment.
CIRM recognizes the need for continued innovation. Researchers must lower treatment costs, reduce patients’ treatment burdens, and improve access.
CIRM and NHLBI co-fund two clinical trials that test different gene therapy approaches for severe sickle cell disease.
David A. Williams, MD, of Boston Children’s Hospital, leads a pivotal Phase 2 trial.
Mark Walters, MD, of UCSF Benioff Children’s Hospital Oakland, leads a Phase 1 trial.
In Williams’s Phase 2 trial, researchers will collect each patient’s blood stem cells.
They will insert an engineered gene that silences abnormal hemoglobin and increases fetal hemoglobin production.
Researchers will then return the modified stem cells to the patient.
The therapy aims to produce normally shaped red blood cells that carry oxygen efficiently.
This result could reduce the disease’s severity.
“As a researcher and clinician, I am delighted that the FDA approved two effective genetic therapies,” Williams said.
“I look forward to patients with severe disease accessing these treatments. I also welcome additional curative treatment options.”
Williams has consulted for Vertex Pharmaceuticals and bluebird bio.
In Walters’s Phase 1 trial, patients will receive their own blood stem cells.
Researchers will edit those cells with CRISPR-Cas9 to correct the disease-causing beta-globin gene mutation.
The 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.