New Small Molecule Pill Offers Safer Treatment for Sickle Cell Disease, Published in Science Magazine

Jul 4, 2024, 06:20 PM

A groundbreaking discovery in the treatment of sickle cell disease has been published in Science Magazine. Researchers have developed a small molecule pill that acts as an epigenetic modifier to inhibit fetal hemoglobin, offering a less dangerous and potentially more accessible alternative to CRISPR genome editing, such as Casgevy. This new approach could open up treatment options globally, reducing the need for toxic conditioning. The study, led by a team including Pamela Ting and Jay Bradner, marks a significant advancement in chemical biology and the development of molecular glue degraders.