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spotlight

MARAC Advisory Statement: COVID-19 Vaccines

News is evolving rapidly about COVID-19 and COVID-19 vaccines. Early results from the COVID-19 vaccine trials are very promising, although the true benefits and risks will not be known until a larger number of people receive the vaccine.

The Centers for Disease Control and Prevention (CDC) lists sickle cell disease (SCD) as one of the populations vulnerable to severe COVID-19. Sickle cell disease raises the risk for serious problems with COVID-19, especially when compared to the same age in the general population.

Dec 15, 2020
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clinical trials and research

FDA Clears Graphite Bio to Begin Trial for Gene Therapy in Sickle Cell Disease

In response to Graphite Bio’s investigational new drug (IND) application for its gene editing therapy, GPH101, the FDA will allow the pharmaceutical company to initiate a phase I/II trial in patients with sickle cell disease (SCD).

Aimed at curing SCD by targeting the gene mutation that causes damaged and sickled red blood cells, GPH101 uses CRISPR technology to cut out this mutation in the beta-globin gene and paste in the wild-type DNA sequence. The CEDAR trial will evaluate the safety, preliminary efficacy, and pharmacodynamics of GPH101 in adult and adolescent patients with severe SCD. “We are eager to initiate enrollment for the CEDAR clinical trial in early 2021, which will be a historic milestone as the first experimental treatment designed to correct the mutation that is the underlying cause of sickle cell disease,” said Graphite Bio CEO Josh Lehrer, MPhil, MD.

Dec 20, 2020