Next Generation Gene-Editing
GPH102 RD Beta-Thalassemia; 2 add'l RD programs
Graphite Bio is a gene-editing company developing therapeutics for a wide range of diseases. CRISPR technology offers the potential to rewrite genes and cure diseases following one therapeutic dose. Using their UltraHDR platform, Graphite is identifying CRISPR machinery that offers incredible precision in the gene-editing process and works to correct, replace and insert genes anywhere in the genome. Graphite’s proprietary technology has the potential to deliver individualized therapies going beyond personalized medicine. Currently, Graphite’s most advanced product, GPH101, is being tested to correct Sickle cell disease, with other programs focusing on Beta-thalassemia and AAT deficiency.