Just as the world’s first CRISPR-based gene editing therapy gained approval for treating sickle cell disease late last year, Pfizer waswith an investigational oral drug for the same condition. Regulatory backing for Casgevy received far more attention, given its historic significance and dramatic effects on relieving acute symptoms of the condition. But for many people living with sickle cell disease an oral drug such as GBT021601 might be more important.
Pfizer hopes that GBT021601’s higher affinity for oxygen will translate into a superior safety and efficacy profile over that of Oxbryta. “I do think ’601 has the potential to be more efficacious at a lower dose,” says Smith-Whitley.data Pfizer unveiled at the American Society of Hematology’s 2023 annual meeting. After 12 weeks’ therapy, patients in the high-dose and low-dose arms of an ongoing phase 2/3 study achieved mean increases in hemoglobin of 3.17 g/dL and 2.67 g/dL, respectively. .
Several other targets linked to HbF expression have recently emerged. A team of scientists at Basel, Switzerland-based Novartis has used phenotypic screening tothat degrade a transcription factor, Wiz, thereby de-repressing γ-globin expression and boosting HbF production in patient-derived cells. Others are attempting to drug BCL11A directly.
In vivo gene or gene editing therapy would bypass these problems. That may seem a remote prospect at this point, but it is the long-term ambition of a partnership between the Innovative Genomics Institute at the University of California, Berkeley, led by Jennifer Doudna, and Pioneer Science, a Brazilian philanthropic organization based in Rio de Janeiro.
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